GenEdit believes its polymer nanoparticles have the potential to avert the safety problems of some gene therapy delivery methods, and the five-year-old biotech now has $26 million to work on proving it, thanks to an investor group that includes the likes of Eli Lilly.
Derived out of a lab at the University of California, Berkeley, GenEdit also shared new in vivo data on its polymer nanoparticles showing tissue-selective delivery and the ability to maintain functional activity after repeat dosing, the biotech said Thursday.
The goal is to overcome the safety problems that can come with adeno-associated virus, or AAV, a viral vector used to deliver gene therapies. Problems with that mode of delivery triggered an FDA advisory committee meeting earlier this month, where experts discussed the future of preclinical research and how to deliver the treatments safely.
“The data presented today indicates we can overcome the historic challenges in the field of gene therapy and establishes the feasibility of using GenEdit’s polymer nanoparticles to deliver genetic medicines to a variety of tissues, including the [central nervous system], with the potential for delivering a therapeutic effect,” said Kunwoo Lee, Ph.D., CEO and cofounder, in a statement.
GenEdit’s platform houses thousands of polymers that are chemically distinct and can deliver DNA, RNA or CRISPR ribonucleoprotein, depending on whether the goal is to add, delete, edit or silence a gene.
The financing and data come a day after GenEdit named Romuald Corbau, Ph.D., as chief scientific officer and Aaron Mishel as chief financial officer. Corbau previously led the research team working on AAV-based gene therapies at Freeline, and before that he was translational lead at Spark Therapeutics. Mishel joins from Magnetic Insight, where he held the same post.
Lilly joins the South San Francisco biotech’s roster of backers, which includes DCVC Bio, Sequoia Capital, Korea Investment Partners and nearly a dozen other investors.
GenEdit has been working off an $8.5 million seed financing from December 2018.