Barth Syndrome Foundation (BSF) Company Profile

Background

The Barth Syndrome Foundation (BSF) is a nonprofit organization dedicated to saving lives through education, advancing treatments, and finding a cure for Barth syndrome—a rare, life-threatening genetic disorder primarily affecting males. Established in 2000 and headquartered in Larchmont, NY, BSF serves as the only global network uniting families, healthcare providers, and researchers to combat this disease.

Key Strategic Focus

BSF's strategic objectives encompass:

Research Advancement: Funding and facilitating research to understand Barth syndrome's pathology and develop effective treatments.

Patient Support: Offering resources, educational conferences, and individualized support to affected families.

Awareness and Advocacy: Raising public and professional awareness and advocating for policies that support the Barth syndrome community.

Financials and Funding

BSF operates as a 501(c)(3) tax-exempt organization. In 2023, the foundation reported:

Revenue: $1,334,806

Expenses: $1,421,717

Net Assets: $5,285,354

The foundation has funded over $6.6 million in research and catalyzed more than $37.7 million from other sources to advance scientific discoveries.

Pipeline Development

BSF supports various research initiatives, including:

Strategic Initiatives Program (SIP): A flexible funding vehicle designed to advance the Barth syndrome research and development pipeline.

Technological Platform and Innovation

BSF collaborates with biotech firms to explore innovative treatments. Notably:

Partnership with Scenic Biotech: In March 2022, BSF partnered with Scenic Biotech to explore genetic modifiers for tailored treatments, leveraging Scenic's Cell-Seq discovery platform.

Leadership Team

Emily Milligan: Executive Director since 2019, overseeing strategic initiatives and operations.

Katherine McCurdy: Chair of the Board, providing governance and strategic direction.

Leadership Changes

No recent significant leadership changes have been reported.

Competitor Profile

Market Insights and Dynamics

The Barth syndrome treatment market is characterized by a dynamic competitive landscape with a mix of established pharmaceutical companies and emerging biotechnology firms. Major players such as Sarepta Therapeutics and Sangamo Therapeutics are actively involved in developing innovative therapies targeting the underlying genetic causes of the disorder. These companies are leveraging advancements in gene therapy and enzyme replacement therapy to create effective treatment options. Additionally, collaboration among research institutions, universities, and patient advocacy groups is fostering an environment conducive to innovation and the discovery of new therapeutic approaches. The relatively small patient population poses challenges for market players, but it also presents opportunities for specialized companies focused on rare diseases. Ongoing clinical trials and increased funding for research initiatives are further enhancing competition, as companies strive to bring effective treatments to market. Overall, the competitive landscape is evolving, driven by advancements in medical research and heightened awareness of Barth syndrome.

Competitor Analysis

Stealth BioTherapeutics: Focused on mitochondrial dysfunction therapies, developing elamipretide for Barth syndrome.

Sarepta Therapeutics: Engaged in gene therapy research for rare diseases.

Sangamo Therapeutics: Specializes in genomic medicine, including gene therapy and genome editing.

Strategic Collaborations and Partnerships

BSF has established significant collaborations to advance research:

Scenic Biotech: Partnered to explore genetic modifiers for tailored treatments.

Stealth BioTherapeutics: Advocated for the FDA review of elamipretide, a potential treatment for Barth syndrome.

Operational Insights

BSF's strategic initiatives, such as the Strategic Initiatives Program, aim to bridge gaps in research funding and expertise, accelerating the development of potential therapies.