GenEdit

GenEdit, spun-out of the University of California, develops novel therapies through targeted in vivo gene delivery of genetic medicines (gene editing done inside the body). It leverages its proprietary NanoGalaxy platform based on non-viral, non-lipid polymer nanoparticles that are reportedly safer than commonly used delivery vectors such as adeno-associated virus (AAV). The platform accommodates a library of thousands of novel nanoparticles capable of delivering the medicine payloads to the relevant tissues to treat a disease. 

The company boasts several benefits to its platform including simpler and cost-effective manufacturing process compared to viral systems; the ability to redose medicine due to lack of immune response; payload flexibility to deliver various elements such as ribonucleic acid (RNA), DNA, CRISPR, and AAV (of any size); and tissue selectivity to perform in vivo delivery to any desired tissue. GenEdit is also developing its own pipeline of therapeutic candidates targeted at central nervous system (CNS) diseases, which is yet to be revealed.

Key customers and partnerships

Most recently, in January 2024, GeneEdit entered a multi-year partnership with Genentech , a Roche Group member, to develop therapies for autoimmune diseases. The partnership leveraged GenEdit's NanoGalaxy platform to develop hydrophilic nanoparticle (HNP) medications for autoimmune diseases. Genentech agreed to take charge of preclinical, clinical, regulatory development, and product commercialization. As a result, GenEdit received an upfront payment of USD 15 million in advancements and milestone payments, along with royalties from global net sales.

The company also secured a partnership with Sarepta Therapeutics in February 2022 to license its delivery technology for four of Sarepta’s neuromuscular disease indications. It was eligible to receive more than USD 57 million in near-term payments and further milestone payments from Sarepta. Similarly, in December 2019, the company partnered with Editas Medicine to offer its delivery technology to Editas for CRISPR-based therapeutic delivery.

Funding and financials

In January 2024, the company raised USD 24 million in a Series A1 funding round which saw participation from both existing and new investors. The funding was set aside to support the continued development of the NanoGalaxy platform and the company’s pipeline of preclinical therapeutic candidates.