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The adeno-associated virus is a workhorse of genetic medicine and serves as a carrier for a number of approved and experimental therapies. But side effects are a known risk of AAV; Injuries and even deaths to patients in clinical trials have sparked several clinical problems over the past year. GenEdit aims to avoid these issues by taking 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 wide variety of genetic loads to different tissues. The approach has attracted the interest of at least one clinical-stage genetic drug company. Now it is also piquing investor interest. On Thursday, GenEdit unveiled $ 26 million in Series A funding from a consortium that includes pharmaceutical giant Eli Lilly.
The GenEdit technology is called NanoGalaxy. It consists of a library of thousands of chemically different polymers, each with properties that allow it to be targeted to different tissues and cell types. GenEdit searches this library to identify the polymer best suited to deliver a particular genetic payload to a particular type of tissue to treat a disease. Computer analysis and medicinal chemistry are then used to optimize the polymer structure, making the polymer fabric selective. The company says its technology can deliver DNA, RNA, or a CRISPR ribonucleoprotein, whichever approach is required to achieve a therapeutic effect.
In addition to targeted delivery of genetic cargo to specific tissues, GenEdit says its technology is suitable for repeated dosing when more doses are needed. This is difficult with AAV-based therapies because patients develop antibodies to the virus that would render subsequent doses ineffective. In some cases, people already have antibodies to AAV. Another advantage over AAV is the manufacturability. GenEdit says its polymers are made in a simpler and cheaper process compared to viral technologies.
GenEdit uses its technology to develop an in-house pipeline of therapies for central nervous system disorders. To date, the company has not disclosed any specific disease targets, but some of the new funding will be used to select therapeutic candidates to enter the clinic. On Thursday, GenEdit CEO and co-founder Kunwoo Lee presented animal data at the TIDES conference showing how the company’s nanoparticles enable tissue-selective administration of a genetically dosed intravenous infusion or an injection into the spinal canal.
“The data presented today shows that we can overcome the historic challenges in gene therapy and demonstrate the feasibility of using GenEdit’s polymer nanoparticles to deliver genetic drugs to a wide variety of tissues, including the CNS, with the potential for one deliver therapeutic effect. “Said Lee in a prepared statement.
Lee and GenEdit co-founders, Hyo Min Park and Niren Murthy, worked with Jennifer Doudna of the University of California, Berkeley, who won the Nobel Prize in Chemistry last year for her CRISPR research. GenEdit was introduced in 2016. In 2017, the scientists published research in Nature Biomedical Engineering showing that the company’s nanoparticles could deliver CRISPR ribonucleoproteins into a range of tissues from mice and correct 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 as additional research showing that the technology could bring CRISPR into the brain of a mouse model of Fragile X syndrome.
Thanks to funding for early research, GenEdit raised $ 8.5 million in seed funding at the end of 2018. The early work also paved the way for research alliances. In 2019, the CRISPR editing biotech company Editas Medicine received an exclusive license for GenEdit’s technologies, which are based on the CRISPR enzyme Cpf1. No financial terms were announced, but the agreement provides that the two companies will work together to develop Cpf1-based technologies using GenEdit’s platform. When Editas, based in Cambridge, Massachusetts, commercializes a therapy based on the technology, it pays its partners royalties on the sale.
The new investors in GenEdit’s Series A round include Lilly 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 round of funding.
Photo: BlackJack3D, Getty Images