The National Institutes of Health estimates that:

  • 30 million people in the United States are living with rare diseases. This equates to 1 in 10 Americans or 10% of the U.S. population
  • Europe has approximately 30 million people living with rare diseases

Worldwide, it is estimated that more than 300 million people suffer from rare diseases – half of them children. In fact, rare diseases together are among the most life-threatening and debilitating conditions children face.

Advancing Treatments For Rare Diseases

BioPontis Alliance is a nonprofit therapeutics product development organization focused on the needs of these hundreds of millions of patients.

Our mission is to partner with rare disease patient organizations and academic scientists, with the goal of translating their research results into viable treatments.

We do this by:

  • Identifying and selecting promising therapeutic approaches
  • Doing the work necessary – the complex product development work to prepare new treatments for the clinical trials required to prove the product will safely help patients
  • Developing these new treatments to the point where pharmaceutical companies are able to take them forward through the final human trials needed to get them approved for use in patients, and the final commercial development to ultimately get them in the hands of doctors and patients

We are creating a new ecosystem needed to overcome the challenges of developing new treatments for rare disease, conducting the business of translating good science into potential medicines. By providing the necessary skills and resources, we serve as a bridge connecting rare disease basic research with product development science, patent creation, business partnerships and regulatory compliance.

We have one simple goal: Bring new treatments to patients suffering from rare diseases that will improve their lives.

Our work carries the hope of a treatment or cure for millions of patients with rare disease – people who otherwise are unnoticed and neglected by the medical R&D enterprise. We harness the philanthropic drive to strive for cures, and use it to drive practical, effective programs that deliver long-overdue treatments to patients in need.

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