Rectify Pharmaceuticals

Rectify Pharmaceuticals is a private, preclinical-stage biotech leveraging its proprietary PFM platform to rectify membrane protein dysfunction, an historically challenging drug target. The company's pipeline is led by a dual-targeted ABCB4/BSEP program for PSC, currently in IND-enabling studies, with parallel programs targeting ABCC6 for vascular calcification and ABCD1/ABCD2 for adrenoleukodystrophy. Backed by venture capital investors like Atlas Venture, Forbion, and Longwood Fund, Rectify aims to deliver first- and best-in-class oral therapies for diseases with high unmet need across hepatobiliary, cardio-renal-metabolic, and neurological therapeutic areas.

Proprietary Positive Functional Modulator (PFM) platform for discovering oral small molecules that restore or enhance the function of dysfunctional membrane proteins, with demonstrated success against ABC transporters and applicability to SLCs, GPCRs, and ion channels.

In PSC, competition includes other novel mechanisms (FXR agonists, FGF19 analogs) and anti-fibrotics, though no therapy is approved. In broader cholestasis, several approved drugs exist for PBC. The PFM approach is differentiated by its direct target engagement to restore function, but it competes with established drug classes and emerging modalities like gene therapy, particularly in rare diseases like ALD.