AI-enabled protein design company Profluent has leveraged artificial intelligence to design an open-source gene editor called OpenCRISPR-1, demonstrating the technology can be used to create molecules with the power to edit human DNA.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, developed more than a decade ago, allows scientists to modify DNA sequences within living organisms precisely.

Potential applications range from treatments for genetic disorders to researching disease mechanisms.

The molecules it designs are fully synthetic and do not exist in nature, in contrast to previous technologies in gene editing, such as CRISPR-Cas9.

The company is open-sourcing OpenCRISPR-1 for free ethical research and commercial use and published the science behind the protein’s development in a preprint publication.

“Attempting to edit human DNA with an AI-designed biological system was a scientific moonshot,” Ali Madani, Profluent cofounder and CEO, said in a statement. “Our success points to a future where AI precisely designs what is needed to create a range of bespoke cures for disease.”

WHY THIS MATTERS

AI was at the heart of this achievement, with the company training large language models (LLMs) on massive scale sequence and biological context.

The Profluent team developed 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.

This enabled the LLM to create novel gene editors from scratch as it learned through examples found in nature.

After narrowing down the results, they identified OpenCRISPR-1, a protein performing similarly to Cas9 but with far less impact on off-target sites. This makes it more precise and causes minimal damage to DNA.

The goal of open-sourcing OpenCRISPR-1 is to encourage the use of AI for ethical research and commercial use, particularly in developing medicines leveraging CRISPR.

“We believe by doing so, we can help accelerate the pace of discovery and innovation in the field,” Madani said. “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.”

He added that the company intends to partner with cutting-edge research institutions and drug developers working across the drug development lifecycle to enable CRISPR medicines to become available to a greater number of patients and for a greater number of disorders.

THE LARGER TREND

Gene editing technologies, including SHERLOCK and DETECTR, are transforming digital diagnostics, enabling rapid detection of infectious diseases such as COVID-19.

Companies including Atomwise, Deep Genomics and Valo are incorporating gene editing into drug discovery processes, revolutionizing treatment development.

Beyond gene editing, AI is powering everything from bone marrow analysis software to drug discovery and platforms to help pair patients with the right cancer-treatment drugs.