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July 11, 2024

Beyond CRISPR, gene-editing advances are stacking up

From AI-based CRISPR tools to alternatives that promise greater precision, there’s lots of good news for life-saving medical advancements.

In the news

CRISPR gene-editing caused a big splash in 2015 when Science magazine named it the breakthrough discovery of the year. It made headlines again recently when the first CRISPR treatment was approved in the UK and US.

But as it turns out, there’s more than one way to edit a gene. And to no one’s surprise—given all the attention on generative AI of late—one of the newest gene-editing techniques to recently hit the presses uses the same technological methods that fuel ChatGPT.

Profluent, a startup headquartered in Berkeley, Calif., is using artificial intelligence to create customizable gene-editing proteins—from scratch. Whereas CRISPR uses biological materials found in nature to edit DNA strands, Profluent’s OpenCRISPR initiative creates its own gene editors using AI.

While these AI-created gene editors were inspired by nature and closely modeled on existing proteins, they have never before existed on Earth, as one professor said after reading Profluent’s research paper.

Similar to how ChatGPT was trained on enormous amounts of text data, Profluent trained its programs on massive databases of protein sequences. The result is a program that can generate hundreds of thousands of CRISPR-like proteins and instructional RNA molecules imbued with the ability to make edits to the human genome. The foundational molecules can be further improved, paving the way to even more accessible, cost-effective gene-editing therapies, the company says.

Profluent is open-sourcing one of its top-performing gene editors, OpenCRISPR-1, in hopes of “extending the reach of CRISPR gene-editing tools” to develop new therapies.

Not to be outdone, scientists at the University of Sydney have developed a gene-editing tool that they claim boasts “greater accuracy and flexibility” than CRISPR.

Where CRISPR creates a break in both strands of targeted DNA and requires other proteins to insert the new sequence, SeekRNA is designed to more accurately cleave the target site and insert the new DNA sequence without the need for other proteins.

The Cognizant take

An important attribute of the Profluent announcement is the “democratization” of gene editing. Researchers and developers can freely license OpenCRISPR-1 for ethical research and commercial purposes. The hope is this accessibility will accelerate the development of gene therapies for a broader range of diseases, including rare genetic disorders with high unmet needs.

It’s also a breakthrough for generative AI. Profluent is reportedly generating nearly five times more protein clusters than are found in “natural” CRISPR-Cas families, which bodes well for the ability of generative AI to expand the boundaries of what’s possible in genetic engineering.

Clinical trials will be needed to validate the technology’s efficacy and safety. But ideally, implications of the breakthrough extend beyond the research lab in the form of personalized medicine. It’s not unrealistic to foresee custom cures tailored to an individual’s genetic makeup. This could greatly affect healthcare by making CRISPR medicines available to more patients, addressing a wider spectrum of disorders.

It remains to be seen whether SeekRNA is a superior alternative for gene editing. But this type of competition can only be good news for patients and the healthcare sector.

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