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Sensorion has filed clinical trial applications in Canada and France for SENS-601, its AAV-based gene therapy targeting GJB2 mutations — the most common genetic cause of congenital deafness — while discontinuing development of its OTOF-targeting program, SENS-501. Sensorion, the French clinical-stage biotechnology company focused on hearing loss therapies, has elevated SENS-601 to lead program status and submitted clinical trial applications (CTAs) in Canada and France for the AAV-based gene therapy, which targets hearing loss caused by mutations in the GJB2 gene. The announcement marks a significant strategic shift for the company, which is simultaneously ending recruitment in its Audiogene trial and discontinuing clinical development of SENS-501, its gene therapy candidate for OTOF-related hearing loss. The Hearconnex trial — the clinical study name for SENS-601 — will evaluate the safety, tolerability, and efficacy of intra-cochlear administration of SENS-601 in pediatric patients with GJB2 gene-mediated hearing loss. France's national drug agency, ANSM, granted the CTA a Fast Track designation, which provides a notably reduced regulatory assessment period compared to the standard pathway. Sensorion also reports that IND submission with the FDA in the US, and a submission in Australia, remain on track for completion by year-end 2026. https://hearingreview.com/inside-hearing/research/sensorion-provides-update-on-hearing-loss-gene-therapies GJB2 mutations are responsible for approximately 50% of autosomal recessive non-syndromic hearing loss and represent the most common cause of genetic congenital deafness. No approved gene therapy for GJB2-related hearing loss currently exists. Sensorion notes that recent research has also linked GJB2 mutations to early-onset forms of severe presbycusis in adults, meaning SENS-601 has the potential to address three distinct pathologies: pediatric congenital deafness, progressive hearing loss in children, and early-onset presbycusis in adults. "GJB2-related hearing loss is the most common cause of genetic deafness, affecting a large patient population," says Fred Chereau, CEO of Sensorion, in a release. "With the regulatory progress announced today, built on years of dedicated work by our teams and the strength of our partnership with the Institut Pasteur – Institut reConnect/Institut de l'Audition, we are entering a genuinely exciting phase for the program and the Company, as SENS-601 is positioned to be among the leading gene therapies to enter clinical development for this debilitating hearing loss disorder." Strategic Pivot Away from OTOF Program The decision to discontinue SENS-501 follows a strategic pipeline review prompted in part by the competitive landscape shift in OTOF-related hearing loss — an ultra-rare condition for which a competing gene therapy has recently demonstrated meaningful clinical efficacy. With the unmet need for GJB2-related hearing loss described by the company as "an order of magnitude larger," Sensorion's board and management concluded that concentrating resources on SENS-601 was the more viable path. https://hearingreview.com/hearing-loss/hearing-disorders/sensorineural/fda-grants-accelerated-approval-to-otarmeni-gene-therapy-for-otof-related-hearing-loss Sensorion emphasizes that the scientific and operational platform built through the Audiogene trial — conducted in close collaboration with Prof. Christine Petit's team at Institut Pasteur — is directly transferable to SENS-601. The company also confirmed it will maintain long-term follow-up for all patients enrolled in the Audiogene trial in accordance with applicable regulatory requirements. Robust Preclinical Data Underpins Confidence SENS-601 was developed in collaboration with Prof. Christine Petit and her team at the Institut reConnect, Institut de l'Audition, Institut Pasteur, Inserm, and CNRS. Preclinical data generated in clinically relevant animal models has demonstrated significant hearing restoration following SENS-601 administration. "GJB2 mutations are the most common cause of genetic hearing loss, and the work we have conducted with Sensorion over many years has built a strong foundation for what I believe will be a pivotal program," says Christine Petit, professor at Institut Pasteur – Institut reConnect/Institut de l'Audition and professor emeritus at the Collège de France, in a release. "We have generated extremely robust, comprehensive data demonstrating significant hearing restoration after SENS-601 administration, in clinically relevant animal models developed in our laboratories. The scientific and clinical advances of Audiogene are directly guiding and increasing our confidence in SENS-601, confirming the very promising results my team first obtained in mice back in 2019." Petit is also a laureate of the Kavli Prize in Neurosciences. Financial Runway and Program Funding Sensorion reports that its cash runway extends through the end of 2027, a timeline the company says is sufficient to support advancement of SENS-601 through clinical development and the generation of initial first-in-human data. The SENS-601 program is partially funded by the French State as part of the France 2030 investment plan through the ConnexGene project, with Bpifrance. https://hearingreview.com/inside-hearing/industry-news/sensorion-3 Sensorion's portfolio also includes SENS-401 (Arazasetron), a clinical-stage small molecule that has progressed through three Phase 2 proof-of-concept studies, including in cisplatin-induced ototoxicity, residual hearing preservation in cochlear implant candidates in partnership with Cochlear Limited, and sudden sensorineural hearing loss. Featured image: Dreamstime

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Sensorion Names SENS-601 Lead Gene Therapy Program, Files Clinical Trial Applications for GJB2-Related Hearing Loss

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