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Dual-targeted ABCD1/ABCD2 PFM

Adrenomyeloneuropathy (AMN), Adrenoleukodystrophy (ALD)

Pre-clinicalActive

Key Facts

Indication
Adrenomyeloneuropathy (AMN), Adrenoleukodystrophy (ALD)
Phase
Pre-clinical
Status
Active
Company

About 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.

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