About Barth Syndrome Foundation: 

The Barth Syndrome Foundation (BSF) is a 501(c)(3) nonprofit, founded in 2000 with a steadfast international mission of “Saving lives through education, advances in treatments, and finding a cure for Barth syndrome.” As the only organization focused on Barth syndrome, this means that we not only educate and empower affected individuals and their families to be able to live their best lives with this devastating disease today, but we also aggressively pursue scientific and medical advancements to give them hope for a better tomorrow. BSF makes strategic investments in basic, translational, and clinical research to bolster fundamental understanding of the disease mechanism, pursues novel and repurposed drugs as well as genetic approaches for treatment, and supports clinical trials and the development of guidelines for clinical care. With the expanding ecosystem of experts that we engage in our quest, together we will find and make accessible treatments for this life-threatening and life-limiting, ultra-rare, genetic, metabolic disorder.

Position Summary:

Reporting to the Executive Director, the Director of Research is a senior and trusted staff member who directs the creation, development, expansion, and management of the Foundation’s research portfolio. Primary responsibilities focus on cultivating basic and preclinical efforts, with additional technical support allocated for translational and clinical trials. He/she/they leads strategic planning efforts to inform resource allocation across new and established research and development programs; implements scientific priorities though a variety of BSF funding vehicles; and effectively communicates scientific information to the lay public (and especially our families, the BSF staff and Board of Directors, and our donors). The Director of Research helps recruit and partners closely with the world-class Scientific and Medical Advisory Board (SMAB) and other ad hoc expert advisors, who review BSF seed grant applications, advise on scientific and medical policies of the organization, and provide expert guidance on an array of specific scientific and medical programs and projects undertaken by the organization, such as disease natural history augmentation.

Specific Responsibilities:

• Identifies, analyzes, evaluates, monitors, and suggests action regarding science trends and opportunities of strategic importance. Responsible for executing impactful scientific investment portfolio.
• Collaborates with the Executive Director to identify resources needed and develop strategies for partnerships with appropriate research institutions and for-profit institutions.
• Oversees BSF-funded basic and translational annual seed grants program, including recruitment of applicants, review of proposals, interaction with the principal Investigators, management of the review process with the SMAB and outside expert reviewers who have been recruited, presentation of the applications to the mostly lay BSF Board of Directors.
• Actively manages post-award portfolio and plans for success by exercising foresight to avoid funding gaps and maintain R&D momentum.
• Reviews Federal requests for applications and analyzes the impact of those opportunities regarding potential research concerning Barth syndrome.
• Fosters engagement and relationship development with federal agencies, especially NIH and FDA in the US and equivalent organizations internationally in concert with BSF affiliates.
• Cultivates working relationships with biotechnology and pharmaceutical companies to advance treatments for Barth syndrome.
• Creates and develops BSF-funded strategic investments, including informing contracts and cooperative agreements, with other institutions doing work on Barth syndrome related projects.
• Organizes and manages the scientific and medical sessions of the biennial Barth Syndrome International Scientific, Medical and Family Conference, in conjunction with the Organizing Committee who is recruited for each conference.
• Works virtually but travels on a limited basis as required to represent BSF at specific national and international scientific meetings and to present at other BSF events throughout the year (including major donor meetings).

Required Education, Experience, and Aptitudes:

• Required Ph.D., M.D. or equivalent doctoral degree and extensive experience in working on complex disorders affecting multiple organ systems (with experience in rare, metabolic, or mitochondrial disorders a plus).
• Required ten years or more of prior related work experience in research review and management. Experience in drug development or healthcare a plus.
• Previous experience in grant writing, scientific peer review, and contract management desired.
• Demonstrated ability to develop and maintain collaborative relationships with key stakeholders, including scientists, doctors and administrators from academic institutions, employees from for-profit companies, other expert advisors, colleagues from peer organizations, and lay volunteers.
• Excellent communication with many diverse audiences – scientists, physicians, business people, donors, and, very importantly, affected families.
• Proven propensity to think creatively and strategically to identify various resources needed for projects.
• Effective and efficient project creation and management, with ability to multi-task in a team environment.
• Ability to display high degree of emotional intelligence and interest interfacing with members of the affected population.
• Knowledge of regulatory practices as they pertain to therapeutic development and industry operations in terms of business development is a plus.

We recognize that no applicant fits a job description perfectly, so we encourage candidates with most, but not all, of the above qualifications to apply.

Please direct expressions of interest to Emily Milligan, BSF Executive Director at: Emily.milligan@barthsyndrome.org