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      Congruence Awarded Grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to Advance Novel GCase Activators and Correctors for Parkinson's Disease with GBA1 Mutations

      (PRNewsfoto/Congruence Therapeutics)

      News provided by

      Congruence Therapeutics

      Jul 24, 2025, 09:00 ET

      Share this article

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      -Congruence's Revenir™ computational drug discovery engine has identified activators and correctors of GCase designed to target the key biological dysfunction in Parkinson's disease patients with GBA1 mutations-

      -Congruence plans to file an IND for its lead GCase program in 2026-

      MONTREAL, July 24, 2025 /PRNewswire/ -- Congruence, a computationally-driven biotechnology company building a unique pipeline of correctors for diseases of protein misfolding, including MC4R-deficient genetic obesity, GBA-driven Parkinson's disease and Alpha-1 antitrypsin deficiency, announced today it has received a research grant of $5 million dollars from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance its GCase-targeting small molecules for GBA1 Parkinson's disease.

      Mutations of the GBA1 gene, encoding the enzyme GCase, represent the single largest genetic risk factor for Parkinson's Disease.  Congruence has leveraged its powerful computational engine Revenir™ to discover small molecules that are designed to correct biological deficits resulting from mutations in the GBA1 gene.  Currently, there are no disease-modifying therapies available for the treatment of GBA1-driven Parkinson's disease.

      "We are honored to receive this grant from The Michael J. Fox Foundation. Their confidence strengthens our commitment to apply Congruence's computational drug discovery tools toward the development of GCase-targeting therapies that bring hope to the Parkinson's community," stated Dr. Clarissa Desjardins, CEO of Congruence.

      In GBA1 Parkinson's disease, GCase deficiency leads to the accumulation of α-synuclein and subsequent death of neuronal cells that are hallmarks of Parkinson's.  The MJFF grant will support the advancement of Congruence's efforts for GBA1 Parkinson's disease, including progressing its novel development candidates through lead optimization towards the clinic.  At the GBA1 Meeting 2025, which gathered stakeholders from both academia and industry engaging and specializing in GBA1 research, Congruence scientists delivered an oral presentation on the discovery of potent, orally active and brain-penetrant allosteric GCase activator and corrector molecules for clinical evaluation in Parkinson's disease.  The GBA1 Meeting 2025 was hosted by McGill University and supported by The Michael J. Fox Foundation. 

      "The Michael J. Fox Foundation supports a broad portfolio of research and drug development around important Parkinson's disease biology such as GBA1," said MJFF chief scientist Brian Fiske, PhD. "Congruence's efforts to advance novel therapies contribute critically to our shared commitment to deliver new treatments for people with Parkinson's."

      About Revenir™ Drug Discovery Platform

      Revenir™, Congruence's proprietary computational drug discovery platform, captures the dynamic biophysical changes caused by mutations in proteins, offering unique insights into protein defects and their correction. By examining surface features and a spectrum of biophysical descriptors across an ensemble of protein conformers, Revenir™ predicts small molecule induced correction of the underlying pathogenic defect in proteins.

      About Congruence Therapeutics

      Congruence is a computationally-driven biotechnology company building a unique pipeline of transformative small molecule correctors rationally designed to rescue aberrant protein function. Our proprietary scalable platform, Revenir™, captures the biophysical features of proteins across their conformational ensembles, in order to identify novel allosteric and cryptic pockets which are virtually screened to generate novel chemical matter. Congruence has leveraged its proprietary discovery engine to launch a pipeline of wholly owned drugs directed to high-value, genetically validated, difficult-to-drug targets spanning a variety of indications including genetic obesity, GBA Parkinson's Disease, and α1-Antitrypsin Deficiency. Congruence is further deploying its discovery platform to support two recently executed multi-target research collaborations with large pharmaceutical companies focused on the discovery of small molecule correctors for the treatment of solid tumors and metabolic diseases.

      For more information, please visit www.congruencetx.com.

      Company Contact
      Charles Grubsztajn
      Chief Operating Officer
      [email protected]

      Media Contact
      Amy Conrad
      Juniper Point
      [email protected] 
      858-366-3243

      SOURCE Congruence Therapeutics

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