Scribe, Sanofi Report Milestone for In Vivo Program Collaboration
Although specifics about the milestone were not immediately provided, Scribe is eligible to receive more than $1.2 billion for certain research, development, regulatory, and commercial benchmarks.
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Scribe Therapeutics, a genetic medicines company based in Alameda, Calif., announced on Jan. 13, 2025 that it had achieved a success milestone for one of the targets in a research collaboration with Sanofi (1). That partnership, which centers around Scribe’s focus on clustered regularly interspaced short palindromic repeats (CRISPR) technology for cell therapies, began in 2022 and was expanded upon in 2023 with the intention of advancing in-vivo medicines for genomic diseases (2).
Scribe’s CRISPR by Design is a data-driven design and engineering approach that was developed to optimize the company’s CRISPR-based platforms and assets (1). It works by engineering bacterial immune systems into a suite of genome and epigenome editing tools, with the end goal of providing safer and more effective genetic therapies by improving activity, specificity, and deliverability, according to a press release from Scribe.
The release did not reveal exact details of the milestone reached, but the company said it is eligible to receive payments of more than $1.2 billion for the achievement of certain research, development, regulatory, and commercial milestones, as well as royalties described in the “high-single-digit to mid-teen” range (1).
“We are excited about the progress we have made towards creating potentially curative in vivo genetic medicines with a partner like Sanofi,” said Benjamin Oakes, PhD, co-founder and chief executive officer of Scribe, in the press release. “The complementary expertise of our teams has enabled the program to move quickly and attain early validation of our CRISPR genome editing technologies. We look forward to further advancement of this program with a direct line of sight towards bringing breakthrough CRISPR-based therapeutics to patients with significant unmet need.”
“Advancing our in vivo program with Sanofi speaks to the versatility and strength of Scribe’s CRISPR-based platforms,” said Svetlana Lucas, PhD, chief business officer at Scribe, in the release. “We are pleased to reach this important milestone and continue progressing our collaboration to accelerate and expand our patient impact globally.”
The 2023 expansion, previously reported, combined Scribe’s proprietary CRISPR XE, or X-Editing, technologies with Sanofi’s targeted, non-viral delivery technologies (2). Scribe received $40 million up front, and it was at that time that the company said the additional $1.2 billion might eventually be available if certain milestones were hit.
At the time, the genomic medicines being worked on through this partnership were primarily concerned with in vivo treatment of sickle cell disease, although Scribe said other diseases would also be addressed (2).
“We’re encouraged by what we’ve accomplished to date with Scribe in creating ex-vivo NK [natural killer] cell therapies and now look forward to accelerating our ability to effectively leverage genome editing in vivo through Sanofi’s innovative research work in targeted lipid nanoparticles (LNPs), for in-vivo therapies with the potential to dramatically improve treatment outcomes and ultimately to change patients’ lives,” Christian Mueller, global head of the Genomic Medicine Unit at Sanofi, said in the 2023 release (2).
References
1. Scribe Therapeutics. Scribe Therapeutics Achieves Milestone for In Vivo Program in Collaboration with Sanofi. Press Release. Jan. 13, 2025.
2. Scribe Therapeutics. Scribe Therapeutics Expands Collaboration with Sanofi to Advance In Vivo Genetic Medicines for Sickle Cell and Other Genomic Diseases. Press Release. July 17, 2023.
