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’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 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 Clinical Development Medical Director to join AskBio team in our Research Triangle Park (Durham), NC and be part of our continued success!
The Clinical Development is responsible for the clinical sciences and biostatistics of product development, including design, medical oversight of execution, and reporting of clinical trials. Clinical Development is accountable for clinical dossiers to enable interactions with regulatory agencies and IND/BLA submissions.
The Clinical Development Medical Director (CDMD) is the global clinical leader responsible for one or more clinical programs across indications, involving one or multiple molecular entities. Under the guidance of the Vice President, Clinical Development (CDVP) and partner with the Therapeutic Sector Leader, the CDMD owns the risk benefit assessment for the program(s), and is accountable for the design, medical oversight of execution, and reporting of a clinical development program(s) to support internal governance decision, regulatory submissions, and market access. The CDMD may lead a highly matrix and cross-functional clinical development team to develop and implement a Clinical Development Plan (CDP). The Medical Director will report to the Vice President, Clinical Development.
Responsibilities & Accountabilities
- Represent Clinical Development on the Global Program/Project Team
- Partner with Therapeutic Sector Leader or Global Project Leader to develop and execute the clinical strategy
- Partner with the Therapeutic Sector Leader and others to develop the Clinical Development Plan (CDP), in line with the Target Product Profile (TPP), for successful global regulatory approvals and market access for one or multiple indications
- Partner with the Therapeutic Sector Leader, Biostatistics and other functions to design and report clinical trials
- Lead the creation of clinical components of key documents (e.g., Clinical Trial Protocols (CTPs), Investigator’s Brochures, Inform Consent Form (ICF), Risk-Based Medical Monitoring Plan, Clinical Study Reports (CSRs), regulatory documents including maintenance of product licenses, registration dossiers, value dossiers, pharmacoeconomic dossiers) with high quality and consistency with IDP and TPP
- Partner with Clinical Operations to provide medical oversight of trial execution, assure timely initiation of studies, support recruitment and retention activities and reach study timelines/ milestones
- May support technical due diligence in Business Development & Licensing (BD&L) activities
- Together with Medical Safety Monitor, ensure continuous evaluation of drug safety profile, including safety monitoring of clinical studies and signal detection from post-marketing surveillance
- Serve as a core member of the Safety Management Team (SMT)
- As the medical expert, participate in interactions with external stakeholders (e.g., regulatory authorities, KOLs, Data Monitoring Committee (DMC), advisory boards, patient advocacy groups), internal stakeholders (e.g.Therapeutic Sector Leaders, Clinical Operations, Biostatistics, Regulatory Affairs, Project Management, Research, Technology Development, Manufacturing, Translational Medicine, Global Medical Affairs, Marketing and Market Access, Business and Corporate Development), and internal governance committees
- May serve as project leader to strategize and execute a development program under the guidance of the Therapeutic Sector Leader
- May organize and manage scientific or clinical advisory boards
- MD or equivalent (based on the current World Directory of Medical Schools) is preferred; PhD, PharmD, or DVM with relevant clinical research experience and achievements will be considered
- Advanced clinical training/knowledge or clinical research experience in a medical/scientific area, preferably aligned with AskBios’ focused therapeutic areas (cardiovascular, CNS, neuromuscular, metabolic, and genetic disorders), is required
- At least 5 years of experience in clinical research in an academic institution or 3 years in an industry environment spanning clinical activities in Phases I through III/IV, including IND and NDA submission dossiers is required
- In-depth knowledge of ICH-GCP, clinical trial design, statistics, and regulatory/clinical development process
- Experience with regulatory submissions and interactions with health authorities is required
- Demonstrated ability to establish strong scientific partnership with internal and external key stakeholders
- Demonstrated leadership and strong communication skills with a documented track 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
- Hands-on experience in organizing and managing scientific or clinical advisory boards
- Advanced knowledge in gene therapy, training or extensive experience in genetic disorders, neurosciences, and/or heart failure, with the capability to innovate in clinical development study designs that provide robust clinical evidence to internal decision-makers and regulatory agencies is preferred
- Non-clinical research and development experience and achievements (e.g. research, translational medicine, medical affairs, medical safety, regulatory affairs) in academia or biopharmaceutical industrial is preferred.