Senior Director Regulatory Strategy - Gene Therapy & Ultra Rare Disease

Regulatory Strategy Lead - Individualized Genetic Medicine (Gene Therapy / ASO) Early-stage, venture-backed | West Coast preferred | Reports directly to the Founder/CEO About the company An early-stage company building the operating system for personalized genetic medicine - helping rare and ultra-rare disease families move from mutation to dose. The model sits closer to a concierge CRO than a traditional biotech: AI-native, lean internal team, most functions outsourced, and roughly 10 individualized genetic medicine programs running today. The opportunity This is the first dedicated regulatory hire, working side by side with the founder. You'll own the regulatory dimension of both the company's programs and its core client deliverable. Ground-floor role in a category that doesn't exist yet at scale - real ownership, not a seat in a large regulatory department. What you'll do Own regulatory strategy and execution for n-of-1 and ultra-rare genetic medicine programs, serving as the primary point of contact with FDALead agency interactions end to end - INTERACT, pre-IND, Type B/C, briefing books, IND submissions, and all correspondenceBuild the regulatory layer of the client blueprint so it answers what a program actually needs (e.g. whether an 18-week tox study is relevant) and what the submission path looks likePursue expedited and designation pathways - Orphan Drug, Rare Pediatric Disease, Fast Track - and navigate plausible-mechanism and expanded access frameworksCompress timelines by running manufacturing and regulatory in parallel rather than in sequenceAdvise on ex-US pathways and health authority strategy outside the USBring regulatory knowledge in-house, replacing external consultants who don't understand the n-of-1 model What they're looking for Regulatory strategy across gene therapy and/or ASO / oligonucleotide programsDirect ownership of pre-IND and IND submissions - personally led, not supportedDeep rare / ultra-rare development experience; n-of-1 or individualized medicine is the single strongest filterHas personally led agency interactions and knows how to get through CBER - neuro a strong plusBackground in biotech, CRO, consultancy, or similarly lean environment; comfortable being a team of oneOrphan Drug Designation experience in gene therapyEx-US regulatory awareness - able to advise on pathways outside the USMid-level seniority: senior enough to have led agency interactions, hands-on enough to still execute If you're interested and meet the criteria above, please reach out to me on LinkedIn or at sydney.hewitt@epmscientific.com.