Job Purpose Summary: This position is split between two 50% roles: The candidate will serve as the Scientific Director of the Sickle Cell Regulatory Science Consortium and Scientific Director supporting additional rare disease programs (Duchenne Regulatory Science Consortium and Rare Disease Cures Accelerator- Data and Analytics Platform). The Scientific Director will contribute to the overall strategic planning and management of collaborative research endeavors with various stakeholders to accomplish the objectives of C-Path's Sickle Cell Regulatory Science Consortium, as well as supporting specific projects in other rare disease programs. Through presentations and scientific publications, the Scientific Director communicates the vision of C-Path's sickle cell and other rare disease initiatives to key stakeholders, including pharma, academic, patient foundations, and government participants; existing and potential funders; strategic partners and the scientific community. The Scientific Director will also work with other scientific staff to produce regulatory documents and other materials to support this work.
Essential Job Duties and Responsibilities:
Provide scientific leadership and coordination as well as administrative oversight of rare disease activities.
Provide scientific input to individual workgroup discussions dealing with clinical and scientific issues relating to the work of the consortium/initiative including the sharing and analysis of genetic data as well as medical product development tools.
Foster relationships with key individuals among regulatory bodies, companies, academics, patient groups, and other research initiatives or other collaborative efforts in the field.
Coordinate efforts of a broad range of stakeholders to develop evidentiary considerations for evaluating the safety and effectiveness of gene therapies.
Guide the execution of a detailed work plan and its milestones to develop, evaluate, and prepare applications for submission to the FDA/EMA for qualification of quantitative disease models, biomarkers, COAs, etc. for a specific use in medical product development as appropriate.
Establish and lead qualification projects (where appropriate), including developing research plans, task lists, milestones and timelines.
Recruit new organizations from pharmaceutical, biotechnology and device companies for potential membership or engagement in projects; enlist other research-performing organizations that are doing work in the area to benefit from their expertise and data.
Create and execute detailed research work plans and revise as appropriate to meet changing needs and requirements.
Prepare annual budget and manage expenditures.
Work with C-Path administration management to provide financial oversight of any grants or funds utilized to execute the work of the Initiative.
Communicate consortia/programs progress via scientific publications and presentations.
Seek collaborations/connectivity with other consortia and external organizations
Travel on occasion for out-of-town meetings (approximately 10%).
Other duties and responsibilities may be assigned.
Participates in the supervision and oversight of project manager(s), administrative support staff, consultants and vendors.
Experience with the design, conduct, and reporting of experiments or studies relevant to the preparation of IND applications: for example, the applications of novel efficacy or safety biomarkers, or preclinical efficacy models.
Working knowledge of assay development and validation, and biostatistical analysis.
Working knowledge of regulatory approval and the medical product development process.
Ability to drive collaboration and facilitate the scientific activities of diverse groups of stakeholders toward improving the conduct of regulated medical product studies.
Ability to communicate clearly with a broad range of audiences (scientists, regulators, industry, patients) through clear presentation skills and writing skills.
Ability to provide clear and concise messaging to consortium members with multiple demands for their time and attention.
Knowledge of FDA/EMA/PMDA regulations and requirements.
Working knowledge of good clinical practices.
Apply effective management, interpersonal, negotiation, strategic planning and problem-solving skills.
Adapt to shifting priorities, demands, and timelines.
Track best practices and lessons learned and operationalize those within the team.
Identify opportunities for improvement and make constructive suggestions for change.
Practice highest level of integrity and core value system consistent with C-Path's code of conduct.
Ability to meet target deadlines and manage time effectively.
Strong critical thinking and analytical skills.
Ability to successfully anticipate issues or challenges and proactively address without being specifically directed.
Use sound judgment when working with critical or confidential information.
Education and Training:
A PhD (or equivalent doctoral degree) in Genetics or related scientific discipline. Training or experience in genetic therapies and rare diseases is highly desirable.
Four or more years' experience in medical product development (discovery, pharmacology, safety assessment, clinical development, or regulatory affairs) in academia, industry and/or government.
Broad scientific, clinical, technical, and regulatory understanding of the functions involved in the development of medical products, diagnostics, and/or devices.
Internal Number: SCIDIR SC
About Critical Path Institute
C-Path is a globally recognized nonprofit whose mission is to improve the medical product development process by advancing drug development tools, through knowledge and data sharing via pre-competitive consortia and other collaborative platforms with regulatory agencies, industry, and academia.