Curran M.s. on muscular atrophy
From Gene Therapy for Rare and Prevalent Conditions: ARM Studios Interview with Curran Simpson · · Alliance for Regenerative Medicine
“One of the biggest challenges has been to change muscular atrophy. We have made really good progress in the last 18 months, safely dosing up to our pivotal dose, and our construct produces micro-dystrophin with a CT domain different from others, which we hope will result in functional benefit to patients.”
On , Curran M.s., President, Chief Executive Officer & Director at REGENXBIO INC, spoke about muscular atrophy during Gene Therapy for Rare and Prevalent Conditions: ARM Studios Interview with Curran Simpson on Alliance for Regenerative Medicine.
About Curran M.s.
Curran Simpson, president and CEO of Regenxbio, discussed the company's evolution and pipeline in a December 2024 interview. He stated that Regenxbio began as a licensing organization and that the first approved gene therapy, Zolgensma, originated from its technology. Simpson said the company shifted to internal development and now has late-stage programs, including a wet age-related macular degeneration (AMD) compound for which it plans to file in 2026, which he described as potentially the largest gene therapy population treated worldwide. He also noted progress in a Duchenne muscular dystrophy program, saying the company has safely dosed up to a pivotal dose and intends to leverage the FDA's accelerated approval pathway. Simpson also spoke about his role as an ambassador for the ARM Grow program, which aims to diversify the workforce in the gene therapy sector. He said the program selects candidates for internships and that many have gone on to work within the network, describing the initiative as successful in developing talent.