Profluent Launches OpenCRISPR-1: The World’s First AI-Designed, Open-Source CRISPR Gene Editor Successfully Edits Human Genome

Recently, Profluent, a rising company in the AI pharmaceutical field, launched OpenCRISPR.^TMPlan, and released the world's first open-source artificial intelligence (AI)-generated gene editor. Profluent announced that it has successfully achieved precise editing of the human genome for the first time using a customized gene editor designed from scratch by AI.

Specifically, Profluent's released OpenCRISPR-1 is an AI-created gene editor, consisting of Cas9-like proteins and guide RNA, and fully developed using Profluent's large language models (LLMs).

During the training of OpenCRISPR, Profluent utilized AI technology to learn from large-scale sequences and biological contexts, thereby generating millions of different CRISPR-like proteins.(These proteins do not exist in nature), thereby exponentially expanding nearly all known CRISPR families.

Profluent has launched OpenCRISPR-1 as the initial open-source version, allowing AI-designed gene editors to be licensed for free for ethical research and commercial use (https://github.com/Profluent-AI/OpenCRISPR). OpenCRISPR-1 retains the prototype structure of the Type II Cas9 nuclease but is more than 400 mutations away from SpCas9 and nearly 200 mutations away from any other known natural CRISPR-associated protein. Profluent has also utilized its LLMs to generate a synthetic guide RNA.

In the paper published on the preprint platform biorxiv, Profluent provided a detailed description of the generation and characteristics of OpenCRISPR-1. After being delivered to HEK293T cells, OpenCRISPR-1 demonstrated comparable editing efficiency and higher specificity compared to SpCas9.

When combined with an AI-generated deaminase, OpenCRISPR-1 is capable of performing base editing, demonstrating robust A-to-G editing at the target site.

Profluent, founded in 2022 and headquartered in Berkeley, California, is dedicated to developing deep generative models to design and validate novel functional proteins, revolutionizing biomedicine.

In August 2023, Profluent announced the completion of a $9 million seed funding round; in March 2024, it announced the completion of the latest $35 million financing round, with investors including OpenAI, Google, and DeepMind's Chief Scientist Jeff Dean (who also participated in the company’s angel round investment).

Profluent believes that the uniqueness of artificial intelligence lies in its ability to enable researchers to reimagine and construct gene editing systems from scratch, which traditional protein engineering methods cannot achieve. The empowerment of AI technology is expected to significantly expand the range of diseases treatable by CRISPR and accelerate the development of new CRISPR gene editing therapies.

As Nature magazine wrote in a report titled “‘ChatGPT for CRISPR’ creates new gene-editing tools”: With the advancement of generative AI, scientists may now only need to press a button to design new CRISPR editing systems.

Notably, in addition to Profluent, in February this year, a team of scientists from Stanford University and the Arc Institute also announced a model trained on microbial genomes via a preprint platform.(Evo, trained on 80,000 genomes from bacteria, archaea, and other microorganisms, totaling 300 billion DNA bases), for the purpose of designing new CRISPR systems.

Profluent Co-founder and CEO Ali Madani stated, "Using AI-designed biological systems to edit human DNA is a scientific 'moonshot' project. Our hope is that AI-designed gene-editing tools will be better suited for medical applications than the current CRISPR systems. Profluent also aims to collaborate with companies dedicated to gene-editing therapies to jointly advance the development of AI-generated CRISPR treatments."

- Scroll up and down to view references -