We are pleased to announce the publication of a new white paper co-authored by Cilcare and CBSET, featured in World Pharma Today, titled: “Gene Therapy for Hearing Loss: A Translational Development Roadmap”
Authored by Lise Byelyayeva (Cilcare) and Ellie Khadir (CBSET), this article outlines a strategic, step-by-step roadmap for translating gene therapy research into clinical treatments for hearing disorders—an area that, despite major advances in gene therapy, still lacks approved therapies.
The paper highlights the unique challenges of developing gene therapies for inner ear indications and offers a roadmap to address scientific, regulatory, and translational hurdles. It also includes an overview of current clinical trials targeting Otoferlin-related hearing loss, illustrating real-world progress.
Key topics include:
- Vector selection and delivery routes for inner ear gene therapy
- Surgical techniques for preserving auditory function during vector administration
- Preclinical models and assessements
- Clinical trials designs and outcome measures for early-stage studies
- Translational & regulatory strategies to bridge the gap between discovery and clinical development
Read the full article here: https://www.worldpharmatoday.com/articles/gene-therapy-for-hearing-loss-a-translational-development-roadmap/
This publication further reinforces Cilcare-CBSET’s commitment to advancing breakthrough therapies for hearing loss and supporting our partners through every step of the development journey.
