Scribe Therapeutics, a biotech firm focused on developing next-generation gene-editing technology, has raised $20 million in its first major round of financing, backed by Andreessen Horowitz. The firm separately unveiled a deal with Biogen to develop CRISPR-based treatments for amyotrophic lateral sclerosis (ALS).

Scribe was cofounded in 2018 by several University of California, Berkeley, scientists, including gene-editing pioneer Jennifer Doudna and protein engineer Benjamin Oakes, who at the time was an entrepreneurial fellow at the Innovative Genomics Institute, where Doudna is president. Their goal was to engineer a newly discovered class of Cas proteins to make them behave better as therapies than the original CRISPR-Cas9 gene-editing system.

The original system was found in bacteria, which use it to recognize and chop up DNA from invading pathogens. Scientists, including Doudna, quickly realized the system could be co-opted to make precise cuts to human DNA. The tool set off a race among companies trying to use it to address the genetic mutations underlying many diseases.

But even with its promise, the CRISPR-Cas9 system comes with evolutionary baggage, Oakes, who is now CEO of Scribe, says. Those systems arent designed to work within the context of the human cell or even the human genome, he says, complicating efforts to turn the technology into drugs.

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Scribe Therapeutics launches to explore next-generation CRISPR technology - Chemical & Engineering News

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