Director, Regulatory Affairs - AAV

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

Rocket Pharma is seeking an experienced and highly motivated Director, Regulatory Affairs for AAV, who enjoys a fast-paced, dynamic work environment at the forefront of innovation.

This role will be responsible for providing US regulatory leadership in support of the development, registration, and life-cycle management of our innovative gene therapy programs.

The responsibilities include providing US regulatory leadership and direction for Rocket’s gene therapy products, including developing long and short-term planning of regulatory projects that align with the company's business plan, and developing strategies to ensure effective achievement of regulatory / business objectives.

The individual will be responsible for staying current in the US regulatory environment and assess and communicate impact of changes on business and product development.

The position will report to the Associate Vice President of Global Regulatory Affairs.

Responsibilities

• Provides US regulatory leadership in support of the development, registration, and life-cycle management of Rocket’s products.
• Provides strategic regulatory direction including developing the US regulatory plan, advising on US regulatory requirements for development plans, study designs and marketing approval, conducting risk assessments, and managing critical issues and FDA interactions.
• Writing regulatory documents (. new INDs, briefing book content, RMAT Designation request, Priority Review Requests, ODD);

reviewing core documents to support IND, in conjunction with EUL or GRL as needed.

• Foster positive relationships with FDA
• Ensures the successful implementation and execution of regulatory plans.
• Harnesses knowledge of FDA and ICH regulatory requirements to strategically and operationally resolve regulatory issues impacting development programs to ensure business goals are met.
• Builds partnerships with key senior stakeholders from other functions to ensure that strategic business goals are met through the sharing of knowledge, expertise and the provision of appropriate resources.
• Liaises and negotiates with US FDA as needed for all aspects pertaining to drug development including novel regulatory pathways for the company's gene therapy products, resolution of key regulatory issues and to expedite approvals of products.
• Takes a hands-on approach in leading the completion of IND / CTA and other global submission documents.
• Serves as a subject matter expert on relevant US regulations, stays abreast of changes in the US regulatory environment, and assesses impact of changes on business and product development programs.
• Proactively manages critical issues, taking leadership for regulatory contribution.

Qualifications

• MS degree and 10+ years Pharmaceutical industry experience. PharmD / PhD Degree preferred
• 5+ years of regulatory filing (IND / CTA / BLA / MAA) strategy preferred.
• We encourage applicants with strong expertise in AAV gene therapy, experience with IND submission, and particularly those with PharmD or PhD, to apply.

This skill set and educational background may outweigh the requirement for a specific number of years of experience.

• Hands-on regulatory experience in submission of INDs
• Extensive experience in interacting with Health Authorities.
• Ability to develop and implement US regulatory strategies.
• Rare disease and / or gene therapy experience is desirable.
• Strong scientific foundation, including a strong understanding of preclinical, cardiac, gene therapy products and development.
• Practical understanding, interpretation, and application of relevant ICH and FDA guidelines and regulations.
• Excellent verbal and written skills; able to analyze, define and effectively convey difficult and complex issues in a way that accurately and persuasively communicates issues to internal and external stakeholders, including Senior Management.
• Ability to think creatively and develop creative solutions.
• Ability to prioritize and handle multiple projects simultaneously.
• Sense of urgency and perseverance to achieve bringing curative therapies to patients.
30+ days ago