Entrada Therapeutics Shares Updates on Its Exon 45 and Exon 44 Skipping Programs in Duchenne

Exon 45 and Exon 44 Skipping Program Updates

As an early investor in Entrada Therapeutics, we are pleased to share updates on their Exon 45 and Exon 44 skipping programs in Duchenne.

Exon 45:  ENTR-601-45, an experimental therapeutic designed to skip exon 45, was safe at the initial dose of 5 mg/kg given once every 6 weeks.  Treated participants demonstrated a 0.10% increase in dystrophin, but the company expects significant increases and functional benefits in future cohorts receiving higher doses.  Cohort 2, which will receive 10 mg/kg every 6 weeks, is fully enrolled, with data expected in the first half of 2027.   Read press release here

Exon 44:  An independent Data Monitoring Committee has reviewed the data from Cohort 2 receiving 12 mg/kg of  ENTR-601-44, an experimental therapeutic designed to skip exon 44, and recommends initiation of Cohort 3 at the top dose of 18 mg/kg given once every 6 weeks.  Data from Cohort 1’s open-label period is expected by year-end 2026, and data from Cohort 2 is expected Q1 2027.   Read the press release here

Link to community newsletter HERE 

Link to press releases:

Positive Results From First Cohort of ELEVATE-45-201

DMC Recommends Initiation of Cohort 3 at Top Dose of 18 mg/kg in ELEVATE-44-201