Lilly’s Prevail, Doudna’s Scribe Partner on Genetic Treatments for Neuro Diseases
Pictured: Illustration of CRISPR-Cas9 editing DNA / iStock, Artur Plawgo
Tuesday, Jennifer Doudna-founded Scribe Therapeutics and Eli Lilly subsidiary Prevail entered into a strategic collaboration to develop CRISPR-based genetic medicines for neurological and neuromuscular indications.
Under the terms of the agreement, Prevail will make a $75 million upfront payment and equity investment, as well as pledge more than $1.5 billion in development and commercial milestones. Scribe will also be entitled to additional research financing and tiered royalties up to low double-digits on net future sales.
The gene editing start-up can also opt-in and co-fund one collaboration program under the deal, which would entitle it to share in its profits.
At the core of the research collaboration are Scribe’s proprietary X-editing (XE) technologies, which allow the company to develop in vivo treatments with three key attributes: deliverability, specificity and greater editing activity. In turn, these treatments directly modify their target genes in the body to achieve a therapeutic effect while also limiting safety concerns, according to the company.
XE was developed using Scribe’s CRISPR by Design platform, which leverages and harnesses the bacterial immune system to produce a suite of genome editing technologies.
The Prevail partnership focuses on neurological and neuromuscular indications. Scribe lists several such indications on its website, including Huntington’s disease, spinal muscular atrophy and Parkinson’s disease as potential targets for its technology. The company is also working to develop genomic tools for these diseases, along with other indications such as sickle cell disease, glaucoma and Duchenne muscular dystrophy.
Co-founded by Doudna, who pioneered the CRISPR/Cas9 gene editing system with Emmanuelle Charpentier, Scribe emerged from stealth in October 2020 to develop and advance novel CRISPR-based therapies.
At the time, the start-up also signed a $15 million deal with Biogen that had the potential to balloon to $400 million. The partners deepened their relationship in May 2022 and expanded the collaboration to a second disease target.
Scribe picked up another powerhouse partner in September 2022, when it bagged a $1 billion cell therapy deal with Sanofi. The agreement will leverage Scribe’s CRISPR technologies to develop natural killer cell-based medicines for cancer.
The start-up secured 18th place on BioSpace’s Top Life Science Startups to Watch in 2021.
In March 2021, Scribe closed its Series B funding round, counting $100 million in capital, which the company funneled into its pipeline of gene therapies for neurodegeneration and other diseases with high unmet need, as well as into the development of its gene editing and delivery technologies.
In addition to Doudna, Scribe is backed by several big supporters and investors, including Avoro Ventures, OrbiMed Advisors and Perceptive Advisors.
Tristan Manalac is an independent science writer based in metro Manila, Philippines. He can be reached at email@example.com or firstname.lastname@example.org.