Predicting the Treatment Evolution of Hemophilia B – Managed Healthcare Executive

Steven Pipe, MD, describes his hopes for Hemophilia B treatment in the future.

EP. 1 : An Overview of Hemophilia B

EP. 2 : The Economic Burden of Hemophilia B From a Payer Perspective

EP. 3 : Defining the Impact of Gene Therapy

EP. 4 : Managing Hemophilia B Using Gene Therapy

EP. 5 : Predicting the Treatment Evolution of Hemophilia B

Steven Pipe, MD: The current platform looks promising for durable correction in factor 9 levels that are sufficient to prevent most bleeding events and improve health-related quality of life, with a substantial elimination of the economic and personal treatment burden associated with factor 9 prophylaxis. However, with the eligibility limitations we've also indicated, the biggest one, for me, is that this is only for adults. By adulthood, a substantial portion of patients already have established joint disease. We would have a much bigger impact from gene therapy if we could do an earlier intervention in the pediatric age group. Unfortunately, with the way the AAV mechanism occurs by targeting the liver, the DNAthat's the transientis delivered into the nucleus and remains episomal, which means it's primarily outside of the genome. When dividing liver cells, the transgene doesn't get propagated to the daughter cells. What that would mean is, if we did AV in a pediatric age group with a growing, dividing liver, the effect of the gene therapy will be deluded over time. We need different approaches for gene therapy to impact the pediatric age group.

Some of the things I've seen that show promise are non-viral approaches. By using a non-viral approach, you have the option to re-treat patients. That is not an option for patients who get current AV therapy because once you've delivered the AV once, the immunological consequences of that would preclude getting another AV dose sometime later. Right now, we're asking patients to make a once-in-a-lifetime treatment decision to receive gene therapy, but other approaches could also involve gene editing. When you do gene editing, you're either inserting the transgenes somewhere in the genome or modifying the factor 9 gene in such a way that you can restore factor 9 expression. Because you're editing directly in the genome, that would be propagated to daughter cells in a dividing liver. I'm already seeing some new protocols come to the forefront [that will be implemented] in the clinic soon. If these show their utility in adult patients, we'll see that these offered to younger and younger patients and, hopefully, the pediatric age groups where we can completely abrogate the development of joint disease.

The other thing I'm excited about is the evolving treatment landscape as applied to hemophilia B, not just invested in gene therapy approaches. There are several innovative treatment platforms that have been tested over the last few years. The ones I'm particularly excited about are non-factor placement therapies. These are cross-platform therapeutics that can work for hemophilia A or B regardless of whether the patient has had any prior inhibitor development to the factor 8 or factor 9 proteins. These primarily target the natural anticoagulants of the hemostatic process. They rebalance hemostasis without replacing the factor 9 molecule. Data that was presented at this most recent ISTH Congress [International Society of Thrombosis and Haemostasis] in July of this year showed encouraging results that can be applied to patients with hemophilia B. These are treatments that can be delivered subcutaneously. They have a lower intensity. Some of them are given either weekly, monthly, or once every other montha low burden of treatment. They [provide] effective prophylaxis that, in some studies, is superior to traditional factor 9 replacement therapy. I'm excited about the evolving treatment landscape. Hemophilia B gene therapy [carries] great promise, but these other novel therapeutics are hopefully going to be available to us in the clinics quite soon.

Transcript edited for clarity.

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Predicting the Treatment Evolution of Hemophilia B - Managed Healthcare Executive

Building Europes gene therapy ecosystem POLITICO

Gene therapy is a transformative field of medicine that could halt diseases before patients become symptomatic, saving lives and improving quality of life for millions of people who suffer from conditions caused by genetic abnormalities1. But these breakthrough treatments require new approaches to pricing and reimbursement2.

While the science behind gene therapy has existed for decades, the first generation of treatments is now emerging for debilitating conditions including spinal muscular atrophy3 and inherited retinal diseases4 5. Although Europe was at the forefront6 of science a decade ago, other regions have invested heavily in research and Europe has work to do to re-gain7 its leadership role.

A cell is the fundamental biological building block of all living things8. Genes, found within cells, are small sections of DNA that carry the cells genetic information. Genes are the cells blueprint for making proteins that support functions such as muscle strength, and mutations in genes contribute to a range of diseases including those caused by recessive gene disorders, like hemophilia and sickle cell anemia, acquired genetic diseases such as certain cancers, and some viral infections like AIDS9.

Gene therapies improve and/or replace mutated genes, most often via the technique of recombinant DNA technology in which a molecular carrier known as a vector is used to carry a healthy copy of the gene and introduce the new genetic material into the cell11. Most commonly, gene therapy allows cells to add an additional genetic unit, but new engineering techniques now exist to switch off, allow modification to or correct the problematic DNA sequence12.

Europes gene therapy leadership

Academic researchers, biotech and pharmaceutical companies in Europe have been instrumental in developing the field of gene therapy13. Between 2017 and 2019, the lead author of 120,000 papers in cell and gene therapy (CGT) publications was affiliated with a European institution, compared to 72,000 and 100,000 in the U.S. and China respectively14. Professor Emmanuelle Charpentier at the Max Planck Unit for the Science of Pathogens in Berlin was a joint winner of the Nobel Prize in Chemistry 2020, a recognition of her contribution to the development of tools for gene editing15.

The European Unions medical regulatory institution, the European Medicines Agency (EMA), has been a world leader in assessing advanced therapy medicinal products (ATMPs), a class of innovative biological products of which gene therapy is part, approving a tissue-engineering product in 2009, a year before the U.S.s first ATMP approval16. It also approved the worlds first gene therapy in 201216. The EMA has granted orphan drug status to the majority of CGT drugs currently in development and performed accelerated assessments for a number of cell and gene therapies14.

But the region needs to build on its momentum to take gene therapy from the lab to patients. The total number of gene-, cell- and tissue-based therapeutic developers worldwide lags behind the North American and Asia-Pacific regions17.

Europes gene therapy sector could be boosted by introducing more flexibility and agility to the methodologies that are used to evaluate and reimburse breakthrough treatments.

Because the diseases that cell and gene therapies are aimed at are rare, trials are conducted in a far smaller number of patients than is normal for a new medicine. Decisions to license the treatment will be based on the data from one small trial7.

Health Technology Assessment (HTA) bodies, who ultimately determine whether a new therapy is reimbursed, expect to review randomized controlled trials against a standard of care18. Yet gene therapies are a new approach that can effectively intercept, rather than manage, a disease and there is therefore no standard of care to compare against19.

Cost-effectiveness is commonly evaluated based on the direct financial burden of a disease on the health system18. Yet many genetic diseases biggest impact is indirect inherited blindness, for instance, is costly for the individual, their family and carers, and most costs are borne in the social and welfare system, rather than in clinics and hospitals20. One review by Deloitte estimated the total cost attributable to inherited retinal diseases at 523.3 million in 2019, across economic and wellbeing costs (see chart). Health system costs were the lowest share20.

Gene therapies that have the potential to intercept a disease entirely, are more difficult to quantify in terms of benefits19. Conventional HTA methods discount a benefit over time as a patients quality of life deteriorates due to aging and its other attendant health consequences21. This makes little sense in the case of a gene therapy that can avoid blindness emerging in a young person, say, thereby allowing them to lead a full and active adult life21.

Unlike rare disease treatments that are regularly administered over decades, gene therapy would be administered only once, providing many years, if not a lifetime, of biological activity and clinical benefit2. Under current reimbursement systems, this therapy would be paid once at the time it is administered. To encourage investment in the development of these therapies, payments in excess of $1 million may be needed2. The fact that one intervention can provide a cure, replacing long-term costs of treatment, makes investment in gene therapy a problem that multiple stakeholders need to work together to solve2. The industry is developing new approaches to pricing and reimbursement, such as outcome-based payment models22, orannuity payments23 based upon duration and efficacy, which could help payers accommodate the one-time nature of gene therapies.

Europe is already moving towards a coordinated response to review the challenges of breakthrough therapies. In December 2021, Europes Regulation on Health Technology Assessment was adopted to help member states to make timely and evidence-based decisions on patient access to innovative health technologies24. Gene therapies are amongst the medical breakthroughs that European reform dialogue is focused on to address unequal access across the continent25.

Building on this momentum can bring forth the required change to value assessment methodologies and help to realize the full potential of gene therapies. It will ensure gene therapy, long confined to research laboratories, can finally reach patients, and open a new chapter in medical history21.

Follow our journey at https://www.politico.eu/evolution-of-health-care/

References

1Mayo Clinic Staff. (2017). Gene Therapy. Available at:https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619

2Brennan, T. and Wilson, J. (2014). The special case of gene therapy pricing. Available at:https://www.nature.com/articles/nbt.3003

3Vamshi K. et al. (2018). Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease. Available at: https://www.jmcp.org/doi/pdf/10.18553/jmcp.2018.24.12-a.s3

4EMA Europe. (2018). Luxturna, European Medicines summary of product characteristics. Available at https://www.ema.europa.eu/en/documents/product-information/luxturna-epar-product- information_en.pdf

5Maldonado, R. et al. (2020). Curative gene therapies for disease. Available at: https://link.springer.com/article/10.1007/s12687-020-00480-6

6Cressey, D. (2012). Europe Nears First Approval for Gene Therapy Treatment. Available at: https://www.scientificamerican.com/article/europe-nears-first-approval-gene-therapy-treatment/

7Collis, H. (2022). Patients with rare diseases hope for Brexit hastened treatments. Available at: https://www.politico.eu/article/uk-gene-cell-therapy-brexit-science-research-rare- diseases/

8NCBI. (1989). The Fundamental Biological Unit of All Living Organisms is the Cell. Available at: https://www.ncbi.nlm.nih.gov/books/NBK217797/

9NCBI. (2017). Gene therapy: advances, challenges and perspectives. Available at:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5823056/

10American Society of Gene + Cell Therapy. (2021). Gene, Cell, & RNA Therapy Landscape. Available at: https://asgct.org/global/documents/asgct-pharma-intelligence-quarterly-report-july-20.aspx?_zs=sisac&_zl=Uu4h2

11Britannica. (2022). Recombinant DNA Gene therapy. Available at:https://www.britannica.com/science/recombinant-DNA-technology/Gene-therapy

12Medline Plus. (2022). What is gene therapy?. Available at:https://medlineplus.gov/genetics/understanding/therapy/genetherapy/#:~:text=A%20newer%20technique%2C%20called%20genome,existing%20DNA%20in%20the%20cell

13AuWerter, T. et al. (2020). Biopharma portfolio strategy in the era of cell and gene therapy. Available at: https://www.mckinsey.com/industries/life-sciences/our-insights/biopharma-portfolio-strategy-in-the-era-of-cell-and-gene-therapy

14Loche, A. et al. (2021). A call to action: Opportunities and challenges for CGTs in Europe. Available at: https://www.mckinsey.com/industries/life-sciences/our-insights/a-call-to-action-opportunities-and-challenges-for-cgts-in-europe

15Rogers, K. (2020). Emmanuelle Charpentier. Available at: https://www.britannica.com/biography/Emmanuelle-Charpentier

16NCBI. (2019). Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States. Available at:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6728416/

17Alliance for Regenerative Medicine. (2022). Regenerative Medicine: Disrupting the status quo. Available at:http://alliancerm.org/wp-content/uploads/2022/03/ARM_AR2021_FINAL-singles.pdf

18EUPATI. (2022). HTA systems in Europe. Available at: https://toolbox.eupati.eu/resources/hta-systems-in-europe/

19Pochopie M et al. (2021). An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8592603/

20Deloitte Access Economics & Retina International. (2019). The socio-economic impact of inherited retinal dystrophies (IRDs) in the United Kingdom. Available at: https://www2.deloitte.com/content/dam/Deloitte/au/Documents/Economics/deloitte-au-economics-cost-illness-irds-uk-030919.pdf

21Salzman, R. et al. (2018). Addressing the value of gene therapy and enhancing patient access to transformative treatments. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277509/

22Jrgensen, J. and Kefalas, P. (2019). The use of innovative payment mechanisms for gene therapies in Europe and the USA. Available at: https://www.futuremedicine.com/doi/10.2217/rme-2020-0169

23Cook, F. et al. (2018). Regenerative Medicine is Here: New Payment Models Key to Patient Access. Available at: https://alliancerm.org/wp-content/uploads/2018/07/ARM_WhitePaper3_IV1807_LRS.pdf

24HTA Regulation Press. (2021). Health Technology Assessment: Commission welcomes the adoption of new rules to improve access to innovative technologies. Available at: https://ec.europa.eu/commission/presscorner/detail/en/ip_21_6771

25Cornetta, K. et al. (2022). Gene therapy access: Global challenges, opportunities, and views from Brazil, South Africa, and India. Available at: https://www.sciencedirect.com/science/article/pii/S1525001622002301

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Building Europes gene therapy ecosystem POLITICO

FDA approval brings first gene therapy to the United States

For Immediate Release: August 30, 2017

This release was updated on Aug. 30, 2017 to correctly identify the FDA designations granted to Kymriah.

Espaol

The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, said FDA Commissioner Scott Gottlieb, M.D. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, were committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patients own T-cells, a type of white blood cell known as a lymphocyte. The patients T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes. The disease progresses quickly and is the most common childhood cancer in the U.S. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year. ALL can be of either T- or B-cell origin, with B-cell the most common. Kymriah is approved for use in pediatric and young adult patients with B-cell ALL and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 percent of patients.

Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research (CBER). Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.

The safety and efficacy of Kymriah were demonstrated in one multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was 83 percent.

Treatment with Kymriah has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events. Both CRS and neurological events can be life-threatening. Other severe side effects of Kymriah include serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). Most symptoms appear within one to 22 days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also destroy those normal B cells that produce antibodies, there may be an increased risk of infections for a prolonged period of time.

The FDA today also expanded the approval of Actemra (tocilizumab) to treat CAR T-cell-induced severe or life-threatening CRS in patients 2 years of age or older. In clinical trials in patients treated with CAR-T cells, 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of Actemra.

Because of the risk of CRS and neurological events, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified. As part of that certification, staff involved in the prescribing, dispensing, or administering of Kymriah are required to be trained to recognize and manage CRS and neurological events. Additionally, the certified health care settings are required to have protocols in place to ensure that Kymriah is only given to patients after verifying that tocilizumab is available for immediate administration. The REMS program specifies that patients be informed of the signs and symptoms of CRS and neurological toxicities following infusion and of the importance of promptly returning to the treatment site if they develop fever or other adverse reactions after receiving treatment with Kymriah.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

CAR T-cell therapy approved to treat certain children and young adults with B-cell acute lymphoblastic leukemia

Andrea Fischer 301-796-0393

Angela Stark 301-796-0397

888-INFO-FDA, OCOD@fda.hhs.gov

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FDA approval brings first gene therapy to the United States

Hope For Leukemia Patients, Where Other Treatments Have Failed – Longevity LIVE – Longevity LIVE

Two experts from Cleveland Clinic explain how a new cell-based gene therapy is helping patients with certain types of leukemia, with research into potential wider applications continues.

As World Leukemia Day approaches on 4 September, experts from global health system Cleveland Clinic are working on new immunotherapy treatments for blood cancers such as leukemia, expanding treatment options and bringing hope to patients for whom other treatments have failed.

Leukemia is a group of cancers characterized by rapid, uncontrolled growth of abnormal blood cells called leukemia cells. It can occur in children and adults and according to the World Cancer Research Fund International, it is the 13th most prevalent cancer globally.

According to Jan Joseph Melenhorst, PhD, a translational immunologist who is Director of Cleveland Clinics recently established Cell Therapy and Immuno-Engineering Program, chimeric antigen receptor (CAR) T-cell therapy is profoundly changing the treatment landscape, bringing the promise of durable remission for many patients with blood cancers such as leukemia.

Dr-Jan Joseph-Melenhorst

CAR T-cell therapy is a specialized treatment using a patients own T-cells a type of white blood cell that forms part of the immune system.

The T-cells are extracted and genetically modified so that they can recognize and destroy cancer cells, before being multiplied and infused back into the bloodstream, explains Dr. Melenhorst, who is also Vice Chair of the Center for Immunotherapy and Precision Immuno-Oncology at Cleveland Clinic.

He added that while CAR T-cell therapy has brought new hope for many patients, there are currently several challenges to overcome in administering the treatment.

In particular, it may be more expensive than other therapies. Additionally, it has potentially serious side effects.

This limits access to treatment as the therapy needs to be administered on an inpatient basis at a specialized facility where the side effects can be managed. We are working with Clevelands Case Western Reserve University and other parties and collaborating with various manufacturers to address issues such as accessibility, safety, and costs, he says.

Dr. Melenhorst and his team are also aiming to improve the efficacy of existing CAR T-cell therapies while developing new versions for a wide range of blood and other cancers.

Their colleague, Craig Sauter, MD, is a hematologist and Director of Blood and Marrow Transplant at the Cleveland Clinic who has been using CAR T-cell therapy to successfully treat patients with leukemia and other blood cancers where other treatments have failed.

The standard of care in initial therapy for many acute leukemia patients remains cytotoxic chemotherapy, with the aim of achieving remission. In cases where first-line chemotherapy or other treatments have failed, however, CAR T-cells provide another potential treatment option for patients with B-cell acute lymphoblastic leukemia. In the FDA-approved indications, it has proved much more effective than the previous standard, which was a further round of chemotherapy, he says.

The U.S. Food and Drug Administration (FDA) has approved commercial CAR T-cell products for several types of blood cancers including acute lymphocytic leukemia (ALL) in patients who have been resistant to other treatments or whose cancer has returned after a period of remission. Clinical trials are also underway at institutions including Cleveland Clinic for CAR T-cells to treat acute myeloid leukemia (AML).

ALL and AML are two of the four main types of leukemia. The disease is classified as acute or chronic based on how rapidly the disease spreads in the body, and as myeloid or lymphocytic depending on whether the leukemia cells arise from myeloid cells, which develop in bone marrow, or from lymphoid cells, which are related to the immune system.

Dr. Craig Sauter

Explaining how the procedure works in practice,

Dr. Sauter says the first step is to extract the patients lymphocytes and insert an inactive virus that delivers new genetic instructions to the T-cells to start producing chimeric antigen receptors targeting proteins that live on the malignant cells.

Researchers take a small batch of these newly altered CAR T-cells and induce them to grow and multiply until there are enough to effectively target cancer cells.

The CAR T-cells are frozen and stored until the patient is ready to receive them. To prepare for the infusion, the patient receives a mild form of chemotherapy to prevent the immune system from rejecting the CAR T-cells.

According to Dr. Sauter, most people need to stay in the hospital for one to two weeks so their response to the treatment can be monitored and any side effects treated. The two most common side effects of CAR T-cell therapies are cytokine release syndrome (CRS) and neurological problems such as headaches, confusion, or difficulty speaking during the treatment period.

Dr. Sauter points out CAR T-cell therapy is in its early phase, but he is cautiously optimistic about its potential. In future, as a result of further research and carefully conducted studies, there may be an opportunity to identify high-risk groups who may benefit from having CAR T-cell therapy over chemotherapy in earlier lines of treatment, he says. The possibility of its application being extended to treat other forms of cancer is also very exciting.

Cleveland Clinic is a nonprofit multispecialty academic medical center that integrates clinical and hospital care with research and education. Located in Cleveland, Ohio, it was founded in 1921 by four renowned physicians with a vision of providing outstanding patient care based upon the principles of cooperation, compassion and innovation. Cleveland Clinic has pioneered many medical breakthroughs, including coronary artery bypass surgery and the first face transplant in the United States.

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Hope For Leukemia Patients, Where Other Treatments Have Failed - Longevity LIVE - Longevity LIVE

A cure must be a cure for all: why more HIV cure research in Africa is needed – aidsmap

Only one in eight HIV cure studies are being conducted in sub-Saharan Africa, but a few important studies have got off the ground in recent years. In South Africa, young women with very recent infection are testing broadly neutralising antibodies and vesatolimod, while gene therapy research is underway in Uganda. In the prevention field, early work on an mRNA vaccine is being done in Rwanda and South Africa.

The studies were discussed at a pre-conference meeting on cure held before the 24th International AIDS Conference (AIDS 2022), as well as at the conference itself.

Curing complex conditions like cancer and inherited diseases can involve equally complex, expensive drugs. This is especially problematic with HIV, an infectious disease targeting, in general, the poorest and most disadvantaged people in the world. Sub-Saharan Africa accounts for almost 70% of the global HIV burden and 59% of new global infections in 2021.

To eliminate a disease or a condition in an individual, or to fully restore health. A cure for HIV infection is one of the ultimate long-term goals of research today. It refers to a strategy or strategies that would eliminate HIV from a persons body, or permanently control the virus and render it unable to cause disease. A sterilising cure would completely eliminate the virus. A functional cure would suppress HIV viral load, keeping it below the level of detection without the use of ART. The virus would not be eliminated from the body but would be effectively controlled and prevented from causing any illness.

A type of experimental treatment in which foreign genetic material (DNA or RNA) is inserted into a person's cells to prevent or fight disease.

A neutralising antibody (NAb) is an antibody that fully defends its target cell from an antigen. A broadly neutralising antibody (bNAb) is a neutralising antibody that has this effect against a wide range of antigens. A number of broadly neutralising antibodies have been isolated from persons living with HIV. Some of them are being studied and, in some cases, used in clinical trials, to defend humans against HIV infection, treat HIV infection, and kill HIV-infected CD4+ T cells in latent reservoirs.

A unit of heredity, that determines a specific feature of the shape of a living organism. This genetic element is a sequence of DNA (or RNA, for viruses), located in a very specific place (locus) of a chromosome.

Dr Elizabeth Barr of the US National Institutes of Health told the pre-conference meeting that they had counted 162 HIV cure studies in 2019. Of these, three-quarters were being conducted in North America (76, almost all in the US) or in Europe (44, with France leading the way at 12). There were 23 in Asia and Oceania and eight in Latin America and the Caribbean, and only 19 (12%) in sub-Saharan Africa.

In addition, crucial groups, most notably women, were under-represented. Only one in six cure study participants were cisgender women and 1.4% were transgender people (mainly women), even though women respond to HIV differently from men. People over 50 only formed 7%. The survey did not cover babies and children taking part in studies.

But how to involve people in the global South in cure research now? We need to do this not only to ensure universal applicability if one is developed and to ensure fair inclusion, but also to counter myths and misunderstandings (for instance, that a particular therapy is a cure rather than a single step on a long road).

Perhaps the best example of paying attention to the needs and expectations of participants in cure research in lower-income settings is provided by the FRESH cohort in KwaZulu-Natal, South Africa (the FRESH acronym stands for Females Rising through Education, Support, and Health). Based on a similar cohort in Thailand, it recruits young women aged 18-23 in one of the highest-incidence areas for HIV in the world.

It tests its participants for HIV very frequently twice a week. The object is to treat them in the very earliest detectable states of HIV infection (Fiebig 1 and 2, before antibodies appear) to find out if this limits the progression of HIV later, and what the immunological signature of the young women is, to see if it can be reverse-engineered into a vaccine or cure for others.

Dr Krista Dong of FRESH told the meeting that so far it has recruited 2916 young women of whom 90 have tested HIV positive. The median number of days, it is estimated, between infection and the first positive test is only four an extraordinary achievement in itself. All 90 are on antiretroviral therapy (ART).

Of these, 29 have so far consented to leukapheresis, the process of extracting lymphocytes so their immune function can be tested.

But FRESH is a lot more than just a test-early-and-treat project. It also offers an empowerment and life-skills curriculum for its participants with computer training, literacy and numeracy classes, and workshops. The women are tested for HIV twice a week over a nine-month period when they come to attend the empowerment classes.

It is partly the mark of the painstaking consideration that went into FRESH that clinical trials of potential prevention and cure therapies only started in 2021 even though the cohort started recruiting in 2012. (It is also due to a year under COVID lockdown during which tests were suspended, floods cut the road to the research clinic, and political unrest led to the wrecking of the clinic which re-established itself the next day in the car park of a local hospital.)

The first study was a prevention one, of using a Lactobacillus supplement to treat bacterial vaginosis and see if it helped prevent infection.

But the second study is a phase 2a acceptability and tolerability cure study: in partnership with Gilead Sciences, 25 participants are being given two broadly neutralising antibodies, VRC07-523 and CAP256V, in combination with Gileads TLR-7 agonist, vesatolimod. ART will be stopped for up to 43 weeks (or longer if viral suppression is maintained). The first participant received her first ART plus antibody infusions on 13 July this year.

Innovative HIV vaccine studies are also underway in sub-Saharan Africa. Dr Kundai Chinyenze of the International AIDS Vaccine Initiative (IAVI) told AIDS 2020 that phase I clinical trials to evaluate the safety and immune response of an mRNA HIV vaccine antigen (mRNA-1644) have started in Rwanda and South Africa.

Led by a team of African scientists, IAVI G003 has enrolled 18 healthy HIV-negative adult volunteers to test whether vaccine injection with eOD-GT8 60mer, delivered via Modernas mRNA platform, can induce similar immune responses in African populations as were seen in IAVI G001.

Results from IAVI G001 (in US adults) demonstrated that vaccination with the HIV immunogen eOD-GT8 60mer safely targeted nave B cells with specific properties in 97% of recipients. This process is known as germline targeting. Once stimulated, this specific class of B cells can generate broadly neutralising antibodies (bnAbs). The induction of bnAbs is widely considered a goal of an efficacious HIV vaccine, and this B-cell activation is the first step in that process.

All participants of the IAVI G003 will receive two doses of eOD-GT8 60mer mRNA, and their immune responses will be examined to evaluate whether the targeted responses have been achieved. Novel sampling techniques being used in this trial include ultrasound-guided fine needle aspiration, where two weeks after each immunisation, a thin needle will be used to access the lymph nodes of participants to monitor in real-time B-cell responses in germinal centres. Another technique is leukapheresis, where eight weeks after the second dose, white blood cells will be extracted from participants to allow deep characterisation of the B-cell responses.

From Uganda, Dr Cissy Kityo told the conference that the Joint Clinical Research Centre in Kampala has teamed up with Fred Hutchinson Center in Seattle to develop a gene therapy treatment for HIV that will be ready for testing in Uganda by 2024. This will be the first gene therapy trial in Africa. The licensed product which will be evaluated in Uganda is the Anti-HIV duoCAR-T cell therapy; it modifies T cells such that they may be able to directly control HIV in the absence of therapy. This product is currently being evaluated in Phase 1 clinical trials at the University of California, San Francisco.

Uganda is also currently developing genetic engineering policy and the regulatory approval pathway. One Ugandan scientist is currently being trained to manufacture gene therapy products at the Fred Hutchinson Center.

But what do people living in Africa want from an HIV cure? What will it need to cost? What can be practically given in non-urban clinics? Are interventions like gene therapy acceptable? These are the questions Professor Sharon Lewin, the new president of the International AIDS Society, wants researchers to consider when undertaking HIV cure trials in Africa.

She also pointed out that scientific issues like HIV subtype, genetics unique to the dominant African population, and common prevalent co-morbidities must be considered by HIV cure research scientists in Africa.

While strengthening HIV cure research in Africa is a top priority because of the high burden and distinct characteristics of HIV in the region, a cure which is scalable, affordable and available will only be achieved if researchers engage and work with affected communities, local scientists, and local HIV care advocates.

Read more here:
A cure must be a cure for all: why more HIV cure research in Africa is needed - aidsmap

Why We Chose Oral Placement Therapy to Address AADC Deficiency… – AADC News

When I dont know how to do something, Im the type of person to go figure it out. YouTube has taught me skills ranging from fixing a leaky faucet to trail running. My wife and I applied the same method when it came to speech therapy training.

Speech seemed like a distant goal for our daughter, Rylae-Ann, who has aromatic l-amino acid decarboxylase (AADC) deficiency. This is because AADC deficiency causes more severe symptoms that take precedence. However, we tried our best to work on speech when we had time. Thankfully, my wife is a special education teacher and has some training, but even she was at a loss in terms of developing a treatment plan for our daughters unique case.

When Rylae-Ann was diagnosed at 8 months old, we knew she would have speech problems. We wanted to meet this challenge head-on and begin early intervention. We sought outside assistance at speech therapy clinics, but the doctor told us that Rylae-Ann was still developing, and therefore too young to receive treatment.

It seemed like we were being told to wait until there was a more significant problem before proceeding. This method did not sit well with us, and there was no way we would sit idly by. We found many helpful videos on YouTube, but one led us to a specific form of speech therapy called oral placement therapy, or OPT.

There was no speech intervention guide for AADC deficiency, so we also connected with other rare disease support groups and researched programs that supported cases involving similar symptoms and challenges as our daughter. OPT was mentioned within the groups and seemed like a perfect match.

OPT is a speech therapy approach that combines visual, auditory, and tactile stimulation to improve speech and feeding skills. It focuses on all the muscles involved in speech, not just the mouth.

What caught our attention about this program was that it looks at early intervention and offers treatment to young children before they have speech problems. This is exactly what we were looking for.

We visited the website of TalkTools, an organization that provides resources and training for oral motor placement therapy, and appreciated all of the helpful information, much of it free of charge. The site also links to a directory of licensed OPT speech therapists around the world. It encouraged us to take things a step further by enrolling in a program and completing their training courses.

We wanted to learn and apply the strategies directly. The courses arent free, and we also needed to pay for equipment sold separately. But doing so empowered us to confidently provide treatment for Rylae-Ann at home. This, combined with our other therapies, led to noticeable improvements by our daughter.

While completing oral placement therapy training, Richard and his wife, Judy, began using the assigned tools with their daughter, Rylae-Ann. (Courtesy of Richard E. Poulin III)

TalkTools was founded by Sara Rosenfeld-Johnson, an internationally recognized speech and language pathologist. She also was the teacher for our course.

Rosenfeld-Johnson began our first class by sharing a story about a baby with Down syndrome. The baby was experiencing feeding difficulties but was not receiving speech therapy intervention due to young age. Rosenfeld-Johnson was surprised about this and felt action should be happening from birth, especially when it was known that the child would have speech difficulties later. This story felt almost identical to what my wife and I had experienced.

In a 1997 article in Advance Magazine,titledThe Oral-Motor Myths of Down Syndrome, Rosenfeld-Johnson explained how the facial features of a child with Down syndrome are not a symptom of the disease but a manifestation of not taking early action, and therefore allowing the body to develop the characteristic myths of Down syndrome. The stereotypical characteristics were not apparent in the cases she had worked with, because OPT uses an early intervention and prevention approach.

The goal of OPT is to prevent the symptoms from occurring in the first year of life. So, although children cannot yet speak, speech therapy is used. OPT relies on understanding the mechanics and physiology of the mouth to achieve certain goals. These goals may be initially related to sucking and feeding, but the same muscles are used for speech.

We began when Rylae-Ann was 8 months old, but it should have started when she was born. By beginning sooner, mothers of newborns with AADC deficiency can experience successful breastfeeding, which leads to improved feeding for toddlers. They wouldnt have to worry about weight gain or providing their child with a feeding tube. Eventually, speech has a greater likelihood of happening.

Rylae-Ann improved her feeding due to oral placement therapy, which eventually led to speech. (Courtesy of Richard E. Poulin III)

Another reason we believed OPT was the best match for our daughter is because it aids several special needs cases, including Down syndrome, cerebral palsy, autism spectrum disorder, attention-deficit/hyperactivity disorder, and those with motor-sensory impairments. This list featured similar challenges faced by our daughter. In addition, the multisensory approach aligned with our experience with early education and learning support curriculums.

Special needs children benefit from tactile and proprioceptive components, which are very different from traditional speech therapy. In special education, educators use a similar technique of combining multiple senses and learning styles to reach individual children. So while we were working on speech, we also improved her senses, overall articular awareness, placement, stability, and muscle memory.

The results for us began slowly, but we did not rush the sequence. Rylae-Anns first milestone was allowing us to use the tools in and around her mouth to help her build muscle awareness. Her sensory processing issues did not make this an easy task, but she did it.

Then, she quickly learned to drink from a straw and blow bubbles. This was a huge leap, and the progress continued.

After she had gene therapy, the progress was even quicker, which I believe was due to early intervention with OPT speech therapy.

Success with this method strengthened our resolve to support our daughter and other parents who want to do the same with their children. Oral placement therapy is one component of speech and language. To maximize results, it should be led by a licensed therapist and not done in isolation. All paramedical therapies work together to allow a child to make the most significant progress possible. As always, consult your healthcare team before making any treatment or therapy decisions.

Thanks to Rylae-Anns dedication and hard work during speech therapy, she is able to eat independently while talking with her friends at lunch. (Courtesy of Richard E. Poulin III)

Note:AADC Newsis strictly a news and information website about the disease. It does not provide medical advice,diagnosis, ortreatment. This content is not intended to be a substitute for professional medical advice,diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those ofAADC Newsor its parent company, BioNews, and are intended to spark discussion about issues pertaining toaromatic l-amino acid decarboxylase deficiency.

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Why We Chose Oral Placement Therapy to Address AADC Deficiency... - AADC News

Enzyme Replacement Therapy Market Worth $15.18 Billion by 2028 at 6.8% CAGR Led by Hospitals Segment (49% Market Share in 2021) Global Analysis by The…

The Insight Partners

The global enzyme replacement therapy market size is expected to grow from $9.67 Billion in 2021 to $15.18 Billion by 2028; it is estimated to grow at a CAGR of 6.8% from 2022 to 2028.

New York, July 22, 2022 (GLOBE NEWSWIRE) -- The Insight Partners published latest research study on Enzyme Replacement Therapy Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Enzyme Type (Alglucosidase Alfa, Agalsidase Beta, Imiglucerase, Idursulfase, Galsulfase, Velaglucerase Alfa, and Other Enzymes), Therapeutic Conditions (Gaucher's Disease, Fabry's Disease, MPS, Pompe's Disease, SCID, and Other Therapeutic Conditions), Route of Administration (Parenteral and Oral), and End User (Hospitals, Infusion Centers, and Others), the global enzyme replacement therapy market research report provides deep dive regional and country level analysis of 18+ countries across 5 key regions, 30+ key company profiles, detailed SWOT and PEST analysis, in-depth market segmentation and industry landscape to provide latest market initiatives and competitive landscape, impact of COVID-19 pandemic on ecosystem.

Download PDF Brochure of Enzyme Replacement Therapy Market Size - COVID-19 Impact and Global Analysis with Strategic Developments at: https://www.theinsightpartners.com/sample/TIPRE00008474/

Enzyme Replacement Therapy Market Report Scope & Strategic Insights:

Report Coverage

Details

Market Size Value in

US$ 9.67 Billion in 2021

Market Size Value by

US$ 15.18 Billion by 2028

Growth rate

CAGR of 6.8% from 2022 to 2028

Forecast Period

2022-2028

Base Year

2021

No. of Pages

226

No. Tables

111

No. of Charts & Figures

86

Historical data available

Yes

Segments covered

Enzyme Type, Therapeutic Conditions, Route of Administration, and End-User

Regional scope

North America; Europe; Asia Pacific; Latin America; MEA

Country scope

US, UK, Canada, Germany, France, Italy, Australia, Russia, China, Japan, South Korea, Saudi Arabia, Brazil, Argentina

Report coverage

Revenue forecast, company ranking, competitive landscape, growth factors, and trends

Global Enzyme Replacement Therapy Market: Competitive Landscape and Key Developments Takeda Pharmaceutical Company Limited; Sanofi S.A.; AbbVie Inc.; BioMarin Pharmaceutical Inc.; Amicus Therapeutics; Alexion Pharmaceuticals, Inc. (AstraZeneca); Janssen Pharmaceuticals (Johnson & Johnson Services, Inc.); Recordati S.p.A.; Pfizer Inc.; and CHIESI Farmaceutici S.p.A are among the key companies operating in the global enzyme replacement therapy market. Leading players focus on expanding and diversifying their market presence and clientele, thereby tapping prevailing business opportunities.

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In September 2021, Takeda Pharmaceutical Company Limited signed a collaboration and partnership arrangement with JCR Pharmaceuticals Co., Ltd. to commercialize JR-141 outside of the US to treat Hunter syndrome.

In June 2019, BioMarin Pharmaceutical Inc. announced that Vimizim (elosulfase alfa) has been approved by the National Medical Products Administration (NMPA) for the treatment of patients suffering from mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.

Enzyme Replacement Therapy Market: Key Insights The enzyme replacement therapy market growth is attributed to the rising prevalence of lysosomal storage diseases (LSDs) and rapid regulatory approval with other marketing benefits for a drug with orphan drug designation. However, cutthroat competition among market players hinders overall market growth.

North America dominates the global enzyme replacement therapy market. The US holds the largest share of the market in the region due to the rising prevalence of LSDs and increasing regulatory approval of enzyme replacement therapy products. Intravenous (IV) infusions are used in enzyme replacement therapy to treat the underlying enzyme shortage that underlies rare diseases, including Gaucher, Fabry's, and Hunter's syndrome. Therefore, rising cases of these rare diseases propel the need for developing novel treatments.

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According to the National Organization for Rare Disorders (NORD), in 2021, males and females are equally affected by various types of Gaucher disease. Around 6,000 people in the US are suffering from Gaucher disease. Gaucher disease is the most prevalent genetic illness in the people of Ashkenazic Jewish origin. Its prevalence potentially hit 1 in 450 births. According to statistics released by the National Institute of Neurological Disorders and Stroke in August 2021, there are an estimated 32,950 instances of Pompe disease in the US, affecting one in 40,000 persons. The National Fabry Foundation estimates that there were around 7,713 Fabry patients in the US as of May 2020. Thus, the vast majority of rare diseases remain beyond possibilities of treatment and with continued research, it would be able to offer therapies to the affected patients. Therefore, this rise in the prevalence of rare diseases would drive the growth of the regional market in the coming years.

Rising Prevalence of LSDs and Rapid Regulatory Approval Positively Influence Overall Market Growth: Enzyme replacement therapy is performed to treat congenital enzyme deficiencies by using an enzyme or protein having enzymatic activity. The therapy is generally applied in rare and lysosomal storage diseases, including Pompe disease, Tay-Sachs disease, Fabry disease, Gaucher disease, and Hurler syndrome. Researchers and players are actively involved in the enzyme replacement therapy industry and are constantly focusing on advancing technologies that can offer better alternatives to conventional techniques.

In August 2021, the US Food and Drug Administration (FDA) approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients aged one year and above suffering from late-onset Pompe disease. Nexviazyme is an ERT designed to specifically target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of enzyme replacement therapy in Pompe disease. Nexviazyme has been shown in clinical trials to provide patients with improvements in respiratory function and walking distance.

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In July 2019, Takeda Pharmaceutical Company launched an enzyme replacement therapy portfolio for LSD in India, expanding its range of rare disease therapies in the country. Under lysosomal storage disorders, India has been recording the prevalence of hunter syndrome, gaucher disease, and fabry disease. To address these, Takeda brought idursulfase for hunter syndrome, velaglucerase alpha for gaucher disease, and agalsidase alfa for fabry disease to the market. Such advancements and the rise in investments are propelling the global enzyme replacement therapy market growth significantly.

Global Enzyme Replacement Therapy Market: Segmental Overview Based on enzyme type, the global enzyme replacement therapy market is segmented into alglucosidase alfa, velaglucerase alfa, agalsidase beta, imiglucerase, galsulfase, idursulfase, and other enzymes. The others enzyme segment held the largest market share in 2021 and is anticipated to register the highest CAGR during 20222028. Based on therapeutic conditions, the market is segmented into Gaucher's disease, Fabry's disease, Pompe's disease, SCID, MPS, and other therapeutic conditions. The Gauchers disease segment held the largest market share in 2021 and is expected to register the highest CAGR during 20222028. Based on the route of administration, the global enzyme replacement therapy market is bifurcated into oral and parenteral. The parenteral segment held a larger market share in 2021 and is anticipated to register a higher CAGR during 20222028. Based on end users, the global enzyme replacement therapy market is segmented into hospitals, infusion centers, and others. The hospitals segment held the largest market share in 2021. However, the infusion centers segment is expected to register the highest CAGR during 20222028.

SARS-CoV-2 infection can cause a high risk for patients suffering from inborn errors of metabolism (IEM), since they often suffer from a multisystem disease, including respiratory and cardiovascular system involvement. Due to multi-systemic involvement, including crucial organs, people affected by rare metabolic abnormalities were at severe risk during the pandemic because of lysosomal dysfunction and inflammatory cascades activation. The major reason involved was the fear of getting infected at the hospital or during traveling to the hospital by using public transportation.

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Many patients missed their infusions in March 2020, due to the inadequacy of hospital services dealing with the burden of COVID-19 patients. Patients claimed to feel anxious during hospital admissions or regarding drug supply, and studies revealed that they had depression and an increased amount of anxiety. Thus, the facts mentioned above indicate the overall negative impact of the COVID-19 pandemic on the global enzyme replacement therapy market.

Browse Adjoining Reports: Pompe Disease Treatment Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, Non-Classic infantile-onset Pompe Disease); Therapy (Enzyme Replacement Therapy, Gene Therapy, Others) and Geography

Pompe Disease Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Therapy Type (Enzyme Replacement Therapy, Substrate Reduction Therapy); Molecule Type (Biologics, Small Molecules); Route of Administration (Oral, Parenteral); Dosage Forms (Solid, Liquid) and Geography

Gaucher Disease Treatment Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Disease Type (Neuropathic, Perinatal Lethal, Slow Neurogenic Decay); Treatment Approach (Enzyme Replacement Therapy, Substrate Reduction Therapy, Surgery); End Users (Hospitals, Ambulatory Surgical Centers, Clinical research institutes) and Geography

Gaucher Disease Drugs Market Forecast to 2028 - Covid-19 Impact and Global Analysis - By Disease Type ( Type I, Type II, Type III ); Treatment Type ( Enzyme Replacement Therapy (ERT), Substrate Reduction Treatment (SBT) ) and Geography

Fabry Disease Treatment Market Forecast to 2028 - Covid-19 Impact and Global Analysis - by Drug Type (Enzyme Replacement Therapy (ERT), Chaperone Treatment, Substrate Reduction Therapy (SRT), Others)

Mucopolysaccharidosis Treatment Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Treatment (Enzyme Replacement Therapy, Stem Cell Therapy); MPS Type (MPS-I, MPS-II, MPS-IV, MPS-VI, MPS-VII, Others); End User (Hospitals, Specialty Clinics, Others) and Geography

Metabolic Disorders Therapeutics Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type (Gene Therapy, Drug Therapy, Enzyme Replacement Therapy, Cellular Transplantation, Small Molecule Based Therapy, Substrate Reduction Therapy); Indication (Lysosomal Storage Diseases, Diabetes, Obesity, Inherited Metabolic Disorders, Hypercholesterolemia); Route of Administration (Oral, Parenteral, Others), and Geography

Metabolism Drugs Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Therapy Type (Enzyme Replacement Therapy, Substrate Reduction Therapy, Small Molecule Drugs, Others); Application (Glycogen Metabolism Disease, Lipid Metabolism Disease, Amino Acid Metabolism Disease, Others); Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies) and Geography

Lysosomal Acid Lipase Deficiency Treatment Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Disease Indication (Cholesteryl Ester Storage Disease (CESD), and Wolman Disease (WD)); Treatment Type (Liver Transplant, Enzyme Replacement Therapy, Lipid-Modifying Agents, and Others) and Geography

About Us: The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We specialize in industries such as Semiconductor and Electronics, Aerospace and Defense, Automotive and Transportation, Biotechnology, Healthcare IT, Manufacturing and Construction, Medical Device, Technology, Media and Telecommunications, Chemicals and Materials.

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Contact Person:Sameer Joshi E-mail: sales@theinsightpartners.com Phone:+1-646-491-9876 Press Release: https://www.theinsightpartners.com/pr/enzyme-replacement-therapy-market

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Enzyme Replacement Therapy Market Worth $15.18 Billion by 2028 at 6.8% CAGR Led by Hospitals Segment (49% Market Share in 2021) Global Analysis by The...

The Importance of Patient Centricity in Clinical Research and Cell / Gene Therapy Development – Contract Pharma

Recently, a patient centricity concept has spread across all corners of the healthcare community with a straightforward goal: to improve and save patients lives through innovative and effective drug therapies adapted to patients' individual needs. However, for this concept to truly come to life, it is necessary to count on temperature-controlled supply chain logisticslike CRYOPDP to turn what could be a complex operation into a highly efficient process. This is extremely vital, because a patients life could literally be on the line with every shipment. Patient centricity in the clinical trial community involves meeting the patients where they are. The clinical trial industry is globalising and demands international healthcare logistics partners that can serve its growing and continuously evolving needs. For decades, temperature-controlled logistics experts have operated behind the scenes with patients barely aware of the hard work that went into their medicines journey. Thanks to the Covid-19 vaccine distributions impressive performance and its universal effect on people's health across the globe, it has become apparent to the world just how critical and important a logistics providers mission is for both patients and the industry. The endless pandemic lockdowns meant that patients could not travel to hospital sites for clinical trials due to travel and access restrictions. This led to a seismic shift where sponsors quickly moved to a direct-to-patient model to try to continue trials and keep patient treatments on schedule. As part of our response to this unique situation, CRYOPDP as a specialist in temperature-controlled logistics, with the mission to improve people's healthcare options, had to go further and dive deeper to better understand customers perceptions of the impact of the pandemic and work with them to provide those options. With the patient always at the centre of its business and considering the huge development of decentralised clinical trials, the development and implementation of a new service such as Direct-to-Patientseems to be the most natural evolution for CRYOPDP. We have been offering this turnkey solution completely adapted to patients needs, with the same efficiency and total peace of mind, to the benefit of many customers. This model makes life a lot easier for patients and their families, as they dont have to make multiple trips to a clinic or hospital that is potentially a great distance from their home. It is also beneficial for patients who may be too ill to travel, as well as saves patients time and money. Because of these benefits, the direct-to-patient model has increased clinical trial recruitment by up to 60 percent and helped to maintain patient retention by over 95 percent. Drug developers can also gain access to a larger patient population by onboarding those who are not located near participating hospitals or clinics. Patient centricity becomes more evident with the new generation of cell and gene therapies. From day one, CRYOPDP temperature-controlled logistics solutions have always been essential to improve and save patients lives. But, when speaking about cell and gene therapies in particular, this gains an even more significant meaning. The increase in personalised medicine, advanced therapies, and improved access to healthcare in the developing world are influencing future supply chain solutions development. Cell and gene therapies are enabling the healthcare community to shift the arrow and think about patients in a whole new way. Cell and gene therapies demand rigorous and precise temperature control to ensure that the therapies maintain their viability. And maintaining temperature control calls for flawless implementation and execution. From designing the best transportation route to selecting the correct packaging, every detail is critical to keep product integrity high under all conditions, and for this to happen, the healthcare community can count on CRYOPDP specialists who can meticulously handle the entire supply chain process. We've been supporting the life sciences and healthcare communities and focused on improving patient centricity with innovative temperature-controlled logistics solutions because thinking about the patient and the outcome of our work is what moves and inspires us to be better every day.

In all the geographies of the world that we cover, around 150 countries, we produce an operational performance of 99.96%. And to deliver such operational performance, we count on our employees, the specialists around the globe, to follow our quality standards and protocols in detail, so we can deliver a quality service.

When dealing with patients lives, there is no room for errors. Its all about quality of service making sure that lifesaving samples are distributed on time, within the correct specifications and at the right temperature to ensure the patient's health is never compromised. This commitment has helped CRYOPDP to win numerous industry awards, including Best Clinical Trial Logistics Provider in APAC at the Bioprocessing Excellence Awards 2021 and Most Advanced Healthcare Solution Providers from Europe 2021 by Healthcare Insights Magazine. Our achievements are being recognised across the industry, and as we continue to improve our services for healthcare communities, it will be the end patient that benefits the most.

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The Importance of Patient Centricity in Clinical Research and Cell / Gene Therapy Development - Contract Pharma

Global Gene Therapy Market to Garner a Revenue of About USD 6 Billion by 2031 by Growing with a CAGR of ~34% During 2022-2031 – GlobeNewswire

New York, July 13, 2022 (GLOBE NEWSWIRE) -- According to the World Health Organization (WHO), around 10 million deaths, or nearly 1 in 6 deaths, were caused by cancer in 2020, making it the top cause of death globally. Breast, lung, colon, rectum, and prostate cancers are the most prevalent types of cancer. If found early and appropriately treated, many tumors (30% to 50%) are curable. According to the American Cancer Society (ACS), 1,918,030 new cancer cases and 609,360 cancer deaths are expected in 2022, with lung cancer as the primary cause of death accounting for about 350 of those fatalities daily in the United States.

In recent research titled Global Gene Therapy Market, Kenneth Research provided a brief overview of market elements including growth drivers, restraint factors, current market trends, and potential for future growth. The influence of COVID-19 and its effects on end-users are both thoroughly examined in the market research report, which covers the forecast period, i.e., 2022-2031. In addition, the research study examines the product portfolios and market expansion plans of the principal competitors. In 2020, according to the World Cancer Research Fund (WCRF), there were 18 million new cases of cancer worldwide. 9.3 million of these instances involved men, while 8.8 million involved women. The growth of the global gene therapy market can be attributed on account of the rising prevalence of cancer cases. Also, the adoption of gene therapies for the treatment of cancer is predicted to grow the market further. For instance, at the University of Pennsylvania, the first trial for testing a CRISPR-created cancer medicine was launched in the United States in 2019.CRISPR is a gene-editing-tool, that can modify any DNA segment within the 3 billion letters of the human genome. The global gene therapy market is expected to gather around USD 6 billion in revenue by 2031 and grow with a CAGR of ~34% over the forecast period. Get A Sample Copy of This Report @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented on the basis of region into North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. On the back of rapid rising cancer incidence rates, and the availability of high disposable income, the market in North America is predicted to experience significant expansion over the course of the forecast period. For instance, the Cancer Facts & Figures 2021 by the American Cancer Society, the study estimates that 1.9 million new instances of cancer were diagnosed and 608,570 cancer deaths in the United States in 2021. Also, an increase in the demand for gene-therapy-related R&D activities further helps the growth of the market. According to the World Bank Data, the domestic general government healthcare expenditure in the U.S. was 5,552.60 IN 2019 whereas in Canada the domestic general health care expenditure was 3,873.70 in 2019. Thus, a rise in government health care support is expected to expand the gene therapy-related R&D activities and further aid on to improve the market in the region. On the other hand, the global gene therapy market in the Asia Pacific region is anticipated to experience the greatest CAGR throughout the forecast period owing to the growing population in the region and increased approval and availability of gene therapy products. According to the World Bank data, the total population of China was 1.41 billion in 2020 whereas, India had 1.38 billion people in 2020. As the population grows, the likelihood of contracting a disease increases. Additionally, it is anticipated that increased government efforts to upgrade the health care infrastructure and rising healthcare costs in that region are expected to expand the industry. Also, the health care expenditure in Japan in 2019 was 10.74% whereas, in China, the GDP was 5.35%. In addition to that, the domestic general government health expenditure per capita for Japan was 3,846.54 in 2019 and China was 492.72 in 2019. Thus, growing health care expenditure and government support in health care expansion are further expected for the growth of the market in the region.

Browse to access In-depth research report on Gene Therapy Market with detailed charts and figures: https://www.kennethresearch.com/report-details/gene-therapy-market/10070542

The study further incorporates Y-O-Y Growth, demand & supply and forecasts future opportunities in North America (U.S., Canada), Europe (U.K., Germany, France, Italy, Spain, Hungary, Belgium, Netherlands & Luxembourg, NORDIC[Finland, Sweden, Norway, Denmark], Poland, Turkey, Russia, Rest of Europe), Latin America (Brazil, Mexico, Argentina, Rest of Latin America), Asia Pacific(China, India, Japan, South Korea, Indonesia, Singapore, Malaysia, Australia, New Zealand, Rest of Asia Pacific), Middle East and Africa(Israel, GCC[Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of the Middle East and Africa). The global gene therapy market is segmented by indication into cancer, metabolic disorders, eye disorders, cardiovascular diseases, and others. Among that the cancer segment is predicted to hold the largest share over the forecast period. On account of the growing widespread presence of cancer cases, the growth of the market can be accredited. The estimated number of new cases of cancer patients in India was around 11,57,294 cases which had risen to 13,24,413 total cases in 2020. In addition to that, the total number of cancer patients was 1,708,921 in 2018 in the U.S., according to the Centers for Disease Control and Prevention (CDC) which got increased to an estimated rate of 1.8 million new cases in 2020. The statistical studies exhibit an increasingly widespread of the disease worldwide which is expected to drive the growth of the segment. Gene therapies are used to treat a variety of malignancies, including those of the brain, lung, breast, pancreatic, liver, prostate, bladder, head & neck, skin, and ovary. For instance, according to the World Cancer Research Fund (WCRF), the most common cancers around the world were breast and lung cancers, accounting to 12.5% and 12.2% respectively of all new cases that were expected to be diagnosed in 2020. Also, there were 1.9 million new instances of colorectal cancer, accounting for 10.7% of all cancer cases in 2020.

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The global gene therapy market is segmented by end-user into pharma & biotech, and academia. Numerous ongoing researches and studies have been conducted in the pharma and biotech sector which is anticipated to account for the growth of the segment. For instance, based on a study by PhRMA, there were 289 gene therapies done in clinical development by biopharmaceutical companies in 2018 which had increased to 362 gene therapies in 2020. Also, 6 diseases were already being treated using gene therapy, whereas 362 cell and gene therapies were in the development stage in 2020. In addition to that, 9 cell or gene therapy products have been approved by U.S. Food and Drug Administration (FDA) as of February 2020; they are used to treat cancer, eye conditions, and uncommon inherited diseases.

The global gene therapy market is also segmented on the basis of technology and application.

Global Gene Therapy Market, Segmentation by Technology:

Global Gene Therapy Market, Segmentation by Application:

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Some of the well-known leaders in the global gene therapy market that are included in our report are Kineta, Inc., Orchard Therapeutics plc, SIBIONO, Questex, CRISPR Therapeutics, Editas Medicine, and others.

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Global Gene Therapy Market to Garner a Revenue of About USD 6 Billion by 2031 by Growing with a CAGR of ~34% During 2022-2031 - GlobeNewswire

Terumo Blood and Cell Technologies and GenCure collaborate to advance cell and gene therapy manufacturing solutions – Marketscreener.com

Lakewood, Colorado, US - Terumo Blood and Cell Technologies, a medical technology company specializing in a portfolio of products, software and services for blood component collection, therapeutic apheresis and cellular technologies, today announces it has signed a new collaborative agreement with GenCure, a subsidiary of BioBridge Global, to extend and unify cell and gene therapy manufacturing solutions.

The latest agreement between Terumo Blood and Cell Technologies (Terumo) and BioBridge Global follows a series of long-running activities between the organizations for more than 20 years, including with a number of BioBridge Global's subsidiaries. It is anticipated that this latest agreement will extend into a broader collaboration with the full array of BioBridge Global's subsidiaries. Both parties have signed the new agreement to further combine technologies and expertise to cater to the demand across the cell and gene therapy (CGT) industry for smart systems that are automated and closed. These smart-systems allow for real-time response and refinement to the living therapies being produced inside the systems.

This collaboration will initially utilize expertise from Terumo and GenCure to establish a reference manufacturing center for BioBridge Global. This includes both organizations jointly developing core process and manufacturing operations. The reference site will enable GenCure to deliver a full solution for cell and gene therapy developers. It will be based at GenCure's process development and cGMP manufacturing facility at the VelocityTX campus in San Antonio, Texas, US.

Terumo will contribute a variety of products from its portfolio to allow in-plant optimization of processes and user feedback, along with investment in new products and process utilization. GenCure will leverage its highly developed expertise in Terumo Blood and Cell Technologies' cell collection, cell and gene therapy manufacturing and fill-finish equipment. This will significantly enhance its portfolio for developers requiring process optimization and manufacturing support.

The collaboration is scheduled to last three years and is in two phases. The first phase will enable Terumo to select from its portfolio of automated and closed-cell and gene therapy manufacturing devices and share the range of services offered at GenCure. This combination will help solve the critical industry gaps in manufacturing capacity and operational expertise. The second phase will allow GenCure and other BioBridge Global subsidiaries to use Terumo's data management solutions and value-added services to seamlessly transition between clinical and manufacturing sites by enabling chain-of-custody/identity and consistency of leukapheresis products.

Terumo selected GenCure as a key collaborator that has access under the BioBridge Global umbrella to all of the services needed for cell and gene therapy manufacturing that originates from blood products. These include donor recruitment, cell collections, cell selection and manufacturing facilities and biological testing services. The collaboration allows Terumo to link blood and cell collections with cell and gene therapy manufacturing. There is currently a critical lack of skills and knowledge bridging these areas.

"Despite the rapid evolution of the cell and gene therapy sector, critical challenges such as management between collection, manufacturing sites and logistics still remain unresolved. For over 20 years, Terumo has been working with BioBridge to provide safe, high-quality blood products," said Delara Motlagh, General Manager, Cell Therapy Technologies, Terumo Blood and Cell Technologies. "We are now combining our product portfolios, expertise and management to provide a vital link between cell collection sites and cell and gene therapy manufacturing sites, while addressing capacity and ensuring GMP production. This will provide a route to deliver cutting-edge cell and gene therapies through pursuing new standards of collection and manufacturing, including quality, consistency and demonstrated scalability."

Martin Landon, Chief Executive Officer of BioBridge Global said: "Across the cell and gene therapy sector, collaborations are now proving vital to the rapid development of solutions to a number of key challenges. Our donor-to-patient solutions, which include cell collection, manufacturing and testing, require the best technologies and expertise available in the market. Working with an innovator like Terumo Blood and Cell Technologies will allow us to provide a leading-edge end-to-end solution for cell and gene therapy manufacturing that will be vital to deliver advanced therapies to patients."

ENDS

About Terumo Blood and Cell Technologies

Terumo Blood and Cell Technologies is a medical technology company. Our products, software and services enable customers to collect and prepare blood and cells to help treat challenging diseases and conditions. Our employees around the world believe in the potential of blood and cells to do even more for patients than they do today. This belief inspires our innovation and strengthens our collaboration with customers.

Terumo Blood and Cell Technologies' customers include blood centers, hospitals, therapeutic apheresis clinics, cell collection and processing organizations, researchers and private medical practices. Our customers are based in over 130 countries across the globe. We have more than 750 granted patents, with more than 150 additionally pending.

We have global headquarters in Lakewood, Colo., US., along with five regional headquarters, seven manufacturing sites and six innovation and development centers across the globe. Terumo Blood and Cell Technologies is a subsidiary of Terumo Corporation (TSE: 4543), a global leader in medical technology.

About GenCure

GenCure, a subsidiary of San Antonio-based nonprofit BioBridge Global, provides process development and optimization expertise and cGMP manufacturing services for cell and cell-based advanced therapies companies as the anchor tenant for the VelocityTX landmark biotech innovation corridor complex. Working with its sister subsidiaries, GenCure understands and provides support for donor-to-patient services with a foundation in the critical elements related to clinical practice standards, access to source materials, testing services, research and development, and clinical research support. Learn more at GenCure.org.

About BioBridge Global

BioBridge Global (BBG) is a San Antonio, Texas-based 501(c)(3) nonprofit advanced therapies enterprise that offers diverse services through its subsidiaries - South Texas Blood & Tissue, QualTex Laboratories, GenCure and The Blood and Tissue Center Foundation. BBG provides products and services, donor recruitment and sample collection for a wide range of blood and tissue types, processing and manufacturing support, and testing of both donor and finished products for clients in the United States and worldwide. BBG is committed to saving and enhancing lives through the healing power of human cells and tissue. It enables advances in the field of regenerative medicine by providing access to human cells and tissue, testing services and biomanufacturing and clinical trials support. Learn more at BioBridgeGlobal.org.

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Terumo Blood and Cell Technologies and GenCure collaborate to advance cell and gene therapy manufacturing solutions - Marketscreener.com