All articles tagged with #cell therapy
The FDA issued a draft guidance showing how sponsors can reuse existing public data—such as CMC, nonclinical and clinical results—to streamline regulatory submissions for somatic genome-editing therapies, with the goal of accelerating access for patients with rare or life-threatening diseases while maintaining safety and efficacy; sponsors are encouraged to engage early (e.g., INTERACT) and submit comments within 90 days.
Eli Lilly agreed to pay up to $7 billion for Kelonia Therapeutics, marking another high-profile in vivo CAR-T deal as Big Pharma bets on the space; analysts say deal activity may slow due to a shrinking pool of viable targets, with Umoja Biopharma identified as a prime remaining buyout candidate and offshore players like Starna Therapeutics likely to surface. The pace will hinge on Kelonia’s data and whether the platform can extend beyond oncology into autoimmune indications.
UCB agreed to acquire Neurona Therapeutics for $650 million in cash plus up to $500 million in milestones, expanding into regenerative medicine with Neurona's NRTX-1001 neuronal cell therapy in phase I/II trials for drug-resistant mesial temporal lobe epilepsy, with closing expected by quarter's end.
Researchers wrapped mitochondria in red blood cell membranes to shield them from destruction and facilitate cellular entry. In a Leigh syndrome mouse model, this capsule-like delivery increased survival by about two weeks (roughly 20%), with ~80% of cells taking up the mitochondria—significantly higher than naked mitochondria. The approach preserves the mitochondria’s membrane potential, marking a major efficiency advance, though some scientists caution that claims such as preventing Parkinson’s disease in mice may be overstated.
Johnson & Johnson announced more than a $1 billion investment to build a next-generation cell-therapy manufacturing facility in Lower Gwynedd, Montgomery County, Pennsylvania, expanding U.S. manufacturing capacity, creating 500+ biomanufacturing jobs and up to 4,000 construction roles, as part of a broader $55 billion U.S. investment through 2029 to advance cancer, immune-mediated, and neurological therapies.
Researchers have developed a technology to reprogram rogue T cells into protective Treg cells, demonstrating targeted therapy for autoimmune diseases like pemphigus vulgaris, inflammatory bowel disease, and graft-versus-host disease in mouse models, potentially leading to safer, personalized treatments.
Bristol Myers Squibb is acquiring Orbital Therapeutics for $1.5 billion to enhance its cell therapy portfolio with next-generation RNA medicines, including the investigational CAR T-cell therapy OTX-201, aimed at autoimmune diseases, leveraging Orbital's innovative RNA platform for more accessible and effective treatments.
The FDA's biologics center has released new draft guidances aimed at helping developers streamline the creation of innovative cell and gene therapies for small patient populations.
Researchers have successfully implanted CRISPR-edited pancreatic cells into a person with type 1 diabetes, enabling the cells to produce insulin and evade immune detection without immunosuppressive drugs, marking a significant step toward a potential cure for the disease. However, the study involved only one patient and more research is needed to confirm efficacy.
The FDA rejected Capricor Therapeutics' application for its cell therapy deramiocel aimed at treating Duchenne muscular dystrophy, citing insufficient evidence of effectiveness and requesting more clinical data.
Recent leadership changes at the FDA, including the removal of top officials overseeing cell and gene therapies, have created uncertainty in the biotech sector, impacting stock prices and raising questions about the future regulatory landscape for genetic medicines, especially following disagreements over drug approvals like Sarepta's Elevidys.
The FDA's chief regulator of cell and gene therapies was ousted following a disagreement over the review process for a Duchenne muscular dystrophy therapy and ongoing management tensions, highlighting internal conflicts within the agency.
At a June 5 FDA roundtable, officials and experts discussed streamlining regulations to accelerate the development of cell and gene therapies, with suggestions including adopting China's two-tier system and reducing bureaucratic delays, amid concerns over international competition and innovative breakthroughs like CRISPR treatments for rare diseases.
Chinese scientists have developed a pioneering CAR-T cell therapy that offers the potential for a one-time treatment for asthma, a condition that currently requires lifelong management. This therapy, which targets key cytokines driving asthma, has shown promising results in animal studies, providing long-term relief from symptoms. While the findings are encouraging, experts stress the need for further research to ensure the therapy's safety and efficacy in humans.
Chinese scientists have reported a potential cure for diabetes using innovative cell therapy, successfully treating a 59-year-old man with type 2 diabetes who became insulin-independent within eleven weeks of a cell transplant. The patient has remained medication-free for over 33 months, marking a significant breakthrough, though further large-scale trials are needed to confirm the treatment's efficacy and safety.