BioPontis Alliance Develops Translational Research Readiness Tool with Rare Disease Patients Organizations

BioPontis Alliance Develops Translational Research Readiness Tool with Rare Disease Patients

February 15, 2017

FOR IMMEDIATE RELEASE – Raleigh, NC, USA and Brussels, BE – BioPontis Alliance for Rare Diseases, an international charitable organization, is announcing the introduction of its Translational Research Readiness Tool (TRRT). The TRRT is the first of a series of initiatives by BioPontis to help rare disease patient’s organizations manage their research programs toward the essential data and tools needed to start the journey from basic disease knowledge to drug discovery. BioPontis is extending its therapy discovery mission to more effectively utilize the wealth of great discovery science being supported by public agencies and by rare disease patient advocacy organizations to generate a more robust flow of new potential therapies.

The TRRT is intended to provide guidance to patient organizations on the types of research investments that they and their researchers can carry out to more effectively move from detailed knowledge about a disease to potential therapeutic strategies for counteracting the causes of that disease and set up the basis for the search for actual treatment candidates. “By using the TRRT with patient groups and their advisors in basic science and clinical practice, BioPontis can add the drug discovery and development focus to highlight promising paths to new therapies”, explains BioPontis’ Chief Science Officer, David Spencer, PhD.

The TRRT is available for download at Patient organizations are invited to download the tool and contact BioPontis with questions and suggestions for how it can be best applied in their specific organization’s strategic planning and management. June Kinoshita, Chief Operating Officer of the Facioscapulohumeral (FSH) Society, helped to pilot the TRRT and remarks; “Working with BioPontis on the TRRT was a highly educational and rewarding experience. The exercise gave the FSH Society an opportunity to review the critical benchmarks that the field needs to meet in order to proceed with drug discovery and development and to have several scientific experts weigh in on the current status of the field. This process brought to the surface the need to invest more in the development of cell lines, for example, and establishing other resources that are more easily available to researchers. We’ll be able to continually update the TRRT to measure our progress and efficacy of our funding.”

As a nonprofit organization performing the earliest stages of drug discovery and development, BioPontis Alliance for Rare Diseases partners with academic research institutions and patient organizations with a dual mission: translate science into clinical candidates for rare neurologic diseases, and engage patient organizations to develop novel partnerships that enable patients’ participation on development teams. BioPontis is uniquely positioned to explore how patients’ representation and engagement could be practiced in treatment development.

This initiative has been supported by generous contributions from Shire, GlaxoSmithKline, the Baillet Latour Fund and private donors to BioPontis Alliance.

About BioPontis Alliance for Rare Diseases

BioPontis Alliance for Rare Diseases is a 501c(3) public charity in the US (North Carolina) and a foundation of public utility (fup/son) in Belgium (Brussels). Managed by an international Board and executive team, BioPontis partners with patient organizations and clinical researchers to bridge the gap between promising science and new medicines. BioPontis Alliance for Rare Diseases is being established as a global non-profit, inviting corporate, private and foundation philanthropic support. More information, including how contributions to support our programs and mission can be made, at



Erik Tambuyzer, Chairperson
+32 475 615711 (Brussels, Belgium)

Barbara Handelin, CEO
+1 610.636.2332 (Philadelphia, USA)

Richard Basile, COO
+1 919.345.5386 (Raleigh, USA)