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North Carolina, United States
Remote only
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.
The Medical Director, Movement Disorders Specialist, GDNF Program will provide strategic and hands-on clinical leadership for gene therapy programs targeting CNS movement disorders across Phase 1–3 development. This role has study-level accountability for clinical strategy, benefit–risk assessment, and execution, and serves as a medical leader within the organization. The Movement Disorders Specialist Medical Director will guide cross-functional teams, support regulatory interactions, and ensure inspection-ready, high-quality clinical development from first-in-human studies through registrational trials and long-term follow-up.
Drive the overall clinical development strategy in alignment with function, program team and timelines for the GDNF gene therapy program across Phase 1–3 with specific focus on movement disorders
Serve as a Parkinson’s clinical expert in state-of-the-art, randomized, double-blind clinical trials with emphasis on maintenance of the blinding status of the trial
Serve as the medical lead for clinical trial design, protocol development, and endpoint strategy for movement disorders indications
Provide medical oversight for clinical trials, ensuring patient safety, data integrity, and regulatory compliance
Lead clinical risk–benefit assessments, including safety signal evaluation, dose escalation decisions, and go/no-go recommendations
Authoring support for regulatory interactions, including FDA meetings, briefing documents, and inspection readiness activities
Oversee long-term follow-up (LTFU) strategies required for gene therapy products, including delayed safety monitoring and post-treatment commitments
Guide and mentor Medical Directors and other clinical team members; contribute to clinical capability building
Partner with Clinical Operations, Biostatistics, Regulatory, and CMC to ensure integrated program execution
Present clinical strategy and data to senior leadership, governance committees, and external stakeholders
Ensure a culture of inspection readiness, quality, and continuous improvement across the clinical organization
Support the strategic vision for clinical development for the GDNF program and contribute to broader organizational clinical strategy as appropriate
Foster and maintain strong relationships with key opinion leaders (KOLs), investigators, and external scientific and clinical stakeholders
Oversee the integration of clinical development activities with cross-functional partners, including Regulatory, CMC/Manufacturing, Commercial, and Research, to ensure seamless program advancement
Represent the organization as a clinical and scientific leader in high-level industry forums, scientific meetings, advisory boards, and conferences
MD from an accredited U.S. medical school or equivalent international medical degree with 6+ years relevant clinical experience with Movement Disorders patients
Completion of clinical training with board certification or eligibility in Neurology or a closely related specialty with movement disorders specialization and clinical experience
Minimum of two years of industry experience or related experience in clinical development within the pharmaceutical, biotechnology, medical device, or CRO/CDMO environment
Demonstrated leadership in the design and execution of clinical trials across multiple phases, including oversight of clinical monitoring activities
Proven experience supporting FDA or global health authority inspections, audits, or formal inspection readiness initiatives
Working knowledge of GCP, regulatory requirements, and trial quality management systems
Ability to independently lead clinical programs and make program-level medical decisions in a regulated development environment
Strong strategic leadership and cross-functional influence.
Ability to support FDA inspection readiness activities, including review of critical trial documentation, monitoring outputs, and CAPAs
Expertise in risk-based monitoring and clinical trial quality oversight
Strong analytical judgment applied to complex clinical and safety issues
Excellent communication and executive presentation skills
Ability to operate effectively in a fast-paced, evolving biotech environment
Proven track record of successful clinical program leadership, including advancing programs through critical development milestones
Exceptional strategic and operational planning skills, with the ability to translate long-term vision into executable clinical plans
Strong leadership presence with the ability to influence, inspire, and align cross-functional teams
Established professional network within the gene therapy and/or CNS development community
Understanding of market dynamics, competitive landscape, and evolving trends relevant to gene therapy and movement disorders
Prior experience in CNS, neurology, or movement disorders clinical development
Experience leading or significantly contributing to Phase 1+2 and Phase 3 registrational trials for Parkinson’s disease or similar disorders
Experience in gene therapy, cell therapy, or advanced therapeutic modalities, including long-term safety considerations
Direct experience overseeing long-term follow-up (LTFU) studies for gene therapy products
Experience working in rare disease or small patient population settings
Experience mentoring Medical Directors & clinical staff with non-neurology background
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 careers@askbio.com.
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.
