Ambulero’s Investigational Gene Therapy to Treat Vascular Disease Supported by NIH Funding

June 12, 2023—Ambulero, Inc. announced that the National Heart, Lung, and Blood Institute (NHLBI)’s Catalyze Program of the National Institutes of Health (NIH) approved a second phase of funding for investigational new drug–enabling work conducted by Ambulero’s Chief Medical Officer Omaida C. Velazquez, MD, and Chief Scientific Officer Zhao-Jun Liu, MD. Ambulero is developing a first-in-class gene and cell therapy platform for treating serious vascular diseases and nonhealing wounds where amputation is a care option.

According to the company, lead investigators Dr. Velazquez and Dr. Liu, both with the University of Miami Miller School of Medicine in Miami, Florida, are focused on advancing E-selectin/adeno-associated virus (AAV) gene therapies for the treatment of arterial occlusive diseases such as peripheral artery disease with chronic limb-threatening ischemia. In 2021, Ambulero’s AMB-301 E-selectin/AAV gene therapy product received a key Orphan Drug designation from the FDA as a novel treatment candidate for Buerger disease, a rare vasculitis that can lead to limb amputation.

The academic sponsor is the University of Miami; the AAV development partner is the University of Florida Powell Gene Therapy Center, directed by AAV expert Dr. Barry Byrne; and the accelerator industry partner is Ambulero. The grant award numbers are Catalyze NIH/NHLBI 1R61HL156152-01A1 and Catalyze NIH/NHLBI 4R33HL156152-02.

As noted in the company’s press release, Drs. Velazquez and Liu are coinventors of the intellectual property being used in the study and have consulting, equity, and board service relationships with Ambulero, Inc. Additionally, Dr. Velazquez, Dr. Liu, and the University of Miami stand to gain royalties from the commercialization of the intellectual property.