MONTPELLIER, FRANCE — Sensorion announced that it has selected SENS-601, its gene therapy candidate for GJB2-related hearing loss, as the company’s lead gene therapy program after achieving several key regulatory milestones.
The company has filed Clinical Trial Applications in Canada and France, with additional submissions planned in the United States and Australia by the end of 2026.
The announcement also marks a strategic shift for the company, which has decided to discontinue development of its SENS-501 program targeting OTOF-related hearing loss and instead concentrate its resources on SENS-601, a therapy designed to address the most common cause of genetic deafness.
According to Sensorion, the planned clinical study will evaluate the safety, tolerability, and efficacy of intra-cochlear administration of SENS-601 in pediatric patients with GJB2 gene-mediated hearing loss. The study will also assess the safety, performance, and ease of use of the company’s delivery system.
The French regulatory agency, ANSM, has granted the program a Fast Track procedure, providing a significantly shorter review timeline than the standard pathway. Sensorion said the regulatory submissions follow pre-submission discussions with authorities and that it has confidence in the completeness and scientific quality of its application package.
The decision to prioritize SENS-601 reflects both scientific opportunity and strategic focus. While OTOF-related hearing loss represents an ultra-rare condition, GJB2 mutations account for the most common form of inherited deafness, affecting a substantially larger patient population and currently lacking an approved gene therapy.
“On behalf of Sensorion, I wish to express our sincere gratitude to the patients and their families for their courage and trust, and to the investigators for their exceptional dedication in the Audiogene trial. In my early weeks at Sensorion, working closely with the Board, management team, and our scientific partners, I am confident that dedicating our gene therapy development resources to SENS-601 is the right strategic decision. GJB2-related hearing loss is the most common cause of genetic deafness, affecting a large patient population. The scientific, clinical, and operational foundation built through SENS-501 gives us a meaningful head start in advancing SENS-601 toward the clinic. 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. I am fully committed to executing on this opportunity and delivering on the promise of gene therapy for patients and families.”
—Fred Chereau, CEO of Sensorion
The program was developed through Sensorion’s collaboration with researchers at the Institut Pasteur – Institut reConnect/Institut de l’Audition, where years of preclinical work demonstrated significant hearing restoration in animal models carrying GJB2 mutations.
“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” said Dr Christine Petit, Professor at Institut Pasteur – Institut reConnect/Institut de l’Audition, and Professor Emeritus
at the Collège de France, laureate of the Kavli Prize in Neurosciences. “We have generated extremely robust, comprehensive data demonstrating significant hearing restoration after SENS-601 administration, in clinically relevant animal models developed in our laboratories. I look forward to continuing this collaboration and to the prospect of providing patients with a meaningful therapeutic option. 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. The rigor with which the multidisciplinary teams from the Institut Pasteur-Institut reConnect/Institut de l’Audition, Necker Enfants-Malades Hospital (AP-HP), the Fondation Pour l’Audition and Sensorion have conducted this program is a credit to everyone involved.”
Sensorion noted that while it is ending recruitment in the Audiogene trial evaluating SENS-501, it remains committed to the long-term follow-up of all enrolled patients. The scientific and operational experience gained through that program will be directly applied to the development of SENS-601.
The company also announced that concentrating its resources on the GJB2 program extends its projected cash runway through the end of 2027, supporting the clinical advancement of SENS-601 and the generation of first-in-human data.
About SENS-601 (GJB2-GT)
SENS-601 (GJB2-GT) is an innovative AAV-based gene therapy program developed in collaboration with Prof. Christine Petit’s team (Institut reConnect, Institut de l’Audition, Institut Pasteur, Inserm, CNRS) to treat hearing loss linked to mutations in the GJB2 gene, which plays a critical role in maintaining the ionic balance necessary for sound transduction in the inner ear. GJB2 mutations represent the most common cause of genetic congenital deafness, responsible for approximately 50% of autosomal recessive non-syndromic hearing loss. Recent research has also established that GJB2 mutations are found in early onset forms of severe presbycusis in adults, which appear to be monogenic potentially treatable by gene therapy. As such, SENS-601 has the potential to address three distinct pathologies: paediatric congenital deafness, progressive forms of hearing loss in children, and early onset of presbycusis in adults. With no approved gene therapy currently available for GJB2-related hearing loss, SENS-601 has the potential to be among the first gene therapy addressing GJB2 mutations. This program is partially funded by the French State as part of the France 2030 investment plan (ConnexGene project, with Bpifrance).
About SENS-501
SENS-501 (OTOF-GT) is an innovative gene therapy program developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. This gene plays a key role in the transmission of auditory signals between the hair cells of the inner ear and the auditory nerve. When this gene is defective, affected individuals are born with severe to profound hearing loss.
The aim of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). This therapy aims to restore the normal process of converting sound into electrical signals, enabling patients to regain their hearing ability. This gene therapy for patients suffering from otoferlin deficiency has been developed in the framework of RHU AUDINNOVE, a consortium composed of Sensorion with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l’Audition. The project is partially financed by the French National Research Agency, through the “investing for the future” program (ref: ANR-18-RHUS-0007). The OTOF gene targeted by the Audiogene trial was discovered in 1999 at the Institut Pasteur, by Prof. Christine Petit’s team (Institut reConnect, Institut de l’Audition, Institut Pasteur), who also unravelled the pathophysiology of the corresponding deafness (DFNB9).
About Sensorion
Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates. SENS-601 (GJB2-GT) is the Company’s lead gene therapy program, targeting hearing loss related to mutations in the GJB2 gene to address important hearing loss segments in adults and children developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur.
Sensorion’s portfolio also comprises programs of a clinical-stage small molecule, SENS-401 (Arazasetron), for the treatment and prevention of hearing loss disorders. Sensorion’s small molecule progressed in three Phase 2 proof of concept clinical study: firstly, in Cisplatin-Induced Ototoxicity (CIO) for the preservation of residual hearing, with analysis completed in Q1 2026. Secondly, with partner Cochlear Limited, a study of SENS-401 for the residual hearing preservation in patients scheduled for cochlear implantation, completed in 2024. Thirdly, a Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in 2022
Source: Sensorion
