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 sitsΒ ...
President, Chief Executive Officer & Director, Regenxbio
Search every verified Curran M.s. interview, podcast appearance, and on-the-record quote β 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.
“Ken Mills was CEO for Regenxbio for 15 years. We started out early on as a licensing organization, and the license for the first gene therapy ever approved, Zolgensma, originated out of our technology.”
“Rather than license out technologies, the intent was to put AAV8 and AAV9 into the hands of as many developers as we could and start internally developing programs, which now are late-stage and moving into phase three, with one possibly becoming a commercial program next year.”
“We will be filing for our wet AMD compound in 2026, which could be the largest gene population treated by gene therapy worldwide.”
“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.”
“The accelerated approval of the Elus program was great for the sector and showed a lot of flexibility on the part of the FDA. We intend to leverage the accelerated approval pathway for our program.”
“The combination of unmet need, safe administration, good safety profile, and positive efficacy is key to a successful gene therapy program. Programs struggle when there is an existing therapy that meets the need, even if inconvenient.”
“For our RGX-314 ocular program, we have to wrestle with whether it is an iterative improvement that will supersede treatments like Lucentis and Eylea. Investigators see a real need for gene therapy, especially for patients who miss regular treatments and start to lose vision.”
“I feel really strongly about the opportunity to be an ambassador for the ARM Grow program. The goal is to diversify our workforce, bring new ideas into organizations, and enrich resumes to ensure a diverse population applies for new positions.”
“The candidates selected for the Grow program are brilliant and motivated. Many have gone on to work for companies within the network, showing the program's success in developing talent for the sector.”
Gene therapy holds much promise to not only treat difficult rare diseases, but also prevalent conditions. ARM's Mark Battaglini sitsΒ ...
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