Clinical Trials

About Drug Development and Clinical Trials

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.

Ionis Pharmaceuticals / Antisense Oligonucleotides (ASOs)

Not currently enrolling – watch for updates.

Read Ionis Pharmaceuticals' Community statement

Learn about antisense oligonucleotide targeting of PRNP mRNA as 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.

Prion protein monoclonal antibody (PRN100)

Not currently enrolling

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.

Sangamo Therapeutics / Zinc Fingers

Not currently enrolling

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”