MIAMI, Oct. 19 (Korea Bizwire) — Ambulero Inc., a biotechnology company (“Ambulero” or the “Company”) developing new cell and gene therapy treatments for patients suffering from severe vascular disease, today announced that the U.S. FDA’s Office For Orphan Products Development (OOPD) granted the company’s request for orphan drug status for its gene therapy candidate, AMB-301, to treat Buerger’s Disease (BD), also known as Thromboangiitis Obliterans (TAO).
AMB-301 is a first-in-class gene therapy vector encoding a cell adhesion molecule (E-selectin). Over a decade of research in animal models of vascular disease suggests that providing E-selectin to damaged blood vessels promotes therapeutic angiogenesis, robust tissue regeneration and improved limb function.
BD is a debilitating vascular disease that can lead to severe limb damage and amputation, often in relatively young patients. There are no effective treatments and existing therapies are largely ineffective. AMB-301 is a promising gene therapy candidate for enhancing blood vessel formation, restoring tissue integrity and eliminating the need for amputation as a treatment option.
The FDA’s orphan drug designation provides AMB-301 with seven (7) years of market exclusivity for the treatment of BD if approved. The FDA grants orphan designations to medical products showing promise to treat diseases that affect 200,000 or fewer Americans. The company is also eligible for certain tax credits and regulatory benefits with the FDA designation.
“Ambulero is honored to receive this important FDA recognition. Should clinical testing be successful, we believe our first-in-class gene therapy platform will provide new treatment options to patients suffering from BD and potentially other vascular diseases. The Orphan Drug Designation should also help expedite our BD clinical trial timeline,” said Robert L. Buchanan, Chief Executive Officer of Ambulero, Inc.
“It is very rewarding to offer a potential new therapy to so many patients facing amputation as the only treatment option for their disease,” said Carlton Anderson, Chief Operating Officer of Ambulero, Inc.
Ambulero’s Chief Regulatory Officer, Dr. Khemraj (Raj) Hirani added, “Receiving Orphan Drug Designation for AMB-301 is a significant step forward for BD patients and confirms the potential of our novel E-selectin based platform technology to accelerate clinical development.”
Ambulero is a privately-held biotechnology company advancing a new platform of cell and gene therapies to treat serious vascular diseases. The company is a recent spin-out of the University of Miami co-founded by Randy Berholtz, Robert L. Buchanan, Omaida C. Velazquez and Zhao-Jun Liu. Ambulero’s cell therapy program uses stromal cells engineered to express E-selectin to promote tissue repair. The company’s gene therapy program uses established approaches to administer E-selectin directly to injured vascular tissues. Ambulero is currently sourcing investors for a Series A round. See www.ambulero.com
1951 NW 7th Street, STE. 600
Miami, FL 33136
Dr. Carlton Anderson
Chief Operation Officer
Source: Ambulero via GLOBE NEWSWIRE