PORTEFEUILLE

Rare genetic disorders affect millions of patients worldwide, yet 95% lack effective treatments. Gene editing — the ability to correct faulty DNA directly in living cells — holds enormous promise, but existing tools are imprecise, often unable to reach the culprit mutation, and associated with unwanted off-target DNA edits. Nerai Bio set out to solve this problem by building a high-throughput protein engineering platform that designs better, safer gene editors from the ground up.

Over the course of this project, Nerai’s proprietary MORPHEME platform completed multiple rounds of directed evolution and generated hundreds of novel CRISPR base editors. From this pool, Nerai selected NB-301, an adenine base editor optimized for citrullinemia type 1 (CTLN1) — a severe liver metabolic disorder caused by a single DNA letter change. NB-301 demonstrated meaningful editing efficiency in human cultured cells and is now advancing toward in vivo validation in a mouse model.
Beyond the lead candidate, the project validated MORPHEME as a scalable, disease-agnostic platform capable of generating tailored editors for any monogenic indication. Results from the platform underpinned a publication in Nature Methods (2024) and additional manuscripts currently in preparation. Nerai also secured follow-on funding and was recognized externally through a 2nd Place and Audience Award at the »»venture»» competition.
The GRS InnoBooster grant provided critical early-stage support that allowed Nerai to generate key proof-of-concept data packages to unlock subsequent investments, effectively translating a research idea into a fundable therapeutic pipeline.