Jennifer Doudna-founded in vivo gene editing biotech launches with $82M financing

Gene editing biotech Azalea Therapeutics announced its official launch and completion of $82 million in seed and Series A financing to advance its proprietary Enveloped Delivery Vehicle (EDV) technology.

The $82 million funding, which includes a recently closed $65 million Series A financing, was led by Third Rock Ventures, with participation from RA Capital Management, Yosemite, Sozo Ventures and select individual investors. Funds will be used to advance Azalea’s CD19-based in vivo CAR-T therapy for B cell malignancies and autoimmune diseases through IND-enabling studies and into the clinic, progress its BCMA-targeted in vivo CAR-T program for multiple myeloma and an undisclosed program for solid tumors, while exploring expansion of the platform to other cell types.

Currently based at UC Berkeley’s Bakar Bio Labs, Azalea grew out of collaborative research conducted at the Innovative Genomics Institute in Dr. Jennifer Doudna’s laboratory and in Dr. Justin Eyquem’s laboratory at UC San Francisco, as part of the Gladstone/UCSF Institute for Genomic Immunology.

According to the company, its EDV technology selectively targets cells, delivering transient CRISPR-Cas9 cargo to mediate programmable genome editing. Combining this with a highly efficient T cell-tropic AAV to deliver a promoterless homology-directed repair template enables programmable, site-specific large gene insertion at defined genomic sites within the T cell. This dual-vector platform provides multiple layers of precision by achieving cell- and genomic site-specific gene insertion under regulatory control of a native promoter, with the potential to increase durability, efficacy and safety.

Azalea has utilized this approach to insert CAR genes under an endogenous, T cell-restricted promoter in vivo — enabling durable therapeutic benefit while eliminating the need for ex vivo manufacturing and lymphodepletion.