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Gene Therapy Delivery Company Gains $26M in Early Funds

DNA puzzle

(Arek Socha, Pixabay)

23 Sept. 2021. A developer of delivery methods for gene therapies promising greater safety for patients is raising $26 million in its first venture funding round. GenEdit Inc., a five year-old enterprise in South San Francisco, is spun-off from drug delivery research labs at University of California in Berkeley.

GenEdit aims to make gene therapies safer for patients. Gene therapies deliver healthy or edited genes to produce beneficial proteins or correct genetic disorders, which are often difficult to treat or incurable for many people. In most cases today, gene therapies are delivered with benign viruses, such as adenoviruses, naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease. However, adenoviruses can also generate an immune response, dangerous for patients with weakened immune systems and those with respiratory or cardiac disorders.

GenEdit is commercializing research from the lab of UC-Berkeley professor Niren Murthy that studies delivery of small molecule and biologic therapies with micro- and nanoscale particles. Murthy, with doctoral students at the time Kunwoo Lee and Hyo Min Park, demonstrated the use of gold nanoparticles to deliver Cas9 enzymes with the gene-editing technique Crispr to edit the relevant genes in lab mice induced with the inherited diseases Duchenne muscular dystrophy and fragile X syndrome, a common single-gene form of autism spectrum disorders. Murthy, Lee, and Park are co-founders of GenEdit, with Lee and Park now serving as CEO and senior vice-president respectively.

Produce nanoparticles with specific properties

In August 2018, the UC-Berkeley team demonstrated delivery of more precise Crispr Cpf1 enzymes with nanoscale particles made of polymers instead of gold. The use of polymer particles became the basis of GenEdit’s technology, called NanoGalaxy. The company says it created a library of water-soluble and biocompatible polymer nanoparticles, rather than viruses or lipids, to deliver gene therapies designed to interact with specific cells and tissue in nerves, muscle, liver, and blood. And GenEdit says it can produce precise nanoparticles designed with physical or chemical properties to meet specific genetic payloads or therapeutic needs.

CEO Lee is scheduled to describe advances in the company’s technology later today at the TIDES 2021 conference in Boston. Lee will offer preclinical data showing GenEdit’s polymer nanoparticles can be delivered to specific tissue types with intravenous infusions and to the central nervous system or CNS. Findings also show GenEdit’s polymer nanoparticles can be dosed multiple times without losing functional activity of their payloads.

“The data presented today,” says Lee in a company statement, “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.”

GenEdit is raising $26 million in its first venture financing round from new and current investors. The company’s new funders are drug maker Eli Lilly & Co., KTB Network, Ltd., Company K Partners, Korea Investment Partners, DAYLI Partners, KB Investment, IMM Investment, and TIMEFOLIO Asset Management. They join existing investors DCVC Bio, SK Holdings, Bow Capital, and Sequoia Capital. Science & Enterprise reported on GenEdit’s seed funding in December 2018, where it raised $8.5 million.

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