Director, Regulatory Affairs CMC

Asklepios BioPharmaceutical, Inc. (AskBio) is a leading, clinical-stage gene therapy company founded in 2001 based on the work of adeno-associated virus (AAV) gene therapy visionary and pioneer, Jude Samulski, PhD. Since that time, our commitment to developing life-saving medicines that can potentially cure genetic diseases has never wavered. At AskBio, we’ve built a foundation of therapeutic programs, AAV manufacturing and a rich portfolio of patented technology. Our therapeutic focus includes neuromuscular, central nervous system, cardiovascular and metabolic diseases with clinical trials underway for Pompe disease, Parkinson’s disease, and congestive heart failure. Our gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. We became a wholly-owned, independently operated, subsidiary of Bayer AG in late 2020 as a cornerstone of its newly formed Cell & Gene Therapy (CGT) platform. Our global headquarters is in Research Triangle Park, NC. Additionally, we have significant operations in Philadelphia, PA, Columbus, OH, Scotland, France, Germany, and Spain. Our vision:

Pioneering science to create transformative molecular medicines. Our mission:

Lead innovative science and drive clinical outcomes to transform people's lives. Our principles: Advance innovative science by pushing boundaries.

Bring transformative therapeutics to patients in need.

Provide an environment for employees to reach their fullest potential.

Our values: Be a Pioneer. We are not afraid of the impossible and to innovate to make gene therapies accessible to those in need.

Cultivate Collaboration. Strive to be the best teammate, actively listen, openly communicate, and embrace diverse points of view.

Embrace Responsibility. We are humbled by the enormity of our mission. We hold a relentless commitment to advance science and clinical outcomes for our patients, families, and caregivers.

Raise the Bar. Continuously drive improvements and efficiencies. Seek and provide constructive feedback. Have a bias for learning and action.

Act with Uncompromising Integrity. Be honest, transparent, and committed to doing what’s right in every situation. Make clear commitments and follow through.

Position Summary The

Director, Regulatory Affairs CMC

is responsible for managing the regulatory CMC strategy and coordinating regulatory operations and logistics for all assigned projects. This role will assure appropriate development and timely registration of gene therapy product candidates. Reporting to the Vice President, Regulatory Affairs CMC, the role will be responsible for the progression and submission of regulatory filings for gene therapy projects, in the US and internationally. The individual will drive the preparation and submission of high‑quality CMC sections of briefing documents, IMPDs, GMOs, INDs, CTAs, and eventual original marketing applications (BLAs/MAAs), in close collaboration with analytical, CMC and Quality teams, contract manufacturing organizations, and external consultants and collaborators. This opportunity will offer you the chance to grow your Regulatory Affairs CMC expertise across a range disciplines with diverse regulatory requirements from novel gene therapy platforms in an evolving regulatory landscape. Job Responsibilities Support innovative, comprehensive regulatory strategies and associated timelines for assigned projects to ensure timely global registration of Company’s product candidates in gene therapy.

Provide regulatory CMC strategic support to the development of multiple innovative gene therapy products for the treatment of a broad range of diseases.

Manage and implement planning, authoring and submission of high-quality CMC‑related sections of briefing documents and clinical trial applications in the US, EU and ROW.

Assist with negotiations with regulatory agencies to resolve CMC issues and shepherd proactive interactions with regulators globally; prepare and submit responses to CMC questions; support interactions with regulatory agencies during GMP and pre-approval inspections.

Drive adherence to CMC regulatory guidelines relevant for the development of gene therapy products; author, review and approve, as necessary, internal documentation to ensure conformance with regulations and existing regulatory approvals.

Support development of manufacturing strategies for AskBio’s therapeutics, including assessing proposed manufacturing process changes and provide strategic regulatory guidance.

Support the development of regulatory processes and standards.

Ensure operational alignment and on-time completion of all registration-directed activities through close collaboration with Project Management colleagues and senior management.

Assist in the preparation of project teams/colleagues for discussions with health authorities, including but not limited to USFDA, EMA, MHRA, PMDA, and HC. Lead team discussions with regulators to facilitate positive outcomes.

Guide project teams on appropriate and accurate interpretation of health authority regulations and guidelines, approval processes, special mechanisms to expedite development and registration globally.

Partner and work very closely with CMC, Analytical Development, Quality, Nonclinical, Clinical and Regulatory to ensure achievement of submission deadlines and obtain timely approvals of CMC‑related applications.

Partner with Quality Assurance colleagues to ensure regulatory compliance with applicable local and international laws and regulations, as well as industry standards.

Minimum Requirements Bachelor’s Degree in a scientific discipline with a minimum of 10 years of experience in drug development, process or analytical R&D and manufacturing is required with experience in biologics, vaccines, gene or cell therapy. Equivalent experience on the review/agency side may also be considered.

At least 5 years in a regulatory CMC role and demonstrated increasing level of responsibility.

Experience working with cell therapy, gene therapy products or mRNA-based therapeutics.

Proven record of contributions to successful IND/CTA and NDA/BLA/MAA submissions and approvals.

Preferred Skills & Abilities Advanced degree (MS, MD, PhD, PharmD) in a scientific discipline or engineering.

Excellent negotiation skills and demonstrated experience modeling positive communications, interpersonal, and relationship-building skills.

Experience in advanced therapeutic technologies (e.g., gene therapy, cell therapy).

Deep and broad knowledge of FDA, EMA, MHRA, PMDA, HC, etc. laws, regulations, directives, guidance and practices.

Demonstrated problem-solving skills and learning agility.

Enthusiasm and drive to lead special assignments that enhance AskBio’s excellence in gene therapy product development, registration, and commercialization.

Proficiency with MS Office products and applications commonly used in Regulatory Affairs.

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