CRISPR/CasX based gene therapy (Biogen/Scribe Therapeutics)

Sanofi is upping its investment in gene editing, expanding an existing collaboration with California-based Scribe Therapeutics with a $40 million deal.Scribe and Sanofi initially inked a partnership in September for the pharmaceutical company to gain access to the startup's ex vivo gene editing technology. Its plan was to use the platform to develop cancer cell therapies.Under the new deal, Sanofi gets to use Scribe's CRISPR-based gene editing technology to develop in vivo therapies as well.Everyone wants to bet on gene editing but the right players are betting in the right areas, said Benjamin Oakes, Scribes co-founder and CEO.As Scribe and Sanofi collaborated on cancer therapies, Oakes said it became apparent there were drug discovery opportunities to explore together.Sanofis first target will be sickle cell disease, according to a Monday statement that was originally set to be released Tuesday morning.Scribe is eligible for payments of up to $1.2 billion if certain development and commercial milestones are hit.Were encouraged by what weve accomplished to date with Scribe in creating ex vivo [natural killer] cell therapies, Christian Mueller, global head of genomic medicine at Sanofi, said in a statement.Spun out of CRISPR pioneer Jennifer Doudnas lab in 2017, Scribe has also struck deals with Prevail, an Eli Lilly subsidiary, and Biogen. Those collaborations focus on drug candidates for neurological diseases and ALS, respectively.Theyre large players who can develop through the clinic and commercialize these drugs in ways that would likely be difficult for us to do, Oakes said.Scribe has raised $120 million across its Series A and B rounds, from investors such as Andreessen Horowitz, Avoro Ventures and OrbiMed.Scribe has studied gene editing using casX enzymes DNA-cutting proteins different than the more familiar cas9 that the company hopes could make more specific and easily deliverable medicines.Editor's note: This story has been updated after publication with comments from Scribe CEO Benjamin Oakes. '

Predicated on the progress of the companies’ first collaboration, Sanofi has selected Scribe’s novel CRISPR by Design™ approach to drive forward a new wave of in vivo genetic medicines Sanofi and Scribe will collaborate to combine Scribe’s proprietary CRISPR X-Editing (XE) technologies with Sanofi’s targeted non-viral delivery technologies for the treatment and prevention of genomic diseases, beginning with the in vivo treatment of sickle cell disease Scribe to receive $40 million upfront, with the potential for more than $1.2 billion in milestones across all programs, and royalties tiered into the double digits with an option to co-promote and profit share on one program coming out of the collaboration ALAMEDA, Calif.--(BUSINESS WIRE)-- Scribe Therapeutics Inc., a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced an expanded collaboration with Sanofi (NASDAQ: SNY). Under the agreement, Sanofi receives an exclusive license to use Scribe’s CRISPR X-Editing (XE) 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 XE technologies and Sanofi’s capabilities in non-viral delivery to address an initial target for sickle cell disease. Scribe’s 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. “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,” said Benjamin Oakes, Ph.D., co-founder and Chief Executive Officer of Scribe. “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.” 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 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, Global Head of Genomic Medicine Unit at Sanofi. “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.” “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, Ph.D., Chief Business Officer at Scribe. “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. 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.” Deal Terms Under the terms of the agreement, Scribe will receive $40 million in 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. About Scribe Therapeutics Scribe Therapeutics is revolutionizing the development of optimized genetic medicines that have the potential to durably treat disease at scale. Our CRISPR by Design™ approach engineers bacterial immune systems into a premier suite of genome editing tools built for unique molecular advantages in activity, specificity and deliverability that translate into safer and more effective genetic therapies. Co-founded by Nobel Prize winner Jennifer Doudna and backed by leading life sciences investors, Scribe is engineering the future of genetic medicine. To learn more, visit . View source version on businesswire.com: Contacts Investor Contact: Stern IR for Scribe Therapeutics investors@scribetx.com Source: Scribe Therapeutics Inc. View this news release online at: