Sensorion Reveals Breakthrough Candidate for GJB2 Gene Therapy Program

Today, Thursday, April 6, 2023, Sensorion (FR0012596468 – ALSEN) – a pioneering clinical-stage biotechnology company that specializes in gene therapies for the inner ear – is hosting an R&D Day to present new data in support of its GJB2-GT program candidate selection to address hearing loss related to mutations in the GJB2 gene. With its cutting-edge technology, Sensorion is setting out to revolutionize the treatment of hearing loss.

The candidate has crafted a precise viral vector, utilizing an adeno-associated virus (AAV) capsid, that is tailored to target the key cells in the ear that naturally express GJB2 and is designed to protect against ototoxicity.

Sensorion is taking the next step in developing a potential therapy for three pathologies related to GJB2 mutations – early onset of age-related hearing loss in adults, progressive forms of hearing loss in children, and pediatric congenital deafness – by advancing into IND-enabling activities to enable clinical development.

Sensorion is thrilled to announce the selection of a promising candidate for its GJB2-GT program, a gene therapy developed in collaboration with the renowned Institut Pasteur. With the potential to offer long-term therapeutic solutions to hearing loss for those with GJB2 mutations, this marks the second gene therapy candidate in Sensorion’s expanding portfolio. As Chief Medical Officer, Dr. Géraldine Honnet proudly remarks, “This is a remarkable breakthrough in the fight against hearing loss.”

Sensorion is thrilled to be taking strides towards clinical development with our promising gene therapy candidate. We are confident that this is the optimal candidate, and our internal CMC capabilities will allow for a smooth tech transfer to our CDMOs. We strongly believe that gene therapy has the potential to revolutionize lives, and are proud to be part of this journey.

Sensorion, in collaboration with the Institut Pasteur, has achieved an exciting breakthrough in the treatment of GJB2-related hearing loss. Positive Proof of Concept (POC) data was generated in both Non-Human Primates and mouse models, demonstrating the efficacy of the candidate in both congenital and progressive conditions. This is a major step forward in the search for a viable treatment for this condition.

Christine Petit, Professor at the Institut Pasteur and the Collège de France, has highlighted the importance of gene therapy in treating genetic forms of hearing loss. Through the knowledge of the genetic architecture of deafness, treatments based on gene therapy have become increasingly feasible.

However, the development of these treatments requires appropriate models that simulate the various pathogenic variants of deafness genes. By doing so, the optimization and definition of the range of application of gene therapy agents is possible, helping to make hearing loss more manageable.

By working together with Sensorion on gene therapy for different types of deafness caused by GJB2 gene defects, we have been able to make significant strides in preclinical development of a potential gene therapy that targets the cochlear cells which rely on connexin 26. Through our collaboration, we have been able to create mouse models that accurately replicate the clinical indications associated with GJB2 deficits.

GJB2 mutations are the single most common cause of congenital deafness, with an estimated 300,000 sufferers in the US, Europe and Japan. The severity of hearing loss ranges from mild to profound, but is usually severe to profound. Those affected by GJB2 mutations often face a life of profound deafness and reduced communication.

This quarter, Sensorion is taking a major step forward in its mission to treat children born with hearing loss caused by otoferlin deficiency, filing a Clinical Trial Application for its revolutionary OTOF-GT program, a dual-vector AAV gene therapy.

Already granted Orphan Drug Designate status by both the US Food and Drug Administration and the European Medicines Agency, OTOF-GT is poised to revolutionize the lives of those born with this condition, delivering hope and healing to countless families.

R&D Day showcasing gene therapy for inner ear diseases

Today, 6 April 2023, Sensorion invites you to join their R&D Day at the Hearing Institute (Institut de l’Audition), an Institut Pasteur center in Paris, France. Starting at 8am EDT / 2pm CEST, this exciting event will provide you with a unique chance to be part of an interactive experience and learn more about Sensorion’s cutting-edge research and development.

You can follow along with a live webcast, or catch up later with a replay of the presentation. Don’t miss this incredible opportunity – join Sensorion’s R&D Day today!

Sensorion is proud to present its world-class team of management professionals, including CEO Nawal Ouzren, Chief Medical Officer Géraldine Honnet, M.D., Preclinical Development Head Laurent Désiré, Ph.D., and CMC Gene Therapy Head Christine Le Bec, Ph.D.

This dynamic group will be providing insight into the Company’s upcoming milestones and development programs for 2023. Get ready to witness the groundbreaking work of Sensorion’s top minds!

KOLs from the field of hearing therapeutics will be in attendance to present on relevant topics, such as patient needs, therapeutics, and the market landscape of hereditary monogenic forms of deafness. These KOLs are sure to provide an insightful look into the current and future state of the industry, giving attendees a comprehensive knowledge base to draw from.

About Sensorion

Sensorion is a groundbreaking clinical-stage biotech company that is on a mission to revolutionize the treatment of hearing loss disorders. By developing cutting-edge gene therapies, they are striving to make a significant impact on the global medical landscape and address a major unmet medical need.

Sensorion has developed a cutting-edge research and development technology platform to explore the causes and symptoms of inner ear-related diseases. With this platform, they are able to identify the most effective targets and mechanisms of action to develop drug candidates that could improve the quality of life of those affected.

Pluristem Therapeutics is pioneering in the field of gene therapy, with two programs targeting hereditary monogenic forms of deafness. In collaboration with the Institut Pasteur, these programs, OTOF-GT and GJB2-GT, aim to correct hearing loss related to mutations in the otoferlin and GJB2 genes, for both adults and children. In addition, the Company is working to identify biomarkers to improve diagnosis of these underserved illnesses.

Sensorion has developed an impressive portfolio of small molecule programs for the treatment and prevention of hearing loss disorders, with some of them in the clinical-stage of development. By exploring these therapeutic options, Sensorion is paving the way for new treatments to help people hear better and live better.

Sensorion is making strides in the clinical-stage with its Phase 2 product, SENS-401 (Arazasetron). Currently, the company is conducting a Proof of Concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, in partnership with Cochlear Limited, in a study of SENS-401 in patients scheduled for cochlear implantation.

Furthermore, Sensorion completed a Phase 2 study of SENS-401 in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022, showing great promise for its development.

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