DEBRA Research gGmbH, a global non-profit organization dedicated to advancing research and drug development for those affected by Epidermolysis bullosa (EB), and Nonsense Therapeutics (Nonsense Tx), a biotechnology company developing read-through therapies based on small molecules targeting nonsense mutations, announced the formation of ReadOn Therapeutics, a new non-profit company dedicated to advancing treatments for EB, initially focusing on Dystrophic EB, one of the more severe forms of EB caused by defective type VII collagen.

ReadOn Therapeutics represents a pioneering joint venture between patient advocacy and biotech innovation in the EB field. The initiative is designed to progress promising preclinical science through the critical early stages of development, de-risking programs so they are ready to advance into clinical development.

Alex Hersham, Managing Director of ReadOn Therapeutics and Co-founder of Nonsense Tx, commented: “As the father of a child with dystrophic EB, I know firsthand the urgency of finding new treatments. ReadOn Therapeutics was born from that urgency – a patient-driven mission that combines the power of advocacy, cutting-edge science, and entrepreneurial execution. By working together, we can move beyond ideas and bring real hope closer to patients and families who need it most.”

Focus on nonsense mutations in dystrophic EB now – potential for many rare diseases

Around one-third of patients with recessive dystrophic EB carry nonsense mutations in the collagen VII gene, which lead to incomplete, non-functional protein products and thus often cause the most severe cases of the disease. ReadOn Therapeutics will focus on the development of small molecules that target the translation machinery, enabling full protein expression. The approach aims to restore type VII collagen production that could significantly reduce - or potentially suppress - disease symptoms. In this regard, ReadOn Therapeutics will carry out a comprehensive preclinical program and IND-enabling studies, intending to be ready for the clinic in less than 3 years. Since nonsense mutations not only cause EB but also many other genetic diseases, this approach could be translated to other rare conditions if successful.

Innovative business model

As a non-profit biotech, ReadOn Therapeutics has a unique structure that allows it to fund and advance multiple preclinical programs in parallel, in ways traditional biotech or non-profit models cannot. By comparing and evaluating several therapeutic approaches from diverse sources, the company can select the most promising candidates for accelerated advancement into early clinical trials, thereby opening multiple pathways, including spin-out opportunities attracting venture capital, and out-licensing opportunities for interested pharma and biotech companies.

Dr. Martin Steiner, Managing Director of ReadOn Therapeutics and DEBRA Research, concluded: “Through this innovative model, DEBRA Research consolidates drug development activities for read-through molecules within a dedicated non-profit biotech. This structure allows us to focus resources and expertise on advancing promising therapeutic strategies that could benefit many patients in urgent need of effective treatment. Our approach directly addresses critical gaps in the therapeutic landscape of EB, while also serving as a blueprint for future focused initiatives and a catalyst for further collaboration with scientists, entrepreneurs, biotechs and pharma driving rare disease innovation.”