VP, GDNF Clinical Development

Asklepios BioPharmaceutical, Inc. (AskBio)•Durham, NC

About The Position

AskBio Inc., a subsidiary of Bayer AG, is a gene therapy company focused on developing life-saving medicines. The company has a pipeline of investigational therapeutics for various diseases, including congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio utilizes a proprietary cell line manufacturing process (Pro10™) and an extensive array of capsids and promoters. The Vice President (VP), GDNF Clinical Development, will report to the head of Global Clinical Development and be a core member of the GDNF Integrated Product Team (IPT). This role is responsible for developing and implementing the clinical development strategy for clinical programs across all stages of product development. The VP will collaborate with various cross-functional groups, including Program Management, Clinical Operations, Regulatory Affairs, Medical Affairs, Commercial, Nonclinical, Drug Delivery, and CMC. The position requires establishing and overseeing global clinical development strategies to support regulatory approval and commercial launches in key territories like the US, EU, and UK. The VP will build and manage a team of medical directors and co-chair the IPT’s Clinical Sub-team (CST) with a Clinical Operations co-chair. The CST is responsible for developing and executing the overall clinical strategy, ensuring high-quality execution of complex clinical trials, and aligning timelines with the overarching IPT clinical program strategy. The CST also oversees clinical research activities, including the development of core documents such as clinical development plans, investigator brochures, clinical trial protocols, statistical analysis plans, clinical study reports, and regulatory filings. The VP will partner with Safety and Pharmacovigilance teams for medical monitoring and drug safety reporting, and work with Clinical Operations, Regulatory, Medical Affairs, and Patient Advocacy teams on clinical study planning and resource management. Responsibilities include analyzing, interpreting, and reporting clinical study data to support BLA/MAA filings and global product launch. The VP will also act as a Subject Matter Expert (SME) to maximize product positioning and value generation in preparation for commercial launches, working closely with medical affairs, market access, and commercial product teams. Additionally, the role involves expanding and supporting global relationships with clinical sites, investigators, patient and community organizations, internal functional teams, and third-party vendors.

Requirements

MD, DO or equivalent (based on the current World Directory of Medical Schools), in addition to Neurology residency training
At least 10 years of clinical development experience in an industry environment spanning Phases I through III/IV
Strong track record and demonstrated excellence in IND/NDA/BLA submissions and approvals
Advanced clinical training, expertise and clinical research experience in a medical/scientific area, preferably with a neurology focus
In-depth knowledge of ICH-GCP, clinical trial design, biostatistics, and regulatory framework for global strategy development
Demonstrated ability to establish strong scientific partnership with internal and external key stakeholders
Demonstrated strong leadership and effective communication skills with a documented record of delivering high quality projects/submissions in a global matrix environment in pharmaceutical or biotech industry or trial conduct in academia or clinical CRO is required
Firsthand experience in organizing and managing scientific or clinical advisory boards
Experience working with Patient Advocacy Groups and other external stakeholders
Demonstrated experience with direct line management; overseeing team performance, fostering professional development, driving clinical development excellence within the organization

Nice To Haves

Advanced knowledge in gene therapy drug development, training or experience in neurosciences with the capability to innovate in clinical development study designs that provide robust clinical evidence to internal decision-makers and regulatory agencies
Movement disorders fellowship training with clinical research experience
Experience with radiographic evaluation of neurologic disease and biomarker development

Responsibilities

Lead the global strategic clinical development initiatives for AskBio’s GDNF IPT, fostering cross-functional alignment via open and collaborative discussions
Lead, manage, and mentor a team of clinical development physicians
Contribute to budget planning and budget management related to clinical development activities aligned with the IPT goals
Provide clinical development input and maintain alignment with the CNS Drug Delivery IPT and GDNF IPT development plans
Contribute to development and execution of the clinical strategy across GDNF and other CNS programs, ensuring that the overall scientific and medical content of each clinical study is scientifically rigorous, cost-effective sound and designed for success
Proactively work to identify clinical risks and implement risk mitigation strategies for complex gene therapy studies
Provide input to CDP development, in line with the Target Product Profile (TPP), for successful global regulatory approvals and market access
Partner cross-functional colleagues (including Clinical Operations, Medical Affairs, Pharmacovigiliance, Regulatory, Commercial, Nonclinical, Drug Delivery and CMC) to design, execute and report clinical trials
Partner with Clinical Operations to co-chair the IPT CST to oversee, execute and deliver the end-to-end clinical development strategy in alignment with the overall product strategy and objectives
Provide clinical development input as a member of the IPT Brand Subteam
Manage the creation and maintenance of critical study documents assigned to Clinical Development (e.g., Clinical Study Protocols (CSPs), Investigator’s Brochures (IB), Informed Consent Forms (ICF), Risk-Based Medical Monitoring Plan, Clinical Study Reports (CSRs), and other key regulatory documents) with high quality and consistency with CDP, TPP and AskBio SOPs
Partner with Pharmacovigilance and Safety colleagues to provide continuous evaluation of drug safety profile, including safety monitoring of clinical studies and signal detection from post-marketing surveillance. May serve as a core member of the Safety Management Team (SMT)
Lead the GDNF Clinical Development team during interactions regarding drug development, safety, marketing, regulatory filing, and other aspects of a CNS gene therapy product life cycle
As the neurology medical expert, participate in interactions with external stakeholders (e.g., regulatory authorities, KOLs, Data Monitoring Committee (DMC), advisory boards, patient advocacy groups)
Work collaboratively with Medical Affairs to prepare and present abstracts, manuscripts and presentations for external meetings and publications
May support technical due diligence in Business Development & Licensing (BD&L) activities