Sanofi turns to startup Scribe to CRISPR edit new NK cell therapies for most cancers


 

Sanofi’s technology for making natural killer (NK) cell therapies came from an M&A move. The pharmaceutical giant just struck a deal that adds a CRISPR-editing technology to its to toolkit for developing NK cell-based therapies for cancer.

The CRISPR technology is from Scribe Therapeutics, a startup co-founded by CRISPR technology pioneer Jennifer Doudna. Whereas CRISPR’s initial development employed the Cas9 cutting enzyme, Alameda, California-based Scribe’s platform is comprised of gene-editing and delivery tools based on CasX, a protein discovered in Doudna’s lab. CasX is a smaller protein, which makes it a better fit for the in vivo gene-editing applications that are the focus of the startup’s internal research.

Sanofi will apply the Scribe technology to ex vivo NK cell therapies. According to financial terms announced Tuesday, the pharma giant is paying $25 million up front. Development and commercialization milestone payments to Scribe could top $1 billion; Scribe would also receive royalties from sales of any approved products that stem from the collaboration.

NK cells are a type of immune cell endowed with tumor-killing enzymes. NK cells seek out cancer cells to carry out their work, and they have application across many tumor types. Two years ago, Sanofi licensed an off-the-shelf NK cell therapy from Kiadis Pharma that was in preclinical development. The stated goal was to pair that drug with Sarclisa, a Sanofi antibody drug for multiple myeloma.

Months after the licensing deal was announced, Sanofi agreed to acquire the biotech outright for €308 million cash. Sanofi said Kiadis’s NK cell therapies would be developed as standalone treatments and in combinations with the pharma giant’s drugs. Sanofi’s pipeline currently lists one clinical-stage NK program from Kiadis: SAR445419, an off-the-shelf therapy, is in Phase 1 testing for acute myeloid leukemia.

Companies conducting NK cell research are working to bring cell therapy to solid tumors. The first cell therapies, based on engineering a patient’s own T cells, have worked only on blood cancers so far. Frank Nestle, Sanofi’s global head of research and chief scientific officer, said in a statement that his company sees NK cells having applications in both solid tumors and blood cancers.

“This collaboration with Scribe complements our robust research efforts across the NK cell therapy spectrum and offers our scientists unique access to engineered CRISPR-based technologies as they strive to deliver off-the-shelf NK cell therapies and novel combination approaches that improve upon the first generation of cell therapies,” he said.

Scribe emerged from stealth in 2020, backed by a $20 million Series A round of financing. Last year, Scribe closed a $100 million Series B round to finance further development of its technology platform and to advance its pipeline of CRISPR-based therapies for neurodegenerative disorders. The biotech’s neuroscience work previously led to a research partnership with Biogen focused on developing CRISPR-based therapies for amyotrophic lateral sclerosis. In May, the two companies announced that Biogen had exercised its option to expand the collaboration to an additional disease target in gene therapy. That target was not disclosed.

Photo: Nathan Laine/Bloomberg, via Getty Images



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