RARE Daily

Scribe Expands Collaboration with Sanofi to Advance In Vivo Genetic Medicines for Sickle Cell and Other Genomic Diseases

July 18, 2023

Rare Daily Staff

Genetic medicines biotech Scribe Therapeutics expanded its collaboration with Sanofi give the pharma giant an exclusive license to use Scribe’s CRISPR X-Editing genome editing technologies for the development of in vivo therapies, including sickle cell disease.

The agreement follows the launch of the companies’ existing collaboration focused on ex vivo editing of natural killer (NK) cell therapies for the treatment of cancer.

The in vivo collaboration will leverage Scribe’s precisely engineered CRISPR X-Editing technologies and Sanofi’s capabilities in non-viral delivery to address an initial target for sickle cell disease. Scribe says its proprietary CRISPR by Design approach powers the only platform that uses holistic engineering to transform bacterial immune systems into therapeutically relevant genome editing technologies. By combining Scribe’s novel technologies with Sanofi’s proven expertise in developing and manufacturing therapeutics on a global scale, the companies will seek to advance potentially breakthrough genomic medicines for the in vivo treatment of sickle cell and other diseases.

“This in vivo collaboration further demonstrates the versatility of Scribe’s design-based approaches to CRISPR, which enables greater activity, specificity, and deliverability, ultimately accelerating the development of life-changing therapeutics,” said Benjamin Oakes, co-founder and CEO of Scribe.

By directly modifying genes within the body, Scribe’s tools for in vivo genome editing offer several key benefits over existing treatment options and late-stage investigational therapies. The innovative in vivo approach being explored under the collaboration has the potential to streamline the treatment process for sickle cell disease, minimizing complications associated with investigational ex vivo autologous treatments, such as conditioning regimen toxicities and a long, complex cell manufacturing process. Importantly, in vivo genome editing also promises to reduce both the cost and time required for treatment, thereby improving access for patients.

“We’re encouraged by what we’ve accomplished to date with Scribe in creating ex vivo NK 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,” said Christian Mueller, global head of Genomic Medicine Unit at Sanofi.

Under the terms of the agreement, Scribe will receive $40 million in an upfront payment and is eligible to potentially receive more than $1.2 billion based on the successful completion of certain development and sales milestones. Scribe will also be eligible to receive tiered royalties that range from high single digits to low double digits on net future sales on any products that may result from this agreement. Scribe has a right to opt-in to development cost sharing, as well as co-promotion and profit and loss sharing in the U.S. on one future program.

Photo: Benjamin Oakes, co-founder and CEO of Scribe

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