#solidbio

pharmaphorum

@pharmaphorum.bsky.social

3 days ago

Regenxbio, Solid report data with Duchenne gene therapies Regenxbio and Solid Bio say their gene therapies for Duchenne muscular dystrophy show no sign so far of the liver issues seen with Sarepta's Elevidys.

This week saw both #Regenxbio and #SolidBio report new #data with their #genetherapy candidates for #Duchenne muscular dystrophy (DMD), vying to join Sarepta's Elevidys on the market.

DMD WarrioR

@dmdwarrior.com

1 year ago

Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 Solid Biosciences, a clinical-stage biotechnology company, shared positive initial data from its Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy for Duchenne muscular dystrophy. Clinical Data of Duchenne Gene Therapy Candidate SGT-003 📌Average microdystrophin expression of 110% (N=3) and significant improvements in multiple additional muscle health biomarkers observed support the potential of SGT-003 as a next-generation, best-in-class Duchenne muscular dystrophy gene therapy candidate. 📌Encouraging early signals of potential cardiac benefit observed. 📌SGT-003 has been well-tolerated in the 6 participants dosed as of February 11, 2025, with no serious adverse events observed. 📌Participant enrollment continues, with the 7th participant dosed on February 17, 2025; Company expects to dose approximately 20 total participants by Q4 2025.

Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003

👉 Read More: dmdwarrior.com

#dmd #duchenne #solidbio #sgt-003 #genetherapy