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.”
Sheila Bond, MD, talked about the latest trends regarding integration of AI in healthcare.
The FDA has approved UCB’s Kygevvi for treating children and adults with thymidine kinase 2 deficiency, an inherited mitochondrial disorder. It’s the drugmaker’s first ultra-rare disease product.
Sarepta Therapeutics attributed the Phase 3 failure to the Covid-19 pandemic, during which many patients missed multiple doses of its Duchenne muscular dystrophy therapies. The company plans to discuss with the FDA traditional regulatory approvals based on the totality of data that includes real world evidence from the years these drugs have been commercially available under accelerated approvals.
The FDA clinical hold covers two Phase 3 studies for nexiguran ziclumeran, or nex-z, an experimental gene-editing therapy for transthyretin amyloidosis. Nex-z uses the CRISPR gene-editing technology to inactivate the gene that codes for the protein driving this rare disease.
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.
How Apella leverages technology to increase OR efficiency.
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.
Ionis Pharmaceuticals plans a 2026 FDA submission for zilganersen as a treatment for Alexander disease, an ultra-rare inherited neurological disorder. The antisense oligonucleotide developer has been moving toward commercializing drugs on its own rather than seeking partners.
With positive Phase 1 results for the metabolic disorder drug MZE782, Maze Therapeutics plans to advance the small molecule to Phase 2 tests in phenylketonuria and chronic kidney disease. Following the data readout, Maze raised $150 million in a private placement.
Cemdisiran, a small interfering RNA drug that Regeneron Pharmaceuticals licensed from Alnylam Pharmaceuticals, met the goals of its pivotal test in generalized myasthenia gravis. The rare neuromuscular disease has several new therapies, but analysts see cemdisiran as having advantages over one particular class of drugs.
Richard Fu details the company's approach to nutrition therapy and strategy for patients using GLP-1s.
Precigen’s Papzimeos is the first FDA-approved drug for the rare disease recurrent respiratory papillomatosis, or RRP. While this immunotherapy carries blockbuster expectations, Precigen’s financial reports indicate budgetary challenges.
The FDA approved Ionis Pharmaceuticals’ Dawnzera for preventing swelling attacks caused by the rare disease hereditary angioedema. A Takeda Pharmaceutical product dominates this market, but Ionis has clinical data showing patients had better outcomes after switching to Dawnzera from Takeda’s drug and other currently available HAE medications.
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.
Though anselamimab failed its Phase 3 test in light chain amyloidosis, AstraZeneca points to encouraging results in a pre-defined patient subgroup — which may not be a winning strategy. Prothena resurrected its failed drug candidate for the disease based on a subgroup analysis, only to see that antibody fall short in a pivotal study.
Capricor Therapeutics said the FDA asked for more data supporting the efficacy of Deramiocel, the biotech’s off-the-shelf cell therapy for treating the heart complications of Duchenne muscular dystrophy. Those data could come from an ongoing Phase 3 study expected to yield preliminary results soon.