Research Scientist, Rare Disease

About RTW Foundation (RTWF) RTW Foundation (RTWF) was founded in 2018 as the philanthropic arm of RTW Investments, LP a biotech investment firm. RTWF has two major focuses: improving health equity in New York City and advancing ultra-rare disease research. Our overarching goal is to improve the health of underserved communities, whether that be from historically underinvested communities in New York City (including communities of color and migrant populations) to patients and families facing ultra-rare diseases. Our rare disease initiatives focus on conditions often overlooked due to small patient populations and limited commercial incentives, working directly with patient-led foundations to move science toward the clinic. Role Overview 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. Why Join Us? This role offers a unique opportunity to bridge scientific research, therapeutic development, and strategic advising in the rare disease space. At RTW Foundation, you’ll play a direct role in shaping research strategies that impact patients and families facing ultra-rare diseases. If you're passionate about applying your scientific expertise to drive meaningful change, we encourage you to apply. Key Responsibilities Scientific Advising & Research Support: 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. Research Oversight & Team Management 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. Grant & Funding Support: 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. Cross-functional & External Engagements: 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. Deliverables 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. Qualifications and Skills Education & Experience: 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. Skills & Attributes: 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). The duties and responsibilities described are not a comprehensive list and additional tasks may be assigned to the employee from time to time. Compensation & Benefits The base salary for this New York-based position ranges from $120,000 to $150,000, depending on experience, skills, and responsibilities. Actual salary may vary based on qualifications and the level at which the role is hired. 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. Some community events will require a commitment outside of typical working hours, for example our quarterly Days of Action are typically held on Saturdays. To Apply Please submit a resume and a personal statement highlighting any previous non-profit or rare disease research experiences that might be relevant to the role outlined above.