Cambridge, Mass. — Sensorion has selected SENS-601 as its lead gene therapy program after reaching key regulatory milestones for the treatment of GJB2-related hearing loss.
The company said clinical trial applications have been filed in Canada and France to evaluate the safety, tolerability and efficacy of intra-cochlear administration of SENS-601 in pediatric patients with GJB2 gene-mediated hearing loss. The French National Agency for Medicines and Health Products Safety, or ANSM, has granted the application a Fast Track procedure.
Sensorion said it remains on track to submit an investigational new drug application to the U.S. Food and Drug Administration and a submission in Australia by the end of 2026.
SENS-601, also known as GJB2-GT, is an AAV-based gene therapy program developed through Sensorion’s collaboration with Prof. Christine Petit’s team at Institut Pasteur, Institut reConnect, Institut de l’Audition, Inserm and CNRS. The program targets hearing loss linked to mutations in the GJB2 gene, which Sensorion said is the most common cause of genetic congenital deafness.
As part of a strategic review, Sensorion said it will discontinue clinical development of SENS-501 for OTOF-related hearing loss and end recruitment in the Audiogene trial. The company said it will focus its gene therapy development resources on SENS-601, extending its cash runway through the end of 2027.
Fred Chereau, Chief Executive Officer of Sensorion, thanked patients, families and investigators involved in the Audiogene trial and said the company is confident that focusing resources on SENS-601 is the right strategic move.
“GJB2-related hearing loss is the most common cause of genetic deafness, affecting a large patient population,” Chereau said. “The scientific, clinical, and operational foundation built through SENS-501 gives us a meaningful head start in advancing SENS-601 toward the clinic.”
Christine Petit, Professor at Institut Pasteur – Institut reConnect/Institut de l’Audition, and Professor Emeritus at the Collège de France, said Sensorion and its academic partners have built a strong foundation for the program.
“We have generated extremely robust, comprehensive data demonstrating significant hearing restoration after SENS-601 administration, in clinically relevant animal models developed in our laboratories,” Petit said. “I look forward to continuing this collaboration and to the prospect of providing patients with a meaningful therapeutic option.”
Sensorion said OTOF-related hearing loss is an ultra-rare condition and that the recent availability of a gene therapy showing meaningful clinical efficacy in that indication has changed the development landscape for SENS-501. The company said it remains committed to long-term follow-up for all patients enrolled in the Audiogene trial.
SENS-601 is designed to address hearing loss associated with mutations in the GJB2 gene. Sensorion said the program has potential applications in pediatric congenital deafness, progressive forms of hearing loss in children and early-onset presbycusis in adults. The program is partially funded by the French State as part of the France 2030 investment plan through the ConnexGene project with Bpifrance.
