C-Path to Lead Comprehensive Task Force Focused on Accelerating Drug Development for Limb-Girdle Muscular Dystrophies

TUCSON, Ariz., September 30, 2024 — Critical Path Institute (C-Path) today announced the formation of a new task force under its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), dedicated to advancing therapeutic development for limb-girdle muscular dystrophies (LGMDs). This initiative brings together leading organizations and experts in a concerted effort to tackle the challenges associated with LGMDs drug development. LGMDs are a group of disorders that affect voluntary muscles, primarily those around the hips and shoulders.Continue reading

C-Path’s Inaugural Global Impact Conference Charts the Future of Drug Development

WASHINGTON, Sept. 12, 2024 — Critical Path Institute (C-Path) successfully concluded its inaugural Global Impact Conference (CGIC) on September 11, 2024, at the Washington Marriott at Metro Center in Washington, D.C. This landmark event brought together industry leaders, regulatory agencies, academic experts, and patient advocacy groups, all dedicated to advancing drug development and regulatory science for rare diseases. Continue reading

FDA Issues ‘Letter of Support’ Encouraging Use of Synuclein-based Biomarker, αSyn-SAA, in Clinical Trials in Parkinson’s and Related Diseases

  • U.S. regulatory agency recognizes potential of alpha-synuclein seed amplification assay (αSyn-SAA) to accelerate clear outcomes in drugmakers’ trials
  • Letter is latest milestone in the journey of the biomarker, discovered in 2023, to transform drug development focused on synuclein pathology — a biological hallmark of Parkinson’s, Dementia with Lewy Bodies and related neurodegenerative conditions
  • FDA notes critical role of the Parkinson’s Progression Markers Initiative in generating and validating data and studies that made this landmark step forward possible

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C-Path Awarded $1.5 Million Grant from Ara Parseghian Medical Research Fund at Notre Dame to Enhance Collaboration in Niemann-Pick Disease Research and Drug Development

TUCSON, Ariz., August 22, 2024 — Critical Path Institute (C-Path) is thrilled to announce its Critical Path for Lysosomal Diseases (CPLD) Consortium has been awarded a grant from the Ara Parseghian Medical Research Fund (APMRF) at the University of Notre Dame.Continue reading

C-Path’s TRxA Announces $250,000 Grant for Drug Development Project on Antibiotic-Resistant Gram-Negative Bacteria

TUCSON, Ariz. August 13, 2024– Critical Path Institute’s (C-Path) Translational Therapeutics Accelerator (TRxA) announced today that Kenneth Keiler, Ph.D., Professor of Molecular Biosciences at the University of Texas at Austin, has been awarded a research grant for his innovative project titled “Inhibitors of the Gram-negative Cell Envelope Stress Response as Anti-Infectives and Antibiotics.” Continue reading

C-Path’s TRxA Announces $250,000 Award for Drug Development Project in ALS

TUCSON, Ariz., August 8, 2024 – Critical Path Institute’s (C-Path) Translational Therapeutics Accelerator (TRxA) announced today a new research grant awarded to Daniela C. Zarnescu, Ph.D., Professor of Cellular and Molecular Physiology at Penn State University College of Medicine, in collaboration with Jon Njardarson, Ph.D., Professor at the University of Arizona. Continue reading