Author Archives: Marisa Wexler MS

Pivotal Phase 3 Trial of CAP-1002 Therapy for DMD Launching Soon

A potentially pivotal Phase 3 clinical trial called HOPE-3, which will test the effectiveness of the experimental cell therapy CAP-1002 as a treatment for Duchenne muscular dystrophy (DMD), is expected to launch soon. Capricor Therapeutics, the company developing CAP-1002, announced that the U.S. Food and Drug Administration…

Elamipretide as Add-on May Boost Efficacy of Exon-skipping Therapies

Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for Duchenne muscular dystrophy (DMD), its developer, Stealth BioTherapeutics, reported. “Our new preclinical data increases our optimism that elamipretide may help address the unmet need for therapies in DMD,” Reenie…

Atamyo Cleared in UK to Test ATA-100 for Limb-Girdle MD Type R9

Atamyo Therapeutics has been given the go-ahead to start clinical testing of ATA-100, the company’s investigational gene therapy for a specific form of limb-girdle muscular dystrophy (LGMD) called type R9 (LGMDR9). With approval from the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA), Atamyo now plans to start dosing…

Santhera, ReveraGen Advancing Plans for Vamorolone Application

Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S. approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD). The companies announced Tuesday they had completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA).

Atamyo Seeking Approval for LGMD Gene Therapy Trial in Europe

Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a type of limb-girdle muscular dystrophy (LGMD). The company, a spin-off from Genethon, a Paris-based genetics research laboratory, has submitted an application asking three European…

FDA Puts Investigational DM1 Therapy AOC 1001 on Fast Track

The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for myotonic dystrophy type 1, known as DM1, a late-developing muscle disorder. The designation will enable AOC 1001’s developer, Avidity Biosciences, to have more frequent interactions with the FDA throughout…