Author Archives: Patricia Inacio PhD

Pivotal CMT1A Trial to Begin Patient Enrollment Soon

An international Phase 3 clinical trial investigating Pharnext‘s investigational therapy PXT3003 for treating Charcot-Marie-Tooth disease type 1A (CMT1A) is on track and expected to enroll its first participants in March, the company announced. Dubbed PREMIER, the trial will include about 350 patients with mild-to-moderate CMT1A across 50 clinical…

Passage Bio Launches New Gene Therapy Program for CMT2A

Passage Bio has acquired the rights for a gene therapy developed at the University of Pennsylvania (Penn) that aims to restore the function of the MFN2 gene, which is defective in people with Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A). Developed under Penn’s Gene Therapy Program, Passage Bio…