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.
By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures.
By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures. The channels are expressed when the promoter is turned on by excessive neuronal activity, and so “we can’t stop the first seizures,” Dimitri Kullmann told BioWorld.
By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures.
Abeona Therapeutics Inc. is on the road to filing a BLA with the U.S. FDA after posting positive top-line phase III data in wound healing and also to the bank with a new $35 million private placement financing. The data for EB-101, an autologous cell therapy, came from a pivotal study of treating recessive dystrophic epidermolysis bullosa, an ultra-rare connective tissue disorder. Results showed the study met its two co-primary endpoints in wound healing and for reducing pains in large, chronic wounds caused by the disorder.
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