Sensorion (FR0012596468 – ALSEN) a clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat, and prevent within the field of hearing loss disorders, announced the addition of a new gene therapy target, GJB2 coding for the Connexin 26 protein, to its development portfolio. The target is the third candidate to emerge from Sensorion’s R&D collaboration with Institut Pasteur, according to Sensorion. The GJB2 program will focus on major new markets with an estimated patient population (prevalence) of 300,000 children and adults in Europe and in the United-States alone.
New research (Boucher et al., Proc Natl Acad Sci U S A. 2020;117(49):31278-3128) published by scientists at the Institut Pasteur shows that anomalies in GJB2 are not only the most common cause of congenital deafness (prevalence of around 200,000 individuals in the US and in Europe alone) but also occur in adult cases of severe age-related hearing loss (around 100,000 adults in the same geographies). Although the types of GJB2 mutations in children and adults may differ, gene therapy could potentially provide a solution to both.
Sensorion’s GJB2 gene therapy program “has the potential to target three pathologies related to GJB2 mutations: age-related hearing loss in adults, progressive forms of hearing loss in children, and pediatric congenital deafness.” Initially, the focus will be on the first two populations with GJB2-associated hearing loss, making Sensorion “the first company to address these important medical needs in adults and potentially large market opportunities.”
Current scientific understanding suggests that mutations in GJB2 alter a gap junction protein widely expressed in the inner ear, disturbing intercellular exchanges of molecules and leading to hearing loss that is severe-to-profound in a majority of cases.
“The emergence of a new gene therapy target candidate validates our conviction that long-term solutions for restoring hereditary hearing loss will arise from an in-depth analysis of the ‘genetic landscape’ of hearing loss,” said Nawal Ouzren, CEO of Sensorion. “It was clear that mutations in the GJB2 gene are important in severe-to-profound childhood hearing loss. However, the new discovery made by our collaborators at Institut Pasteur shows that alteration of this gene in adults offers new opportunities for Sensorion. It marks significant potential expansion of our pipeline and supports our goal of becoming a global leader in the field of gene therapies for hearing loss disorders.”
“Until now, the genetics of late forms (age-related deafness or presbycusis) was considered to involve multiple variants in each individual,“ said Professor Christine Petit, director of the French Hearing Institute, an Institut Pasteur Center. “We have shown that the same genes underlying congenital or childhood deafness are also involved in severe forms of early presbycusis. The presence of these very rare genetic variants makes these forms of presbycusis appear to be monogenic types of hearing loss which can therefore be potentially treated by gene therapy.”
Sensorion’s collaboration with Institut Pasteur initiated in 2019 has already led to gene therapy candidate programs in two other indications – Otoferlin deficiency and Usher Syndrome Type 1. GJB2-GT is the third program under this collaboration and represents the largest gene therapy opportunity for Sensorion to date.