Ambulero’s Gene Therapy Candidate AMB-301 Granted US Orphan Drug Designation For Treatment of Severe Vascular Disease

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.

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