Profluent Launches OpenCRISPR-1: World’s First AI-Designed Gene Editor Successfully Edits Human Genome

Apr 24, 2024 11:20 CST Updated 11:20

Profluent

Protein Designer

Recently, Profluent launched the OpenCRISPR™ initiative,Released the world’s first AI-generated gene editor —— OpenCRISPR-1, which successfully achieved precise editing of the human genome.Its core components include a Cas9-like protein and guide RNA, entirely developed by Profluent's large language models (LLMs). During the training of OpenCRISPR, Profluent's AI system learns from extensive data such as large-scale sequences, creating millions of diverse CRISPR-like proteins that do not exist in nature.

OpenCRISPR-1 Gene Editor Structure

Profluent Releases OpenCRISPR-1 as the Initial Open-Source Version, Allowing Free Access to This AI-Designed Gene Editor for Ethical Research and Commercial Use。Ali Madani, co-founder and CEO of Profluent, stated that using AI-designed biological systems to edit human DNA is a bold scientific endeavor. This success indicates that AI will be able to precisely create a range of customized therapies for diseases in the future. To promote innovation in the field of gene editing and realize this vision as soon as possible, the company has decided to make the results of this project public.

Profluent hopes that CRISPR drugs can benefit more patients in need and be applied to a wider range of disease treatments. The unique advantage of AI lies in enabling researchers to reimagine and construct gene editing systems from scratch, which traditional protein engineering methods cannot achieve. The application of AI will pave the way for broader accessibility and lower costs of gene editing therapies.

Chief Commercial Officer of ProfluentHilary EatonIt was pointed out that although the first batch of CRISPR-based genetic disease therapies, such as the treatment for sickle cell disease, have significantly improved the quality of life for patients receiving the therapy, there is still an urgent need to accelerate the development of this technology for the thousands of diseases that currently have no cure. OpenCRISPR aims to collaborate with cutting-edge research institutions and drug developers to provide a powerful and practical approach to safely and rapidly advance the development of novel CRISPR gene therapies.

Peter Cameron, Vice President and Head of Profluent's Gene Editing Department, stated that the release of OpenCRISPR-1 initiates an iterative process for building the next generation of gene therapies. He encouraged the gene editing community to rigorously test OpenCRISPR-1, and if any areas for improvement or optimization are discovered during practical applications, Profluent is willing to be informed and collaborate on optimizing it.

In addition, Profluent pointed out thatFollowing ethical standards, the OpenCRISPR license agreement will explicitly prohibit certain uses, such as human germline gene editing.

Recently, Profluent published an article titled “Design of highly functional genome editors by modeling the universe of CRISPR-Cas sequencesThe article revealed the scientific principles behind the OpenCRISPR initiative andYesOpenCRISPR-1. The gene editor has been extensively characterized, confirming its highly efficient functional activity. Experimental data show that OpenCRISPR-1 exhibits higher activity at target sites compared to SpCas9 (OpenCRISPR-1: 55.7%, SpCas9: 48.3%). Moreover, strikingly, off-target editing by OpenCRISPR-1 is reduced by 95% compared to SpCas9 (see figure below), indicating that OpenCRISPR-1 possesses higher on-target editing efficiency and lower off-target activity.