Adeno-associated virus is a workhorse in genetic medicine, serving as a delivery vehicle for a multitude of approved and experimental therapies. But side effects are a known risk of AAV; injuries and even deaths of patients in clinical trials have triggered several clinical shutdowns over the past year. GenEdit aims to avoid these issues by pursuing a completely different delivery approach.
The South San Francisco-based startup uses polymer nanoparticles to deliver genetic drugs. GenEdit has animal data showing that its technology can deliver a variety of genetic cargoes to different tissues. The approach has attracted interest from at least one clinical-stage genetic drug company. Now it is also arousing the interest of investors. On Thursday, GenEdit unveiled $ 26 million in Series A funding from a syndicate that includes pharmaceutical giant Eli Lilly.
GenEdit technology is called NanoGalaxy. It consists of a library of thousands of chemically distinct polymers, each with properties to target different tissues and cell types. GenEdit examines this library to identify the polymer best suited to deliver a particular genetic payload to a specific type of tissue to treat disease. Computer analysis and medicinal chemistry are then used to optimize the structure of the polymer, making the polymer tissue selective. The company claims that its technology can deliver DNA, RNA, or CRISPR ribonucleoprotein, depending on the approach needed to produce a therapeutic effect.
In addition to the targeted delivery of genetic cargoes to specific tissues, GenEdit says its technology is suitable for repeated dosing if additional doses are needed. This is difficult to do with AAV-based therapies because patients develop antibodies against the virus, which would make subsequent doses ineffective. In some cases, people already have pre-existing antibodies to AAV. Another advantage over AAV is the possibility of manufacturing. GenEdit claims that its polymers are produced in a simpler and cheaper process than viral technologies.
GenEdit uses its technology to develop an internal pipeline of therapies to treat disorders of the central nervous system. So far, the company has not disclosed specific targets for the disease, but 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 an intravenous infusion dosed with a genetic drug or drug. ‘an injection into the spinal canal.
âThe data presented today indicates that we can overcome historic challenges in the field of gene therapy and establish the feasibility of using GenEdit’s polymer nanoparticles to deliver genetic drugs to a variety of tissues, including the CNS. , with the potential to produce a therapeutic effect, âLee said in a prepared statement.
Lee and fellow GenEdit co-founders Hyo Min Park and Niren Murthy worked with Jennifer Doudna of the University of California, Berkeley, a scientist who received the Nobel Prize in Chemistry last year for her CRISPR research. GenEdit was launched in 2016. In 2017, scientists published research in Nature Biomedical Engineering showing that the company’s nanoparticles could deliver CRISPR ribonucleoproteins to a range of tissues in mice, correcting the mutation that causes muscular dystrophy. Duchenne. The following year, they published research demonstrating the provision of gene editing therapies in vitro and in vivo, as well as additional research showing that the technology could deliver CRISPR in the brain of a mouse model for the syndrome. fragile X.
By encouraging early research in hand, GenEdit was able to raise $ 8.5 million in seed funding at the end of 2018. Early work also paved the way for research alliances. In 2019, CRISPR Editas Medicine biotechnology was granted an exclusive license to GenEdit technologies based on the CRISPR Cpf1 enzyme. No financial terms were disclosed, but the deal calls for the two companies to work together to develop Cpf1-based technologies with GenEdit’s platform. If Cambridge, Massachusetts-based Editas commercializes technology-based therapy, it will pay its partner royalties on the sales.
Besides Lilly, GenEdit’s new Series A investors include KTB Network, Company K Partners, Korea Investment Partners, DAYLI Partners, KB Investment, IMM Investment and TIMEFOLIO Asset Management. They joined previous investors DCVC Bio, SK Holdings, Bow Capital and Sequoia Capital in the startup’s latest funding round.
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