The U.S. FDA named the first nine recipients of the recently unveiled commissioner’s national priority voucher (CNPV) program aimed at addressing unmet public health needs by shortening regulatory review times to as little as one to two months. For one of those firms, Disc Medicine Inc., which submitted an NDA for bitopertin for rare genetic disorder erythropoietic protoporphyria in September, that could mean a potential approval before the end of 2025.
Rznomics Inc. scored a potential ₩1.9 trillion (US$1.35 billion) global license option agreement with Eli Lilly and Co. to codevelop a novel RNA editing gene therapy to treat hereditary hearing loss.
Rznomics Inc. scored a potential ₩1.9 trillion (US$1.35 billion) global license option agreement with Eli Lilly and Co. to codevelop a novel RNA editing gene therapy to treat hereditary hearing loss.
In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss, 10 out of 11 have shown improvements after being treated with a gene therapy. “What is really remarkable about this type of therapeutic approach is that the first people who are going to see the impact are not actually the physicians – it’s the families,” Jonathon Whitton, vice president and Regeneron’s auditory global program head, told BioWorld.
Six years of collaboration is ending in a buyout, as Decibel Therapeutics Inc. agreed to be acquired by Regeneron Pharmaceuticals Inc. in a deal valued at up to $213 million, including $109 million in equity, with additional payments via contingent value rights linked to clinical and regulatory milestones for DB-OTO, the lead gene therapy program targeting hearing loss.
Decibel Therapeutics Inc. has received clearance of its clinical trial application (CTA) by the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of gene therapy DB-OTO in pediatric patients with congenital hearing loss due to an otoferlin deficiency.
The volatile gene therapy space is getting a boost with Eli Lilly and Co.’s acquisition of Akouos Inc., which only a month before had received the first IND from the U.S. FDA for an adeno-associated virus (AAV)-based hearing loss treatment. Lilly plans to pay about $610 million for the company to get at AK-OTOF, Akouos’ lead candidate for treating hearing loss due to mutations in the otoferlin gene.
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.
