Lead regulatory strategy for a rare disease gene therapy heading towards a BLA submission, with Breakthrough Therapy designation already granted.
The role
The company develops AAV gene therapies for inherited metabolic disorders, with its lead programme in a pivotal Phase 3 trial and two further candidates in Phase 1. Topline data are expected in the second half of next year, followed by a Biologics License Application under Breakthrough Therapy designation. Regulatory work has so far been led by the Chief Medical Officer with support from consultants, and the company now needs a full-time head of the function. You will report to the CMO and join a senior leadership team of 12.
What you will do
Own global regulatory strategy for the portfolio, starting with the BLA and a parallel marketing authorisation application to the EMA.
Lead all interactions with the FDA’s Office of Therapeutic Products, including the pre-BLA meeting.
Build a team of four over the next year across regulatory strategy, CMC and operations.
Work with manufacturing and quality on the CMC sections, where gene therapy filings most often stall.
Advise on paediatric plans, expedited pathways and labelling.
What you will bring
At least 12 years in regulatory affairs, including direct leadership of a BLA or NDA through to approval.
Experience with cell or gene therapy, or another complex biologic.
A record of leading FDA meetings yourself, not supporting them.
Working knowledge of EMA procedures, including PRIME.
The ability to explain regulatory risk clearly to the board and to investors.
The package
Base of $290,000 to $330,000, a 30% target bonus, an equity grant and full benefits. Three days a week in the Kendall Square office. The process includes a meeting with the CEO and a presentation to the leadership team, and should complete within five weeks.
