Category Archives: Stem Cell Clinic

Cell Therapy Company Raises $160 Million to Advance iPSC-Derived Therapies to Clinic – BioSpace

Century Therapeuticsreceived a $160 million infusion of cash to advance the companys pipeline of induced pluripotent stem cell (iPSC)-derived cell therapies for cancer.

This morning, the Philadelphia-based company announced a Series C financing round that will drive its preclinical pipeline, which includes multiple iPSC-derived CAR-iT and CAR-iNK cell products, into the clinic over the next 12 months. The companys assets are designed to resist host rejection, enhance cell persistence, and allow repeat dosing to provide durable responses in all patients. Century Therapeutics, which launched in 2019, anticipates clinical testing to begin in 2022 and also predicts it will generate multiple Investigational New Drug applications over the next several years.

The companysgenetically engineered, iPSC-derived iNK and iT cell products are designed to specifically target hematologic and solid tumor cancers. Centurys iPSCs, which are stem cells that can be generated from adult stem cells, have unlimited self-renewing capacity, which enables multiple rounds of cellular engineering. According to the company, these engineering rounds will produce master cell banks of modified cells that can be expanded and differentiated into immune effector cells to supply vast amounts of allogeneic and homogeneous therapeutic products. This platform differentiates Century from competitors that are developing cell therapies made from non-renewable donor-derived cells.

The Series C financing round was led by Casdin Capital and include a number of new investors, including Fidelity Management & Research LLC, the Federated HermesKauffmannFunds, RA Capital, Logos Capital, OrbiMed,Marshall Wace, Qatar Investment Authority, Avidity Partners, and Octagon Capital.Founding investorsVersant Ventures and Leaps by Bayer also participated in the latest fundraising.

We are fortunate to be surrounded by such a top-tier group of investors, whose support will enable the acceleration of Century's technology platform into the clinic, Lalo Flores, chief executive officer of Century Therapeutics said in a statement. With this new investor partnership, we are well-positioned to capitalize on the tremendous potential of our integrated iPSC, cell engineering and manufacturing capabilities to develop safer, more effective and more affordable next generation allogeneic cancer therapies.

Eli Casdin, chief investment of Casdin Capital, who joined the Century Therapeutics Board of Directors following this Series C, said he was excited to partner with the cell therapy company.

It's a remarkable and transformative time in the field, with the ability to engineer cells for therapeutic impact now a commercial reality. At the same time, iPSC technology has matured and is now leading the transition from bespoke autologous products to off-the-shelf allogeneic ones, Casdin said in a statement.

For Century Therapeutics, the financing round was announced about one month after the company expanded its capabilities with new laboratory manufacturing facilities in Pennsylvania and New Jersey. In addition to thePennsylvaniaandNew Jerseylocations, Century has a laboratory inHamilton, Ontariospecifically focused on targeting glioblastoma, and recently opened aSeattle-based innovation hub to help advance the company's novel iPSC platform and support the continued pipeline growth and development.

Last year, Century Therapeutics acquired Empirica Therapeutics to leverage its iPSC-derived allogeneic cell therapies against glioblastoma, one of the most common types of primary brain tumor in adults.

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Cell Therapy Company Raises $160 Million to Advance iPSC-Derived Therapies to Clinic - BioSpace

Retracing the Lineage of Cancer Cells – Technology Networks

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There is no stronger risk factor for cancer than age. At the time of diagnosis, the median age of patients across all cancers is 66. That moment, however, is the culmination of years of clandestine tumor growth, and the answer to an important question has thus far remained elusive: When does a cancer first arise?

At least in some cases, the original cancer-causing mutation could have appeared as long as 40 years ago, according to a new study by researchers at Harvard Medical School and the Dana-Farber Cancer Institute.

Reconstructing the lineage history of cancer cells in two individuals with a rare blood cancer, the team calculated when the genetic mutation that gave rise to the disease first appeared. In a 63-year-old patient, it occurred at around age 19; in a 34-year-old patient, at around age 9.

The findings, published in the March 4 issue ofCell Stem Cell, add to a growing body of evidence that cancers slowly develop over long periods of time before manifesting as a distinct disease. The results also present insights that could inform new approaches for early detection, prevention, or intervention.

"For both of these patients, it was almost like they had a childhood disease that just took decades and decades to manifest, which was extremely surprising," said co-corresponding study author Sahand Hormoz, HMS assistant professor of systems biology at Dana-Farber.

"I think our study compels us to ask, when does cancer begin, and when does being healthy stop?" Hormoz said. "It increasingly appears that it's a continuum with no clear boundary, which then raises another question: When should we be looking for cancer?"

In their study, Hormoz and colleagues focused on myeloproliferative neoplasms (MPNs), a rare type of blood cancer involving the aberrant overproduction of blood cells. The majority of MPNs are linked to a specific mutation in the gene JAK2. When the mutation occurs in bone marrow stem cells, the body's blood cell production factories, it can erroneously activate JAK2 and trigger overproduction.

To pinpoint the origins of an individual's cancer, the team collected bone marrow stem cells from two patients with MPN driven by the JAK2 mutation. The researchers isolated a number of stem cells that contained the mutation, as well normal stem cells, from each patient, and then sequenced the entire genome of each individual cell.

Over time and by chance, the genomes of cells randomly acquire so-called somatic mutations--nonheritable, spontaneous changes that are largely harmless. Two cells that recently divided from the same mother cell will have very similar somatic mutation fingerprints. But two distantly related cells that shared a common ancestor many generations ago will have fewer mutations in common because they had the time to accumulate mutations separately. Cell of origin

Analyzing these fingerprints, Hormoz and colleagues created a phylogenetic tree, which maps the relationships and common ancestors between cells, for the patients' stem cells--a process similar to studies of the relationships between chimpanzees and humans, for example.

"We can reconstruct the evolutionary history of these cancer cells, going back to that cell of origin, the common ancestor in which the first mutation occurred," Hormoz said.

Combined with calculations of the rate at which mutations accumulate, the team could estimate when the JAK2 mutation first occurred. In the patient who was first diagnosed with MPN at age 63, the team found that the mutation arose around 44 years prior, at the age of 19. In the patient diagnosed at age 34, it arose at age 9.

By looking at the relationships between cells, the researchers could also estimate the number of cells that carried the mutation over time, allowing them to reconstruct the history of disease progression.

"Initially, there's one cell that has the mutation. And for the next 10 years there's only something like 100 cancer cells," Hormoz said. "But over time, the number grows exponentially and becomes thousands and thousands. We've had the notion that cancer takes a very long time to become an overt disease, but no one has shown this so explicitly until now."

The team found that the JAK2 mutation conferred a certain fitness advantage that helped cancerous cells outcompete normal bone marrow stem cells over long periods of time. The magnitude of this selective advantage is one possible explanation for some individuals' faster disease progression, such as the patient who was diagnosed with MPN at age 34.

In additional experiments, the team carried out single-cell gene expression analyses in thousands of bone marrow stem cells from seven different MPN patients. These analyses revealed that the JAK2 mutation can push stem cells to preferentially produce certain blood cell types, insights that may help scientists better understand the differences between various MPN types.

Together, the results of the study offer insights that could motivate new diagnostics, such as technologies to identify the presence of rare cancer-causing mutations currently difficult to detect, according to the authors.

"To me, the most exciting thing is thinking about at what point can we detect these cancers," Hormoz said. "If patients are walking into the clinic 40 years after their mutation first developed, could we have caught it earlier? And could we prevent the development of cancer before a patient ever knows they have it, which would be the ultimate dream?"

The researchers are now further refining their approach to studying the history of cancers, with the aim of helping clinical decision-making in the future.

While their approach is generalizable to other types of cancer, Hormoz notes that MPN is driven by a single mutation in a very slow growing type of stem cell. Other cancers may be driven by multiple mutations, or in faster-growing cell types, and further studies are needed to better understand the differences in evolutionary history between cancers.

The team's current efforts include developing early detection technologies, reconstructing the histories of greater numbers of cancer cells, and investigating why some patients' mutations never progress into full-blown cancer, but others do.

"Even if we can detect cancer-causing mutations early, the challenge is to predict which patients are at risk of developing the disease, and which are not," Hormoz said. "Looking into the past can tell us something about the future, and I think historical analyses such as the ones we conducted can give us new insights into how we could be diagnosing and intervening."

Reference:Egeren DV, Escabi J, Nguyen M, et al. Reconstructing the lineage histories and differentiation trajectories of individual cancer cells in myeloproliferative neoplasms. Cell Stem Cell. 2021;28(3):514-523.e9. doi:10.1016/j.stem.2021.02.001

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Retracing the Lineage of Cancer Cells - Technology Networks

QC Kinetix (San Antonio) Offers Non-Surgical Regeneration Therapy To Treat Aches, Pains, and Injuries In Individuals Around San Antonio – Press…

San Antonio, CA - QC Kinetix (San Antonio) offers a range of regenerative treatments to individuals living in San Antonio and surrounding areas to treat acute and chronic pain and sports injuries. Their regenerative therapies help treat knee pain, shoulder pain, Carpal Tunnel Syndrome, Arthritis, Plantar Fasciitis, Tendonitis, Elbow Pain, Sciatica, Finger and Toe Pain, Foot and Hand Pain, Wrist Pain, Ankle Pain, Low Back Pain, Nerve Pain, Hip Pain, Tendon, Ligament, and Muscle Pain, and Low Levels of Testosterone in individuals.

The clinic uses stem cell therapy with BMAC and other regenerative treatments like comprehensive regenerative cell therapy injections using amniotic membrane tissue, A2M therapy, and PRP therapy. The team ensures they diagnose the patients' condition and discuss the different treatment options before proceeding with treatment. If they feel any patient is not a candidate for therapy at their clinic, they advise him/her of other options.

The patients at QC Kinetix (San Antonio) range from young athletes to active senior citizens. The doctors and treatment providers have extensive therapy in pain treatment using regenerative medicine. They speak openly with patients about their symptoms and don't try to hide anything. They aim to ensure individuals can maximize their body's ability to repair and heal itself and function efficiently. This, in turn, helps improve the individuals' overall quality of life.

The talented and well-experienced treatment providers at QC Kinetix (San Antonio) know how frustrating it can get for patients with limited mobility. They know patients can get tired of taking addictive pain medications without any positive results. They realize all of these failed attempts at treating physical ailments can have a physical and emotional toll on them.

All treatment providers at QC Kinetix (San Antonio) are board-certified and have years of regenerative medicine experience. They keep themselves updated with the latest development and technology in this field and adapt depending on their condition and requirement.

All patients that visit QC Kinetix (San Antonio) are treated equally, irrespective of age and condition. Whether individuals have mild symptoms or unbearable pain, the team at QC Kinetix (San Antonio) will ensure the patient gets full attention and is treated with care. The team knows that many people get overwhelmed at the thought of surgery and will prefer another treatment option where surgery is not required. They do their best to help individuals get treated without surgery through regenerative medicine. This place is ideal for anyone looking for San Antonio stem cell treatment.

QC Kinetix (San Antonio) has successfully treated many patients from the time they have been established. All patients who get treated at this facility go back home happier and healthier than before.

QC Kinetix (San Antonio) is located at 18707 Hardy Oak Pavilion, Suite 445, San Antonio, TX, 78258. Individuals interested in knowing more about their treatments and therapies can visit their website or contact them on (210) 571-0318 to request an appointment.

Media Contact Company Name: QC Kinetix (San Antonio) Contact Person: Casey Sietsema Email: Send Email Phone: (210) 571-0318 Address:18707 Hardy Oak Blvd Suite 445 City: San Antonio State: TX Country: United States Website: https://qckinetix.com/san-antonio/

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QC Kinetix (San Antonio) Offers Non-Surgical Regeneration Therapy To Treat Aches, Pains, and Injuries In Individuals Around San Antonio - Press...

QC Kinetix (Myrtle Beach) Offers Stem Cells Therapy, An Alternative to Surgery For Pain Management in Myrtle Beach, SC – Press Release – Digital…

Myrtle Beach, SC - Often, the only solution is a surgical intervention for people who suffer from pain in their muscles, joints, and tendons. However, the professional team at QC Kinetix (Myrtle Beach) offers a better and non-invasive alternative by using a regenerative medicine approach to treating chronic pain and related symptoms in Myrtle Beach, SC. At QC Kinetix (Myrtle Beach), the experts stay abreast of the latest advances in the stem cell and regenerative medicine world to keep delivering effective results to patients.

Rated one of the top pain clinics in the area, QC Kinetix (Myrtle Beach) ensures that each patient who comes into the clinic is offered a unique approach to diagnosing and treating the conditions suffered. QC Kinetix (Myrtle Beach) and its team employ a cutting-edge approach to handling all pain conditions to ensure effective results.

The spokesperson for QC Kinetix (Myrtle Beach), while describing the regenerative medicine practice, said: Pain in your muscles, joints, and tendons can greatly affect your daily life and deter you from doing all the activities you once loved. Too often, surgery is looked at as the best solution to relieve pain. But surgery comes with long recovery times, drugs, and other less-than-desirable effects. The QC Kinetix Myrtle Beach office takes a different approach. We are a regenerative medicine clinic focusing on non-surgical methods of joint pain treatment.

The regenerative medical approach offered at the clinic focuses on whole-body healing using the blood, cells, and tissues that help repair all damaged areas, lessen inflammation and speed up the recovery process. Using stem cell therapy, the team at QC Kinetix (Myrtle Beach) can help patients combat all kinds of pain, especially orthopedic pain affecting the deeper areas of the muscle and tendons.

QC Kinetixs Myrtle Beach stem cells treatment is focused on different areas of the body, including the shoulder, elbow, wrists, hip, knee, ankle, feet, and low back region. Patients who suffer from chronic knee pain and instability, bone-on-bone osteoarthritis, knee arthritis, decreased range of motion, cracking, popping, or snapping sounds from the knee; torn ACL, MCL, and LCL, or torn Meniscus can take advantage of the wide range of regenerative medical treatments offered at QC Kinetix (Myrtle Beach).

Patients will be exposed to class IV laser therapy, which delivers instant relief to the affected areas; Platelet-Rich Plasma (PRP injections) which decreases inflammation and initiates the process of regeneration; regenerative cell therapy, which kickstarts the process of regeneration; stem cell therapy / BMAC, which aids in the tissue repair process, as well as ultrasounds which aid in proper diagnosis and procedural precision.

QC Kinetix (Myrtle Beach) is located at 8210 Devon Ct Suite A, Myrtle Beach, SC 29572, USA. Schedule an appointment with their providers via phone at (843) 310-2703 or visit their website.

Media Contact Company Name: QC Kinetix (Myrtle Beach) Contact Person: Adam Rose Email: Send Email Phone: (843) 310-2703 Address:8210 Devon Ct Suite A City: Myrtle Beach State: SC Country: United States Website: https://qckinetix.com/myrtle-beach/

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QC Kinetix (Myrtle Beach) Offers Stem Cells Therapy, An Alternative to Surgery For Pain Management in Myrtle Beach, SC - Press Release - Digital...

Caribou nets $115M to boost CRISPR tech, advance off-the-shelf cell therapies – FierceBiotech

On the heels of a new partnership with AbbVie, Caribou Biosciences is reeling in $115 million to keep improving its CRISPR technology and shepherd its pipeline of allogeneic, or off-the-shelf, cell therapies into and through the clinic.

Caribous most advanced program is CB-101, an anti-CD19 CAR-T therapy that's in phase 1 in B cell non-Hodgkin lymphoma. Its second program, CB-011, is a BCMA-targeting CAR-T in development for the treatment of multiple myeloma. A third asset targets CD371 for the treatment of acute myeloid leukemia. The company is developing all three programs for patients whose cancer has come back despite undergoing other treatments or did not respond to those treatments in the first place.

RELATED: After ditching Editas, AbbVie taps Caribou for new CRISPR, CAR-T pact

Learn about the key factors that are essential to creating a collaborative and productive working relationship to advance pre-clinical drug discovery programs.

The FDA has approved four CAR-T treatments for blood cancers from Novartis, Gileads Kite unit and Bristol Myers Squibb, but all four treatments are autologous, meaning they are made with a patients own cells. Caribou and other companies taking the allogeneic tack are building their treatments with donor cells in hopes that this will clear some hurdles that come with the autologous route.

Autologous treatments can be complex and time-consuming to makecells must be taken out of the patient, modified to fight cancer and then put back into the patientand some patients simply dont have that much time. Other patients may not have enough T cells, or T cells of good enough quality, to make those treatments.

And Caribou isnt stopping at CAR-T. It is also working on an allogeneic natural killer cell therapy derived from induced pluripotent stem cells for solid tumors.

Caribou has built a remarkable and highly differentiated technology platform along with a pipeline of novel therapeutic candidates which hold breakthrough potential, said Santhosh Palani, Ph.D., partner at PFM Health Sciences, who joined Caribous board as part of the financing.

RELATED: Bristol Myers Squibb's CAR-T liso-cel wins long-delayed FDA nod

PFM led the series C round alongside Farallon Capital Management and Ridgeback Capital Investors. Caribous backers returned for the financing, including Heritage Medical Systems, Maverick Ventures, and Pontifax Global Food and Agriculture Technology Fund, while several newcomers also joined in, including AbbVie Ventures, Janus Henderson Investors andThe Leukemia & Lymphoma Society Therapy Acceleration Program.

In addition to propelling Caribous pipeline, the capital will also bankroll the development of the CRISPR technology it uses to make its cell therapies. The funding follows the AbbVie deal, which saw Caribou snag $40 million upfront, with $300 million promised down the line.

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New Research Study Investigates Metformin as a Therapy to Promote Brain Repair and Reduce Disability in Children and Young Adults with MS -…

February 02, 2021 06:00 ET | Source: Multiple Sclerosis Society of Canada

multilang-release

TORONTO, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Today the Multiple Sclerosis Society of Canada announced $400,000 in funding to support a pilot clinical trial to investigate the use of metformin as a therapy for children and young adults with multiple sclerosis (MS). In partnership with Stem Cell Network (SCN) and Ontario Institute for Regenerative Medicine (OIRM), this investment increases the funding for the trial to $1 million. These partnerships are critical for leveraging more research dollars and increased impact for the benefit of Canadian health. Metformin, a widely-used and safe drug prescribed for type 2 diabetes, is a therapeutic agent that may promote brain repair and reduce disability following white matter damage in people living with MS.

Damage to white matter in the brain occurs in people with MS resulting in sensory, motor, and cognitive problems. Preclinical evidence shows metformin enhances oligodendrocytes cells that produce myelin and promotes white matter repair in animal models and in youth with radiation-induced brain injury. This research aims to translate these preclinical findings from animal and human studies into clinical practice by conducting a pilot feasibility trial.

Dr. Ann Yeh (The Hospital for Sick Children [SickKids], University of Toronto) is the lead investigator of the study and she will be joined by a team of nine Canadian researchers.

Our team is very excited about being able to move this trial forward, and for the collaborations across multiple different scientific areas that the collaborative grant will allow. This is truly a team effort that started at the bench at SickKids in the lab of Dr. Freda Miller and has set the stage for a clinical trial that could potentially improve outcomes for children and young adults living with MS, says Dr. Ann Yeh, Staff Physician, Division of Neurology and Senior Associate Scientist, Neurosciences & Mental Health at SickKids.

This trial is a unique opportunity to advance novel therapies that target regeneration and repair to help reverse the progressive nature of MS by re-purposing a safe, low-cost treatment, says Dr. Pamela Valentine president and CEO, MS Society of Canada. The MS Society is thrilled to partner with SCN and OIRM in funding this project that has the potential to change the life course, and transform treatment and care for youth and people living with MS.

The trial is the outcome of several years of work and support among researchers and partners involved in this project. It is set to run for three years and aims to recruit 30 participants between the ages of 10 to 25 who have been diagnosed with MS.

To learn more about this research trial, click here.

About multiple sclerosis and the MS Society of Canada

Canada hasone ofthe highest rates of multiple sclerosisinthe world. On average,12 Canadians arediagnosed every day.MS is a chronicautoimmunedisease of the central nervous system (brain, spinal cord).It is considered an episodic disabilitymeaningthat the severity and duration of illness and disability can vary and are often followed by periods of wellness. It can also be progressive.Most people are diagnosedwith MSbetween the ages of 20 and 49 and the unpredictable effects ofthe disease willlast for the rest of their lives.The MS Society providesinformation, support and advocacy to people affected by MS,and funds research tofind the cause and cure for the disease, bringing us closer to a world free of MS. Please visitmssociety.caor call 1-800-268-7582 for more information, to get involved, or to support Canadians affected by MS bymaking a donation. Join the conversation and connect with the MS community online. Find the MS Society onTwitter,Instagramor like our page onFacebook.

About the Ontario Institute for Regenerative Medicine (OIRM)

OIRM is a non-profit stem cell institute funded in part by the Ontario government and dedicated to transforming discoveries into clinical trials and cures. Through our commitment to collaboration and partnerships, we leverage our resources to fund and support promising advances. OIRM is a passionate champion for investigators and their patients as we build a healthier future for Ontario, Canada and the world. http://www.oirm.ca

About the Stem Cell Network (SCN) Tomorrows health is here. SCN is a national non-profit that supports stem cell and regenerative medicine research, training the next generation of highly qualified personnel, and delivering outreach activities across Canada. SCNs goal is to advance science from the lab to the clinic for the benefit of Canadians. SCN has been supported by the Government of Canada since inception in 2001. This strategic funding valued at $118M has benefitted approximately 196 world-class research groups and 3,300 trainees and has catalyzed24clinical trials. stemcellnetwork.ca

CONTACT: Jennifer Asselin MS Society of Canada 1-800-268-7582 ext. 3144 jennifer.asselin@mssociety.ca

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New Research Study Investigates Metformin as a Therapy to Promote Brain Repair and Reduce Disability in Children and Young Adults with MS -...

#FillTheSeats to save lives this February with Canadian Blood Services – Sportsnet.ca

Help Fill the Seats to save lives this February!

Canadian Blood Services is encouraging new and existing blood donors to help fill up to 6,690 appointments in Calgary and to consider making a Plasma or Steam cell donation. Currently, the need for plasma and steam cell donations is urgent.

Plasma is needed more than four times what is donated in Canada. It could take over 100 plasma donations to help treat one patient for a year. These donations go to patients who need transfusions, as well as to fractionators for manufacturing specialized medicines needed by Canadians with conditions such as bleeding disorders, burns and immunodeficiency. The winter season is typically a challenging time for blood and plasma collections due to winter hazards and weather impacts.

Stem Cell transplants can treat over 80 different diseases and disorders. Each year, hundreds of patients in Canada with cancers and blood diseases need a stem cell transplant to save their life many of whom are facing challenges finding a matching donor. A stem cell transplant replaces the patients unhealthy stem cells with a matching donors healthy stem cells. There are three sources of stem cells used in transplant:

Potential volunteer stem cell donors and expectant moms can also help save lives by donating cord blood.

Blood & Plasma donations are by appointment only. To book yours go to blood.ca, right click on find a location, enter Eau Claire Market and choose Calgary permanent clinic. To learn more about Stem Cell transplants, join the stem cell registry at https://www.blood.ca/en/stemcells.

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#FillTheSeats to save lives this February with Canadian Blood Services - Sportsnet.ca

Vertex Announces FDA Clearance of Investigational New Drug (IND) Application for VX-880, a Novel Cell Therapy for the Treatment of Type 1 Diabetes…

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND, enabling the company to proceed with initiating a clinical trial for VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell therapy to treat T1D. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2021 in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

As we celebrate the 100th anniversary of the discovery of insulin this year, we are excited to bring a first-in-class cell therapy to the clinic with the potential to meaningfully impact people living with T1D, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We look forward to getting our clinical program underway and testing our unique approach of replacing pancreatic islet cells, which are destroyed in people with type 1 diabetes, with our stem cell-derived fully differentiated insulin-producing pancreatic islet cells.

About VX-880 VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the bodys ability to regulate glucose levels by restoring pancreatic islet cell function, including insulin production.

The VX-880 clinical trial will involve an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.

About the Phase 1/2 Clinical Trial The clinical trial is a Phase 1/2, single-arm, open-label study in subjects who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This will be a sequential, multi-part clinical trial to evaluate the safety and efficacy of different doses of VX-880. Approximately 17 patients will be enrolled in the clinical trial.

About Type 1 Diabetes T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death. Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in patients with T1D. Hypoglycemia remains a critical limiting factor in glycemic management, and severe hypoglycemia can cause loss of consciousness, coma, seizures, injury, and can be fatal.

There are currently limited treatment options beyond insulin for the management of T1D.

About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Bastiano Sanna, Ph.D., in this press release, statements regarding the development, plans and expectations for our T1D pipeline program, including our plans to initiate a Phase 1/2 clinical trial in people with T1D and expected timeline of our clinical trials, statements regarding patient enrollment and dosing, statements regarding potential clinical trial results and anticipated benefits of VX-880, and our plans to provide further updates on our T1D pipeline program. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the FDA may not approve our IND, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs may not support registration or further development due to safety, efficacy or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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Vertex Announces FDA Clearance of Investigational New Drug (IND) Application for VX-880, a Novel Cell Therapy for the Treatment of Type 1 Diabetes...

ClearPoint Neuro, Inc. Announces Expansion of Pre-Clinical and Translational Development Team to Support Gene and Stem Cell Therapy Partners -…

January 26, 2021 16:05 ET | Source: ClearPoint Neuro, Inc.

IRVINE, Calif., Jan. 26, 2021 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT), a global therapy-enabling platform company providing navigation and delivery to the brain, today announced that Ernesto Salegio, PhD will join the Company as Vice President, Segment Leader Translational and Pre-Clinical Research as part of ClearPoints Biologics and Drug Delivery team on March 1, 2021. Dr. Salegio brings over 19 years of experience in translational neuroscience with direct pre-clinical central nervous system (CNS) expertise in the delivery of therapeutics to the brain and spinal cord, including over 16 years of gene therapy experience working with adeno-associated viral vectors (AAV).

ClearPoint Neuros Biologics and Drug Delivery team was established in 2020 to provide turn-key medical device innovation, therapy delivery development and clinical services customized for both pharmaceutical and academic partners working on gene and stem cell therapies to the brain. This team also offers comprehensive services to help its partners navigate existing regulatory guidance and stay abreast of anticipated changes to guidance that will inevitably come.

Our current and prospective partners working on gene and stem cell therapies have an enormous unmet need for pre-clinical support, commented Jeremy Stigall, Vice President, Biologics & Drug Delivery Development. Ernestos extensive pre-clinical expertise in the intraparenchymal administration of therapeutics under image-guidance, as well into cerebrospinal fluid (CSF), will allow ClearPoint to add crucial translational consulting services for our biologics partners. When we establish relationships with pharmaceutical partners well before the clinic, ClearPoint products and services can be incorporated throughout the entire development process, delivering consistent, predictable performance, and providing government agencies assurance that the navigation and delivery platform will remain constant from bench-to-bedside. We aim to become their comprehensive medical device partner, allowing them to focus on more traditional pharmaceutical challenges, while ClearPoint delivers state-of-the-art medical device and therapy delivery.

I am thrilled to become a member of the ClearPoint team, commented Dr. Salegio. After spending almost two decades focusing on translational research, image-guided delivery protocols, complex surgical procedures and in the development of CNS therapies, I am delighted to drive the expansion of ClearPoints capabilities. I am eager to leverage their current pre-clinical and clinical portfolio, as well as to their pipeline of innovative tools on the horizon, to provide customized solutions to current and future ClearPoint pharmaceutical partners.

About ClearPoint Neuro

ClearPoint Neuros mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep-brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics, and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and is installed in over 60 active clinical sites in the United States and the EU. The Companys SmartFlow cannula is being used in partnership or evaluation with 25 individual biologics and drug delivery companies in various stages from preclinical research to late-stage regulatory trials. To date, more than 4,000 cases have been performed and supported by the Companys field-based clinical specialist team which offers support and services for our partners. For more information, please visit http://www.clearpointneuro.com.

Forward-Looking Statements

Statements herein concerning the Companys plans, growth and strategies may include forward-looking statements within the context of the federal securities laws. Statements regarding the Company's future events, developments and future performance, as well as management's expectations, beliefs, plans, estimates or projections relating to the future, are forward-looking statements within the meaning of these laws. Uncertainties and risks may cause the Company's actual results to differ materially from those expressed in or implied by forward-looking statements. Particular uncertainties and risks include those relating to the impact of COVID-19 and the measures adopted to contain its spread; future revenues from sales of the Companys ClearPoint Neuro Navigation System products; the Companys ability to market, commercialize and achieve broader market acceptance for the Companys ClearPoint Neuro Navigation System products; and estimates regarding the sufficiency of the Companys cash resources. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section of the Companys Annual Report on Form 10-K for the year ended December 31, 2019, and the Companys Quarterly Report on Form 10-Q for the three months ended September 30, 2020, both of which have been filed with the Securities and Exchange Commission, and the Companys Annual Report on Form 10-K for the year ended December 31 2020, which the Company intends to file with the Securities and Exchange Commission on or before March 31, 2021.

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ClearPoint Neuro, Inc. Announces Expansion of Pre-Clinical and Translational Development Team to Support Gene and Stem Cell Therapy Partners -...

Cell Therapy Market 2020 Industry Size, Share, Segmentation, Comprehensive Analysis and Forecast by 2025 KSU | The Sentinel Newspaper – KSU | The…

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Competitors Analysis:

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In market segmentation by types, the report covers: Stem Cell, Non-Stem Cell

In market segmentation by applications, the report covers the following uses: Hospital, Clinic

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Cell Therapy Market 2020 Industry Size, Share, Segmentation, Comprehensive Analysis and Forecast by 2025 KSU | The Sentinel Newspaper - KSU | The...