Vice President, GDNF Clinical Development

We are a gene therapy company changing lives with every genetic advancement.

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 cure genetic diseases has never wavered.

As the only end-to-end gene therapy company in the industry, we have 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 Parkinson’s disease, multiple system atrophy, limb girdle muscular dystrophy 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 Platform. Our global headquarters are in Research Triangle Park, North Carolina, a thriving biotechnology hub, and we maintain additional research and development operations in Scotland and France and gene therapy manufacturing in Spain.

We have an opportunity for the Vice President (VP), GDNF Clinical Development to join the AskBio team and be part of our continued success!

The Role

The Vice President (VP), GDNF Clinical Development, reporting to the Integrated Product Team (IPT) Leader for AskBio’s Parkinson’s disease (PD) and Multiple System Atrophy (MSA) programs, with dotted line functional reporting to AskBio’s Chief Medical Officer. Operationally the VP, GDNF Clinical Development will be a core member of the GDNF IPT and be responsible for developing and implementing the IPT’s clinical development strategy for clinical programs spanning all stages of product development. As a core member of the GDNF IPT, the VP for GDNF Clinical Development will work with all members of the IPT including the Program Manager, and functional leads for Clinical Operations, Regulatory Affairs, Medical Affairs, Commercial, Nonclinical, Drug Delivery and CMC. The incumbent will be responsible for establishing and overseeing global clinical development strategies in alignment with the company’s mission and values that will support regulatory approval and commercial launches in multiple territories including the US, EU and UK. He or she will build and manage a team of medical directors and provide leadership as co-chair of the IPT’s Clinical Sub-team in collaboration with a co-chair leading Clinical Operations. The Clinical Sub-team provides oversight of clinical research activities, including development of core documents such as clinical development plans, investigator brochures, clinical trial protocols, statistical analysis plans, clinical study reports, and regulatory filings to meet program timelines.

The VP, GDNF Clinical Development will partner with AskBio’s Safety and Pharmacovigilance teams to oversee medical monitoring and drug safety reporting, and will work closely with the Clinical Operations, Regulatory, Medical Affairs and Patient Advocacy teams on clinical study planning and resource management to execute global clinical studies. Furthermore, he or she will be positioned to expand and support the company’s global relationships with clinical sites, investigators, patient and community organizations, internal functional teams, and third-party vendors.

 Responsibilities & Accountabilities

• Provide global strategic clinical development leadership to AskBio’s GDNF IPT, working to secure cross-functional alignment via active and transparent discussions. 
• Recruit, lead, and mentor a team of development physicians and clinical scientists.
• Partner with the IPT to source clinical, biostatistics and KOL support.
• Contribute to budget planning and budget management related to clinical development activities aligned with the IPT goals.
• Liaise with the AskBio CNS Drug Delivery IPT to offer clinical development input and maintain constant alignment with GDNF IPT development plans.  
• Contribute to development and execution of the clinical strategy, 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.
• Provide input to CDP development, in line with the Target Product Profile (TPP), for successful global regulatory approvals and market access.
• Partner with IPT colleagues (including Clinical Operations, Medical Affairs, Commercial, Nonclinical, Drug Delivery and CMC) and other functional leaders to design, execute and report clinical trials.
• Partner with Clinical Operations to co-chair the IPT Clinical Subteam to provide medical oversight of study design and execution, assure timely initiation of studies, support recruitment and retention activities, and achieve program milestones on time and within budget.
• Manage the creation and maintenance of critical study documents assigned to Clinical Development (e.g., Clinical Trial Protocols (CTPs), Investigator’s Brochures (IB), Informed Consent Forms (ICF), Risk-Based Medical Monitoring Plan, Clinical Study Reports (CSRs), 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 medical expert, participate in interactions with external stakeholders (e.g., regulatory authorities, KOLs, Data Monitoring Committee (DMC), advisory boards, patient advocacy groups)
• Work collaboratively 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.

About you

• MD, DO or equivalent (based on the current World Directory of Medical Schools) is required. Neurology training with movement disorder experience is highly preferred.  
• At least 8 years of clinical development experience in an industry environment spanning clinical activities in Phases I through III/IV is required
• 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/movement disorder focus is highly preferred
• In-depth knowledge of ICH-GCP, clinical trial design, statistics, and regulatory/clinical development process
• 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/trials in a global/matrix environment in pharmaceutical or biotech industry or academia or clinical CRO is required
• Firsthand experience in organizing and managing scientific or clinical advisory boards
• Advanced knowledge in gene therapy, 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 is preferred