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

Minimum 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

Preferred Education, Experience and Skills

• 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

AskBio Inc. (AskBio) is an Equal Opportunity Employer and does not discriminate against any employee or applicant for employment because of race, color, religion, gender, sexual orientation, gender identity, national origin, age, disability, veteran status or any other protected status prohibited under Federal, State or local laws. All employment decisions are based on valid job‐related requirements. If you are a qualified individual with a disability or a disabled veteran and are unable or limited in your ability to use or access our website, you may request a reasonable accommodation to express interest in a specific opening by calling us at (919) 561-6210 or sending us an email at [email protected].

Agencies:  Please do not contact any employee at AskBio about this requisition.  Any resume submitted by a recruitment agency to any employee at AskBio, through any medium, will be deemed the sole property of AskBio unless the agency was engaged by AskBio Talent Acquisition team to recruit for that position.  All agencies must have a prior executed service agreement with AskBio prior to any search engagement.  If a candidate who was submitted outside of the AskBio agency process is hired by AskBio, no fee or payment of any kind will be paid to the agency.