Azalea Therapeutics has emerged from stealth mode with $82 million in financing to advance what the company claims is the first precision in vivo genome engineering platform capable of creating therapeutic cells directly inside patients. The Berkeley-based biotechnology company announced its official launch alongside completion of seed and Series A funding rounds led by Third Rock Ventures, with participation from RA Capital Management, Yosemite, Sozo Ventures and select individual investors.
The company's proprietary Enveloped Delivery Vehicle (EDV) technology represents a potential paradigm shift in cell and gene therapy by enabling the engineering of functional CAR-T cells within the patient's body, bypassing the complex and costly ex vivo manufacturing processes that currently limit access to these treatments.
Revolutionary Dual-Vector Platform
Azalea's EDV technology selectively targets cells, delivering transient CRISPR-Cas9 cargo to mediate programmable genome editing. The platform combines this targeted delivery with a highly efficient T cell-tropic AAV that delivers a promoterless homology-directed repair template, enabling programmable, site-specific large gene insertion at defined genomic sites within T cells.
This dual-vector approach provides multiple layers of precision by achieving both cell-specific and genomic site-specific gene insertion under regulatory control of a native promoter. The company has demonstrated the ability to insert chimeric antigen receptor (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.
"At Azalea, we are aiming to make cell therapy as simple as dosing a medicine," said Jenny Hamilton, Ph.D., co-founder, president and chief executive officer of Azalea Therapeutics. "By combining cell-selective delivery with site-specific genome integration, we can create potent and durable in vivo CAR-T and other cell-based therapies inside the body and extend the reach of genome engineering to many more patients."
Distinguished Scientific Leadership
The company was founded by a team of leading scientists including Jennifer Doudna, Ph.D., the 2020 Nobel laureate in chemistry and Li Ka Shing Chancellor's Chair in Biomedical and Health Sciences at UC Berkeley. Doudna serves alongside co-founders Michael Fischbach, Ph.D., the Liu Family Professor of Bioengineering at Stanford University; Justin Eyquem, Ph.D., associate professor of medicine at UCSF; and Jenny Hamilton, Ph.D., who serves as CEO.
Azalea grew out of collaborative research conducted at the Innovative Genomics Institute in Dr. Doudna's laboratory and in Dr. Eyquem's laboratory at UC San Francisco, as part of the Gladstone/UCSF Institute for Genomic Immunology. The early academic work was supported by philanthropic grants from the Yosemite team and the Parker Institute for Cancer Immunotherapy.
"Azalea is advancing a powerful concept – delivering precision genome editing tools to specific cells inside the body," said Justin Eyquem, Ph.D. "The ability to directly engineer cells in vivo opens new possibilities for treating disease and for bringing genomic medicines to patients in a safer and more scalable way."
Clinical Pipeline and Development Plans
The $82 million funding, which includes a recently closed $65 million Series A financing, will be used to advance Azalea's lead CD19-based in vivo CAR-T therapy for B cell malignancies and autoimmune diseases through IND-enabling studies and into the clinic. The company is also progressing 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's company launch was supported by a $1 million non-dilutive award from the HS Chau Women in Enterprising Science program, which fosters the translation of breakthrough academic research into biotech startups.
Upcoming Data Presentation
Azalea will present its latest data on November 20th at the American Society of Gene and Cell Therapy (ASGCT) Breakthroughs in Targeted In Vivo Gene Editing conference. The presentation, titled "In vivo generation of TRAC CAR-T cells by leveraging enveloped delivery vehicles," will be delivered by CEO Jenny Hamilton during the oral abstracts session.
"Azalea is at the forefront of realizing in vivo CAR-T cell therapy – combining scientific excellence with bold execution to make precise in-patient cell engineering a reality," said Andrea van Elsas, Ph.D., board director of Azalea and partner at Third Rock Ventures. "The team is uniquely positioned to deliver on the promise of off-the-shelf genome engineering with curative intent and a clear path toward the clinic."
