Rocket Pharmaceuticals received the rare pediatric disease priority review voucher with the accelerated FDA approval for Kresladi in March. The non-dilutive capital from the voucher’s sale will support a pipeline that includes a gene therapy in pivotal clinical testing for the rare genetic disorder Danon disease.
The rise of metabolic gene therapies represents a critical juncture for benefits management. These treatments offer genuine hope for patients with serious conditions, but they also pose significant financial challenges.
Regeneron Pharmaceuticals’ Otarmeni is now approved for treating hearing loss from an ultra-rare genetic mutation found in an estimated 50 newborns per year. While Regeneron will offer this gene therapy for free, the approval came with a rare pediatric disease priority review voucher that the company can sell for hundreds of millions of dollars.
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.
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.
Veradigm examines key clinical trends, comorbidity profiles, and treatment trends across adolescence, reproductive years, and peri-/post-menopause. Download it today!
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.
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.”
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
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.
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.