• First company to enable precision in vivo genome engineering, combining both cell-specific delivery and programmable, locus-specific gene insertion, with the potential to achieve physiological, durable gene expression for greater therapeutic impact
• Launching with $82 million in combined seed and Series A financing led by Third Rock Ventures, with participation from RA Capital Management, Yosemite, Sozo Ventures and select individual investors
• Founded by Jennifer Doudna, Ph.D., Michael Fischbach, Ph.D., Justin Eyquem, Ph.D. and Jenny Hamilton, Ph.D. to advance next-generation genomic medicines
• Upcoming oral presentation at ASGCT Breakthroughs in Targeted In Vivo Gene Editing highlights in vivo generation of TRAC CAR-T cells using EDV-mediated, site-specific gene insertion

BERKELEY, Calif., Nov. 04, 2025 (GLOBE NEWSWIRE) -- Azalea Therapeutics, Inc., a biotechnology company redefining precision genomic medicines in vivo, today 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 company’s mission is to engineer therapeutic cells with precision directly inside the patient, transforming how cell and gene therapies are created and delivered.

Azalea’s 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 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. Together, these advances enable precise engineering of functional CAR-T cells within a patient – a monumental advancement in the fields of cell and gene therapy.

“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.”

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. The early academic work was supported by philanthropic grants from the Yosemite team and the Parker Institute for Cancer Immunotherapy to support early de-risking, and 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.

Together with Dr. Hamilton, Azalea’s co-founders bring deep academic and translational expertise across genome engineering, synthetic biology and cell therapy, including:

• Jennifer Doudna, Ph.D., the Li Ka Shing Chancellor’s Chair in Biomedical and Health Sciences and professor of molecular and cell biology at UC Berkeley, Howard Hughes Medical Institute Investigator, founder of the Innovative Genomics Institute and 2020 Nobel laureate in chemistry
  
• Michael Fischbach, Ph.D., the Liu (Liao) Family Professor of Bioengineering at Stanford University, institute scholar of Stanford ChEM-H and director of the Stanford Microbiome Therapies Initiative
  
• Justin Eyquem, Ph.D., associate professor of medicine in the Division of Hematology and Oncology at UCSF, affiliate investigator at Gladstone Institutes and member of 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.”

Together with its distinguished scientific founders, Azalea is supported by leading biotech investors committed to advancing the company’s vision.

“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.”

Azalea will present its latest data on Thursday, November 20^th at the American Society of Gene and Cell Therapy (ASGCT) Breakthroughs in Targeted In Vivo Gene Editing, taking place virtually and in San Diego.

Abstract Title: In vivo generation of TRAC CAR-T cells by leveraging enveloped delivery vehicles
Presenting Author: Jenny Hamilton, Ph.D., Azalea Therapeutics
Session 5: Oral Abstracts
Date/Time: Thursday, November 20, 2025 | 3:45 – 5:00 pm PST

Visit the ASGCT Breakthroughs in Targeted In Vivo Gene Editing website to view the published abstracts.

Azalea’s leadership and board bring deep scientific, operational and investment expertise spanning genome engineering, cell therapy and company building:

Leadership
Jenny Hamilton, Ph.D. – co-founder, president and chief executive officer
Paul Westberg, MBA – chief business officer
Connor Tsuchida, Ph.D. – scientific co-founder
Abdullah Syed, Ph.D. – scientific co-founder

Board of Directors
Andrea van Elsas, Ph.D. – partner, Third Rock Ventures
Mary Lynne Hedley, Ph.D. – venture partner, Third Rock Ventures
Nandita Shangari, Ph.D. – managing director, RA Capital Management
Michael Fischbach, Ph.D. – professor of bioengineering, Stanford University
Justin Eyquem, Ph.D. – associate professor of medicine, UCSF
Jenny Hamilton, Ph.D. – co-founder and chief executive officer

Board Observers
Jennifer Doudna, Ph.D. – professor of molecular and cell biology at UC Berkeley
Reed Jobs, M.A. – investor, Yosemite
Jenna Hebert, Ph.D. – investment director, RA Capital Management

About Azalea Therapeutics
Azalea Therapeutics is a biotechnology company redefining precision genomic medicines in vivo. Its proprietary Enveloped Delivery Vehicles (EDV) platform is engineered to deliver transient CRISPR-Cas9 cargo to specific cells in the body for site-specific genome editing with curative intent. Azalea’s first programs leverage T cell-targeting EDVs and highly efficient T cell-tropic AAVs to enable programmable CAR gene insertion at a defined genomic locus, placing expression under control of the cell’s endogenous promoter for physiologic and sustained activity. This approach aims to generate potent, durable and safe therapies directly inside patients, avoiding the complexity of ex vivo manufacturing and unlocking new treatment modalities – including in vivo CAR-T cell therapies – across cancer, autoimmune disease and genetic disorders. Azalea is headquartered in Berkeley, California. For more information, please visit azaleatx.com and follow us on LinkedIn.

Contact:
Noopur Batsha Liffick, MPH
NBL LifeSci Advisory
noopur@azaleatx.com