A Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Adult Patients With Cystic Fibrosis

EARLY_PHASE1Not yet recruitingINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

May 10, 2025

Primary Completion Date

May 10, 2026

Study Completion Date

May 10, 2027

Conditions
Cystic Fibrosis (CF)Gene Therapy
Interventions
DRUG

inhaled BMD003(CFTR mRNA)

BMD003 is the world's first freeze-dried CFTR Mrna product (stored at 2-8 ℃), which is reconstituted with injection water before use, nebulized through a vibrating sieve atomizer, and inhaled into the lungs through the mouth and nose.

All Listed Sponsors
lead

Peking Union Medical College Hospital

OTHER