PORTEFEUILLE

Gene editing, particularly through CRISPR-Cas9, has revolutionized our approach to diseases and medicine. Millions of patients and families affected by once intractable genetic conditions now have renewed hope. Despite remarkable progress made at an unprecedented pace, these technologies remain in their infancy and encounter limitations that restrict their applications. Specifically, the Cas9 protein only safely targets approximately 20% of all human genes and often with sub-optimal editing efficiency. To address these limitations, Nerai Bio developed an AI-assisted, fully automated platform for high-throughput protein engineering. This programmable platform generates enhanced CRISPR-based tools, introducing novel genome editors of expanded targeting range and increased efficiency, specificity, and safety. Our vision is to break the walls confining genome editing and unleash the future of medicine to patients once deemed uncurable.

Nerai’s core technology fusing AI to programmable biology has been developed and validated through leading academic collaborations. The power of the platform was demonstrated by creating novel CRISPR-Cas9 editors of extreme precision and high performance. Nerai is now focused on the commercial translation of its novel genome editing tools for liver and eye diseases. Nerai secured a significant Innosuisse grant to assess the performance of its therapy candidates for alpha-1 antitrypsin deficiency and phenylketonuria in humanized mice. As the overall medical need in rare genetic disease is vastly greater than one could tackle alone, we aim to join forces with industry leaders, leveraging their resources to accelerate the deployment of Nerai’s cutting-edge therapeutic candidates to patients in need.