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New Trailblazer Profluent Launches its AI-enabled OpenCRISPR-1 to Edit the Human Genome

Profluent successfully combined large language models (LLMs) and CRISPR for an open-source AI gene editing project which could reshape CRISPR treatment options.

Profluent is an AI-first protein design company founded in 2022. Their research aims to develop deep generative models to design and validate novel, functional proteins to transform biomedicine. According to the company's recent press release, Profluent debuted its OpenCRISPR initiative, releasing the world's first open-source, AI-generated gene editor.

CRISPR/Cas9 is a tool that uses guide RNA (gRNA) and a Cas9 enzyme to edit parts of the genome. The Cas9 enzyme cuts two strands of DNA at a specific location. The gRNA finds and binds to a specific sequence in the DNA. The Cas9 enzyme then follows the gRNA to the same location in the DNA sequence where it cuts both strands of DNA.

OpenCRISPR-1 is an AI-created gene editor consisting of a Cas9-like protein and guide RNA, developed using Profluent's LLMs. The Profluent team created a database of 5.1 million Cas9-like proteins and the AI model was trained on this database to create potential proteins for CRISPR use.

The LLM was able to create novel gene editors from scratch by learning from examples found in nature. After analysing the results, the scientists selected OpenCRISPR-1. This protein displayed similar characteristics to Cas9 but with significantly less impact on off-target sites. 

OpenCRISPR-1 demonstrated a 95% reduction in editing at specific off-target sites with a median rate of unwanted genetic insertions and deletions of less than 1%. This gives OpenCRISPR-1 an edge in terms of its precision, and it causes minimal damage to DNA.


Unlike other gene editing techniques like CRISPR-Cas9, the molecules the open-source editor designs are fully synthetic and do not exist in nature. Therefore, this opens up new avenues for developing CRISPR medicines.

Ali Madani, Co-founder and CEO of Profluent summed up his company's mission: "Our vision is to move biology from being constrained by what can be achieved in nature to being able to use AI to design new medicines precisely according to our needs."

Madani has continuously advocated for the ethical use of AI. By making OpenCRISPR free to use, he hopes to show his commitment to democratizing gene editing tools and the importance of promoting inclusivity within the life sciences sector and beyond.

Overall, this innovative tool shows great promise and could have wide implications for gene editing technologies. It could lead to an increase in the availability of CRISPR medicines for a wider range of diseases including rare genetic disorders with high unmet needs. This application contributes to the main challenges facing the cell and gene therapy (CGT) field: greater access and reduced costs for gene editing therapies.