Adeno-associated virus is a workhorse of genetic medicine, serving as the delivery vehicle for a slew of approved and experimental therapies. But adverse effects are a known risk of AAV; injuries and even patient deaths in clinical trials have sparked several clinical holds in the past year. GenEdit aims to avoid those problems by pursuing a different delivery approach altogether.
The South San Francisco-based startup uses polymer nanoparticles to deliver genetic medicines. GenEdit has animal data showing its technology can deliver a variety of genetic cargoes to different tissues. The approach has drawn interest from at least one clinical-stage genetic medicines company. Now it’s attracting investor interest as well. On Thursday, GenEdit unveiled $26 million in Series A funding from a syndicate that includes pharmaceutical giant Eli Lilly.
The GenEdit technology is called NanoGalaxy. It consists of a library of thousands of chemically distinct polymers, each one with properties that enable the targeting of different tissues and cell types. GenEdit screens this library to identify the polymer best suited to deliver a particular genetic payload to a specific type of tissue in order to treat a disease. Computational analysis and medicinal chemistry are then used to optimize the polymer structure, making the polymer tissue selective. The company says its technology can deliver DNA, RNA, or a CRISPR ribonucleoprotein, depending on the approach needed to produce a therapeutic effect.
In addition to targeted delivery of genetic cargo to specific tissue, GenEdit says its technology is suitable for repeated dosing if more doses are needed. That’s difficult to do with AAV-based therapies because patients develop antibodies to the virus, which would make subsequent doses ineffective. In some cases, people already have preexisting antibodies to AAV. Another advantage over AAV is manufacturability. GenEdit says its polymers are produced in a simpler and less expensive process compared to viral technologies.
GenEdit is using its technology to develop an internal pipeline of therapies addressing central nervous system disorders. So far, the company has not disclosed specific disease targets, but it said some of the new funding will be used to select therapeutic candidates to advance to the clinic. On Thursday, GenEdit CEO and co-founder Kunwoo Lee presented animal data at the TIDES Conference showing how the company’s nanoparticles provided tissue-selective delivery of a genetic medicine dosed intravenous infusion or an injection into the spinal canal.
“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 CNS, with the potential for delivering a therapeutic effect,” Lee said in a prepared statement.
Lee and fellow GenEdit cofounders Hyo Min Park and Niren Murthy worked with Jennifer Doudna, the University of California, Berkeley, scientist who was awarded the Nobel Prize in Chemistry last year for her CRISPR research. GenEdit launched in 2016. In 2017, the scientists published research in Nature Biomedical Engineering showing that the company’s nanoparticles could deliver CRISPR ribonucleoproteins to a range of tissue in mice, correcting the mutation that causes Duchenne muscular dystrophy. The following year, they published research demonstrating the delivery of gene-editing therapies in vitro and in vivo, as well additional research showing that technology could deliver CRISPR into the brain of a mouse model for Fragile X syndrome.
With encouraging early research in hand, GenEdit was able to raise an $8.5 million seed financing in late 2018. The early work also paved the way for research alliances. In 2019, CRISPR-editing biotech Editas Medicine gained an exclusive license to GenEdit’s technologies based on the CRISPR enzyme Cpf1. No financial terms were disclosed but the agreement calls for the two companies to work together to develop Cpf1-based technologies with GenEdit’s platform. If Cambridge, Massachusetts-based Editas commercializes a therapy based on the technology, it will pay its partner royalties from sales.
Besides Lilly, new investors in GenEdit’s Series A round include KTB Network, Company K Partners, Korea Investment Partners, DAYLI Partners, KB Investment, IMM Investment, and TIMEFOLIO Asset Management. They joined earlier investors DCVC Bio, SK Holdings, Bow Capital, and Sequoia Capital in the startup’s latest round of funding.
Photo: BlackJack3D, Getty Images
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