Tucson, Arizona — Critical Path Institute’s (C-Path) Duchenne Regulatory Science Consortium (D-RSC) is excited to announce the launch of a groundbreaking model-based Clinical Trial Simulator (CTS), specifically designed to improve design of efficacy studies for potential therapies for Duchenne muscular dystrophy (DMD). This pioneering Drug Development Tool is set to...
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TUCSON, Ariz. — Critical Path Institute (C-Path), a leader in accelerating drug development for rare diseases, today announced the targeted integration of additional Friedreich’s ataxia (FA) datasets into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) as part of a partnership with Friedreich’s Ataxia Research Alliance (FARA). This update...
Tucson, Arizona — 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 Progressive Supranuclear Palsy (PSP). This initiative brings together leading organizations and experts in a concerted effort to tackle...
Tucson, AZ. – Critical Path Institute (C-Path) is pleased to announce the release of a new peer-reviewed publication, titled “Transforming Drug Development for Neurological Disorders: Proceedings from a Multi-disease Area Workshop,” now published in Neurotherapeutics, The Journal of the American Society for Experimental Neurotherapeutics. A distinguished team of C-Path scientists...
WATERTOWN, Mass. — C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, today announced that it has entered into a clinical trial collaboration and supply agreement with Pfizer Inc. Under the terms of the agreement, Pfizer will supply elranatamab (ELREXFIO®), a B-cell...
WATERTOWN, Mass. — C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, today announced that data from its Phase 1 dose escalation clinical trial of cemsidomide in multiple myeloma (MM) will be shared in an oral presentation at the International Myeloma Society...
PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human...
PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today announced positive clinical data and development updates across the RESET-Myositis™, RESET-SSc™ and RESET-SLE trials evaluating rese-cel (resecabtagene autoleucel, formerly...
First cleared CD19-CAR T IND application for generalized myasthenia gravis announced in the U.S. and fourth IND clearance for CABA-201 across a broad range of autoimmune diseases Clearance expands the clinical development of CABA-201 beyond rheumatology into neurology Phase 1/2 clinical trial evaluating CABA-201 in generalized myasthenia gravis features...
PARIS, France – Ipsen announced today that the European Commission has approved Cabometyx® (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues. Most forms of neuroendocrine tumors (NETs) develop slowly,...