CRISPR-Cas9 in-vivo gene editing shows reduction in TTR after a single dose

The study investigated the safety and pharmacodynamics of NTLA-2001, the first-ever systemically administered in vivo CRISPR therapy candidate. The study which Richmond Pharmacology is conducting alongside The Royal Free has the potential to be the first curative treatment for ATTR.

Transthyretin amyloidosis, or ATTR, is a rare, progressive and fatal disease. Hereditary ATTR (hATTR) amyloidosis occurs when a person is born with DNA mutations in the TTR gene, which causes the liver to produce transthyretin (TTR) protein in a misfolded form and build up in the body.

The interim data from a group of six patients demonstrated a reduction in serum TTR protein concentrations and only mild adverse events. Evaluating ongoing dose-escalation of NTLA-2001 in patients living with the condition and will provide an early insight into safety aspects of the drug.

A research participant commented on the study "Since being introduced to Richmond Pharmacology through the Amyloidosis centre it has been a great experience. From reception up to the hospital wards everyone has been fantastic.”

Research Volunteer, on the NTLA-2001 study

CRISPR/Cas9 is a specific, efficient and adaptable gene-editing technology that can harness, modify, delete or correct precise regions of our DNA "We are pleased to be supporting Julian Gillmore of the National Amyloidosis Centre to conduct the first-in-human studies for this first gene editing treatment of TTR amyloidosis making the UK the first site to administer the therapy in this global program. The initial results are as predicted by Intellia. The prospect of a complete cure from one single intervention heralds a new era of medicine. We are excited to be part of this journey."‍

Dr Jorg Taubel, CEO, Richmond Pharmacology 

Read the supporting publication in The New England Journal of Medicine: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Learn more about CRISPR technology 

Learn more about 2021 Peripheral Nerve Society Annual Meeting

Presentation Details

Title: “In vivo CRISPR/Cas9 Editing of the TTR Gene by NTLA-2001 in Patients with Transthyretin Amyloidosis”

Session: Platform Session II

Date and Time: Saturday, June 26, 2021 from 11:15-11:30 a.m. E.T.

Presenter: Dr. Julian Gillmore, M.D, Ph.D., FRCP, FRCPath, Professor of Medicine, National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, U.K., the trial’s national coordinating investigator