Senior Director Regulatory Strategy - Gene Therapy & Ultra Rare Disease

An early-stage, venture-backed 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.

About the role

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.

Responsibilities

• Own regulatory strategy and execution for n-of-1 and ultra-rare genetic medicine programs, serving as the primary point of contact with FDA
• Lead agency interactions end to end—INTERACT, pre-IND, Type B/C, briefing books, IND submissions, and all correspondence
• Build 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 like
• Pursue expedited and designation pathways—Orphan Drug, Rare Pediatric Disease, Fast Track—and navigate plausible-mechanism and expanded access frameworks
• Compress timelines by running manufacturing and regulatory in parallel rather than in sequence
• Advise on ex-US pathways and health authority strategy outside the US
• Bring regulatory knowledge in-house, replacing external consultants who don't understand the n-of-1 model

Requirements

• Regulatory strategy across gene therapy and/or ASO / oligonucleotide programs
• Direct ownership of pre-IND and IND submissions—personally led, not supported
• Deep rare / ultra-rare development experience; n-of-1 or individualized medicine is the single strongest filter
• Has personally led agency interactions and knows how to get through CBER—neuro a strong plus
• Background in biotech, CRO, consultancy, or similarly lean environment; comfortable being a team of one
• Orphan Drug Designation experience in gene therapy
• Ex-US regulatory awareness—able to advise on pathways outside the US
• Mid-level seniority: senior enough to have led agency interactions, hands-on enough to still execute

West Coast preferred. Reports directly to the Founder/CEO.