Category Archives: Induced Pluripotent Stem Cells

The Kyoto University team’s plan to transplant iPS cartilage into knee joints is OK – gotech daily

KYOTO A panel of experts from the Ministry of Health approved a clinical research program proposed by a team from the University of Kyoto on Friday for the transplantation of cartilage from induced pluripotent stem cells [iPS] into damaged knee joints.

Professor Noriyuki Tsumaki and other members of the team are planning to produce 2 to 3 millimeter diameter cartilage using iPS cells, which will be stored at the Universitys Center for iPS Cell Research and Application [CiRA].

The team plans to perform the first transplant this year. According to a clinical study by Asahi Kasei Corp., which supports the project, the technology should be put into practice in 2029.

Four people between the ages of 20 and 70 are transplanted with iPS cell cartilage for their damaged knee joints, with the damage range between 1 cm and 5 cm. The team does not plan to seek additional patients for the program.

Immunosuppressors are not used in the transplant because cartilage usually does not show an immune response.

The team will monitor the four patients for possible tumor development for a year after the operation. If the operations are successful, the transplanted material melts into the existing cartilage.

There are many patients who experience discomfort from cartilage damage, said Tsumaki at a press conference at Kyoto University Hospital on Friday. We will work hard to offer therapy methods.

The team will also try to apply the therapy to patients with osteoarthritis.

In 2014, Riken, a research institute affiliated with the Japanese government, transplanted retina cells made from iPS cells to treat an incurable eye disease in the worlds first transplant of iPS-derived cells.

Similar transplants were later performed by Kyoto University for Parkinsons and Osaka University for corneal diseases.

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The Kyoto University team's plan to transplant iPS cartilage into knee joints is OK - gotech daily

Get rid of the ache within the nerves discovered with the following pointers, learn – Sahiwal Tv

Many folks around the globe will be seen troubled by ache within the veins. And many occasions, even after an excessive amount of therapy, this ache just isnt relieved. But within the coming days you possibly can do away with neuralgia fully, that too with none unwanted effects. Researchers on the University of Sydney have used human stem cells for excessive ache aid in mice.

->Now, theyre shifting in direction of human trials.

Greg Nelly, senior researcher on the Charles Perkins Center, stated that at occasions, extreme strain on the nerves causes them to get broken. For instance, carpal tunnel syndrome is the median nerve within the arms ( median nerve ) Due to extreme strain. As you possibly can think about, nerve accidents can result in insufferable neuropathic ache. There can also be no efficient therapy to alleviate ache in most sufferers.

Therefore, Nelly and colleagues on the University of Sydney developed an efficient remedy. Researchers have been capable of create pain-relieving neurons utilizing human stem cells.

Nelly stated that this success implies that for some sufferers affected by nerve ache, we are able to carry out pain-relieving implants from our cells, which might cease the ache.

In the examine, researchers collected stem cells from grownup blood samples. Then, used human-induced pluripotent stem cells (iPSCs) from the bone marrow to create pain-relieving cells within the laboratory.

To check the efficacy of the therapy, the staff injected neurons that abolished spinal ache in mice affected by extreme neuropathic ache. It was revealed that this therapy supplied full aid from ache to the mice with none unwanted effects.

Co-senior creator Dr. Leslie Caron stated that which means that transplant remedy is more likely to be an efficient and long-lasting therapy for neuropathic ache.

After shut therapy in mice, the University of Sydney staff is shifting ahead for extra in depth research in pigs. Within the subsequent 5 years, theyll check people that suffer from power ache.

Researchers stated {that a} move check in people can be an enormous success. This might point out the event of latest non-opioid, non-addictive ache administration methods for sufferers.

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Get rid of the ache within the nerves discovered with the following pointers, learn - Sahiwal Tv

Astellas and Adaptimmune team up in CAR-T development – European Biotechnology

Adaptimmune Therapeutics plc and Japanese Astellas Pharma, Inc. have signed a discovery partnership to develop off-the-shelf allogeneic T cell-based cancer therapies from stem cells.

At J.P. Morgan conference, the British company announced that Astellas has agreed to co-develop and co-commercialize stem-cell derived allogeneic CAR-T and TCR T-cell therapies against up to three targets. In contrast to current autologous T cell therapies, allogenic T cell therapies might be manufactured in a central facility reducing production cost significantly compared to autologous cell production and logistics.

Under the agreement, Adaptimmune will identify and validate new targets for generating target-specific T-cell receptors (TCRs), chimeric antigen receptors (CARs), and HLA-independent TCRs that recognize surface epitopes independently from the HLA profile of the tumour cell. Astellas subsidiary Universal Cells, Inc will provide its Universal Donor Cell and Gene Editing Platform, which makes use of a stem cell-tropic rAAV vector for engineering humanpluripotent stem cells to contain deletions, insertions, or point mutations at any genomic position.

Adaptimmune has been collaborating with Universal Cells since 2015 on development of gene-edited induced pluripotent stem cell (iPSC) lines that generate proprietary T-cell products without the use of feeder layers.

Under the agreement, Astellas will fund research up until completion of a Phase I trial for each candidate with US$7.5m per year. Subsequently, Astellas and Adaptimmune may opt for co-development and co-commercialization of the candidate, or independent development through a milestone and royalty bearing licence. Under the agreement, Astellas will also have the right to select two targets and develop allogeneic cell therapy candidates on its own.

In case of Astellas would develop the candidates on its own, Adaptimmune may receive up to$897.5m in payments. If Adaptimmune would do so, Astellas may receive up to US$552.5m. If the companies opt for co-commercialisation any T-cell therapy, costs and profits will be shared equally.

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Astellas and Adaptimmune team up in CAR-T development - European Biotechnology

Stem Cell Therapy Market Rising Demand for Digitization in Organizations and Growth till 2027 – Galus Australis

The report provides in-depth analysis on the topic and discuss drivers, restraints and opportunities available in the market. The service is designed to help our clients in their decision support system. The analysis also cover the complete spectrum of the research topic to help our clients meeting their business objective.

Stem cell therapy is a technique which uses stem cells for the treatment of various disorders. Stem cell therapy is capable of curing broad spectrum of disorders ranging from simple to life threatening. These stem cells are obtained from different sources, such as, adipose tissue, bone marrow, embryonic stem cell and cord blood among others. Stem cell therapy is enables to treat more than 70 disorders, including degenerative as well as neuromuscular disorders. The ability of a stem cell to renew itself helps in replacing the damaged areas in the human body.

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Increase in the number of stem cell banking facilities and rising awareness on the benefits of stem cell for curing various disorders are expected to drive the market during the forecast period. Rise in number of regulations to promote stem cell therapy and increase in number of funds for research in developing countries are expected to offer growth opportunities to the market during the coming years.

Top Dominating Key Players:

1. MEDIPOST2. PHARMICELL Co., Ltd3. Holostem Terapie Avanzate S.r.l.4. Mesoblast Ltd5. U.S. Stem Cell, Inc.6. BIOTIME, INC.7. Lonza8. Caladrius9. Takeda Pharmaceutical Company Limited10. KOLON TISSUEGENE INC.

The stem cell therapy market is segmented based on type as, adult stem cell, embryonic stem cell induced pluripotent stem cell and others. The adult stem cells segment is further segmented as hematopoietic, umbilical cord, neuronal and mesenchymal stem cells. Based on treatment, the market is categorized as allogeneic and autologous. The market is categorized by application as, muscoskeletal, dermatology, cardiology, drug discovery & development and others.

The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global stem cell therapy market based on various segments. It also provides market size and forecast estimates from year 2017 to 2027 with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The stem cell therapy market by each region is later sub-segmented by respective countries and segments. The report covers analysis and forecast of 18 countries globally along with current trend and opportunities prevailing in the region.

The report analyzes factors affecting stem cell therapy market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the stem cell therapy market in these regions.

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Stem Cell Therapy Market Rising Demand for Digitization in Organizations and Growth till 2027 - Galus Australis

Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines – Yahoo Finance

Allele Biotechnology and Pharmaceuticals, Inc. (President and CEO: Jiwu Wang, Ph.D., "Allele"), a San Diego-based private company, and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas"), through its Massachusetts-based subsidiary Astellas Institute for Regenerative Medicine (AIRM), entered into a licensing agreement to expand Astellas access to Alleles induced pluripotent stem cell (iPSC) technologies for various cell therapy programs.

Astellas, one of the largest pharmaceutical companies in Japan and already a leader in the development of cell-based therapeutics, has further dedicated to development of the field through its commitment to state-of-the-art iPS cell generation, modification, and manufacturing. iPSC lines can differentiate into all somatic tissue types, enabling a wide variety of therapeutic applications. The field of iPSC-derived cells has seen dramatic growth in clinical trials recently--the majority of the ~12 clinical trials around the world were initiated within the last 18 months and many more are upcoming.

Allele has been developing its core strength in reprogramming somatic cells into iPSCs with granted patents and the first commercial cGMP system it developed over the past 10 years. Allele also engages in more than a dozen different human tissue derivation activities through its own R&D efforts for internal programs and partnerships. To realize the unparalleled potential of iPSC, Alleles researchers and cGMP team are committed to setting up and validating cell assays for product quality control, genome analysis pipelines, closed-system automation for reprogramming, and machine learning in iPSC-related fields.

Under the terms of the new license agreement, Astellas will pay Allele upfront and milestones, product-based royalties, and potentially manufacture fees.

About AlleleAllele Biotechnology and Pharmaceuticals was founded in 1999. In 2015, the company completed an 18,000 square foot state-of-the-art facility in San Diego for the production of GMP-grade human iPSC lines. The facility also supports the production of tissue-specific cells differentiated from these iPSCs, including pancreatic beta cells, neural progenitor cells, and cardiomyocytes.

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Allele Biotechnology and Pharmaceuticals, Inc.Daniel +1 858-587-6645

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Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines - Yahoo Finance

Researchers have managed to bio-print a functional mini-liver in 90 days – FLWL News

Brazilian researchers claim to have bio-printed hepatic organoids. These are miniature versions of livers obtained from human blood cells. However, these mini-organs would be able to perform all the functions of a liver. This innovation gives new hope in terms of organ transplant.

A functional mini-liverIn their publication in the journal Biofabrication of November 27, 2019, researchers from the Human Genome and Stem Cell Institute in Sao Paulo (Brazil) indicated that they obtained a mini-liver through bio-printing. However, the latter would fulfill all the functions hoped for! These include the production of vital proteins, the storage of vitamins and the secretion of bile.

The researchers explained that they combined several bioengineering techniques. Indeed, the culture of pluripotent stem cells and cell reprogramming have been combined with 3D bio-printing. However, there is a difference compared to previous research. In fact, the cells were placed entirely in the bio-ink before being extruded. Previously, it was simply a matter of individual cells.

Relieve waiting for transplantNo less than 90 days were required, from collecting the patients blood to producing the tissue. First, the researchers reprogrammed the patients blood cells into induced pluripotent stem cells. Then, the differentiation of the cells made it possible to change them into liver cells. Finally, their spheroids may have been associated with bio-ink.

You should know that the project directors have bio-printed not one, but three mini-livers. Logically, the stem cells came from three different donors. The objective? Test the method then analyze the functionalities of the organs and the maintenance of cellular contact. As expected, the method worked much better than in the case of previous research incorporating individualized cells. The researchers said the technique could be replicated on a large scale.

Thus, this innovation could open up new hopes in terms of organ transplants. Indeed, the wait for an organ can be very long, which can be problematic. In China, tensions around the field of organ transplants have given rise to questionable research. In 2017, researchers said they wanted to clone pigs to recover their organs. The objective? To successfully transplant humans with these same organs and end the terrible waiting lists.

Lamia spent a couple of years interning at an organization that offered medical consultation before joining the editorial team at FLWL News. An enthusiastic fitness freak in the room, she offers the best amounts of insights and craft-based writing style to keep us up to date about the medicine industry, health and science.

Email:lamia@flwl.orgPhone: +1 512-845-8162

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Researchers have managed to bio-print a functional mini-liver in 90 days - FLWL News

CYTOVIA Therapeutics and the New York Stem Cell Foundation Research Institute enter into a partnership to develop iPSC derived CAR NK Therapeutics -…

Press release content from Globe Newswire. The AP news staff was not involved in its creation.

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NEW YORK, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The New York Stem Cell Foundation (NYSCF) Research Institute today announced a partnership with Cytovia Therapeutics Inc. (Cytovia) to develop new disease treatments that leverage human stem cell research and novel gene editing techniques. NYSCF will be a key partner to Cytovia in using stem cells to advance novel therapeutic targets for cancer.

Cytovia leverages NK cells to make these novel therapeutics more specific to cancer cells. NK or natural killer cells are immune cells that scan the body and attack infected or abnormal cells, often serving as a first line of defense against cancer. CAR (chimeric antigen receptor) NK cells are genetically engineered to better locate and attack tumors. CAR NK-based treatments are currently showing promise in clinical trials and could serve as a potent and cost-efficient alternative to current immunotherapies. Establishing high-quality, stem-cell-derived NKs and CAR NKs will help improve these treatments and accelerate their path to the clinic.

The NYSCF Research Institute is a pioneer and acknowledged leader in stem cell technology, having developed the NYSCF Global Stem Cell Array, the premier automated robotic platform for reprogramming adult cells into induced pluripotent stem cells (iPSCs). These iPSCs carry the genetic blueprint of the person from whom they are derived and can be turned into any cell type in the body, allowing scientists to study disease mechanisms in affected cells or modify them for use in therapeutics.

Our mission is to bring lifesaving treatments to patients around the world and we are excited to further this goal in partnership with Cytovia, says NYSCF CEO and founder Susan L. Solomon. It is critical that we collaborate with partners using our technology and expertise to bring innovative treatments to the market.

We are delighted to collaborate with the NYSCF Research Institute to develop iPSC-derived NK and CAR NK therapeutics, says Dr. Daniel Teper, CEO of Cytovia. By integrating NYSCFs world-class stem cell know-how and the precision gene-editing research conducted at the University of California San Francisco, Cytovia aims to become a leader in NK cell therapeutics for the treatment of cancer.

About The New York Stem Cell Foundation Research Institute The New York Stem Cell Foundation (NYSCF) Research Institute is an independent non-profit organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 190 researchers at leading institutions worldwide, including the NYSCF Druckenmiller Fellows, the NYSCF Robertson Investigators, the NYSCF Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute is an acknowledged world leader in stem cell research and in the development of pioneering stem cell technologies, including the NYSCF Global Stem Cell Array, which is used to create cell lines for laboratories around the globe. In 2019, NYSCF launched its Womens Reproductive Cancers Initiative, which aims to shift paradigms in the way these cancers are studied and treated, in collaboration with leading cancer experts across the globe. NYSCF focuses on translational research in an accelerator model designed to overcome barriers that slow discovery and replace silos with collaboration. For more information, visit

About Cytovia Therapeutics Inc. Cytovia is dedicated to the development of transformational cancer immunotherapies, addressing several of the most challenging unmet medical needs including the prevention of cancer relapse and metastasis. Cytovia focuses on Natural Killer (NK) cell biology and applies precision medicine tools to develop the right therapy for the right patient at the right stage of the disease. Cytovia has secured access to multiple advanced technologies, including allogeneic cell therapy, multispecific antibodies, and cytokines. Cytovia establishes development partnerships to accelerate time-to-market and commercialization alliances in order to optimize rapid adoption of its novel immunotherapies. Learn more at

Contact information:CYTOVIA Therapeutics:Anna Baran-DjokovicVP, Corporate Affairs

Cytovia Media Contact: Charlotte Tomic Cell: 9178825243

NYSCF Research InstituteDavid McKeonChief of Staff

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CYTOVIA Therapeutics and the New York Stem Cell Foundation Research Institute enter into a partnership to develop iPSC derived CAR NK Therapeutics -...

Stem Cells Market Segmentation and Analysis Report, 2025 – Food & Beverage Herald

In theglobalstem cells marketa sizeable proportion of companies are trying to garner investments from organizations based overseas. This is one of the strategies leveraged by them to grow their market share. Further, they are also forging partnerships with pharmaceutical organizations to up revenues.

In addition, companies in the global stem cells market are pouring money into expansion through multidisciplinary and multi-sector collaboration for large scale production of high quality pluripotent and differentiated cells. The market, at present, is characterized by a diverse product portfolio, which is expected to up competition, and eventually growth in the market.

Some of the key players operating in the global stem cells market are STEMCELL Technologies Inc., Astellas Pharma Inc., Cellular Engineering Technologies Inc., BioTime Inc., Takara Bio Inc., U.S. Stem Cell, Inc., BrainStorm Cell Therapeutics Inc., Cytori Therapeutics, Inc., Osiris Therapeutics, Inc., and Caladrius Biosciences, Inc.

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As per a report by Transparency Market Research, the global market for stem cells is expected to register a healthy CAGR of 13.8% during the period from 2017 to 2025 to become worth US$270.5 bn by 2025.

Depending upon the type of products, the global stem cell market can be divided into adult stem cells, human embryonic stem cells, induced pluripotent stem cells, etc. Of them, the segment of adult stem cells accounts for a leading share in the market. This is because of their ability to generate trillions of specialized cells which may lower the risks of rejection and repair tissue damage.

Depending upon geography, the key segments of the global stem cells market are North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. At present, North America dominates the market because of the substantial investments in the field, impressive economic growth, rising instances of target chronic diseases, and technological progress. As per the TMR report, the market in North America will likely retain its dominant share in the near future to become worth US$167.33 bn by 2025.

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Investments in Research Drives Market

Constant thrust on research to broaden the utility scope of associated products is at the forefront of driving growth in the global stem cells market. Such research projects have generated various possibilities of different clinical applications of these cells, to usher in new treatments for diseases.Since cellular therapies are considered the next major step in transforming healthcare, companies are expanding their cellular therapy portfolio to include a range of ailments such as Parkinsons disease, type 1 diabetes, spinal cord injury, Alzheimers disease, etc.

The growing prevalence of chronic diseases and increasing investments of pharmaceutical and biopharmaceutical companies in stem cell research are the key driving factors for the stem cells therapeutics market. The growing number of stem cell donors, improved stem cell banking facilities, and increasing research and development are other crucial factors serving to propel the market, explains the lead analyst of the report.

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Stem Cells Market Segmentation and Analysis Report, 2025 - Food & Beverage Herald

Stem Cell Therapy Market Robust Growth Counted to 2025 – Instanews247

Stem Cell Therapy Market research now available at Industry Stats Report encompasses an exhaustive Study of this business space with regards to pivotal industry drivers, market share analysis, and the latest trends characterizing the Stem Cell Therapy industry landscape. This report also covers details of market size, growth spectrum, and the competitive scenario of Stem Cell Therapy market in the forecast timeline.

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The well-established Key players in the market are:

Gilead Novartis Organogenesis Vericel Others

This report for Stem Cell Therapy Market discovers diverse topics such as regional market scope, product market various applications, market size according to specific product, sales and revenue by region, manufacturing cost analysis, Industrial Chain, Market Effect Factors Analysis, market size forecast, and more.

Reports include the following segmentation: By Product Type Adult Stem Cells Human Embryonic Stem Cells (hESC) Induced Pluripotent Stem Cells Very Small Embryonic Like Stem CellsBy Applications Type Regenerative Medicine Drug Discovery and DevelopmentBy Technology Cell Acquisition Cell Production Cryopreservation Expansion and Sub-CultureBy Cell Therapy Autologous AllogeneicBy Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

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The report outlines the regulatory framework surrounding and governing numerous aspects of the market. At the end, Stem Cell Therapy industry development rival view, the industry scenario, samples, research conclusions are described. The important examination incorporated from 2014 to 2019 and till 2024 makes the report helpful assets for industry officials, promoting, sales, directors, experts, trade consultants, and others looking for key industry information with clearly given tables and charts.

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Stem Cell Therapy Market Robust Growth Counted to 2025 - Instanews247

The Top Biotech Trends We’ll Be Watching in 2020 – Singularity Hub

Last year left us with this piece of bombshell news: He Jiankui, the mastermind behind the CRISPR babies scandal, has been sentenced to three years in prison for violating Chinese laws on scientific research and medical management. Two of his colleagues also face prison for genetically engineering human embryos that eventually became the worlds first CRISPRd babies.

The story isnt over: at least one other scientist is eagerly following Hes footsteps in creating gene-edited humans, although he stresses that he wont implant any engineered embryos until receiving regulatory approval.

Biotech stories are rarely this dramatic. But as gene editing tools and assisted reproductive technologies increase in safety and precision, were bound to see ever more mind-bending headlines. Add in a dose of deep learning for drug discovery and synthetic biology, and its fair to say were getting closer to reshaping biology from the ground upboth ourselves and other living creatures around us.

Here are two stories in biotech were keeping our eyes on. Although successes likely wont come to fruition this year (sorry), these futuristic projects may be closer to reality than you think.

The idea of human-animal chimeras immediately triggers ethical aversion, but the dream of engineering replacement human organs in other animals is gaining momentum.

There are two main ways to do this. The slightly less ethically-fraught idea is to grow a fleet of pigs with heavily CRISPRd organs to make them more human-like. It sounds crazy, but scientists have already successfully transplanted pig hearts into baboonsa stand-in for people with heart failurewith some recipients living up to 180 days before they were euthanized. Despite having foreign hearts, the baboons were healthy and acted like their normal buoyant selves post-op.

But for cross-species transplantation, or xenotransplants to work in humans, we need to deal with PERVsa group of nasty pig genes scattered across the porcine genome, remnants of ancient viral infections that can tag along and potentially infect unsuspecting human recipients.

Theres plenty of progress here too: back in 2017 scientists at eGenesis, a startup spun off from Dr. George Churchs lab, used CRISPR to make PERV-free pig cells that eventually became PERV-free piglets after cloning. Then last month, eGenesis reported the birth of Pig3.0, the worlds most CRISPRd animal to further increase organ compatibility. These PERV-free genetic wonders had three pig genes that stimulate immunorejection removed, and nine brand new human genes to make themin theorymore compatible with human physiology. When raised to adulthood, pig3.0 could reproduce and pass on their genetic edits.

Although only a first clinical propotype that needs further validation and refinement, eGenesis is hopeful. According to one (perhaps overzealous) estimate, the first pig-to-human xenotranplant clinical trial could come in just two years.

The more ethically-challenged idea is to grow human organs directly inside other animalsin other words, engineer human-animal hybrid embryos and bring them to term. This approach marries two ethically uncomfortable technologies, germline editing and hybrids, into one solution that has many wondering if these engineered animals may somehow receive a dose of humanness by accident during development. What if, for example, human donor cells end up migrating to the hybrid animals brain?

Nevertheless, this year scientists at the University of Tokyo are planning to grow human tissue in rodent and pig embryos and transplant those hybrids into surrogates for further development. For now, bringing the embryos to term is completely out of the question. But the line between humans and other animals will only be further blurred in 2020, and scientists have begun debating a new label, substantially human, for living organisms that are mainly human in characteristicsbut not completely so.

With over 800 gene therapy trials in the running and several in mature stages, well likely see a leap in new gene medicine approvals and growth in CAR-T spheres. For now, although transformative, the three approved gene therapies have had lackluster market results, spurring some to ponder whether companies may cut down on investment.

The research community, however, is going strong, with a curious bifurcating trend emerging. Let me explain.

Genetic medicine, a grab-bag term for treatments that directly change genes or their expression, is usually an off-the-shelf solution. Cell therapies, such as the blood cancer breakthrough CAR-T, are extremely personalized in that a patients own immune cells are genetically enhanced. But the true power of genetic medicine lies in its potential for hyper-personalization, especially when it comes to rare genetic disorders. In contrast, CAR-Ts broader success may eventually rely on its ability to become one-size-fits-all.

One example of hyper-tailored gene medicine success is the harrowing story of Mila, a six-year-old with Batten disease, a neurodegenerative genetic disorder that is always fatal and was previously untreatable. Thanks to remarkable efforts from multiple teams, however, in just over a year scientists developed a new experimental therapy tailored to her unique genetic mutation. Since receiving the drug, Milas condition improved significantly.

Milas case is a proof-of-concept of the power of N=1 genetic medicine. Its unclear whether other children also carry her particular mutationBatten has more than a dozen different variants, each stemming from different genetic miscodingor if anyone else would ever benefit from the treatment.

For now, monumental costs and other necessary resources make it impossible to pull off similar feats for a broader population. This is a shame, because inherited diseases rarely have a single genetic cause. But costs for genome mapping and DNA synthesis are rapidly declining. Were starting to better understand how mutations lead to varied disorders. And with multiple gene medicines, such as antisense oligonucleotides (ASOs) finally making a comeback after 40 years, its not hard to envision a new era of hyper-personalized genetic treatments, especially for rare diseases.

In contrast, the path forward for CAR-T is to strip its personalization. Both FDA-approved CAR-T therapies require doctors to collect a patients own immune T cells, preserved and shipped to a manufacturer, genetically engineered to boost their cancer-hunting abilities, and infused back into patients. Each cycle is a race against the cancer clock, requiring about three to four weeks to manufacture. Shipping and labor costs further drive up the treatments price tag to hundreds of thousands of dollars per treatment.

These considerable problems have pushed scientists to actively research off-the-shelf CAR-T therapies, which can be made from healthy donor cells in giant batches and cryopreserved. The main stumbling block is immunorejection: engineered cells from donors can cause life-threatening immune problems, or be completely eliminated by the cancer patients immune system and lose efficacy.

The good news? Promising results are coming soon. One idea is to use T cells from umbilical cord blood, which are less likely to generate an immune response. Another is to engineer T cells from induced pluripotent stem cells (iPSC)mature cells returned back to a young, stem-like state. A patients skin cells, for example, could be made into iPSCs that constantly renew themselves, and only pushed to develop into cancer-fighting T cells when needed.

Yet another idea is to use gene editing to delete proteins on T cells that can trigger an immune responsethe first clinical trials with this approach are already underway. With at least nine different off-the-shelf CAR-T in early human trials, well likely see movement in industrialized CAR-T this year.

Theres lots of other stories in biotech we here at Singularity Hub are watching. For example, the use of AI in drug discovery, after years of hype, may finally meet its reckoning. That is, can the technology actually speed up the arduous process of finding new drug targets or the design of new drugs?

Another potentially game-changing story is that of Biogens Alzheimers drug candidate, which reported contradicting results last year but was still submitted to the FDA. If approved, itll be the first drug to slow cognitive decline in a decade. And of course, theres always the potential for another mind-breaking technological leap (or stumble?) thats hard to predict.

In other words: we cant wait to bring you new stories from biotechs cutting edge in 2020.

Image Credit: Image by Konstantin Kolosov from Pixabay

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