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Gene Editing Therapies

Drug Name
Type of Drug
DMD Mutations Addressed
Sponsor
Current Development Phase
Approval Status
PF-06939926
IV infusion given once only
All with the exception of exons 9-13 or deletion effecting both 29 or 30
Pfizer
Just started Phase 3
Still under investigation
SRP-9001
IV infusion given once only
All with the exception of exons 1-17 and exon 45
Sarepta
Starting Phase 3
Still under investigation
GALGT2
IV infusion given once only
All
Nationwide Children’s Hospital (USA only)
Phase 1 and 2
Still under investigation
SGT-001
IV infusion given once only
All
Solid Biosciences
Phase 1 and 2
Still under investigation
GNT 0004
IV infusion given once only
All but may change soon
Genethon
Phase 1/2/3 but trial currently suspended due to serious adverse events
Still under investigation