Skip to Main Content
Contribute Try STAT+ Today

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).

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED

What is it?

STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.

Create a display name to comment

This name will appear with your comment