Medical Director, GDNF

Asklepios BioPharmaceutical, Inc. (AskBio)

47d

About The Position

AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive array of capsids and promoters. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. 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 Lead and execute the clinical development strategy for gene therapy programs across early- and late-stage development (Phase 1–3) Provide medical and scientific input into clinical trial design, protocol development, dose escalation strategies, and endpoint selection, particularly for CNS and movement disorders Oversee all clinical trials from initiation through completion, ensuring data integrity, patient safety, and regulatory compliance Collaborate with internal cross-functional teams and external partners, including CROs, investigators, vendors, and regulatory agencies Conduct clinical risk assessments and implement appropriate mitigation strategies, including safety monitoring and benefit–risk evaluations Contribute to regulatory interactions, including briefing documents, investigator brochures, and responses to health authority questions Present clinical data and development updates to senior management, governance committees, and external stakeholders Supervise, mentor, and support clinical team members and contribute to a high-performing development organization Establish and maintain oversight of clinical monitoring activities across studies in a highly regulated environment, ensuring compliance with ICH-GCP, regulatory requirements, and internal quality standards Author and review key clinical and regulatory documents, including clinical protocols, investigator brochures, clinical study reports, and regulatory submissions, ensuring scientific rigor, clarity, and compliance with applicable regulatory requirements and internal standards. Collaborate cross-functionally with clinical operations, regulatory affairs, biostatistics, and safety to ensure documents accurately reflect study conduct, data interpretation, and overall clinical strategy Drive innovation in clinical trial execution, particularly in rare disease, CNS, and gene therapy settings

Requirements

MD from an accredited U.S. medical school or equivalent international medical degree with 6+ years’ relevant work experience
Completion of clinical training with demonstrated clinical experience; board certification or eligibility in Neurology or a closely related specialty
Minimum of 3 years of industry experience or related experience in clinical research or clinical development within the pharmaceutical, biotechnology, medical device, CRO/CDMO, or closely related academic or translational research setting
Direct experience contributing to the design and execution of clinical trials, including protocol development and clinical data review
Demonstrated working knowledge of Good Clinical Practice (GCP), including clinical monitoring, safety oversight, and data quality standards
Experience supporting inspection readiness activities, including preparation for or participation in FDA or other health authority inspections, audits, or internal quality reviews
Ability to independently perform the core duties of a Medical Director in a regulated drug development environment
Ability to provide medical oversight for FDA inspection readiness, including review of key trial documentation, monitoring outputs, and safety narratives
Experience reviewing and interpreting monitoring reports, audit findings, protocol deviations, and CAPAs
Strategic oversight of risk-based monitoring and trial quality management approaches
Understanding of gene therapy–specific safety risks, including delayed adverse events and durability considerations requiring extended follow-up
Strong analytical and problem-solving skills applied to clinical, safety, and compliance issues
Excellent written and verbal communication skills, including interaction with senior leadership and regulatory agencies

Nice To Haves

Prior experience in CNS, neurology, or movement disorders clinical development
Experience with gene therapy, cell therapy, or other advanced therapeutic modalities
Experience supporting or leading early Phase development programs including Phase 1 and 2 and/or Phase 3 trials, including registrational strategies
Experience with long-term follow-up (LTFU) studies, including post-treatment safety monitoring required for gene therapy products
Direct experience partnering with CROs and CRAs on monitoring strategy, risk-based monitoring, and vendor oversight
Experience working in rare disease or small patient population studies

Responsibilities

Lead and execute the clinical development strategy for gene therapy programs across early- and late-stage development (Phase 1–3)
Provide medical and scientific input into clinical trial design, protocol development, dose escalation strategies, and endpoint selection, particularly for CNS and movement disorders
Oversee all clinical trials from initiation through completion, ensuring data integrity, patient safety, and regulatory compliance
Collaborate with internal cross-functional teams and external partners, including CROs, investigators, vendors, and regulatory agencies
Conduct clinical risk assessments and implement appropriate mitigation strategies, including safety monitoring and benefit–risk evaluations
Contribute to regulatory interactions, including briefing documents, investigator brochures, and responses to health authority questions
Present clinical data and development updates to senior management, governance committees, and external stakeholders
Supervise, mentor, and support clinical team members and contribute to a high-performing development organization
Establish and maintain oversight of clinical monitoring activities across studies in a highly regulated environment, ensuring compliance with ICH-GCP, regulatory requirements, and internal quality standards
Author and review key clinical and regulatory documents, including clinical protocols, investigator brochures, clinical study reports, and regulatory submissions, ensuring scientific rigor, clarity, and compliance with applicable regulatory requirements and internal standards.
Collaborate cross-functionally with clinical operations, regulatory affairs, biostatistics, and safety to ensure documents accurately reflect study conduct, data interpretation, and overall clinical strategy
Drive innovation in clinical trial execution, particularly in rare disease, CNS, and gene therapy settings