NIH Approves Second Phase of Funding to Support IND-enabling Work for Ambulero Gene Therapy to Treat Vascular Disease

2023-06-12

基因疗法孤儿药临床申请细胞疗法临床研究

MIAMI--(BUSINESS WIRE)-- Ambulero, Inc. is developing a first-in-class gene and cell therapy platform for treating serious vascular diseases and non-healing wounds where amputation is a care option. In support of this effort, the NHLBI CATALYZE program of the National Institutes of Health approved a second phase of funding for IND-enabling work conducted by Ambulero’s Chief Medical Officer (Omaida C. Velazquez, MD., FACS, DFSVS) and Chief Scientific Officer (Zhao-Jun Liu, MD, PhD) of the University of Miami Miller School of Medicine. Drs. Velazquez and Liu are focused on advancing E-selectin/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 US FDA as novel treatment candidate for Buerger’s Disease: a rare vasculitis that can lead to limb amputation. Lead Investigators - Drs. OC Velazquez and ZJ Liu; Academic Sponsor – University of Miami; AAV development partner – University of Florida Powell Gene Therapy Center directed by AAV Expert Dr. Barry Byrne; Accelerator industry Partner - Ambulero, Inc. This grant is supported by the National Heart, Lung, and Blood Institute of the National Institutes of Health under Award Number(s) Catalyze NIH/NHLBI 1R61HL156152-01A1 and Catalyze NIH/NHLBI 4R33HL156152-02. The content of this press release is solely the responsibility of the author and does not necessarily represent the official views of the NIH. Disclosure: Drs. Velazquez and Liu are co-inventors of the intellectual property being used in the study, and also have consulting, equity, and Board service relationships with Ambulero, Inc. Drs. Velazquez, Liu, and the University of Miami stand to gain royalties from the commercialization of the IP. For more information about Ambulero, please see

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