RARE Daily

Profluent Releases AI-Created and Open-Source Gene Editor

April 24, 2024

Rare Daily Staff

Profluent said it demonstrated the first successful precision editing of the human genome with a customizable gene editor designed from scratch with AI as it unveiled its OpenCRISPR initiative to make the gene editor freely available to license for ethical research and commercial uses.

OpenCRISPR-1 consists of a Cas9-like protein and guide RNA, fully developed using Profluent’s large language models. Through the training process for OpenCRISPR, the company’s AI learned from massive scale sequence and biological context to generate millions of diverse CRISPR-like proteins that do not occur in nature, exponentially expanding virtually all known CRISPR families.

“Attempting to edit human DNA with an AI-designed biological system was a scientific moonshot,” said Ali Madani, Profluent co-founder and CEO. “Our success points to a future where AI precisely designs what is needed to create a range of bespoke cures for disease. To spur innovation and democratization in gene editing, with the goal of pulling this future forward, we are open-sourcing the products of this initiative.”

Profluent’s hope is that CRISPR medicines become available to more patients who may benefit, and for a broader range of disorders. The unique power of AI is that it enables researchers to reimagine and build gene editing systems from the ground up, which would be impossible with conventional protein engineering methods. By growing the universe of CRISPR families and releasing foundational molecules that can be further built upon, this application of AI paves the way to greater access and lower costs for gene editing therapies.

“It’s phenomenal that the first CRISPR-based treatments for genetic diseases such as sickle cell disease are already changing the lives of patients, but there remains an urgent need to accelerate the development of this technology for thousands of other currently incurable diseases,” said Hilary Eaton, chief business officer at Profluent. “Our intention with OpenCRISPR is to partner with cutting-edge research institutions and drug developers with a powerful and practical way to safely expedite the development of new CRISPR genetic therapies.”

Photo: Ali Madani, Profluent co-founder and CEO

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