Waskyra is the first FDA-approved gene therapy for the rare disease Wiskott-Aldrich syndrome and the first such approved product from a non-profit applicant, Fondazione Telethon. Data from Waskyra’s pivotal studies were presented this week during the annual meeting of the American Society of Hematology.
The growing use of advanced analytics and artificial intelligence (AI) is reshaping this landscape by enabling data-driven process control, predictive manufacturing, and greater transparency across the development lifecycle.
Alan Murray on improving access for medical transportation.
Eli Lilly is paying $75 million up front for global rights to AAV-AIPL1, a gene therapy on track for U.S. and European regulatory submissions in the rare, inherited eye disorder Leber congenital amaurosis 4, or LCA4. The deal brings to Lilly another late-stage gene therapy for the eyes following the pharma giant’s October acquisition of Adverum Biotechnologies and its lead program for wet AMD.
The six new drugs awarded FDA Commissioner’s National Priority Vouchers brings to 15 the total number of therapies selected for the new pilot program. Under this program, the FDA says the standard 10- to 12-month review time could be shaved down to “within months.”
Recent developments, including a high-profile acquisition and key regulatory changes, point to a paradigm shift. Cell and gene therapy is becoming increasingly commercially viable, with the potential to improve the lives of millions.
Rocket Pharmaceuticals voluntarily withdrew the FDA submission for its Fanconi anemia gene therapy. The move follows a corporate restructuring over the summer that refocused the company on its cardiovascular programs.
Greenway Health blends smart technology with human expertise to elevate revenue cycle.
UniQure plans a first quarter 2026 FDA submission for AMT-130 as a treatment for Huntington’s disease. This gene therapy delivers microRNA designed to silence the mutated gene that produces a protein driving this disorder, which so far has no FDA-approved therapies.
Hansa Biopharma has encouraging early clinical results showing its drug imlifidase, used as a pretreatment, enabled Duchenne muscular dystrophy patients to receive the gene therapy Elevidys. Sarepta is searching for ways to mitigate excessive immune responses to Elevidys that can lead to serious and potentially fatal liver injury.
Sarepta Therapeutics knew about the death of a patient treated with its experimental gene therapy for a type of limb-girdle muscular dystrophy, but said nothing publicly for a month. Financial analysts lambasted Sarepta executives for failing to disclose the fatality when the company announced a restructuring that includes stopping work on this limb-girdle program.
Sarepta’s Elevidys will remain on the market with a black box warning. But this gene therapy for Duchenne muscular dystrophy still faces commercial headwinds and analysts say the company has work to do to win over patients and investors.
In a landscape where complexity has long been the norm, the power of one lies not just in unification, but in intelligence and automation.
Like the first death reported in March, Sarepta Therapeutics attributed the fatality to a case of acute liver failure following dosing of the Duchenne muscular dystrophy gene therapy, Elevidys. The company has proposed an immunosuppressive regimen to mitigate the liver injury risk posed by the therapy.
Rocket Pharmaceuticals adjusted the clinical trial design to include use of approved drug to avert an excessive immune response to its Danon disease gene therapy. The patient who died was one of two who received this drug under the revised protocol.
Johnson & Johnson’s gene therapy for X-linked retinal pigmentosa did not meet the main vision goal of a pivotal study. J&J secured full rights to the program from gene therapy biotech MeiraGTx in 2023.
Abeona Therapeutics makes Zevaskyn by engineering a patient’s biopsied skin cells to express the collagen that they lack, then growing those cells into sheets that can be surgically applied to a wound. Abeona's cell-based gene therapy is just the third FDA-approved treatment for epidermolysis bullosa, a rare disease that leads to skin described as thin and fragile like a butterfly’s wings.
As the FDA’s top biologics official, Peter Marks oversaw regulation of cell and gene therapies. The departure of Marks and mass layoffs throughout the FDA raise concerns about the agency’s ability to review and potentially approve such treatments.