Category Archives: Stem Cell Treatments

From left, T. Denny Sanford, Catriona Jamieson, MD, PhD, and Chancellor Pradeep K. Khosla celebrate the establishment of the UC San Diego Sanford Stem Cell Institute, made possible by a historic gift from Sanford.

Noted businessman and philanthropist T. Denny Sanford has committed $150 million in new funding to expand and, in some ways, quite literally launch stem cell research and regenerative medicine at University of California San Diego into new spaces and endeavors.

The gift will fund the new UC San Diego Sanford Stem Cell Institute and builds upon a $100 million gift in 2013 from Sanford that boldly established UC San Diego as a leader in developing and delivering the therapeutic promise of human stem cells special cells with the ability to develop into many different cell types and which, when modified and repurposed, have the potential to treat, remedy or cure a vast array of conditions and diseases.

Dennys previous generosity spurred discoveries in stem cell research and medicine at UC San Diego that are already benefiting countless patients around the world, said Chancellor Pradeep K. Khosla. His most recent gift adds to our portfolio of stem cell research conducted in Earths orbit that will help us better understand the progression of cancer cells and aging.

Sanfords gift to establish the Sanford Stem Cell Institute is the largest single gift to UC San Diego. This investment enables the team to dream beyond what is possible, said Sanford. The sky is no longer the limit.

In addition to his investment to create the Sanford Stem Cell Clinical Center at UC San Diego Health in 2013, Sanford established the T. Denny Sanford Institute for Empathy and Compassion in 2019, which focuses on research into the neurological basis of compassion, with application toward developing compassion and empathy-focused training for future generations of medical professionals. He also recently made a $5 million gift to support the Epstein Family Alzheimers Research Collaboration, a partnership between UC San Diego and the University of Southern California to spark new collaborative efforts to discover effective therapies for Alzheimers disease.

Sanford was also honorary co-chair of the Campaign for UC San Diego, which concluded in June 2022 having raised more than $3 billion exceeding its initial $2 billion goal. He was honored as a recipient of the 2014 Chancellors Medal, one of the universitys highest honors, in recognition of his exceptional service in support of the campus mission.

Stem cell research will be conducted in a laboratory bay located aboard the International Space Station, pictured here, in low-Earth orbit. Credit: NASA

The new UC San Diego Sanford Stem Cell Institute, under the direction of Catriona Jamieson, MD, PhD, Koman Family Presidential Endowed Chair in Cancer Research in the UC San Diego School of Medicine, will continue three existing stem cell programs at UC San Diego with three new programs.

The new programs to be established with Sanfords gift include:

Existing stem cell programs at UC San Diego in the Sanford Stem Cell Institute include:

We are thrilled to announce the establishment of the UC San Diego Sanford Stem Cell Institute with Denny Sanfords generous support, said Jamieson. This will allow us to keep pace with the growing need for regenerative and stem-cell based therapies and accelerate translational stem cell research and discoveries that will transform human health for years to come.

With three new programs established as part of the Sanford Stem Cell Institute, a key focus of the institute will be leveraging space as a new frontier for stem cell science. Exposure to radiation and microgravity in low-Earth orbit can simulate and speed up aging in stem cells, as well as their transformation into cancer cells. Space-related research may have applications that create better treatments for various cancers and diseases on earth, including blood cancers, as well as neurodegenerative diseases such as Alzheimers and Parkinsons.

To fuel sustained research and education in this promising area, Sanfords gift will establish the Sanford Stem Cell Institute STELLAR Endowed Chair in Regenerative Medicine, the Sanford Stem Cell Institute Endowed STELLAR Exploration Faculty Scholars and Fellows Fund, and the Sanford Stem Cell Institute STELLAR Exploration Discovery Fund.

UC San Diego already has expanded its research capacity in stem cell science to space efforts that will be further amplified with the recent gift.

In late 2021, UC San Diego worked with NASA, Space Tango and the JM Foundation to launch stem cells into space aboard a SpaceX Falcon 9 rocket to study stress-induced aging and how stem cells and their progeny transform into pre-cancer and cancer stem cells associated with leukemia and other blood cancers.

Allyson Muotri, PhD, with human organoid samples

In 2019, Alysson Muotri, PhD, professor of pediatrics and cellular and molecular medicine, and colleagues sent a payload of stem cell-derived human brain organoids to the International Space Station (ISS) orbiting almost 250 miles above Earth to study how these masses of cells organize into the beginnings of a functional brain in microgravity. The first-ever project of its type was dedicated to Sanford, a longtime supporter of Muotris work and others.

When I was designing these experiments, I realized how innovative and cutting edge they were, said Muotri. I thought Denny would be proud of this project, and that I should dedicate this first mission to him. Denny has been a cheerleader for the stem cell community. He is pushing all of us to speed discovery and translate it to help millions of people who suffer from different conditions that could be treated with stem cell-based therapies.

Since its inception in 2013, the Sanford Stem Cell Clinical Center at UC San Diego has yielded a three-fold return on investment by obtaining more than $312 million in funding, including $253.6 million in grants, $15.8 million in clinical trial contracts, $2.7 million in Advanced Cell Therapy Lab (ACTL) service charges and more than $40.2 million in philanthropy all with the goal of discovering new treatments to benefit patients.

Key successes include new pharmaceutical treatments Fedratinib, which was approved by the FDA for the treatment of myelofibrosis in 2019, and Glasdegib, FDA approved for acute myeloid leukemia in 2018.

Meanwhile, clinical trials are ongoing for Cirmtuzumab, a monoclonal antibody-based drug developed by Thomas Kipps, MD, PhD, Distinguished Professor of Medicine and deputy director of research at Moores Cancer Center at UC San Diego Health, and colleagues. Cirmtuzumab targets cancer stem cells and is being tested, alone and in combination with other drugs, to treat chronic lymphocytic leukemia and other blood cancers.

Stem cell research at UC San Diego has been a substantial beneficiary of the California Institute for Regenerative Medicine (CIRM), the states stem cell agency, created in 2004 with the approval of Proposition 71. UC San Diego researchers have garnered 116 awards totaling more than $227 million. Cirmtuzumab is named as a nod to CIRM and its support. In 2020, California voters passed Proposition 14 to continue CIRM operations and funding.

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$150 Million Gift Takes Stem Cell Research to New Heights - University of California San Diego

Immusofts steps to delivering a treatment using engineered B cells. (Screen grab from Immusofts website)

Seattle biotech startup Immusoft has received approval to begin clinical trials of its novel strategy for treating genetic disease, the company announced Thursday. Immusoft says its the first to get permission to use engineered immune system cells called B cells in a human study.

The U.S. Food and Drug Administration (FDA) approved Immusofts Investigational New Drug Application for testing its immunotherapy for a rare, lethal childhood disease called MPS I.

An immune response includes a suite of players, and the B cells are responsible for producing the antibodies that stick to invading bacteria and viruses. The company is able to modify B cells into biofactories that instead crank out missing or non-functioning enzymes and proteins in the cells of patients.

This is a huge achievement for the company and a historic moment in the field of cell and gene therapies, said Sean Ainsworth, Immusofts CEO and chairman, in a statement.

The approach has potential advantages to current strategies for delivering treatments. Therapies that use a virus as its delivery mechanism can trigger immune responses that limit their effectiveness. Treatments using stem cells can have difficulties associated with chemotherapy and stem cell transplants.

Immusofts technology, called ISP-001, uses a patients own B cells, reprogramming them to make needed proteins. Other companies working on B cell therapies include Be Biopharma and Walking Fish Therapeutics.

I dont know if they are going to be successful, but its exciting for all of us that they have gotten permission to start a trial, researcher Richard James told MIT Technology Review. Jamess lab at the University of Washington is also working on B cell engineering.

The trial will be done at the University of Minnesota Medical School and led by Dr. Paul Orchard, a professor in the universitys Division of Pediatric Bone Marrow Transplantation.

Children with MPS I are not able to produce an essential enzyme that helps break down long-chain sugars inside cells. The sugars then build up in cells, causing progressive damage. Severe MPS1 occurs in about 1 in 100,000 births, and symptoms appear within a year.

Immusoft is interested in expanding its therapy to other rare diseases, as well as cardiovascular, autoimmune and central nervous system diseases.

In October 2021, the company announced a collaboration with pharma giant Takeda to develop treatments targeted to the nervous system in a deal worth potentially more than $900 million.

Immusoft was founded in 2009 and has raised more than $50 million in venture capital, according to PitchBook. In 2018, Ainsworth took over leadership from founder Matthew Scholz.

Scholz is now CEO of Oisn Biotechnologies, a startup developing preclinical therapies that target and kill damaged zombie cells. He is still on Immusofts board of directors.

Originally posted here:
Seattle biotech company is the first to receive approval to test B cell gene therapy in humans - GeekWire

SANTA MONICA, Calif.--(BUSINESS WIRE)--Kite, a Gilead Company (Nasdaq: GILD), today announced that the European Commission (EC) has approved its CAR T-cell therapy Tecartus (brexucabtagene autoleucel) for the treatment of adult patients 26 years of age and above with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL).

This approval makes Tecartus the first and only CAR T-cell therapy indicated for this population of patients, addressing a significant unmet medical need, said Christi Shaw, CEO, Kite. This is also the fourth indication in Europe for which a Kite cell therapy is approved, clearly demonstrating the benefits they offer to patients, especially those with limited treatment options.

ALL is an aggressive type of blood cancer; the most common form is B-cell precursor ALL. Globally, approximately 64,000 people are diagnosed with ALL each year. Half of adults living with ALL will relapse, and median overall survival (OS) with current standard-of-care treatments is approximately just eight months.

Adults with relapsed or refractory ALL often undergo multiple treatments including chemotherapy, targeted therapy and stem cell transplant, creating a significant burden on a patients quality of life, said Max S. Topp, MD, professor and head of Hematology, University Hospital of Wuerzburg, Germany. Patients in Europe now have a meaningful advancement in treatment. Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.

The approval is supported by data from the ZUMA-3 international multicenter, single-arm, open-label, registrational Phase 1/2 study of adult patients (18 years old) with relapsed or refractory ALL. This study demonstrated that 71% of the evaluable patients (n=55) achieved complete remission (CR) or CR with incomplete hematological recovery (CRi) with a median follow-up of 26.8 months. In an extended data set of all pivotal dosed patients (n=78) the median overall survival for all patients was more than two years (25.4 months) and almost four years (47 months) for responders (patients who achieved CR or CRi). Among efficacy-evaluable patients, median duration of remission (DOR) was 18.6 months.

Among the patients treated with Tecartus at the target dose (n=100) safety results were consistent with the known safety profile for Tecartus. Grade 3 or higher cytokine release syndrome (CRS) and neurologic adverse reactions occurred in 25% and 32% of patients, respectively, and were generally well managed.

About ZUMA-3

ZUMA-3 is an ongoing international multicenter (US, Canada, Europe), single arm, open label, registrational Phase 1/2 study of Tecartus in adult patients (18 years old) with ALL whose disease is refractory to or has relapsed following standard systemic therapy or hematopoietic stem cell transplantation. The primary endpoint is the rate of overall complete remission or complete remission with incomplete hematological recovery by central assessment. Duration of remission and relapse-free survival, overall survival, minimal residual disease (MRD) negativity rate, and allo-SCT rate were assessed as secondary endpoints.

About Acute Lymphoblastic Leukemia

ALL is an aggressive type of blood cancer that develops when abnormal white blood cells accumulate in the bone marrow until there isnt any room left for blood cells to form. In some cases, these abnormal cells invade healthy organs and can also involve the lymph nodes, spleen, liver, central nervous system and other organs.

About Tecartus

Please see full FDA Prescribing Information, including BOXED WARNING and Medication Guide.

Tecartus is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGIC TOXICITIES

Cytokine Release Syndrome (CRS), including life-threatening reactions, occurred following treatment with Tecartus. In ZUMA-2, CRS occurred in 92% (72/78) of patients with ALL, including Grade 3 (Lee grading system 1) CRS in 26% of patients. Three patients with ALL had ongoing CRS events at the time of death. The median time to onset of CRS was five days (range: 1 to 12 days) and the median duration of CRS was eight days (range: 2 to 63 days) for patients with ALL.. Among patients with CRS, the key manifestations (>10%) were similar in MCL and ALL and included fever (93%), hypotension (62%), tachycardia (59%), chills (32%), hypoxia (31%), headache (21%), fatigue (20%), and nausea (13%). Serious events associated with CRS included hypotension, fever, hypoxia, tachycardia, and dyspnea.

Ensure that a minimum of two doses of tocilizumab are available for each patient prior to infusion of Tecartus. Following infusion, monitor patients for signs and symptoms of CRS daily for at least seven days for patients with MCL and at least 14 days for patients with ALL at the certified healthcare facility, and for four weeks thereafter. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

Neurologic Events, including those that were fatal or life-threatening, occurred following treatment with Tecartus. Neurologic events occurred in 81% (66/82) of patients with MCL, including Grade 3 in 37% of patients. The median time to onset for neurologic events was six days (range: 1 to 32 days) with a median duration of 21 days (range: 2 to 454 days) in patients with MCL. Neurologic events occurred in 87% (68/78) of patients with ALL, including Grade 3 in 35% of patients. The median time to onset for neurologic events was seven days (range: 1 to 51 days) with a median duration of 15 days (range: 1 to 397 days) in patients with ALL. For patients with MCL, 54 (66%) patients experienced CRS before the onset of neurological events. Five (6%) patients did not experience CRS with neurologic events and eight patients (10%) developed neurological events after the resolution of CRS. Neurologic events resolved for 119 out of 134 (89%) patients treated with Tecartus. Nine patients (three patients with MCL and six patients with ALL) had ongoing neurologic events at the time of death. For patients with ALL, neurologic events occurred before, during, and after CRS in 4 (5%), 57 (73%), and 8 (10%) of patients; respectively. Three patients (4%) had neurologic events without CRS. The onset of neurologic events can be concurrent with CRS, following resolution of CRS or in the absence of CRS.

The most common neurologic events (>10%) were similar in MCL and ALL and included encephalopathy (57%), headache (37%), tremor (34%), confusional state (26%), aphasia (23%), delirium (17%), dizziness (15%), anxiety (14%), and agitation (12%). Serious events including encephalopathy, aphasia, confusional state, and seizures occurred after treatment with Tecartus.

Monitor patients daily for at least seven days for patients with MCL and at least 14 days for patients with ALL at the certified healthcare facility and for four weeks following infusion for signs and symptoms of neurologic toxicities and treat promptly.

REMS Program: Because of the risk of CRS and neurologic toxicities, Tecartus is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta and Tecartus REMS Program which requires that:

Hypersensitivity Reactions: Serious hypersensitivity reactions, including anaphylaxis, may occur due to dimethyl sulfoxide (DMSO) or residual gentamicin in Tecartus.

Severe Infections: Severe or life-threatening infections occurred in patients after Tecartus infusion. Infections (all grades) occurred in 56% (46/82) of patients with MCL and 44% (34/78) of patients with ALL. Grade 3 or higher infections, including bacterial, viral, and fungal infections, occurred in 30% of patients with ALL and MCL. Tecartus should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after Tecartus infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 6% of patients with MCL and 35% of patients with ALL after Tecartus infusion and may be concurrent with CRS. The febrile neutropenia in 27 (35%) of patients with ALL includes events of febrile neutropenia (11 (14%)) plus the concurrent events of fever and neutropenia (16 (21%)). In the event of febrile neutropenia, evaluate for infection and manage with broad spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, life-threatening and fatal opportunistic infections have been reported. The possibility of rare infectious etiologies (e.g., fungal and viral infections such as HHV-6 and progressive multifocal leukoencephalopathy) should be considered in patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

Prolonged Cytopenias: Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and Tecartus infusion. In patients with MCL, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 55% (45/82) of patients and included thrombocytopenia (38%), neutropenia (37%), and anemia (17%). In patients with ALL who were responders to Tecartus treatment, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 20% (7/35) of the patients and included neutropenia (12%) and thrombocytopenia (12%); Grade 3 or higher cytopenias not resolved by Day 60 following Tecartus infusion occurred in 11% (4/35) of the patients and included neutropenia (9%) and thrombocytopenia (6%). Monitor blood counts after Tecartus infusion.

Hypogammaglobulinemia: B cell aplasia and hypogammaglobulinemia can occur in patients receiving treatment with Tecartus. Hypogammaglobulinemia was reported in 16% (13/82) of patients with MCL and 9% (7/78) of patients with ALL. Monitor immunoglobulin levels after treatment with Tecartus and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement.

The safety of immunization with live viral vaccines during or following Tecartus treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least six weeks prior to the start of lymphodepleting chemotherapy, during Tecartus treatment, and until immune recovery following treatment with Tecartus.

Secondary Malignancies may develop. Monitor life-long for secondary malignancies. In the event that one occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

Effects on Ability to Drive and Use Machines: Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following Tecartus infusion. Advise patients to refrain from driving and engaging in hazardous activities, such as operating heavy or potentially dangerous machinery, during this period.

Adverse Reactions: The most common non-laboratory adverse reactions ( 20%) were fever, cytokine release syndrome, hypotension, encephalopathy, tachycardia, nausea, chills, headache, fatigue, febrile neutropenia, diarrhea, musculoskeletal pain, hypoxia, rash, edema, tremor, infection with pathogen unspecified, constipation, decreased appetite, and vomiting. The most common serious adverse reactions ( 2%) were cytokine release syndrome, febrile neutropenia, hypotension, encephalopathy, fever, infection with pathogen unspecified, hypoxia, tachycardia, bacterial infections, respiratory failure, seizure, diarrhea, dyspnea, fungal infections, viral infections, coagulopathy, delirium, fatigue, hemophagocytic lymphohistiocytosis, musculoskeletal pain, edema, and paraparesis.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kites singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Gilead and Kite to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving Tecartus; the risk that physicians may not see the benefits of prescribing Tecartus for the treatment of blood cancers; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for Tecartus including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com.

Kite, the Kite logo, Tecartus and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.

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Kite's CAR T-cell Therapy Tecartus Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Acute Lymphoblastic Leukemia...

The findings suggest stem cell-based replacement therapy has the potential to manage blood sugar levels and may eventually replace the requirement for external insulin injections or dosing.

A clinical study presented at ENDO 2022, the Endocrine Societys annual meeting in Atlanta, Georgia, suggests that an investigational stem cell-based therapy called PEC-Direct, intended to function as a replacement pancreas, has the potential to provide blood sugar control in patients with high-risk type 1 diabetes.

The research discovered that numerous individuals receiving the new treatment showed clinically significant increases in C-peptide, a substance produced in the pancreas alongside insulin. Measuring C-peptide can reveal how much insulin the body is producing since they are both released from the pancreas at the same time and insimilar quantities.

This research represents the first instance in multiple patients of clinically relevant increases in C-peptide, indicative of insulin production, with a stem cell-based therapy delivered in a device, according to Manasi Sinha Jaiman, M.D., M.P.H., Chief Medical Officer of ViaCyte, Inc., in San Diego, Calif., the company that makes PEC-Direct.

People with type 1 diabetes gradually lose the ability to produce insulin on their own, which is necessary for blood sugar regulation. Patients must check those levels on a regular basis using finger sticks, administer repeated insulin injections, or carry around cumbersome devices. Additionally, there is a chance that the insulin injection can inadvertently drop blood sugar to unsafe levels.

The PEC-Direct device is intended to provide a consistent, long-term sourceof insulin to control blood sugar levels. The system consists of a pouch containing pancreatic cells produced from stem cells that, when implanted in the body, develop into cells that produce insulin. The devices open membrane enables blood vessels to grow into it to contact the cells. Patients use immunosuppressive drugs to prevent an immune reaction.

The treatment is meant for patients with high-risk type 1 diabetes, who may be especially vulnerable to acute complications due to factors such as recurrent severe low blood sugar, or frequent and extreme blood sugar fluctuations that are difficult to control.

The study included 10 adults with type 1 diabetes who had received their diagnosis at least 5 years prior to the start of the study and were not able to tell when their blood sugar went too low (called hypoglycemia unawareness). Initial data from one patient showed clinically relevant levels of stimulated C-peptide and corresponding improvements in blood glucose control within six months after implantation of PEC-Direct.

Since then, increased C-peptide levels were seen in multiple patients, along with decreases in HbA1C (a blood test that measures average blood sugar levels over the past three months) by as much as 1.5%, and decreases in the amount of insulin patients needed to administer by as much as 70%.

The results suggest stem cell-based replacement therapy has the potential to provide blood glucose control and could one day eliminate the need for injecting or dosing insulin externally, Jaiman said. The study provides further proof-of-concept that continued optimization of PEC-Direct has promise as a functional cure for type 1 diabetes.

Meeting: ENDO 2022

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Could Stem-Cell Based Therapy Treat Type-1 Diabetes? A New Study Demonstrates the Treatments Potential - SciTechDaily

BOSTON and LONDON, Aug. 29, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced seven presentations from across its neurometabolic portfolio will be featured at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, taking place from August 30 to September 2, 2022, in Freiburg, Germany.

Featured presentations include an oral presentation on Libmeldy (atidarsagene autotemcel) from clinical development through approval by the European Commission and treatment of the first patients in a commercial setting in Europe, several accepted abstracts highlighting newborn screening efforts to support the timely and accurate diagnosis of metachromatic leukodystrophy (MLD), as well as an encore clinical data presentation from the companys investigational hematopoietic stem cell (HSC) gene therapy OTL-203 for MPS-IH.

The oral presentation details are as follows:

The poster presentation details are as follows:

About Libmeldy / OTL-200Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies of Libmeldy, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the U.S.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-looking StatementsThis press release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards most recent annual or quarterly report filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

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Orchard Therapeutics Announces Multiple Presentations at 2022 SSIEM Annual Symposium Highlighting Neurometabolic Disease Portfolio - GlobeNewswire

The global cancer stem cells market expected to grow by 2026 due to rising prevalence of cancer. The breast cancer sub-segment is expected to be highly profitable. Market in the North America region is expected to be highly lucrative.

NEW YORK, Aug. 19, 2022 /PRNewswire/ --Research Dive has added a new report to its offering titled, "Cancer Stem Cells Market, By Cancer Form (Breast, Blood, Lung, Brain, Colorectal, Pancreatic, Bladder, Liver), By Application (Targeted CSCs, Stem Cell Based Cancer Therapy): Global Opportunity Analysis and Industry Forecast, 20192026".

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According to the report, the global cancer stem cells market is expected to gather a revenue of $1,722.7 million by 2026, growing at a CAGR of 10.3% during the 2019-2026 timeframe.

Dynamics of the Cancer Stem Cells Market

Drivers: Increasing prevalence of cancer all across the globe is projected to be the primary growth driver of the cancer stem cells market in the forecast period. Additionally, the growth in awareness among people regarding cancer is expected to increase the growth rate of the market.

Opportunities: The extensive focus of various organizations and medical experts on developing new cancer treatments and improving the efficiency of the existing ones is predicted to offer huge growth opportunities to the market. Along with this, growth in the number of cancer patients all over the world is anticipated to push the market forward.

Restraints: Expensive cell therapy treatments may hamper the growth trajectory of the cancer stem cells market.

To get access to the Comprehensive PDF Sample [180-Pages] of Cancer Stem Cells Market Click here!

Segments of the Cancer Stem Cells Market

The report has divided the cancer stem cells market into a few segments based on cancer form, application, and regional analysis.

By cancer form, the breast cancer sub-segment of the cancer stem cells market is estimated to have a dominating share and garner $295.0 million by 2026. Changing lifestyle, hormonal changes, consumption of alcohol, etc. have led to an increase in prevalence of breast cancer in people worldwide, which is predicted to push the market forward.

By application, stem cell based cancer therapy sub-segment is anticipated to have a stunning growth rate and surpass $896.9 million by the end of 2026. Stem cell based cancer therapy comprises of allogenic stem cell therapy and autologous stem cell therapy, which are widely used for treating different types of cancers. This is expected to help the sub-segment grow in the forecast period.

By regional analysis, the cancer stem cells market in the North America region is predicted to flourish immensely and garner a revenue of $783.8 million by 2026. Medical experts and doctors in this region are quite involved in cancer related research and help in promotion of stem cell interventions, which is why the market is expected to grow at such a stunning rate in this region.

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Broader Insights Pertaining (Avail 10%OFF) of Specific Segments or Regions of Cancer Stem Cells Market

Significant Cancer Stem Cells Market Players

Some prominent market players of the cancer stem cells market are

AdnaGen GmbH

Epic Sciences, Inc.

Silicon Biosystems

S.p.A.

Advanced Cell Diagnostics, Inc.

Fluxion Biosciences, Inc.

AVIVA Biosciences Corporation

Rarecells USA, Inc.

Celula, Inc.

These players are developing various business strategies like product development and enhancement, merger and acquisition, partnerships and collaborations to gain a commanding position in the market. Buy the Full Report Here Started at $2999

For example, in January 2022, Century Therapeutics, a leading biotechnology company, announced a partnership with Bristol Myers Squibb, a multinational pharmaceutical company. The collaboration is aimed at developing four induced pluripotent stem cell derived programs in order to tackle malignant tumors. The partnership is expected to hugely benefit both the companies as they will be able to integrate their operations in a much better way in order to develop the intended program.

The report also sums up various crucial facets including SWOT analysis, product portfolio, financial performance of the key market players, and the latest strategic developments.

Checkout the Latest Insights of Cancer Stem Cells Market. Schedule a call with an Analyst

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Global Cancer Stem Cells Market Estimated to Reach $1,722.7 Million by 2026 and Grow at 10.3% CAGR in the 2019 to 2026 Timeframe | [180-Pages] Report...

When the decision is made to have a stem cell or bone marrow transplant, there are several steps in theprocess. The steps are much the same, no matter what type of transplant youre going to have.

You will first be evaluated to find out if you are eligible for a transplant. A transplant is very hard on your body. For many people, transplants can mean a cure, but for some people, problems can lead to severe complications or even death. Youll want to weigh the benefits and risks before you start.

Transplants can also be hard emotionally. They often require being in the hospital, being isolated, and theres a high risk of side effects. Many of the effects are short-term, but some problems can go on for years. This can mean changes in the way you live your life. For some people its just for a while, but for others, the changes may be lifelong. Some of the side effects are really unpleasant and can be serious. Your cancer care team will do everything they can to make you comfortable, but some of the side effects may not be completely controlled or relieved.

Before you have a transplant, you need to discuss the transplant process and all its effects with your doctors. It also helps to talk with others who have already had transplants.

Its also very hard going through weeks and months of not knowing how your transplant will turn out. This takes a lot of time and emotional energy from the patient, caregivers, and loved ones. Its very important to have the support of those close to you. For example, youll need a responsible adult who will be with you to give you medicines, help watch for problems, and stay in touch with your transplant team after you go home. Your transplant team will help you and your caregiver learn what you need to know. The team can also help you and your loved ones work through the ups and downs as you prepare for and go through the transplant.

Many different medical tests will be done, and questions will be asked to try to find out how well you can handle the transplant process. These might include:

You will also talk about your health insurance coverage and related costs that you might have to pay.

You may have a central venous catheter (CVC) put into a large vein in your chest. This is most often done as outpatient surgery, and usually only local anesthesia is needed (the place where the catheter goes in is made numb). Nurses will use the catheter to draw blood and give you medicines.

If youre getting an autologous transplant, a special catheter can be placed that can also be used when your stem cells are being removed or harvested.

The CVC will stay in during your treatment and for some time afterward, usually until your transplanted stem cells have engrafted and your blood counts are on a steady climb to normal.

Younger people, people who are in the early stages of disease, or those who have not already had a lot of treatment, often do better with transplants. Some transplant centers set age limits. Some people also may not be eligible for transplant if they have other major health problems, such as serious heart, lung, liver, or kidney disease. A mini-transplant, described under Allogeneic stem cell transplant in Types of Stem Cell Transplants for Cancer Treatment may be an option for some of these people.

The hospitals transplant team will decide if you need to be in the hospital to have your transplant, if it will be done in an outpatient center, or if you will be in the hospital just for parts of it. If you have to be in the hospital, you will probably go in the day before pre-transplant chemo or radiation treatment begins (see the next section), the transplant team makes sure you and your family understand the process and want to go forward with it.

If you will be having all or part of your transplant as an outpatient, youll need to be very near the transplant center during the early stages. Youll need a family member or loved one to be a caregiver who can stay with you all the time. You and the caregiver will also need reliable transportation to and from the clinic. The transplant team will be watching you closely for complications, so expect to be at the clinic every day for a few weeks. You may still need to be in the hospital if your situation changes or if you start having complications.

To reduce the chance of infection during treatment, patients who are in the hospital are put in private rooms that have special air filters. The room may also have a protective barrier to separate it from other rooms and hallways. Some have an air pressure system that makes sure no unclean outside air gets into the room. If youre going to be treated as an outpatient, you will get instructions on avoiding infection. Usually, people who have transplants are in a separate, special part of the hospital to keep as many germs away as possible.

The transplant experience can be overwhelming. Your transplant team will be there to help you prepare for the process physically and emotionally and to discuss your needs. Every effort will be made to answer questions so you and your family fully understand what will be happening to you as you go through transplant.

Its important for you and your family to know what to expect, because once conditioning treatment begins (see the next section), theres no going back there can be serious problems if treatment is stopped at any time during transplant.

Having a transplant takes a serious commitment from you and your caregiver and family, so it is important to know exactly what to expect.

Conditioning, also known as pre-transplant treatment,bone marrow preparation, or myeloablation, is usually treatment with high-dose chemo and/or radiation therapy. Its the first step in the transplant process and typically takes a week or two. Its done for one or more of these reasons:

The conditioning treatment is different for every transplant. Your treatment will be planned based on the type of cancer you have, the type of transplant, and any chemo or radiation therapy youve had in the past.

If chemo is part of your treatment plan, it will be given in your central venous catheter and/or as pills. If radiation therapy is planned, its given to the entire body (called total body irradiation or TBI). TBI may be given in a single treatment session or in divided doses over a few days.

This phase of the transplant can be very uncomfortable because very high treatment doses are used. Chemo and radiation side effects can make you sick, and it may take you months to fully recover. A very common problem is mouth sores that will need to be treated with strong pain medicines. You may also have nausea, vomiting, be unable to eat, lose your hair, and have lung or breathing problems.

Conditioning can also cause premature menopause in women and often makes people sterile (unable to have children). (See Stem Cell Transplant Side Effects.)

After the conditioning treatment, youll be given a couple of days to rest before getting the stem cells. They will be given through your central venous catheter, much like a blood transfusion. If the stem cells were frozen, you might get some drugs before the stem cells are given. These drugs are used to help reduce your risk of reacting to the preservatives that are used when freezing the cells.

If the stem cells were frozen, they are thawed in warm water then given right away. There may be more than 1 bag of stem cells. For allogeneic or syngeneic transplants, the donor cells may be harvested (removed) in an operating room, and then processed in the lab right away. Once they are ready, the cells are brought in and given to you theyre not frozen. The length of time it takes to get all the stem cells depends on how much fluid the stem cells are in.

You will be awake for this process, and it doesnt hurt. This is a big step and often has great meaning for patientsand their families. Many people consider this their rebirth or chance at a second life. They may celebrate this day as they would their actual birthday.

Side effects from the infusion are rare and usually mild. The preserving agent used when freezing the stem cells causes many of the side effects. For instance, you might have a strong taste of garlic or creamed corn in your mouth. Sucking on candy or sipping flavored drinks during and after the infusion can help with the taste. Your body will also smell like this. The smell may bother those around you, but you might not even notice it. The smell, along with the taste, may last for a few days, but slowly fades away. Often having cut up oranges in the room will offset the odor. Patients who have transplants from cells that were not frozen do not have this problem because the cells are not mixed with the preserving agent.

Other side effects you might have during and right after the stem cell infusion include:

Again, side effects are rare and usually mild. If they do happen, they are treated as needed. The stem cell infusion must always be completed.

The recovery stage begins after the stem cell infusion. During this time, you and your family wait for the cells to engraft, or take, after which they start to multiply and make new blood cells. The time it takes to start seeing a steady return to normal blood counts varies depending on the patient and the transplant type, but its usually about 2 to 6 weeks. Youll be in the hospital or visit the transplant center daily for a number of weeks.

During the first couple of weeks youll have low numbers of red and white blood cells and platelets. Right after transplant, when your counts are the lowest, you may be given antibiotics to help keep you from getting infections. You may get a combination of anti-bacterial, anti-fungal, and anti-viral drugs. These are usually given until your white blood cell count reaches a certain level. Still, you can have problems, such as infection from too few white blood cells (neutropenia), or bleeding from too few platelets (thrombocytopenia). Many patients have high fevers and need IV antibiotics to treat serious infections. Transfusions of red blood cells and platelets are often needed until the bone marrow starts working and new blood cells are being made by the infused stem cells.

Except for graft-versus-host disease, which only happens with allogeneic transplants, the side effects from autologous, allogeneic, and syngeneic stem cell transplants are much the same. Problems may include stomach, heart, lung, liver, or kidney problems. (Stem Cell Transplant Side Effects goes into the details.) You might also go through feelings of distress, anxiety, depression, joy, or anger. Adjusting emotionally after the stem cells can be hard because of the length of time you feel ill and isolated from others.

You might feel as if you are on an emotional roller coaster during this time. Support and encouragement from family, friends, and the transplant team are very important to get you through the challenges after transplant.

The discharge process actually begins weeks before your transplant. It starts with the transplant team teaching you and your primary (main) caregiver about:

For the most part, transplant centers dont send patients home until they meet the following criteria:

(Why Are Stem Cell Transplants Used as Cancer Treatment? has more information about neutrophils, platelets, and hematocrit).

If you do not meet all of these requirements, but still dont need the intensive care of the transplant unit, you might be moved to another oncology unit. When you do go home, you might need to stay near the transplant center for some time, depending on your condition.

The process of stem cell transplant doesnt end when you go home. Youll feel tired, and some people have physical or mental health problems in the rehabilitation period. You might still be taking a lot of medicines. These ongoing needs must now be managed at home, so caregiver and friend/family support is very important.

Transplant patients are followed closely during rehab. You might need daily or weekly exams along with things like blood tests, and maybe other tests, too. During early rehab, you also might need blood and platelet transfusions, antibiotics, or other treatments. At first youll need to see your transplant team often, maybe even every day, but youll progress to less frequent visits if things are going well. It can take 6 to 12 months, or even longer, for blood counts to get close to normal and your immune system to work well. During this time, your team will still be closely watching you.

Some problems might show up as much as a year or more after the stem cells were infused. They can include:

Other problems can also come up, such as:

Your transplant team is still there to help you, even though the transplant happened months ago. Its important that you tell them about any problems you are having they can help you get the support you need to manage the changes that you are going through. They can also help you know if problems are serious, or a normal part of recovery. The National Bone Marrow Transplant Link helps patients, caregivers, and families by providing information and support services before, during, and after transplant. They can be reached at 1-800-LINK-BMT (1-800-546-5268) or online at http://www.nbmtlink.org.

Read more:
Getting a Stem Cell or Bone Marrow Transplant - American Cancer Society

A South Korean biotech company says it will use natural killer (NK) cells, exosomes and brown adipose-derived stem cells (ADSC) as treatment of long COVID.

NK cells are a type of immune cell that have granules or small particles that can kill tumor cells or those infected with a virus. Panacell Biotech said they can be used to treat those with terminal illness as well as those with long COVID or post COVID-19 conditions.

The company isSouth Koreasresearch institute specializing in advanced regenerative medical cell therapy using adipose-derived stem cells (ADSC).

It announced today (August10)that it will soon conduct those cells toxicity tests through clinical trials and laboratory animals.

Currently, inSouth Korea, there are guidelines for plasma treatment that administers plasma from patients who have recovered from COVID-19 to other patients. Although there already exist COVID-19 treatments, such as Paxlovid,aclear therapeutic effect has not been confirmed yet.

There are more than 60 long COVID conditions.

According to the Mayo Clinic, one in four people aged 65 or above suffer from aftereffects of COVID-19.

It has been reported that people with long COVID often experience an extremely broad variety of symptoms, including less well-known side effects such as amnesia, and an inability to perform familiar movements or commands.

Panacell said it is believed that about four million people or 2.4% of the U.S. employed population have reduced ability to work because of long COVID.

Associate professor Gwenalle Douaud at the Nuffield Department of Clinical Neurosciences (NDCN),University of Oxford, said her team observed a greater reduction in grey matter thickness and tissue contract in the orbitofrontal cortex and parahippocampal gyrus.

She said: We saw greater changes in markers of tissue damage in regions that are functionally connected to primary olfactory cortex.

While the long-term effects of COVID-19 on smell remaininconclusive, the study suggests a possible connection between brain changes by COVID-19 and memory.

The company says there have been many clinical results in which the coronavirus causes inflammation in various organs, including the respiratory system, and chronic symptoms persist.

Sun Yanrong, deputy director of the China Biotechnology Development Center, said: We are continuing to monitor the treatment using stem cells. InWuhan, over 200 patients have already been treated with stem cells.

The clinical treatment results show that the stem cell therapy has good safety and has also been confirmed to have a therapeutic effect. It was also effective in recovering the lungs.

Seung-Ho Choi, CEO of Panacell Biotech, added: We expect that this clinical trial will reveal therapeutic effects of stem cell therapy along with these treatments.

Panacell Biotech is a bio institute in advanced regenerative medicine and cell therapy, recognized for its contributions to the development of biotechnology by researching stem cell culture and cell banking, focusing on the development of treatments for various cancers and incurable diseases.

Excerpt from:
Stem cell therapy to be used in treatment of long COVID by Panacell Biotech - Labiotech.eu

Selma Blair reveals how she's changed her approach to beauty and makeup since her 2018 multiple sclerosis diagnosis.

During an interview with InStyle, the 50-year-old actress got candid about her new approach to life and how her chronic disease had inspired her to create a beauty brand after undergoing her medical treatments.

"After I had treatment, I'd honestly, like, stopped looking in the mirror," Blair confessed. "My hair was short, I was bloated, I had alopecia on my lashes and my face. And it was all so much effort I sometimes don't see well so I just stopped, and I didn't think I missed it."

Although the side effects may have kept her from looking in the mirror, the "Legally Blonde" star insists that she never viewed makeup or self-care as "frivolous."

Selma Blair Opens Up About Alleged Assault by Now Ex-Boyfriend That Landed Her in Hospital View Story

Rather, she revealed that beauty, fashion and makeup had always been powerful tools in her self-care kit. Developing products for her brand Guide Beauty that were still accessible to use after the effects of her MS diagnosis became "a real turning point."

"I've never thought of fashion, makeup really, any of our ways of self-care and presenting ourselves as frivolous," she said "It was a real turning point for me, it was like, 'OK, let's start waking up again.'"

Guide Beauty features a collection of makeup and beauty tools that "invite everybody to the table" and are easy to use for people who suffer from MS, chronic diseases or have other disabilities.

"You're not designing for an 'us' or a 'them,'" she explained. "You're just looking to design for that beautiful 'we' moment: When we all get to play in the same world together with makeup."

Back in August 2021, Blair gave an update on her battle with MS during her virtual appearance at Discovery+'s TCA presentation. At the time she revealed that she was "in remission" after she had a stem cell transplant and underwent chemotherapy.

Selma Blair 'Told to Make Plans for Dying' in Incredibly Moving Documentary Trailer View Story

"My prognosis is great. I'm in remission. Stem cell put me in remission," the actress said per PEOPLE. "It took about a year after stem cell [transplant] for the inflammation and lesions to really go down."

"I was reluctant to talk about it because I felt this need to be more healed and more fixed," she added. "I've accrued a lifetime of some baggage in the brain that still needs a little sorting out or accepting. That took me a minute to get to that acceptance. It doesn't look like this for everyone."

"I have really felt unwell and misunderstood for so long that it's just, me," Blair said.

"The Sweetest Thing" actress added that being diagnosed with MS "can be very isolating," but said she was fortunate to have a strong support system.

"People took great care of me. I never really like life. I do now -- strange, huh?" she said. "Just because life's so weird. I was so scared in life. To suddenly start to find an identity and a safety in me, to figure out boundaries, time management and energy. I'm having the time of my life."

Excerpt from:
Selma Blair 'Stopped Looking in the Mirror' After MS Treatments - TooFab

Peer-reviewed publication in Cell Stem Cell unveils a novel differentiation process from Dr. Daleys lab to develop mature immune cells from induced pluripotent stem cells (iPSCs), a potentially disruptive advantage in the development of allogeneic iPSC-derived immunotherapies

New company to leverage ElevateBios unique iPSC platform and ecosystem of enabling technologies and manufacturing capabilities to create allogeneic immune cell therapies

First company to emerge from the previously announced five-year cell and gene therapy collaboration between Boston Childrens Hospital and ElevateBio

WALTHAM, Mass., August 04, 2022--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering transformative cell and gene therapies, today announced that it has formed a new company co-founded by George Daley, M.D, Ph.D., and Boston Childrens Hospital to develop allogeneic immune cell therapies based on a novel platform that generates functionally mature immune cells from induced pluripotent stem cells (iPSCs). This proprietary differentiation process overcomes the tendency of iPSCs to generate immature, embryonic blood cell types, and enables the generation of multiple subtypes of immune cells that display mature molecular signatures similar to T cells from adult blood. The peer-reviewed publication in the journal Cell Stem Cell showed that iPSC-derived mature T cells exhibited antitumor activity and cytokine secretion and could serve as an ideal source for the development of allogeneic "off-the-shelf" therapies.

This is the first company to emerge from the previously announced five-year collaboration between Boston Childrens Hospital and ElevateBio to accelerate the development of novel cell and gene therapies.

"At ElevateBio, we have been rapidly building fully integrated end-to-end cell and gene therapy enabling technologies and capabilities to enable the worlds most prolific scientific innovators and visionaries, such as Dr. Daley, to push the boundaries of the field and deliver powerful new therapeutic modalities for patients," said David Hallal, Chairman and CEO of ElevateBio. "This exciting new company is the first from our collaboration with Boston Childrens Hospital and is a prime example of how our integrated ecosystem our iPSC cell lines and process development capabilities, our gene editing technology, our CAR and TCR constructs, and our scale-up manufacturing capabilities creates the perfect foundation needed to turn these scientific breakthroughs into transformational medicines."

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"CAR-T therapies have revolutionized the treatment of certain blood cancers, with complete responses in many patients undergoing this type of treatment. However, current treatment strategies involve manufacturing a customized CAR-T cell product for each individual patient, which is cumbersome and labor-intensive," said Daley, senior author of the newly published Cell Stem Cell paper and whose lab is at Boston Childrens Hospital. "Our science offers an approach to circumvent these hurdles by offering an entirely new way of making allogeneic immune cell therapies that could pave the way for powerful treatments for a wide range of cancers."

Key findings shared in Cell Stem Cell publication: "EZH1 repression generates mature iPSC-derived CAR T cells with enhanced antitumor activity" (1):

A novel differentiation process discovered in Dr. George Daleys lab at Boston Childrens Hospital has been shown to promote definitive hematopoiesis and supports efficient production of mature T cells

This novel differentiation process incorporates repression of the histone methyltransferase EZH1 during iPSC differentiation to facilitate T cell maturation. The Daley lab showed previously that EZH1 is a negative regulator of lymphoid potential during embryonic blood development.

iPSC-T cells derived in a stroma-free, serum-free system following repression of EZH1, referred to as EZ-T cells, showed a molecular signature more closely resembling mature TCR T cells found in adult blood. Single cell RNA sequencing showed that activated EZ-T cells give rise to high levels of memory T cells, which promotes T cell longevity and may be essential for durable remissions in cancer patients.

In vitro studies showed EZ-T cells engineered to express anti-CD19 Chimeric Antigen Receptors (CARs) exhibited cytotoxic and cytokine-producing effector functions against CD19+ lymphoid tumor cells comparable to CAR-T cells engineered from adult blood.

In a xenograft mouse model injected with CD19+ diffuse large B-cell lymphoma (DLBCL) cells, EZ-T cells expressing anti-CD19 CARs demonstrated increased anti-tumor activity versus traditional iPSC-T cells generated without EZH1 knockdown.

(1) Jing, R., Scarfo, I., Daley, G., EZH1 repression generates mature iPSC-derived CAR T cells with enhanced antitumor activity, Cell Stem Cell, 2022

About ElevateBio:

ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, BaseCamp is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

For more information, visit us at http://www.elevate.bio, or follow Elevate on LinkedIn , Twitter, or Instagram.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220804005758/en/

Contacts

Investors: Catherine Huchu@elevate.bio

Media: Courtney HeathScientPRCourtney@scientpr.com

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ElevateBio Announces the Formation of a New Company With George Daley, M.D., Ph.D., and Boston Childrens Hospital to Develop iPSC-Derived Allogeneic...