Nonsense mutations cause 10-12% of rare disease cases, impacting roughly 40 million people.
These mutations often lead to the most severe disease outcomes.
At Nonsense Tx, we are developing small-molecule therapies to read through premature termination codons caused by nonsense mutations.
Nonsense mutations cause premature termination codons (PTCs) resulting in incomplete translation of the protein.
Proteins made from genes with nonsense mutations are produced in a truncated, non-functional form, leading to diseases such as Recessive Dystrophic Epidermolysis Bullosa (RDEB).
Nonsense mutations across different diseases have a shared therapeutic entry point – premature stop codons can be overcome by therapeutics that cause ribosome readthrough also known as nonsense suppression.
Image adapted from Dabrowski et al. 2018
Our lead program is NTX-001, a small molecule program for Recessive Dystrophic EB.
Beyond RDEB – among the 300 million patients worldwide with rare hereditary or genetic diseases, 10-12% of cases result from nonsense mutations. We are actively exploring other indications for our approach.
Skin literally as fragile as a butterfly wing
Layers of skin tear and blister
Genetic condition
No cure
Affects – 500,000 people worldwide (8,000 of which have DEB
Not contagious
Affects internal organs
Often leads to fatal aggressive skin cancer and chronic wounds
Nonsense mutations cause premature termination codons (PTCs) resulting in incomplete translation of the protein.
Proteins made from genes with nonsense mutations are produced in a truncated, non-functional form, leading to diseases such as Recessive Dystrophic Epidermolysis Bullosa (RDEB).
Nonsense mutations across different diseases have a shared therapeutic entry point – premature stop codons can be overcome by therapeutics that cause ribosome readthrough also known as nonsense suppression.
Father of Abe who has DEB.
Co founder and President of Zencargo, a leading supply chain management company with 130+ employees, ~$80m capital raised.
Previously VP at Cerberus Capital Management & Goldman Sachs.
Previously Partner at Delin Ventures, previously at Versant Ventures.
Serial biotech company builder at Inception, Ridgeline and Delin. Co-inventor of key patent behind our approach to nonsense suppression.
20+ investments in biotech and medtech companies, board roles. Former biotech COO, successfully outlicensed a lead asset.
Proven R&D leadership, managing teams and facilities.
PhD in protein translation and tRNAs, highest distinction from Max Planck Institute/Potsdam University.
Founder/COO of Alys Pharmaceuticals & Venture Advisor at Medicxi.
Prior to this, he held various roles in his 24 years at Galderma, including Global Head of Research and Global Head of Development Strategy and led multiple global approvals of dermatological drugs. He thereafter led the Prescription Medicines Global Business Unit
Our mission is to help patients with EB to live, and to live well.
The mission of Nonsense Tx is deeply personal to me.
My son, Abe, was born in April 2023 with generalised severe Recessive DEB, including a nonsense mutation.