Research Scientist, Rare Disease

RTW Foundation • New York, NY

19h • $120,000 - $150,000 • Hybrid

About The Position

The Research Scientist, Rare Disease is a senior scientific role within RTWF’s Rare Disease Advising Program (RDAP). This position leverages deep training in biomedical science and translational research to guide rare disease foundations through complex research, therapeutic development, and strategic decision-making landscapes.
The Research Scientist conducts in-depth literature reviews, analyzes preclinical and clinical data, and synthesizes insights into disease concept models, therapeutic hypotheses, and actionable research roadmaps. In parallel, this role plays a critical leadership function, managing and mentoring interns and junior researchers, standardizing research processes, and scaling RDAP’s disease review pipeline.
This role blends hands-on scientific analysis with strategic advising, team leadership, and close engagement with patient-led organizations, clinicians, academic investigators, and industry partners.

Requirements

• PhD (preferred) in biomedical sciences, genetics, pharmacology, immunology, neuroscience, or related fields us required.
• Post-PhD experience preferred, including Industry experience in drug development (e.g., biotech, pharma, translational research organizations), or Academic experience with leadership in rare disease or translational research.
• Demonstrated understanding of therapeutic development pipelines, including preclinical models, IND-enabling studies, and clinical trial planning.
• Experience mentoring scientists or managing research teams.
• Deep scientific curiosity with the ability to translate complex research into clear, practical, and actionable recommendations.
• Strong written and verbal communication skills across technical and non-technical audiences, including rare disease families.
• Highly organized and detail-oriented with strong project management capabilities.
• Collaborative, patient-centered mindset aligned with nonprofit mission-driven work.
• Familiarity with research platforms (e.g., PubMed, Scopus), grant management tools (e.g. Salesforce) and productivity software (SharePoint, Outlook).
• Must be able to work from our New York City office (on-site/hybrid).

Responsibilities

• Provide expert-level scientific and translational guidance to rare disease foundations through structured advising engagements.
• Conduct and oversee comprehensive literature reviews and landscape analyses spanning disease biology, natural history, therapeutic modality prioritization, potential research collaborators.
• Develop disease concept models and multi-stage research roadmaps to guide foundation strategy and funding decisions.
• Identify knowledge gaps, preclinical or clinical barriers, and drug development opportunities across rare disease areas.
• Track and synthesize trends across therapeutic platforms (e.g., gene therapy, RNA therapeutics, genome editing, biologics, small molecules) to inform up-to-date recommendations.
• Recruit, train, and supervise internal (in-person) and external (virtual) interns, fellows, and volunteers conducting disease-focused research deep-dives.
• Guide and quality-control team-generated research outputs to ensure scientific rigor, clarity, and strategic relevance.
• Standardize templates, workflows, and training materials to support scalable and repeatable disease review processes.
• Balance individual contributor work with primary responsibility for managing and mentoring others.
• Participate in the scientific review of research grant proposals for scientific rigor and translational potential.
• Support rare disease foundations in refining funding strategies aligned with therapeutic readiness and impact.
• Contribute to the improvement and scaling of internal grant review, tracking, and reporting systems.
• Represent RTW Foundation in scientific meetings, workshops, conferences, and collaborative forums.
• Participate in meetings with patient-led organizations, translating complex science into clear, actionable guidance.
• Collaborate across RTWF programs and with external partners to share insights and inform organizational strategy.
• Regular preparation of disease-specific research slide decks (“deep dives”) summarizing biology, therapeutic landscape, and strategic considerations.
• Development and maintenance of standardized slide and report templates to support RDAP research activities.
• Completion and oversight of a sustained pipeline of disease reviews supporting advising engagements and funding cycles.

Benefits

• In addition to the base salary, the role includes a discretionary incentive compensation program, and a full benefits package, including medical and life insurance, retirement plans, tax-free savings options, and access to other healthcare programs.
• Hybrid work policy offering one day work from home.

What This Job Offers

• Job Type: Full-time
• Career Level: Mid Level
• Education Level: Ph.D. or professional degree