Rocket Pharmaceuticals received FDA approval for Kresladi, a gene therapy developed to treat leukocyte-adhesion deficiency type 1, an inherited immunodeficiency that can become fatal to infants and young children. Kresladi is the first gene therapy for this ultra-rare disease and the first commercial product for Rocket.
As cell-based therapies grow more complex and manufacturing networks expand, resilience can’t be an afterthought, and cryopreservation can no longer just be a step that gets validated during development and left alone. It needs to play an ongoing role in product consistency, potency, and reliability.
MedCity News was at the Vive conference and spoke with executives who shared their insights for the healthcare industry.
Tests of two Regenxbio gene therapies have been placed under an FDA clinical hold after a patient in one of the studies developed cancer. Our recap of recent regulatory news also includes one clinical hold removed, several complete response letters, and drug approvals in the U.S. and Europe.
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.
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.
Artera President Tom McIntyre talks about the practical application of AI in healthcare.
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.
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.
Zelis highlights how employers, payers, and consumers are integrating digital tools to manage healthcare costs.
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.
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.