Full-Time
Vice President Clinical Development
GDNF
AskBio
501-1,000 employees
Clinical-stage gene therapy company developing AAVs
No salary listed
Remote in USA + 1 more
More locations: Durham, NC, USA
Remote
MD
 Biology & Biotech (1) |
|---|
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- MD, DO, or an equivalent medical degree based on the current World Directory of Medical Schools, in addition to Neurology residency training.
- At least 10 years of clinical development experience in an industry environment spanning Phases I through III/IV.
- A strong track record and demonstrated excellence in Investigational New Drug, New Drug Application, and Biologics License Application submissions and approvals.
- Advanced clinical training, expertise, and clinical research experience in a medical or scientific area, preferably with a neurology focus.
- In-depth knowledge of International Council for Harmonisation Good Clinical Practice, clinical trial design, biostatistics, and the regulatory framework for global strategy development.
- Demonstrated ability to establish strong scientific partnerships with internal and external key stakeholders.
- Demonstrated strong leadership and effective communication skills, with a documented record of delivering high-quality projects or submissions in a global matrix environment in the pharmaceutical or biotechnology industry, or trial conduct in academia or a clinical contract research organization.
- Firsthand experience organizing and managing scientific or clinical advisory boards.
- Experience working with patient advocacy groups and other external stakeholders.
- Demonstrated experience with direct line management, overseeing team performance, fostering professional development, and driving clinical development excellence within the organization.
- Lead the global strategic clinical development initiatives for AskBio’s GDNF Integrated Product Team, fostering cross-functional alignment through open and collaborative discussions.
- Lead, manage, and mentor a team of clinical development physicians.
- Contribute to budget planning and budget management related to clinical development activities aligned with the Integrated Product Team goals.
- Provide clinical development input and maintain alignment with the Central Nervous System Drug Delivery Integrated Product Team and GDNF Integrated Product Team development plans.
- Contribute to the development and execution of the clinical strategy across GDNF and other Central Nervous System programs, ensuring that the overall scientific and medical content of each clinical study is scientifically rigorous, cost-effective, and designed for success.
- Identify clinical risks and implement risk mitigation strategies for complex gene therapy studies.
- Provide input to clinical development plan development, in line with the Target Product Profile, for successful global regulatory approvals and market access.
- Partner with Clinical Operations, Medical Affairs, Pharmacovigilance, Regulatory, Commercial, Nonclinical, Drug Delivery, and Chemistry, Manufacturing, and Controls colleagues to design, execute, and report clinical trials.
- Partner with Clinical Operations to co-chair the Integrated Product Team Clinical Sub-team and oversee, execute, and deliver the end-to-end clinical development strategy in alignment with the overall product strategy and objectives.
- Provide clinical development input as a member of the Integrated Product Team Brand Sub-team.
- Manage the creation and maintenance of critical study documents assigned to Clinical Development, including Clinical Study Protocols, Investigator’s Brochures, Informed Consent Forms, Risk-Based Medical Monitoring Plans, Clinical Study Reports, and other key regulatory documents, ensuring high quality and consistency with the clinical development plan, Target Product Profile, and company standard operating procedures.
- Partner with Pharmacovigilance and Safety colleagues to continuously evaluate the 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.
- Lead the GDNF Clinical Development team during interactions regarding drug development, safety, marketing, regulatory filing, and other aspects of a Central Nervous System gene therapy product life cycle.
- Participate as the neurology medical expert in interactions with regulatory authorities, key opinion leaders, Data Monitoring Committees, advisory boards, patient advocacy groups, and other external stakeholders.
- Work collaboratively with Medical Affairs to prepare and present abstracts, manuscripts, and presentations for external meetings and publications.
- Support technical due diligence in Business Development and Licensing activities as needed.
- Advanced knowledge in gene therapy drug development and 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.
- Movement disorders fellowship training with clinical research experience.
- Experience with radiographic evaluation of neurologic disease and biomarker development.
AskBio focuses on developing adeno-associated virus (AAV) gene therapies for genetic and complex disorders. Therapies use AAV vectors to deliver therapeutic DNA into patients’ cells, advancing from research and preclinical work through clinical trials toward commercialization. Its differentiation comes from a clinical-stage portfolio, in-house R&D, and partnerships that emphasize collaboration and shared development. Its goal is to bring gene therapies to patients with unmet medical needs by validating therapies clinically and pursuing strategic collaborations for commercialization.
Company Size
501-1,000
Company Stage
Acquired
Total Funding
$239.1M
Headquarters
Durham, North Carolina
Founded
2001
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See people who can refer or advise you
Simplify's Take
What believers are saying
- AB-1009 dosed its first patient on May 11, 2026, validating Pompe execution.
- GenePHIT completed enrollment on April 1, 2026, de-risking AB-1002’s pivotal next step.
- Viralgen started commercial-scale AB-1005 supply on April 20, 2026, strengthening manufacturing credibility.
What critics are saying
- GenePHIT readouts land in first-half 2027; failure would cripple AskBio’s lead cardiovascular program.
- AB-1009 enrolls only 12 patients; weak efficacy or safety ends Pompe momentum quickly.
- AAV durability, immune reactions, and manufacturing setbacks can destroy investor confidence and Bayer support.
What makes AskBio unique
- AskBio combines Bayer backing with independent operations and internal Viralgen AAV manufacturing.
- Its pipeline spans heart failure, Parkinson’s, MSA-P, LGMD, and Pompe disease.
- AB-1005 uses a surgical brain delivery platform; AB-1002 and AB-1009 target distinct tissues.
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Benefits
Health Insurance
Dental Insurance
Vision Insurance
Life Insurance
Disability Insurance
Health Savings Account/Flexible Spending Account
Unlimited Paid Time Off
Flexible Work Hours
Remote Work Options
Paid Vacation
Paid Sick Leave
Paid Holidays
Sabbatical Leave
Hybrid Work Options
Stock Options
Company Equity
401(k) Retirement Plan
401(k) Company Match
Performance Bonus
Profit Sharing
Employee Stock Purchase Plan
Relocation Assistance
Employee Referral Bonus
Parental Leave
Family Planning Benefits
Fertility Treatment Support
Adoption Assistance
Childcare Support
Elder Care Support
Pet Insurance
Bereavement Leave
Professional Development Budget
Conference Attendance Budget
Training Programs
Tuition Reimbursement
Professional Certification Support
Mentorship Program
Wellness Program
Mental Health Support
Gym Membership
Commuter Benefits
Meal Benefits
Phone/Internet Stipend
Home Office Stipend
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Growth & Insights and Company News
6 month growth
↓ -1%1 year growth
↓ -2%2 year growth
↓ -1%The Speak Foundation announces 2026 LGMD Scientific Summit. Jul 27, 2026, 10:00 ET Global researchers, clinicians, regulators, biotechnology leaders, and patient advocates will convene to advance LGMD research and therapeutic development WASHINGTON, July 27, 2026 /PRNewswire/ - The Speak Foundation today announced the 2026 LGMD Scientific Summit, an international scientific meeting that will convene leading researchers, clinicians, biotechnology innovators, regulators, and patient advocates to discuss recent advances in Limb-Girdle Muscular Dystrophy (LGMD) research, clinical development, and therapeutic innovation. The virtual event will be held July 31, 2026. The Summit comes at a pivotal moment for the LGMD field as multiple therapeutic programs advance through clinical development, new biomarkers emerge, and innovative clinical trial approaches continue to reshape rare disease research. Scientific sessions will highlight gene therapy, regenerative medicine, biomarker qualification, natural history studies, patient-focused drug development, and collaborative strategies designed to accelerate new treatments for individuals living with LGMD. FEATURED SCIENTIFIC LEADERS Featured researchers include Douglas Sproule, MD, MSc (BridgeBio Neuromuscular); Louise Rodino-Klapac, PhD (Sarepta Therapeutics); Barry Byrne, MD, PhD (University of Florida); John Vissing, MD, DMSci (University of Copenhagen); Nicholas Johnson, MD, MSCI, FAAN (Virginia Commonwealth University); Peter Kang, MD (University of Minnesota); Tahseen Mozaffar, MD, FAAN (University of California, Irvine); Simone Spüler, MD (Charité - Universitätsmedizin Berlin); Brad Williams, PhD (Jain Foundation), and Elizabeth McNally, MD, PhD (Northwestern University). Additional scientific and clinical presentations will be delivered by representatives from biotechnology companies and nonprofit research organizations advancing therapies for LGMD, including BridgeBio Neuromuscular, Sarepta Therapeutics, AskBio, Genethon, MyoPax, Edgewise Therapeutics, Advertent Biotherapeutics, Kinea Bio, Myogenica, and Cure Rare Disease. PATIENT-CENTERED DRUG DEVELOPMENT A featured panel will bring together representatives from the U.S. Food and Drug Administration and the patient community to examine patient-centered clinical trial design, stakeholder engagement, and collaborative approaches to accelerating therapeutic development for rare neuromuscular diseases. SCIENTIFIC PERSPECTIVES Nicholas Johnson, MD, MSCI, FAAN Professor and Executive Vice Chair, Department of Neurology Virginia Commonwealth University "We have entered a transformative period for LGMD research. Scientific advances are creating opportunities that were unimaginable only a decade ago, but realizing their full potential will require continued collaboration, rigorous science, and a shared commitment to ensuring that no LGMD subtype is left behind." Douglas Sproule, MD, MSc Chief Medical Officer, BridgeBio Neuromuscular "We are deeply grateful to The Speak Foundation for convening the LGMD community and fostering collaboration among patients, caregivers, clinicians, researchers, and industry, while ensuring the patient perspective remains central to scientific progress. We also thank the individuals and families who have participated in our clinical studies and entrusted us with their time and hope. It is a privilege to contribute to advancing therapies for LGMD, and that trust reinforces our commitment to rigorous science and the urgent development of meaningful new treatments." The Speak Foundation gratefully acknowledges BridgeBio Neuromuscular, Platinum Sponsor of the 2026 LGMD Scientific Summit, and AskBio, Bronze Sponsor, for their support and commitment to advancing LGMD research and therapeutic development. Registration is open to researchers, clinicians, biotechnology professionals, regulators, patients, caregivers, and members of the global LGMD community. To view the program and register, visit TheSpeakFoundation.com/LGMD-Summit. ABOUT THE SPEAK FOUNDATION Founded in 2008, The Speak Foundation is a patient-led nonprofit organization dedicated to improving the lives of individuals and families affected by Limb-Girdle Muscular Dystrophy through advocacy, research, education, and public policy. The Foundation convenes the global LGMD community by bringing together patients, researchers, clinicians, industry, regulators, and policymakers to accelerate research, improve access to specialized care, and advance therapeutic development. Signature initiatives include the International LGMD Conference, LGMD Scientific Summit, LGMD Scientific Workshop, LGMD Day on the Hill, LGMD News Magazine, and the LGMD Centers of Excellence. SOURCE The Speak Foundation
AskBio doses first patient in Phase 1/2 gene therapy trial for late-onset Pompe disease. AskBio has dosed the first participant in a Phase 1/2 clinical trial evaluating AB-1009, an investigational AAV-based gene therapy for late-onset Pompe disease. AskBio has dosed the first participant in PROGRESS-GT LOPD, a Phase 1/Phase 2 clinical trial evaluating investigational gene therapy AB-1009 for the treatment of late-onset Pompe disease (LOPD).[1] The milestone advances the company's efforts to develop a potentially durable gene therapy approach for the rare neuromuscular disorder, which continues to present significant unmet medical needs despite the availability of enzyme replacement therapies (ERTs). AB-1009 is an adeno-associated virus (AAV)-based investigational gene therapy designed to enable sustained production of acid alpha-glucosidase (GAA), the enzyme deficient in patients with Pompe disease.[1] The therapy is being developed by AskBio, a wholly owned and independently operated subsidiary of Bayer AG.[1] The open-label PROGRESS-GT LOPD trial is expected to enroll approximately 12 adult participants across sites in the United States.[2] Investigators will evaluate the therapy's safety, tolerability, and preliminary efficacy in patients with LOPD.[2] Addressing long-term challenges in Pompe disease. Pompe disease is a rare inherited lysosomal storage disorder caused by mutations affecting production of GAA, leading to glycogen accumulation in muscle cells.[3] In LOPD, patients typically experience progressive skeletal muscle weakness and respiratory decline that worsens over time.[3] "Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical." - Tahseen Mozaffar, MD Although multiple recombinant human GAA enzyme replacement therapies have been approved, clinical limitations remain.[1] Some patients experience declining therapeutic response over time, requiring ongoing infusions and increasing treatment burden.[4] Researchers have increasingly explored gene therapy approaches as a potential strategy for sustained enzyme production and reduced dependence on chronic ERT administration. "Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical," said Tahseen Mozaffar, MD, director of the UCI Health ALS & Neuromuscular Center and principal investigator for the AB-1009 clinical program, in the company announcement.[1] According to AskBio, AB-1009 is designed to address the underlying genetic cause of Pompe disease by enabling continuous endogenous production of GAA following a single administration.[1] Regulatory momentum supports program development. The investigational therapy has already received both FDA Fast Track and Orphan Drug designations, reflecting regulatory recognition of the significant unmet need in Pompe disease.[1] The FDA also accepted the investigational new drug application for AB-1009 earlier this year.[1] Fast Track designation is intended to facilitate development and expedite review of therapies targeting serious conditions with limited treatment options.[5] Orphan Drug designation may provide incentives including market exclusivity, tax credits, and regulatory support for therapies addressing rare diseases.[6] "Reaching this milestone reflects the momentum behind our gene therapy platform and our commitment to working toward improving quality of life for people with rare diseases," said Canwen Jiang, MD, PhD, chief development officer and chief medical officer at AskBio, in the company statement.[1] The clinical advancement also highlights continued investment in neuromuscular gene therapies, an area that has seen substantial growth across both biotech and pharmaceutical sectors over the past several years. Gene therapy continues expanding in rare disease. AAV-based gene therapies have emerged as a major area of focus in rare genetic disease research, particularly for neuromuscular disorders caused by single-gene mutations.[7] Developers are increasingly targeting diseases such as Duchenne muscular dystrophy, limb-girdle muscular dystrophy, spinal muscular atrophy, and Pompe disease using viral vector platforms intended to provide long-term protein expression after a single treatment.[7] Despite growing momentum, challenges remain, including durability of expression, immune responses, manufacturing complexity, and long-term safety monitoring.[7] Still, industry investment continues to accelerate as companies pursue therapies capable of transforming chronic rare diseases into potentially manageable conditions with one-time administration approaches. For patients with LOPD, the initiation of the PROGRESS-GT LOPD study represents another step toward evaluating whether gene therapy may offer a more durable alternative to lifelong enzyme replacement treatment.
a-Klotho cell and gene therapy potential. 25 March 2026 Avaí Bio recently announced that it has initiated manufacturing of a master cell bank of genetically modified cells that overexpress the α-Klotho protein, working alongside joint venture partner Austrianova through their shared entity, Klothonova. A Master Cell Bank isn't a research milestone. It's a manufacturing milestone. It's the GMP-compliant, fully characterized starting material from which all working cell banks and final therapeutic products will be produced. It's the moment a cell therapy programme transitions from "we think this could work" to "we're building the production infrastructure to deliver it." There is a key 'master' protein in your body called α-Klotho. The scientific community has been quietly obsessed with it since a Japanese researcher named Makoto Kuro-O discovered in 1997 that mice without the Klotho gene aged rapidly and died young - and mice with extra Klotho lived 30% longer. Since then, the research has only gotten more compelling. Peer-reviewed studies have linked higher Klotho levels to reduced risk of Alzheimer's, certain cancers, cardiovascular disease, and kidney failure. Mayo Clinic research has connected declining Klotho to arterial stiffness and vascular calcification. And here's the part that makes this personal: natural α-Klotho levels drop by approximately 50% after age 40. The protein that protects your brain, your heart, and your kidneys starts disappearing right when you need it most. The question the biotech industry is now racing to answer is simple: can you put it back? The global cell therapy market has surpassed $8.2 billion in 2026. The broader cell and gene therapy sector is forecast to surge from $10.4 billion to more than $45 billion by 2035. And at the intersection of regenerative medicine and longevity science, a small biotechnology company just hit a manufacturing milestone that turns the Klotho thesis from laboratory theory into production reality. The Cell-in-a-Box(R) technology is the delivery mechanism that makes the whole approach viable. When you transplant therapeutic cells into a patient, the immune system typically destroys them within days. Austrianova's encapsulation technology protects the cells inside a porous capsule, allowing the cells to continuously produce and secrete the target protein - in this case, α-Klotho - while protecting the cells from immune rejection. It's backed by over 50 peer-reviewed publications and decades of development. Avaí Bio's dual-programme approach targets both the Klothonova anti-aging platform and the Insulinova diabetes program, each leveraging the same Cell-in-a-Box(R) encapsulation technology. The addressable markets are enormous: Alzheimer's disease alone is projected to reach $32.8 billion by 2033, cardiovascular disease remains the leading cause of death globally, and kidney disease affects 850 million people worldwide.
Through AskBio, a wholly owned subsidiary of Bayer, it is developing a gene therapy as a potential new approach for treating congestive heart failure.
Brain Neurotherapy Bio, Inc (BNB) is pleased to announce its recent merger with Asklepios BioPharmaceutical, Inc (AskBio), a Bayer company.