Richmond Pharmacology is thrilled to confirm plans to conduct and manage the Phase I study of Intellia Therapeutic’s NTLA-2001 for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN)

Intellia’s therapeutic NTLA-2001 could be the first curative treatment for ATTR. By applying the company’s in vivo liver knockout technology, NTLA-2001 allows for the possibility of lifelong transthyretin (TTR) protein reduction after a single course of treatment. The investigational therapy is delivered via Intellia’s proprietary non-viral lipid nanoparticle platform, which the company is also using to develop in vivo treatments for other diseases.

Intellia’s President and Chief Executive Officer John Leonard, M.D. stated “Starting our global NTLA-2001 Phase 1 trial for ATTR patients is a major milestone in Intellia’s mission to develop medicines to cure severe and life-threatening diseases,”

The trial’s primary objectives are to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of NTLA-2001. Richmond Pharmacology’s CEO, Jorg Taubel, M.D. stated “Richmond Pharmacology is pleased to be the first clinical research organisation globally to support Intellia Therapeutics with their ground breaking work. We have a long history of conducting Phase I trials with global partners and demonstrable experience in patient studies. We hope this study will help to improve the lives of patients living with ATTR.”

The study which gained MHRA approval this October, will plan to enrol up to 38 patients and is run in collaboration with National Amyloidosis Centre. Julian Gillmore, Professor of Medicine, National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, commented “I am pleased to be leading the Phase 1 clinical trial in the U.K. for NTLA-2001, which I believe to be a breakthrough, single-course, genome editing therapy with the potential to transform the lives of ATTR patients around the world,” he continued commenting on the suitability of the location “The U.K. is a pioneer and leading authority in genetic diagnosis, research and medicine, which is critical when supporting patients with rare diseases like ATTR.”

The global study plans to begin in late 2020, more information about the NTLA-2001 Phase 1 trial may be found on clinicaltrials.gov when available.

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