Profluent, an AI-first protein design company, has successfully edited the human genome using OpenCRISPR-1, the world's first open-source gene editor designed entirely by artificial intelligence.
The Berkeley-based startup used a large language model trained on massive datasets of biological sequences and contexts to generate millions of diverse CRISPR-like proteins that do not occur naturally. This exponentially expands virtually all known CRISPR families. The company's achievement marks a significant milestone in the field of gene editing and holds promise for accelerating the development of precision treatments for a wide range of genetic diseases.
Profluent's technology could change the landscape of medical research by allowing for more rapid and precise genetic modifications. CRISPR technology has already started to transform the treatment of genetic disorders like sickle cell disease. The AI-generated gene editors promise to be more adaptable and potentially more powerful than those currently available, which are limited by their natural evolutionary designs.
OpenCRISPR-1, the AI-generated gene editor at the heart of this breakthrough, consists of a Cas9-like protein and guide RNA. It demonstrated comparable or improved activity and specificity relative to SpCas9, a widely-used gene editor, despite being hundreds of mutations away from any natural protein. Profluent has made OpenCRISPR-1 freely available to license for ethical research and commercial uses, a move that underscores their commitment to democratizing this transformative technology.
"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."
The implications of this development are far-reaching. By expanding the universe of CRISPR families and releasing foundational molecules that can be further built upon, Profluent's AI-driven approach paves the way for greater access and lower costs for gene editing therapies. This could accelerate the development of treatments for thousands of currently incurable genetic diseases.
However, scientists caution that while AI-generated gene editors hold immense potential, they are unlikely to affect healthcare in the short term. The bottleneck, according to Fyodor Urnov, a gene editing pioneer at the University of California, Berkeley, lies in the cost of pushing these editors through preclinical studies before they can be used on patients.
Nonetheless, the rapid advancements in generative AI technologies offer a glimpse into a future where medicines and treatments could be quickly tailored to individuals. As James Fraser, a professor at the University of California, San Francisco, noted, "The system has learned from nature to create them, but they are new."
Profluent's breakthrough marks the beginning of a new era in gene editing, one where AI plays a central role in designing the tools that could revolutionize medicine. As the technology continues to evolve, it holds the promise of bringing us closer to a world where precision treatments for genetic diseases are more accessible and effective than ever before.