DEBRA Research gGmbH, a global non-profit dedicated to advancing research and drug development for epidermolysis bullosa (EB), has partnered with Nonsense Therapeutics (Nonsense Tx), a biotech developing small-molecule read-through therapies for nonsense mutations, to form ReadOn Therapeutics. The new non-profit biotech will focus on developing treatments for dystrophic EB, a severe form of EB caused by defective type 7 collagen.

ReadOn Therapeutics combines patient advocacy with biotech innovation to accelerate the translation of promising preclinical science into clinical development. The initiative aims to de-risk therapeutic programs, making them ready for early clinical trials.

Alex Hersham, managing director of ReadOn Therapeutics and co-founder of Nonsense Tx, said: “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.”

ReadOn Therapeutics will initially target nonsense mutations, which affect about one-third of patients with recessive dystrophic EB. These mutations in the collagen 7 gene lead to incomplete, non-functional protein, causing the most severe disease symptoms. The company is developing small molecules that target the translation machinery to restore type 7 collagen production, with the potential to significantly reduce or suppress EB symptoms. Preclinical development and IND-enabling studies are expected to be completed within three years, with future potential to expand the approach to other rare genetic diseases caused by nonsense mutations.

The non-profit biotech model allows ReadOn Therapeutics to advance multiple preclinical programs simultaneously, comparing and selecting the most promising candidates for early clinical trials. The structure also supports spin-out opportunities attracting venture capital and out-licensing agreements with pharma and biotech companies.

Dr Martin Steiner, managing director of ReadOn Therapeutics and DEBRA Research, added: “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.”