New In Vivo Cell Therapy Biotech Emerges with Nobel Laureate Backing and $82M Funding

Azalea Therapeutics, a new West Coast biotech company, has entered the competitive in vivo cell therapy space with an impressive $82 million in funding and the support of Nobel Prize-winning CRISPR pioneer Jennifer Doudna, Ph.D. The company aims to revolutionize genome editing with a single-dose approach that could have far-reaching implications for the treatment of various diseases.

Innovative Dual-Vector Technology

At the heart of Azalea's technology is a dual-vector approach that combines two groundbreaking elements:

1. Enveloped Delivery Vehicles (EDVs): Developed in Doudna's lab by co-founder and CEO Jenny Hamilton, Ph.D., these viruslike particles encapsulate DNA-cutting Cas9 enzymes and are decorated with antibody fragments for targeted cell delivery.

2. Adeno-Associated Viruses (AAVs): Contributed by co-founder Justin Eyquem, Ph.D., these viruses carry genes for insertion at specific genomic locations, particularly the T-cell receptor alpha constant (TRAC) locus.

This combination allows for precise genome editing with a single injection, potentially mimicking natural T cell behavior and reducing the risk of editing errors.

Clinical and Financial Outlook

Azalea Therapeutics plans to advance its lead candidate, an in vivo CAR-T therapy for B-cell malignancies, into clinical trials within the next 12 to 18 months. The company's recent $82 million funding round, including a $65 million Series A led by Third Rock Ventures, with participation from RA Capital Management, Yosemite, and Sozo Ventures, will support this ambitious timeline.

Proof-of-concept mouse data for various tumor models will be presented at the upcoming ASGCT Breakthroughs in Targeted In Vivo Gene Editing meeting in San Diego, while primate model experiments are currently underway.

Industry Context and Future Potential

The emergence of Azalea Therapeutics comes amid a surge of interest and investment in in vivo cell therapies. Recent high-profile deals in the sector include Gilead's Kite $1.64 billion agreement with China's Pregene Biopharma and Bristol Myers Squibb's $1.5 billion acquisition of Orbital Therapeutics.

While the initial focus is on in vivo CAR-T therapy, Azalea's technology platform holds promise for engineering other cell types, including hematopoietic stem cells and B cells. The company's ability to precisely insert genes into specific genomic locations sets it apart in the increasingly crowded field of in vivo cell therapies.

Andrea van Elsas, Ph.D., a partner at Third Rock Ventures, noted the exciting convergence of scientific advancements in this area, highlighting the potential of in vivo gene editing to address significant accessibility issues in CAR-T applications.