A former Vertex scientist is setting his sights on an entire family of transporter proteins that can cause scores of rare inherited conditions. His startup, Rectify Pharmaceuticals, recently completed a Series A to solve the problems created when so-called ABC transporters break — but the path ahead isn’t as easy as 1, 2, 3.
There are 48 distinct ABC transporter proteins found in humans, and they each perform essential functions for organs. Some help cells pump out natural and artificial antibiotics; others move lipids; and still more pass peptides through the cell membrane. But when ABC transporters aren’t formed correctly, they give rise to huge problems. A pair of mutated ABC transporter genes, for example, can cause severe genetic diseases such as cystic fibrosis, Stargardt disease, and progressive familial intrahepatic cholestasis (PFIC).
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