MONTPELLIER, FRANCE — Sensorion on Monday reported updated six-month efficacy data from Cohort 2 of its Phase 1/2 Audiogene trial evaluating SENS-501, while also providing a development update on its GJB2 gene therapy program, GJB2-GT (SENS-601).
The company said the six-month follow-up data from Cohort 2 showed sustained early efficacy signals in two of three patients treated with the higher dose of SENS-501. According to Sensorion, those findings were consistent with the improvements previously reported at Month 3 and aligned with a dose-response relationship observed across the first two cohorts.
Across all six patients treated in the dose-escalation phase, the surgical procedure and intra-cochlear administration of SENS-501 continued to be well tolerated, with no serious adverse events or serious side effects reported, the company said.
Based on the dose-response signal seen so far, Sensorion said it is considering a third dose level within the Audiogene trial framework and plans to consult with regulatory authorities while continuing to monitor the broader regulatory and competitive landscape for hearing loss gene therapies.
“The Audiogene trial continues to generate consistent and informative data in a very young patient population. The sustained hearing signals observed at six months in children born with profound deafness, combined with the favorable safety profile and the reproducibility of the surgical procedure with our injection system across patients and dose levels, reinforce our confidence in the potential of gene therapy for genetic hearing loss. These results provide hope for families affected by otoferlin-mediated deafness, and the clinical expertise built through Audiogene will directly benefit patients with other monogenic forms of hearing loss who may benefit from similar gene therapy approaches in the future.”
–Pr. Natalie Loundon, Principal Investigator of the Audiogene trial
Sensorion also said experience gained through the Audiogene program is helping support its broader gene therapy platform, including the development of SENS-601, which targets hearing loss linked to mutations in the GJB2 gene.
GJB2 mutations are described by the company as the most common cause of genetic congenital deafness, accounting for approximately 50% of autosomal recessive non-syndromic hearing loss. Sensorion said CTA-enabling studies for SENS-601 are progressing, with CTA submission on track for the first half of 2026 and an IND submission targeted by the end of 2026.
The company also said it plans to present preclinical safety, biodistribution, and efficacy data for SENS-601 at the American Society of Gene & Cell Therapy annual meeting in May 2026 in Boston.
“The data generated through Audiogene validates the key components of our gene therapy platform, from surgical delivery, and safety through dose escalation to the observation of a dose-response relationship,” commented Amit Munshi, Chairman and Interim Chief Executive Officer of Sensorion.
“This growing body of clinical and procedural experience, together with the relationships we have built with leading clinical centers, regulatory agencies, and our partners, provides further confidence in the advancement of SENS-601, our potential first-in-human gene therapy program targeting GJB2-related hearing loss. Our strengthened financial position following the recent financing, anchored by Sanofi’s strategic investment, positions the Company well to execute on this next phase of growth. Ultimately, our goal is to bring meaningful therapeutic options to the families and patients who today have no targeted treatment for genetic hearing loss, and we remain fully committed to this mission.”
The SENS-501 program has been developed within the RHU AUDINNOVE consortium, which includes Sensorion, 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 its “investing for the future” program.
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. Currently in the clinical research phase, this gene therapy program represents significant hope for families affected by this rare form of genetic deafness. SENS-501 (OTOF-GT) embodies a commitment to scientific innovation in the field of hearing, with the potential to dramatically improve the quality of life of patients suffering from genetic deafness. 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, Pasteur Institute), who also unravelled the pathophysiology of the corresponding deafness (DFNB9).
About the Audiogene Trial
Audiogene aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in infants and toddlers aged 6 to 31 months at the time of gene therapy treatment. By targeting the first years of life, when brain plasticity is optimal, the chances of these young children with pre-linguistic hearing loss acquiring normal speech and language are maximized. The study comprises two cohorts of two doses followed by an expansion cohort at the selected dose. While safety will be the primary endpoint of the first part of the dose escalation study, auditory brainstem response (ABR) will be the primary efficacy endpoint of the second part of the expansion. Audiogene will also evaluate the clinical safety, performance and ease-of-use of the delivery system developed by Sensorion.
About GJB2-GT (SENS-601)
GJB2-GT (SENS-601) is an innovative AAV-based gene therapy program developed in collaboration with the Institut Pasteur 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, GJB2-GT 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 the first-in-human gene therapy addressing GJB2 mutations. CTA-enabling studies are progressing well and the program is on track for CTA submission during H1 2026.
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.
It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve the diagnosis of these underserved illnesses.
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, 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
