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 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. 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.

Rznomics Inc. has received IND approval from the FDA for a phase I/IIa trial of RZ-001 for hepatocellular carcinoma (HCC).

Decibel Therapeutics Inc. has received clearance from the FDA for its IND application to initiate a phase I/II trial of DB-OTO in pediatric patients, potentially in infants younger than 2 years of age.