Gene Therapy for Rare and Prevalent Conditions: ARM Studios Interview with Curran Simpson
Gene therapy holds much promise to not only treat difficult rare diseases, but also prevalent conditions. ARM's Mark Battaglini sits ...
President, Chief Executive Officer & Director, Regenxbio
Search every verified Curran M.s. interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. 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.
Gene therapy holds much promise to not only treat difficult rare diseases, but also prevalent conditions. ARM's Mark Battaglini sits ...
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