Adeno-associated virus is a workhorse of genetic medication, serving because the supply car for a slew of accepted and experimental therapies. However opposed results are a recognized threat of AAV; accidents and even affected person deaths in medical trials have sparked a number of medical holds up to now yr. GenEdit goals to keep away from these issues by pursuing a special supply strategy altogether.
The South San Francisco-based startup makes use of polymer nanoparticles to ship genetic medicines. GenEdit has animal information displaying its expertise can ship quite a lot of genetic cargoes to totally different tissues. The strategy has drawn curiosity from not less than one clinical-stage genetic medicines firm. Now it’s attracting investor curiosity as properly. On Thursday, GenEdit unveiled $26 million in Collection A funding from a syndicate that features pharmaceutical big Eli Lilly.
The GenEdit expertise is named NanoGalaxy. It consists of a library of 1000’s of chemically distinct polymers, each with properties that allow the focusing on of various tissues and cell varieties. GenEdit screens this library to determine the polymer greatest suited to ship a specific genetic payload to a selected sort of tissue with a purpose to deal with a illness. Computational evaluation and medicinal chemistry are then used to optimize the polymer construction, making the polymer tissue selective. The corporate says its expertise can ship DNA, RNA, or a CRISPR ribonucleoprotein, relying on the strategy wanted to supply a therapeutic impact.
Along with focused supply of genetic cargo to particular tissue, GenEdit says its expertise is appropriate for repeated dosing if extra doses are wanted. That’s troublesome to do with AAV-based therapies as a result of sufferers develop antibodies to the virus, which might make subsequent doses ineffective. In some circumstances, folks have already got preexisting antibodies to AAV. One other benefit over AAV is manufacturability. GenEdit says its polymers are produced in a less complicated and cheaper course of in comparison with viral applied sciences.
GenEdit is utilizing its expertise to develop an inner pipeline of therapies addressing central nervous system problems. Thus far, the corporate has not disclosed particular illness targets, but it surely mentioned a few of the new funding can be used to pick out therapeutic candidates to advance to the clinic. On Thursday, GenEdit CEO and co-founder Kunwoo Lee offered animal information on the TIDES Convention displaying how the corporate’s nanoparticles offered tissue-selective supply of a genetic medication dosed intravenous infusion or an injection into the spinal canal.
“The info offered at the moment signifies we will overcome the historic challenges within the subject of gene remedy and establishes the feasibility of utilizing GenEdit’s polymer nanoparticles to ship genetic medicines to quite a lot of tissues, together with the CNS, with the potential for delivering a therapeutic impact,” Lee mentioned in a ready assertion.
Lee and fellow GenEdit cofounders Hyo Min Park and Niren Murthy labored with Jennifer Doudna, the College of California, Berkeley, scientist who was awarded the Nobel Prize in Chemistry final yr for her CRISPR analysis. GenEdit launched in 2016. In 2017, the scientists revealed analysis in Nature Biomedical Engineering displaying that the corporate’s nanoparticles might ship CRISPR ribonucleoproteins to a spread of tissue in mice, correcting the mutation that causes Duchenne muscular dystrophy. The next yr, they revealed analysis demonstrating the supply of gene-editing therapies in vitro and in vivo, as properly further analysis displaying that expertise might ship CRISPR into the mind of a mouse mannequin for Fragile X syndrome.
With encouraging early analysis in hand, GenEdit was in a position to elevate an $8.5 million seed financing in late 2018. The early work additionally paved the best way for analysis alliances. In 2019, CRISPR-editing biotech Editas Medication gained an unique license to GenEdit’s applied sciences primarily based on the CRISPR enzyme Cpf1. No monetary phrases have been disclosed however the settlement requires the 2 firms to work collectively to develop Cpf1-based applied sciences with GenEdit’s platform. If Cambridge, Massachusetts-based Editas commercializes a remedy primarily based on the expertise, it should pay its accomplice royalties from gross sales.
In addition to Lilly, new traders in GenEdit’s Collection A spherical embrace KTB Community, Firm Ok Companions, Korea Funding Companions, DAYLI Companions, KB Funding, IMM Funding, and TIMEFOLIO Asset Administration. They joined earlier traders DCVC Bio, SK Holdings, Bow Capital, and Sequoia Capital within the startup’s newest spherical of funding.
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