As shown below, we are developing two treatment modalities: one that delivers E-selectin as part of an engineered stromal cell (“Supercharged MSCs”) and the other delivering E-selectin as a gene therapy (AAV) vector.  

 

Rare Vascular Disease: Our therapeutic candidate, AMB-101, is a first-in-class regenerative cell therapy candidate. We take allogeneic MSCs and supercharge them ex vivo so that they express E-selectin. Our goal is to amplify these cells and return them to patients in an FDA-authorized clinical trial. Our first indication is a rare form of vascular disease striking the limbs of young people and for which there is no acceptable standard of care (other than amputation). This disease is rare in the US (roughly 50,000 patients) but more widespread elsewhere. AMB-301 is a first-in-class gene therapy candidate that uses a clinically tested AAV vector to deliver E-selectin to diseased tissues.

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Critical Limb Ischemia: Our other therapeutic candidate, AMB-201 is a first-in-class gene therapy candidate that uses an AAV vector to deliver E-selectin directly to ischemic tissues. The company has a strong interest in developing AMB-201 for vascular conditions with large patient populations such as critical limb ischemia (CLI): a major co-morbidity of diabetic patients. We estimate that roughly 2 million people in the US have CLI and about 5-10% must have an amputation as part of their care.