July 2nd 2025
FDA officials have determined that information regarding the risks for six CAR T-cell therapies can be communicated through product labeling, which includes a boxed warning for the risks of cytokine release syndrome and neurological toxicities.
Breakthrough Gene Therapy Saves Infant With Rare Metabolic Disease
May 19th 2025A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ammonia buildup in the blood. The technology used creates an opportunity for the development of personalized treatments for other rare diseases.
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Lack of Social Support Top Barrier to Cell and Gene Therapy Access, Survey Finds
May 12th 2025Healthcare providers are exploring ways to reduce patient travel through a growing trend of expanding sites of administration beyond academic medical centers into community and outpatient settings closer to patients' homes.
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FDA Approves First-Ever Cell Therapy Treatment for Rare Progressive Eye Disease
March 20th 2025Encelto is small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells genetically engineered to produce specific therapeutic proteins. It will be available in June 2025.
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Solid Biosciences Reports Positive Data from DMD Gene Therapy Trial
February 19th 2025Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively to muscle cells.
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Gene Therapy’s Pipeline Has a Steady Flow of More Than 2,000 Candidates
January 31st 2025The majority are in the very early preclinical stage of development, but applications for regulatory approval have been submitted for 11 gene therapies worldwide, and 35 are in phase 3 trials, according to report delineating the gene therapy development landscape.
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Patients Express Concerns Over Costs, Risks of Gene Therapies for Sickle Cell Disease
December 12th 2024Research at the annual meeting of the American Society of Hematology evaluated patient and caregiver perspectives on gene therapies for sickle cell disease, which offer great potential but have had slow uptake.
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Gene Therapy for Rare Pediatric Brain Disease Shows Long-Term Benefit But Also Underscores Risks
October 18th 2024One of two new studies found most patients doing well six years after treatment. Another study found blood cancers in 17% of patients given the therapy using a slightly different protocol.
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