Location: Boston, US

　　Reporting to: Executive Director, Regulatory Science, Head of Early-Stage Programs

　　Job Summary

　　Early-stage programs encompass all Orchard Therapeutics ex vivo gene therapy assets from preclinical stage up to Phase 3 clinical development. After initiation of the registrational clinical study, programs evolve to the late-stage status.

　　This role reports to the Head of Regulatory Science for early-stage programs and the successful candidate will be responsible for the successful development and registration of new ex-vivo Gene Therapy Products in the US and globally.

　　Key Elements and Responsibilities

　　Act as US regulatory lead on assigned early-stage program(s) o Work in close partnership with the Global Regulatory Lead(s) to provide necessary regulatory inputs to the related cross-functional program teams

　　o Develop the US regulatory strategy, including any innovative procedures to expedite the path to approval, in line with corporate goals, in partnership with the Global Regulatory Lead(s) and the program regulatory team(s)

　　o Execute the US regulatory strategy:

　　§ Be the primary liaison with US regulatory authorities for the assigned early-stage programs

　　§ Plan, prepare, and review submissions to FDA, including Investigational Orphan designation applications (ODD), Investigational New Drug (IND) applications, Fast Track/RMAT designations, Pediatric study plans

　　§ Prepare FDA meeting requests, briefing documents, coordinate and prepare cross-functional teams for meetings, and interface with US regulatory authorities

　　§ Coordinate and prepare responses to questions and requests for information from US regulatory authorities

　　§ Ensure that regulatory documents are accurate, complete, compliant with applicable regulatory requirements and prepared in accordance with the defined timelines

　　Actively participate in regulatory compliance and regulatory team development:o Monitor, analyze, and disseminate intelligence on regulatory matters that may affect ongoing development programs

　　o Lead the implementation of new US guidance applicable/relevant to development programs in close collaboration with the Global Regulatory Lead(s) and relevant functional leads

　　o Contribute to updates, improvements and development of processes (including new SOPs as applicable) linked to regulatory activities

　　o Share expertise and learning experiences - “grow together” approach within the regulatory team

　　This position offers growth and development opportunities towards a Global Regulatory Lead role for a global development program.

　　Requirements

　　Required Experience/Knowledge

　　Significant experience in the development and execution of US regulatory strategies for development programs in industry related US Regulatory Affairs

　　Very good knowledge and understanding of US CFR and FDA guidelines is a must – understanding of EMA regulations and guidelines is a plus

　　Previous experience in using US innovative expedited paths to approval in the frame of global development is a plus

　　Track record of INDs preparation and management up to FDA clearance

　　Previous experience of leading a team to prepare for and attending major health authority interactions (e.g. pre-IND meeting, EOP1, EOP2 meetings etc.) is a must

　　Experience of regulatory submission project management

　　Knowledge and experience of development of cell or gene-based therapy is a plus

　　Previous experience in the preparation and submission of BLA is a plus

　　Previous experience with ex-US authorities is a plus

　　Regulatory Affairs certification is a plus

　　Education

　　PharmD or PhD in a scientific disciplineSkills and Abilities

　　Ability to work in a cross-functional team environment; ability to work with remote teams

　　Strong collaboration mindset

　　Strong attention to detail

　　Excellent organizational skills, ability to handle multiple tasks simultaneously and prioritize effectively

　　Proven computer and documentation skills

　　Strong interpersonal skills including verbal and written communication (essential in a highly collaborative work environment and to interact efficiently with Authorities)