CAMBRIDGE, Mass. – December 12, 2022 – Rectify Pharmaceuticals, Inc., (“Rectify”) a biotechnology company developing small molecule disease-modifying therapeutics that restore and enhance ABC transporter function, announced the publication of an article highlighting the therapeutic potential of the ABC transporter class in Trends in Molecular Medicine. The article, titled “ABC transporters: human disease and pharmacotherapeutic potential” is available online via this link.
“At Rectify we believe the ABC transporter superfamily has broad biological relevance to human physiology, and represents a compelling opportunity for the development of small molecule therapies”, said Jonathan Moore, Ph.D., Founder and Chief Scientific Officer. “Our Trends in Molecular Medicine article highlights the important functional role played by this class of transporters across several organs that should spur the development of novel ABC transporter therapies to treat both rare and common disease.”
The review article outlines the biological and functional relevance of the 48 human ABC transporters, and describes the roles that this superfamily of proteins plays in human disease. To date, 21 ABC transporters have been identified as etiological drivers of rare monogenic disease, while many others are linked to the predisposition, severity, and symptomology of complex and common diseases. The publication details both how mutations in 21 ABC transporter genes can give rise to rare disorders of the liver, retina, lung and nervous system (amongst others), and also how ABC transporters can contribute to disease pathogenesis in multigenic or complex diseases. This broad pathophysiological relevance, combined with the pharmacological proof-of-principle demonstrated by currently approved ABC transporter-directed modulators for cystic fibrosis (caused by the ABCC7 gene), points to the value of further research into this target class and their potential to transform the care of myriad human diseases.
About Rectify Pharmaceuticals, Inc. (“Rectify”)
Rectify is developing Positive Functional Modulators (PFMs), disease-modifying therapies that restore and enhance ABC transporter function to address the underlying cause of serious ABC transporter mediated diseases. Rectify is the first company to directly address the untapped therapeutic potential of the ABC transporter target class.
ATP-binding cassette (ABC) transporters are a 48-member superfamily of membrane-bound proteins that actively export a diverse range of substrates (e.g., lipids, ions, peptides, and salts) across lipid membranes. Rectify has built a breakthrough ABC transporter product platform that is enabling efficient and rapid discovery of first-in-class small molecule therapies that restore and enhance ABC transporter function for rare and common liver, biliary, eye and CNS diseases.
Rectify was founded and seeded by Atlas Venture who co-led the $100M Series A round with Omega Funds who were joined by Forbion and Longwood Fund. The company is headquartered in Cambridge, Massachusetts.
For more information, please visit www.rectifypharma.com.
Sarah Sutton/Michael Barron