A new rare disease partnership vision.
We believe that a fundamentally different paradigm is absolutely necessary to find treatments for rare diseases; one that integrates direct patient engagement and a creative system for funding the development of new medicines. BioPontis Alliance for Rare Disease is launching The CMT Therapeutic Alliance, A BioPontis Partnership to advance promising discovery research into treatment for Charcot Marie Tooth Disease.
science and patient organization partnerships
The mission of our Therapeutic Alliance for Charcot Marie Tooth disease begins with patients and great science. We begin each program by engaging with patient organizations who become true partners in the Alliance. Through this partnership we evaluate the readiness of the knowledge base in the rare disease to launch a therapy discovery program.
BioPontis Alliance establishes the technical expertise to plan the development and resources to conduct the experiments for each therapeutic program
BioPontis Alliance has established a global and world class network of contract research resources ready to be assembled around each rare disease project. Our scientists plan and manage the development of treatment candidates
A structure to enable treatment discovery
Each rare disease therapeutics development Program is organized and managed within a special legal entity, a Therapeutic Alliance.
Patients are integral to the Therapeutic alliance. Their partnership is essential to:
- help define which critical aspects of their disease should be addressed by a therapy.
- participate with their medical information and blood/tissue donations to develop essential lab tests to monitor effects of experimental therapies.
- be organized and prepared to become trial subjects.
- help advocate at regulatory agencies at all stages of development and registration.
The Therapeutic Alliance can receive directed philanthropic funding from BioPontis and from outside sources including grants, both public and private.
Intellectual property (patents) can be licensed directly to the Therapeutic Alliance from academic or other sources.
Researchers and clinical experts are recruited into the development team.
Harnessing funding that serves the need to find rare treatments
We begin with philanthropy and government grants to support early steps in the path of developing a new therapy. Later steps may be suitably supported by investments from pharmaceutical companies or by other interested parties. Finally, we will license our therapies that are ready to be proven in human trials to the pharmaceutical companies that await these opportunities to bring new products to patients. They have the resources and capabilities to complete the clinical trials and distribution steps.
Sharing the sustaining economics of success
Because the patient organization(s) and academic scientists are true partners, their benefit is not limited to participation in the development of the ultimate treatment. Each will also have a share in any financial benefits that may come from sale of that therapy to a commercial entity such as a pharmaceutical company. Our partnership approach allows for any payments of royalties or other licensing fees to go back to the patient organizations, academic institutions or other non profit partners in the Therapeutic Alliance. Those funds can then be re-applied in the charitable missions of each organization consistent with their tax exempt status (US 501c3 or European charity regulations).