Portfolio Review of Clinical Candidates


Barth Syndrome Foundation has reached an important milestone in our mission to accelerate treatments and find a cure for Barth syndrome. Presently, three therapeutic ideas are at the cusp of clinical trial implementation:

  1. Gene augmentation therapy (AAV9) with Dr. Barry Byrne;
  2. Enzyme replacement therapy (ERT) with Dr. Michael Chin; and
  3. ALCAT1 inhibitor therapy with Dr. Roger Shi.

Clinical trials will be required to test the safety and efficacy of these potential therapies in order for affected individuals to one day be able to access life-changing options. Beyond the technical and partnering challenges to advance these products, BSF must consider the limited number of affected individuals eligible for participating in clinical trials, the anticipated therapeutic burden and benefit of each proposed therapy, the competitive landscape of therapeutic possibilities, and the capital requirements for BSF to de-risk the development programs.

Meeting Purpose

BSF seeks to understand specific ways, within the next 1-3 years, that the Foundation can strategically advance the above listed clinical candidates through collaborative efforts with the principal investigators (PIs), their academic institutions, and commercial partners. To achieve this goal, PIs will present on several
different considerations that will impact the clinical development of their potential therapies.

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