CM001
Cystic Fibrosis (for patients with little-to-no CFTR protein)
Phase 1Active
Key Facts
Indication
Cystic Fibrosis (for patients with little-to-no CFTR protein)
Phase
Phase 1
Status
Active
Company
About Cystetic Medicines
Cystetic Medicines is a private, preclinical-stage biotech pioneering a 'molecular prosthetics' approach to treat cystic fibrosis, specifically targeting the 'final 10%' of patients with nonsense or non-responsive mutations. Its lead candidate, CM001, is an inhaled dry powder designed to functionally replace the missing CFTR anion channel and has entered Phase 1 clinical trials. Founded by renowned academic scientists Dr. Martin D. Burke and Dr. Michael J. Welsh, the company leverages a repurposed, FDA-approved drug compound and advanced aerosol engineering to create a potentially simple, scalable, and accessible therapy.
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