The Rare Disease Research Unit is looking for a talented senior physician scientist to join our team developing transformational new treatments in rare Neuromuscular, Neurodevelopmental and Neurodegenerative diseases using AAV gene therapy. This individual will focus on the challenging translational, clinical and regulatory problems presented by gene therapy in rare (often) pediatric indications, including natural history studies, novel endpoint development and clinical trial design. The candidate will be accountable for the delivery of translational and clinical development plans for AAV gene therapy programs in multiple rare neurological indications, and will be skilled in early clinical program design and conduct from pre-clinical stages to POC. Similar activities will be required for programs exploiting other treatment modalities. Experience and proven collaborative skills with basic and clinical scientists, investigators, regulators and safety functions are essential.
Lead interactions with colleagues in Global Product Development and Pfizer Innovative Health with regard to clinical development planning and product concept development for AAV gene therapies and other modalities.
Partner with functions in Early Clinical Development as needed to ensure that biomarker and quantitative development plans are enabled in all relevant Rare Neuro programs to help determine early signs of efficacy and proof of concept, patient stratification, etc.
Serve as a clinical champion for early development Rare Neuro programs in AAV gene therapy and other modalities
Help to build, develop, and lead motivated and engaged matrix teams
Provide oversight to clinical teams, including:
Lead preparation of clinical sections of all relevant regulatory filings (IND, Orphan designation, Fast track and breakthrough designations – including NDA/BLA when required).
Oversee management of studies and serve as expert on clinical and medical issues, including medical monitoring where necessary
Oversee review of study data, including safety monitoring and SAE reporting
Oversee writing of final study reports and preparation of documents for submission
Establish and support relationships with external experts, consultants, key opinion leaders, to ensure implementation of latest clinical thinking and guidelines into clinical development plans and study designs
Serve as medical and technical resource for clinical issues raised by internal and external collaborators, investigators, consultants and contractors
Mentor or supervise other clinicians to expand drug development understanding and provide development opportunities
Foster a transparent environment that encourages strong partnerships and mutual trust between teams, sub-teams, and therapeutic areas.
Represent the company at scientific meetings and presentations, interactions with rare disease foundations, as well as other public relations opportunities
Assist in the development of publications, abstracts, and presentations
Support business development activities as needed, including due diligence activities.
Maintain up-to-date knowledge of scientific and clinical published literature in relevant therapeutic areas including the key clinical development issues.
Qualifications -
Requires MD or MD/PhD
Board certified or eligible in Neurology or Pediatric Neurology preferred
Deep understanding of the biopharma environment and the drug development process; 7+ years of relevant experience in drug development with proven track record of success in executing development programs to completion
Strong interpersonal skills and expert team player with demonstrated ability to build consensus and drive change across all levels of the organization, including senior management
A clear working knowledge of the clinical development process and its variation in rare diseases. Experience and knowledge of gene therapy approaches.
A proven track record of success within large pharma or biotech environment with a demonstrated ability to formulate and operationalize clinical strategies
Good understanding of relevant disciplines such as biostatistics, regulatory, clinical pharmacology, pre-clinical toxicology, pharmacogenomics and biomarker based approaches.
Demonstrated scientific productivity (publications, abstracts, etc.).
Good communication and presentation skills as well as proven scientific writing skills.
Results-oriented with demonstrated track record of success in clinical development
Ability to navigate a complex matrixed environment and manage teams that are diverse in terms of geography, background, and expertise
Entrepreneurial mindset with the ability to bring creative solutions to challenges
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