- Sarepta, a biotech best known for its development of gene therapies, has for years been quietly exploring gene editing as well, and on Tuesday revealed a partnership with a South San Francisco startup that's been in place since December 2020.
- The startup, called GenEdit, is working on an alternative method for delivering gene editing medicines into the body than the options that are commonly used by drugmakers. Under the companies' agreement, Sarepta holds an exclusive option to license rights to GenEdit's delivery technology for up to four neuromuscular disease indications.
- GenEdit will get as much as $57 million in near-term payments from Sarepta, and could receive further payment if the research partnership hits unspecified development, regulatory and commercial milestones. Last September, GenEdit raised $26 million in venture funding from Eli Lilly and nearly a dozen other investors, including original backer Sequoia Capital.
The sharp acceleration of research into gene therapy and gene editing over the past several years has been accompanied by new efforts to improve on the tools available for delivering those would-be treatments into patients.
Currently, developers rely on several types of neutered viruses known as viral vectors or on tiny spheres of lipids called lipid nanoparticles to deliver gene therapies or gene editing medicines into cells. The latter method has gained new prominence with the success of the COVID-19 vaccines from Moderna and partners Pfizer and BioNTech.
While viral vectors and lipid nanoparticles have made genetic treatment of many diseases possible, each tool has limitations that restrict what tissues they can reach and what therapeutic "cargo" they can carry. Most infused treatments end up in the liver, for instance, presenting a challenge for companies seeking to treat diseases of other organs, muscles or the central nervous system.
With GenEdit, Sarepta says it was attracted to the non-lipid polymer nanoparticles the company is developing and hopes to use that platform for gene editing treatments aimed at neuromuscular diseases.
According to GenEdit, its polymer nanoparticles — dubbed NanoGalaxy — are able to reach a range of different cell types and tissues, while carrying DNA, RNA or CRISPR ribonucleoproteins.
In a emailed statement, a spokesperson for Sarepta indicated GenEdit's nanoparticles could offer the potential for redosing. They're also designed to reach muscle tissue with more specificity and potentially transport larger therapeutic payloads.
Recent preclinical testing has supported some of that potential for inside-the-body gene editing, Sarepta said.
Much of Sarepta's current drug pipeline is made up of gene replacement therapies, like the treatment for Duchenne muscular dystrophy that the biotech has advanced into late-stage testing.
But over the past several years, Sarepta has struck research partnerships with Duke University and Harvard University, while setting up a gene editing "innovation center" in North Carolina. The GenEdit collaboration adds to those efforts.
"Our gene editing approaches are early, and because we know that systemic non-viral muscle delivery is challenging we do have a few options we are exploring to help increase the chances of success," said the Sarepta spokesperson in an email. "The early data from GenEdit's platform show penetration of muscle tissue and we're excited to continue our work with them."