Role Summary:

The Associate Director, Clinical Scientist is a key member of the Clinical Development team for the planning, conduct, analysis, and reporting of clinical studies. The person in this role plays a key role in designing, implementing, and managing clinical trials to evaluate novel therapies for patients with rare muscle diseases. The person in this role works independently and in collaboration with the Clinical Development Lead and other cross-functional team members in the execution of clinical trial protocols, analysis of clinical trial data, review of regulatory documents, and summary of key data for internal and external presentations.

This role is based in Waltham, MA without the possibility of being remote.

Primary Responsibilities Include:

• In collaboration with the Clinical Development Lead, be accountable for the scientific aspects of the design, implementation, and conduct of clinical trials with both internal and external stakeholders (e.g., investigator meeting presentations, data reviews and summaries, protocol deviation reviews, etc.)
• Contribute to the development of clinical trial protocols, including study design, site selection, patient recruitment strategies, and protocol amendments in close collaboration with the Clinical Development Program Lead and cross-functional team members
• Contribute to the creation and amendment of study-level documents, including drug handling and administration documents, physical therapy manuals, clinical study reports, and preparation of clinical sections of key regulatory documents, including Investigator’s Brochures, briefing books, and submission dossiers
• Collaborate with cross-functional teams including medical affairs, regulatory affairs, biostatistics, and clinical operations to ensure successful planning and execution of clinical trials
• Provide scientific expertise and guidance on study design, data analysis, and interpretation of clinical trial results
• Monitor study progress, identify risks and issues, and develop mitigation strategies to ensure timely and successful completion of clinical trials
• Contribute to high quality clinical safety and efficacy data review and identify clinical data insights through ongoing patient-level review and trends analysis
• Assist with review and interpretation of clinical study data and production of both internal and external facing data presentations
• Contribute to regulatory submissions, including INDs/CTAs, briefing documents, and responses to regulatory queries
• Collaborate with key opinion leaders and investigators to support scientific discussions and presentations
• Assist and collaborate with cross-functional team members to maintain clinical development plans aligned with key strategic goals of the Company
• Stay abreast of the latest scientific developments and therapeutic advances in rare muscle diseases to inform clinical development strategies

Education and Skills Requirements:

• Advanced degree (PhD, MD, PharmD) in life sciences/ healthcare (or clinically relevant degree).
• 8 years of experience in clinical development in the pharmaceutical or biotechnology industry, with a focus on rare diseases preferred.
• 3 years as a Clinical Trial Scientist or comparable role
• Strong familiarization with Good Clinical Practice (GCP) and drug development processes
• Expertise in neuromuscular disease biology and rare disease experience strongly preferred
• Strong knowledge of clinical trial design, execution, and regulatory requirements
• Experience in designing and implementing clinical trials for rare diseases is highly desirable
• Good knowledge of set-up, organization and execution of global clinical studies in a pharmaceutical company or contract research organization (CRO), or comparable experience within academic research institution
• Sound knowledge of principles of clinical data collection and reporting; demonstrated ability to use systems and tools (e.g., EDC systems, Excel, PowerPoint, Veeva CRM) for data collection, analysis and reporting
• Ability to effectively prioritize tasks and comfortable working highly independently in a cross-collaborative matrix environment
• Excellent communication skills with the ability to collaborate effectively with cross-functional teams and external stakeholders
• Proven ability to work independently, prioritize tasks, and meet deadlines in a fast-paced environment
• Flexibility for travel both domestically and internationally for trial site visits, meetings, conferences, etc.~20% travel

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