Drug Development - CJD Foundation

Drug Development and Clinical Trials

The status of prion disease drug development changes regularly. In July 2025, five organizations working toward a treatment for prion disease met with families and spoke on a panel at the CJD Foundation Family Conference in Chicago. Hear more about the information each of those organizations shared at the conference.

Current Programs

Ionis Pharmaceuticals / Antisense Oligonucleotides (ASOs)

Learn about antisense oligonucleotide targeting of PRNP mRNA as a potential treatment for prion disease from this recording of the 2020 Virtual Conference Session presented by Anne Smith, PhD, Executive Director of Clinical Development at Ionis Pharmaceuticals, a California-based biotechnology company focused on RNA-targeting therapeutics.

Broad Institute – Vallabh/Minikel Lab

Launched in Fall 2015, Prions@Broad represents an innovative new model for rare disease drug discovery. Sonia Vallabh and Eric Minikel changed careers to become scientists and devote their lives to prion therapeutics after learning, in December 2011, that Sonia had inherited a fatal genetic mutation in the prion protein gene from her late mother. In our unique model for precision medicine research, these two personally motivated patient-scientists with their disease-specific focus are embedded within a department and an institute equipped with the tools and expertise needed to enable therapeutic discovery.

NobelPharma

Nobelpharma has constantly been aspiring to conduct research and development of drugs, that are not the prime targets for many pharmaceutical companies due to questionable economic viability with a small number of patients (drugs for unmet medical needs).

“We have completed several essential non-clinical studies and expect to initiate clinical studies very soon, probably next year.”

— Kiyoshi Arita, MSc, Project/Product Manager, Research and Development Division, International Clinical Development Department, Nobelpharma Co., Ltd., Tôkyô, Japan, speaking at CJD Foundation Family conference, July 2025

Gate Bioscience

Gate Bioscience is a biotechnology company developing a new class of drugs called Molecular Gates designed to eliminate harmful proteins, like prion protein, before they can cause disease. Molecular Gates have a precise mechanism of action: they selectively block the export of a harmful protein from cells, causing the protein to be degraded instead of secreted. Molecular Gates are small molecule therapeutics, which means they are taken as a pill instead of injected.

Many studies show that reducing prion protein levels in the brain could be an effective way to treat prion disease. Gate Bioscience is developing a Molecular Gate drug to eliminate prion protein from the brain. By reducing the levels of prion protein, the drug eliminates the substrate required for prion multiplication and spreading, thus stopping the disease in its tracks.

Early lab and animal studies have shown promising results: in mice, a lead compound successfully lowered prion protein levels in the brain by 80% after only a week of once daily dosing. With the support of this CJDF grant and in collaboration with the Vallabh Minikel lab at the Broad Institute, Gate Bioscience will now test whether one of their lead compounds can extend survival in a mouse model of prion disease.

If successful, these data will be pivotal in the race to develop a safe and effective prion protein lowering drug that patients and at-risk individuals can pick up from the pharmacy, place in their medicine cabinet, and take from the comfort of their own home to prevent, slow, or reverse this terrible disease.

Sangamo Therapeutics / Zinc Fingers

Learn about a potential future treatment for prion disease from this recording of the 2022 presentation by Brian Zeitler, Senior Director of Gene Regulation, Sangamo Therapeutics: “Engineered zinc finger protein transcription factors potently reduce brain PrP expression and extend survival in prion-infected mice”

Additional Information

Process of Clinical Research and Drug Development

Learn about the process of clinical research and drug development and the role rare disease patient communities can play in the process from this recording of the 2022 Virtual Conference Session presented by Laura Iliescu, MSc, Director, Patient Advocacy Strategy, Center for Rare Diseases.

Prion protein monoclonal antibody (PRN100)

Human prion diseases, including Creutzfeldt–Jakob disease (CJD), are rapidly progressive, invariably fatal neurodegenerative conditions with no effective therapies. Their pathogenesis involves the obligate recruitment of cellular prion protein (PrPC) into self-propagating multimeric assemblies or prions. Preclinical studies have firmly validated the targeting of PrPC as a therapeutic strategy. Read a study in The Lancet Neurology that evaluates a first-in-human treatment programme using an anti-PrPC monoclonal antibody under a Specials Licence. Learn about Prion protein monoclonal antibody (PRN100) therapy for CJD from this recording of the 2022 Virtual Conference Session presented by Prof John Collinge, Professor of Neurology, MRC Prion Unit at UCL, UCL Institute of Prion Diseases.