Catalyst overview
Research summary
This catalyst tracks a Phase 1/2 data readout for PBGENE-DMD in Duchenne Muscular Dystrophy (DMD). The expected timing is based on the latest available company guidance and may change.
9/30/25- File IND and/or CTA by YE 2025. 11/03/25:- Reiterated plan to file IND by YE 2025. Phase 1 initiation in DMD patients expected in H1 2026 with initial data anticipated in H2 2026. 01/12/26:- FUNCTION-DMD IND clearance expected Q1 2026; first dosing late Q1/early Q2; initial data YE 2026. 02/11/26:- FDA Study May Proceed; IRB underway; FUNCTION-DMD Ph1/2 site activation expected H1 2026; initial data YE 2026. 02/18/26:- New preclinical mouse data to be presented in MDA 2026 poster; FUNCTION-DMD Ph1/2 initiation still expected H1 2026. 03/10/26:- Presented new preclinical GLP mouse data at MDA 2026. Reiterated FUNCTION-DMD Ph1/2 site activations in H1 2026 (NCT07429240). 03/12/26:- IND cleared; Fast Track granted; FUNCTION-DMD Ph1/2 sites activating H1 2026; initial multi-patient data expected YE 2026. 04/28/26:- ASGCT 2026 oral preclinical data to be presented May 14, 2026. 05/05/26:- Arkansas Children's site activated; FUNCTION-DMD Ph1/2 enrolling DMD patients; March 2026 preclinical data presented. 08/06/26:- FUNCTION-DMD sites recruiting; initial safety data expected Year-End 2026.
Verification
Confirm timing and program details against the company announcement or filing.