Solid Restarts DMD Gene Therapy Trial, With Encouraging Interim Data To Back It Up

The Phase I/II trial testing SGT-001 in patients with Duchenne muscular dystrophy resumed after FDA lifted a clinical hold on the trial; interim data from the first six patients were encouraging.

Railroad tracks seen from the back of an Adirondack train heading to Montreal, Canada, from New York, USA.
Solid's DMD gene therapy is back on track • Source: Shutterstock

Solid Biosciences Inc. has resumed the Phase I/II IGNITE DMD trial of the gene therapy SGT-001, dosing a seventh patient with Duchenne muscular dystrophy in the study. Meanwhile, the company also announced on 15 March encouraging interim data from the first six patients dosed in the study, improving the outlook for the gene therapy, though not eliminating uncertainty in what is a challenging area of drug development.

Solid is one of several companies in the early race to bring a gene therapy to market for DMD, but has faced several setbacks, including two clinical trial holds. The field remains open, however, with Pfizer Inc

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Clinical Trials

More from R&D

Sanofi R&D Head’s Words Of Wisdom For Biotechs

 
• By 

Houman Ashrafian tells delegates at Swiss Biotech Day that ‘literally only two things matter in our industry – target credentialing and differentiated pharmacology. The rest is execution.’

Marea Eyes Cardioprotective Benefit With ANGPTL4 Inhibition

 
• By 

Phase IIa data for ANGPTL4-targeted antibody shows reduced triglycerides and remnant cholesterol, which Marea asserts indicates potential to protect against cardiovascular outcomes.

First Win For AstraZeneca’s Enhertu In Early-Stage Breast Cancer Treatment

 

Already dominating in later lines of breast cancer treatment, Enhertu could now displace both chemotherapy combinations and Roche’s Kadcyla in early-stage HER2-positive patients.