This page describes what two patient-facing organisations publish about their work with industry. It is information for companies planning engagement. It does not speak for these organisations, and each decides its own terms. Contact them directly.
What does the CJD Foundation do with drug developers?
Its drug development page lists organisations working on treatments, among them Ionis Pharmaceuticals, Prions@Broad at the Broad Institute, NobelPharma, Sangamo Therapeutics, Gate Bioscience and Regeneron. It hosts statements from some of these groups and links the ClinicalTrials.gov entries for the Ionis, Broad Institute and Regeneron trials. It reports on programmes that are not enrolling, naming Sangamo’s zinc finger programme and the PRN100 antibody therapy.
In July 2025, five organisations working on treatments met families and spoke on a panel at the Foundation’s Family Conference in Chicago. The page links to a conference session on the clinical research and drug development process and the role of rare disease communities. Families can reach the Foundation’s helpline on 1-800-659-1991 or at help@cjdfoundation.org. The Foundation also funds research grants, described on the grant calendar, and says one grant supports work at Gate Bioscience.
What does the Prion Alliance do?
In its mid-year update of 16 July 2026, the Alliance says donations help cover travel for trial participants and their caregivers. It describes the NIH NeuroNEXT programme as funding the PRiSM trial and providing its operational infrastructure. It commits to sharing individual patient data from PRiSM and general learnings, and says FDA regulatory documents are already public. It is funding further observational studies in pre-symptomatic and symptomatic people, which it describes as in planning and approval, and it is urging clinicians to speed up diagnosis.
Where do the organisations’ statements differ?
Trial status changes quickly, and the sources are dated differently. The Alliance’s July update says only symptomatic patients were being recruited to PRiSM and that pre-symptomatic people should wait. The CJD Foundation page, citing a statement of 14 October 2026, reports that the pre-symptomatic arm is open. The Alliance also says Ionis has reopened a third PrProfile arm, while the ClinicalTrials.gov record, updated 18 September 2026, lists the study as active, not recruiting. Check the registry and the sponsor for current status.
What should a company consider?
- Keep communication with families to what the sponsor can state, with a date. Eligibility is as stated by the sponsor.
- Do not ask an organisation for a family’s or person’s details. Neither organisation published a route for that, and this site does not name families.
- Plan for travel and caregiver costs. Both the Alliance and the registry records, which require a caregiver in PrProfile and PRiSM, point to this.
What is not known?
- Neither organisation’s pages read for this brief publish terms for industry partnerships, advisory roles or data requests.
- No source read reports how many families each organisation reaches.
See also biomarkers and endpoints, unmet need and patient numbers, the pharma overview and the foundations and patient groups list in the Atlas.