Edgewise Therapeutics (NASDAQ: EWTX) is a clinical‐stage biopharmaceutical company headquartered in Cambridge, Massachusetts. The company is focused on the discovery and development of small‐molecule therapeutics designed to activate chaperone‐mediated autophagy (CMA), a key cellular pathway for the selective degradation of misfolded or damaged proteins. By targeting this pathway, Edgewise aims to address the underlying biology of rare and debilitating genetic disorders that currently lack effective treatments.
The company’s lead development programs target enzyme‐deficient and muscle‐wasting disorders such as Pompe disease (glycogen storage disease type II) and certain forms of limb‐girdle muscular dystrophy. Edgewise’s proprietary platform enables the identification and optimization of compounds that enhance lysosomal function and promote the clearance of toxic protein aggregates. Preclinical studies have demonstrated the ability of these candidates to restore cellular homeostasis, and the company has advanced its most advanced compound into early‐stage clinical trials.
Founded in 2017 as a spin‐out from leading academic research laboratories, Edgewise has attracted venture funding from prominent life‐science investors and formed strategic research collaborations to support its translational pipeline. The company maintains research and development activities in both the United States and Europe, leveraging partnerships with contract research organizations and academic centers of excellence to conduct pharmacology, toxicology, and initial human studies. These alliances help to streamline the path from discovery to clinical proof‐of‐concept.
Edgewise’s leadership team comprises experienced executives and scientists with backgrounds in biotechnology, regulatory affairs, and commercial strategy. Drawing on prior roles at global pharmaceutical firms and specialized biotechs, the management group is committed to advancing innovative treatments for underserved patient populations. As Edgewise progresses through clinical milestones, it remains focused on delivering novel therapeutic options that harness the power of targeted protein degradation.
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