Eli Lilly gets behind the latest approach to solving gene therapy’s delivery problem

This story features GenEdit, a former QB3 Garage@Berkeley company. The incubator space in Stanley Hall features more than 800 square feet of laboratory space for startup companies. 

Kunwoo Lee was a graduate student at UC-Berkeley when gene editing pioneer Jennifer Doudna — who happened to work in the same building where he studied — published a paper on CRISPR/Cas9. So he did what any aspiring bioengineer would do: He ran to her lab, and grabbed a postdoc there.

“We started really thinking about the future coming (for) gene therapy and gene editing,” he said.

Lee’s research with Doudna led him to co-found a small San Francisco-based biotech called GenEdit in 2016, the same year he graduated. After five quiet years, the team is now unveiling a $26 million Series A round with support from some big names like Eli Lilly to fund their work on one of the most pressing challenges in gene therapy: what Lee calls the “delivery problem.”

One of the big issues with adeno-associated viruses (AAV) — the nanometer-sized delivery system used in current gene therapies — is that some patients have a natural immunity. Or, once they’re dosed with an AAV-based therapy, they mount an immune response, making it difficult to re-dose.

GenEdit, though, is working on a non-viral, non-lipid polymer nanoparticle approach, which could make it possible to re-dose gene therapies, among other benefits. Their NanoGalaxy platform consists of a large library of chemically made polymers, which are screened for their ability to selectively target different tissues and cell types. The nanoparticles can carry DNA, RNA, or CRISPR ribonucleoprotein, CSO Romuald Corbau told Endpoints News, depending on whether scientists are looking to delete, edit or silence a gene.

Profile of romuald corbau
Romuald Corbau

Because the nanoparticles are chemically made, patients won’t have a pre-existing immunity, or generate an antibody response, he said. Plus, the particles can carry more cargo, and come with a lower manufacturing cost compared to traditional AAV.

The ability to re-dose could allow scientists to treat some people with a lower dose to start, potentially avoiding some of the safety issues involved with giving higher doses.

“We have generated the very first sets of the data that the iteration process works,” Lee said of the company’s in vivo delivery approach, adding that preclinical data suggest the nanoparticles can be dosed multiple times while maintaining functional activity of the payload.

While Lee declined to provide a timeline for GenEdit’s push toward the clinic, Corbau said the Series A will be used in part to develop an IND-ready drug. He didn’t say much about initial indications, only giving away that the company will be focusing on diseases of the nervous system.

More importantly, the Series A funds will be used to build out the roughly 30-person team. Corbau joined GenEdit about a month ago — less than a year after being promoted to CSO at the AAV-based gene therapy company Freeline. Prior to Freeline, he served as the translational lead at Spark Therapeutics, where he worked on Luxturna, the first-approved gene therapy. Lee also recently announced the addition of Aaron Mishel, who left molecular imaging leader Magnetic Insight to take the CFO spot at GenEdit.

The crowd of companies searching for gene therapy 2.0 is only getting larger. Anjarium uncloaked earlier this month with backing from Pfizer’s venture arm to develop DNA-based gene vectors. Just a few months ago, GlaxoSmithKline vet Brian McVeigh launched Code Biotherapeutics to find a nonviral delivery method. And Generation Bio closed a $230 million IPO last June to fund its preclinical push for a non-viral gene therapy for diseases of the liver and retina.

Eli Lilly, KTB Network, Company K Partners, Korea Investment Partners, DAYLI Partners, KB Investment, IMM Investment, TIMEFOLIO Asset Management, DCVC Bio, SK Holdings, Bow Capital, and Sequoia Capital all chipped into GenEdit’s latest round.

“There are so many other indications that we are currently not able to apply (gene therapies), but if there is a delivery technology, we can unlock all of those parts,” Lee said.

This story was reposted from Endpoints News. Read more here