Neurophth Therapeutics Inc. has received FDA clearance of its IND application for the in vivo gene replacement therapy NFS-02, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized NADH-dehydrogenase subunit 1 (ND1) gene, for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND1 mutation.
Ferring Pharmaceuticals A/S has notched another U.S. FDA approval, this time for a bladder cancer treatment, Adstiladrin (nadofaragene firadenovec). The non-replicating adenovirus vector-based gene therapy’s approval comes only weeks after the FDA’s Nov. 30 approval of the privately held company’s Rebyota (fecal microbiota, live), the first fecal microbiota treatment in the U.S. Adstiladrin is another landmark, as the first FDA-approved gene therapy to treat high-risk, non-muscle-invasive bladder cancer. Saint-Prex, Switzerland-based Ferring said it anticipates the product becoming commercially available in the U.S. in the second half of 2023.
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding.
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
Eikonoklastes Therapeutics Inc. and Forge Biologics Inc. have established a manufacturing partnership to advance Eikonoklastes' adeno-associated viral (AAV)-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS).
The FDA has awarded orphan drug designation to Sensorion SA's OTOF-GT, a dual vector AAV gene therapy, for the treatment of otoferlin gene-mediated hearing loss.
The FDA has awarded orphan drug designation to Tenaya Therapeutics Inc.'s gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC). TN-401 is an adeno-associated virus (AAV)-based gene therapy being developed for the treatment of genetic ARVC caused by plakophilin-2 (PKP2) gene mutations.
The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
The FDA has awarded orphan drug designation to Eikonoklastes Therapeutics Inc.'s ET-101 program for the treatment of amyotrophic lateral sclerosis (ALS).
Cyagen Biomodels LLC has established a strategic collaboration with Neurophth Therapeutics Inc. to codevelop next-generation AAV gene therapy vectors for specific types of genetic ophthalmic disorders.
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