Sanofi and Scribe Therapeutics will partner to develop CRISPR-based cell therapies to fight cancer, the companies said today. The collaboration could generate more than $1 billion for the CRISPR drug developer, whose co-founders include Nobel laureate Jennifer Doudna, PhD, of University of California, Berkeley.
The pharma giant will apply Scribe’s CRISPR genome editing technologies, designed to enable genetic modification of novel natural killer (NK) cell therapies for cancer. Sanofi has been granted non-exclusive rights to Scribe’s proprietary CRISPR platform of wholly owned enzymes for creating ex vivo NK cell therapies.
Scribe has developed a suite of custom engineering genome editing and delivery tools called CasX-Editors, based on the CasX enzyme and other novel foundations. Through the collaboration, Scribe’s tools will support Sanofi’s expanding pipeline of NK cell therapeutics for oncology.
“We’re pleased to provide Sanofi with access to Scribe’s proprietary and enhanced gene editing technologies for use in ex vivo oncology applications distinct from our current pipeline,” Scribe co-founder, president, and CEO Benjamin L. Oakes, PhD, said in a statement.
Sanofi has agreed to pay Scribe $25 million upfront, and potentially more than $1 billion in payments tied to achieving development and commercial milestones, as well as tiered royalties on net future sales on any products developed through the collaboration.
Sanofi is developing a range of NK cell-based next-generation therapies that, according to the company, could be broadly applicable across solid tumors and blood cancers. Sanofi’s pipeline includes SAR445419 (formerly KDS1001), an NK cell therapy candidate being developed for acute myeloid leukemia that is under study in a Phase I trial. Sanofi added the candidate to its pipeline when it acquired Kiadis Pharma for €308 million ($295 million) in 2020.
“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,” added Frank Nestle, Sanofi’s global head of research and chief scientific officer.
Sanofi is one biopharma giant with which Scribe is collaborating on CRISPR-based therapies. Another is Biogen, which Scribe said in May had exercised an option for a second (unnamed) target through which they aim to treat disease. The first is a genetic cause of amyotrophic lateral sclerosis (ALS). Oakes discussed the Biogen collaboration and Scribes’ progress in developing its genome editing technology with GEN’s premium content channel GEN Edge in May.
Headquartered in Alameda, CA, Scribe develops CRISPR-based treatments through its genetic modification platform, designed to build and apply its suite of CRISPR technologies in therapeutic areas that include neurodegenerative diseases; ophthalmological diseases; multi-system, muscle, and metabolic disorders; and hematopoietic disorders. That platform aims to address safety, delivery, poor editing outcomes, and the long-running bitter legal battle over who invented CRISPR-Cas9.
To avoid intellectual property uncertainty, Scribe has engineered its own CRISPR effectors—CasX enzymes—that are more capable of delivering CRISPR packaged in a viral vector because the protein is less than 1,000 amino acids (aa), compared with the 1,200–1,400-aa size of Cas9. Those “X-Editing” (XE) molecules are highly engineered CRISPR-based enzymes designed to provide combined aspects of greater efficacy, specificity, and deliverability than current CRISPR genome editing technologies.
Scribe completed a $100 million Series B financing in March 2021, five months after it raised a $20 million Series A round.protein cell-based