Scribe, Sanofi expand research collaboration

The companies are developing in vivo treatments for SCD and other disorders

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by Mary Chapman |

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Genetic medicines company Scribe Therapeutics has expanded its collaboration with Sanofi to advance the development of a new wave of in vivo treatments for sickle cell disease (SCD) and other disorders.

The collaboration leverages Scribe’s proprietary CRISPR X-Editing technologies and Sanofi’s targeted non-viral delivery technologies for the treatment and prevention of genetic disorders, starting with the in vivo treatment of SCD, an inherited blood disorder thought to affect between 70,000 and 100,000 U.S. residents.

While ex vivo therapies require cells collected from a patient to then be genetically modified in the lab and eventually returned to the patient, in vivo therapies use certain methods that directly modify genes within a patient’s cells.

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Taking a holistic approach to sickle cell disease management

“We are continually impressed by the expertise, rigor, and urgency that Sanofi has brought to our existing ex vivo collaboration, as well as their rapid advancements and innovation in non-viral delivery technologies. Now, we are thrilled to expand our work together into new areas of high unmet need,” Benjamin Oakes, PhD, Scribe’s co-founder and CEO, said in a press release.

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

The agreement follows an ongoing collaboration between the two companies focused on ex vivo editing of immune cells for treating different types of cancer.

Terms of the new agreement

Terms of the new collaboration call for Scribe to receive $40 million upfront, with possible payments totaling more than $1.2 billion contingent upon the successful completion of specific development and sales milestones. Scribe may receive tiered royalties on net future sales of any treatments resulting from the agreement. Scribe also is allowed to seek shared development costs in addition to co-promotion and profit and loss sharing in the U.S. on a single future program derived from the collaboration.

“We are pleased to expand our work with Scribe, an illustration of our shared commitment to advance best-in-class genome editing therapies for patients in need,” said Christian Mueller, Sanofi’s global head of genomic medicine unit.

“We’re encouraged by what we’ve accomplished to date with Scribe in creating ex vivo [immune] cell therapies and now look forward to accelerating our ability to effectively leverage genome editing in vivo through Sanofi’s innovative research work,” Mueller said, adding that this has “the potential to dramatically improve treatment outcomes and ultimately to change patients’ lives.”

By combining Scribe’s technologies with Sanofi’s expertise in developing and producing treatments on a global scale, the companies will strive to advance the development of potentially breakthrough genomic therapies for the in vivo treatment of SCD and other conditions.

CRISPR by Design

Scribe’s CRISPR by Design approach powers a platform that employs holistic engineering to transform a bacterial immune system mechanism into genome-editing technologies that are therapeutically relevant.

Through direct modification of human genes, Scribe’s in vivo genome-editing tools potentially may streamline the protracted and complex cell manufacturing processes associated with current treatment options and late-stage investigational treatments.

The in vivo approach being explored in sickle cell potentially can mitigate certain complications, including conditioning regimen toxicities associated with experimental ex vivo autologous therapies. In vivo genome editing also could decrease the cost and time necessary for treatment, thus improving patient access.

“Scribe’s platform holds tremendous potential to address an unprecedented number of therapeutically relevant targets across the human genome for many serious diseases,” said Svetlana Lucas, PhD, Scribe’s chief business officer.

“We are excited to partner with companies like Sanofi that offer not only disease and drug development expertise but also the resources and technologies enabling the delivery of our molecules in vivo to specific disease tissues, allowing us to develop better therapies,” Lucas added. “We look forward to our continued collaboration with Sanofi to expand the application of Scribe’s best-in-class genome editing tools and fully realize the promise of CRISPR for patients as broadly and rapidly as possible.”