Senior Vice President, Regulatory Affairs

Overview

Work at ROCKET PHARMA and help cure rare diseases!

Rocket Pharma is a fully integrated, clinical-stage company advancing gene therapies with curative potential for multiple rare childhood diseases. Rocket places enormous value on people and considers team member progress and well-being to be as important as the progress of its pipeline. We are looking for hands-on team players who enjoy collaborating with colleagues. We share a passion for seeking gene therapy cures for devastating diseases and making a difference for patients. This is an opportunity to become an integral part of a small, highly productive team and to help grow an entrepreneurial, scientifically driven organization that is increasingly recognized as a leading biotechnology innovator. In addition to a competitive compensation package featuring a generous 401K match and stock options, the position includes excellent health benefits.

Position Summary

As Rocket prepares for continued growth, they are at an inflection point where a senior level regulatory affairs leader will be pivotal. The incoming Senior Vice President (SVP), Regulatory Affairs will be a key member of the senior leadership team, reporting to the Chief Medical Officer. The SVP will develop and drive regulatory strategies to progress Rocket's innovative pipeline of first-in-class adeno-associated viral vector (AAV) and lentiviral vector (LVV) gene therapies.

The ideal candidate will be a dynamic, and astute senior regulatory strategy leader with demonstrated expertise in the rapidly evolving gene therapy industry. This is an opportunity for the successful candidate to make an impact in a critical part of the business. This includes careful evaluation of regulatory and drug development risks or gaps and developing contingency plans with a focus on efficiency and planning for first in human and proof of concept studies through registration. The incoming SVP will be a trusted partner to the leadership team and will be critical to driving Rocket Pharmaceuticals’ development and leadership in regulatory affairs.

The SVP of Regulatory Affairs will be responsible for leading and implementing strategic and operational regulatory plans for the development of current and future indications. They will play an important role in maintaining effective communication and fostering critical relationships with internal/external collaborators as well as regulatory authorities. This role provides the unique opportunity to design and guide regulatory pathways across an innovative portfolio of early to pivotal-stage gene therapy assets. As the company continues to scale, the incumbent will have the ability to contribute to the organizational build and directly impact patients’ lives by gaining approval for, and delivering on, critical gene therapy products for rare and undertreated diseases.

Responsibilities

• Develop and execute regulatory strategies for all clinical, non-clinical and CMC approval pathways across Rocket's pipeline, and ensure compliance with ICH guidance, FDA, EMA, and additional countries’ regulations.
• Leads regulatory interactions with the FDA, EMA, and other Agencies on behalf of Rocket Pharmaceuticals, while overseeing all aspects of label development and negotiation, and effectively negotiating acceptable responses to health authority inquiries.
• Advises leadership team and Board on regulatory strategy and execution for early and late-stage programs, CMC, nonclinical and commercial activities, as well as advice on areas of regulatory risk, and updates on new governmental/regulatory developments.
• Demonstrates a comprehensive understanding of current FDA and EMA gene therapy guidelines and regulations for a variety of submission documents, including INDs, BLAs, MAAs, all types of amendments, their supplements, and annual reports. This includes comprehensive familiarity with Orphan Drug (US/EU), Rare Pediatric, Regenerative Medicine Advanced Therapy (RMAT), Advanced Therapy Medicinal Product (ATMP) and PRIME designations.
• Ensures all outgoing FDA/EMA submissions are complete, accurate, timely and in compliance with current regulations and guidelines.
• Develops trust and strong relationships with Agencies based on frequent & transparent discussions.
• Participates in due diligence and provides regulatory advice and perspective on all potential licensing and M&A deals.
• Gains in-depth scientific knowledge of diseases, as well as non-clinical and clinical data supporting the development of Rocket’s pipeline programs.
• Demonstrates regulatory understanding of CMC gene therapy platform (LVV and AAV), Rocket’s product manufacturing process, and associated analytics knowledge.
• Hires, develops and manages a high-quality regulatory team.
• Develops and oversees regulatory operatio