Sensorion nabs rare pediatric designation for gene therapy; Avalo sells off remaining drug rights for $5M
A French biotech is moving forward on its hearing loss gene therapy, and the FDA is giving the candidate a regulatory boost.
Sensorion put out word Monday morning that the US regulatory agency granted Rare Pediatric Disease Designation to its lead gene therapy candidate, called OTOF-GT. It is being tested to treat otoferlin gene-mediated hearing loss, a hearing loss caused by otoferlin deficiency that affects up to 20,000 people in the US and Europe, per the biotech.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.