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.
There are a number of criteria for selecting the best autism therapy centers. Parents choosing a facility or professionals seeking to improve their offerings should consider certain standards to ensure the right fit.
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.
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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.
Sarepta Therapeutics said the Duchenne muscular dystrophy patient recently had a cytomegalovirus infection, which can damage the liver and may have contributed to the fatality. It’s the first patient death out of more than 800 patients treated with Elevidys, a gene therapy for the rare muscle-wasting disease.
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MeiraGTx’s gene therapy for Parkinson’s disease will enter Phase 3 testing under a new joint venture the biotech is forming with Hologen, a generative AI startup. The joint venture has up to $230 million in committed capital; MeiraGTx gets additional cash for its pipeline.
Neurotech Pharmaceuticals’ surgically implanted cell and gene therapy, Encelto, is now FDA approved to treat macular telangectasia type 2 (MacTel). It’s the first therapy for this rare retinal degeneration disorder.
Carlyle and SK Capital Partners are buying beleaguered gene therapy biotech Bluebird Bio for about $29 million. Meanwhile, Pfizer continued the culling of its gene therapies with the discontinuation of Beqvez for hemophilia B.
Blood centers are neutral partners, as they do not compete with hospitals for patients. Instead, they complement hospitals by providing a focused array of high-quality, specialized, patient-centric services, allowing hospitals to maintain primary relationships with patients and their families.
Astellas Pharma still looks to grow in cancer with a pipeline that includes internally developed programs that could stand out in oncology. But the drugmaker sees additional growth opportunities in ophthalmology and neuromuscular disorders, Chief Medical Officer Tadaaki Taniguchi said.