Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

This 2-part first in human (FIH) study consists of an open-label, single ascending dose Part 1, which may identify the optimal biologically active dose (OBD)

The NEURO-TTRansform Study

The NEURO-TTRansform Study is a Phase 3 clinical trial of an investigational antisense drug called AKCEA-TTR-LRx for people living with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). If you

Gene Editing Technology For Hereditary TTR Amyloidosis (hATTR) In New Zealand

What is hATTR Amyloidosis or FAP? Familial Amyloid Polyneuropathy (FAP) is a rare inherited disease caused by a specific mutation in your  own DNA in a gene which codes for the Transthyretin protein (TTR). If you have this mutation, the TTR  protein becomes sticky and forms clumps of amyloid within your small blood vessels and over many  years this will damage your nerves (causing numbness and weakness) and heart (causing heart  failure).   Inheritance Traits These symptoms do not appear until a person is an adult. FAP is passed on between generations in an autosomal dominant pattern – which means if someone has the disease then half of his/her brothers and sisters will have the disease and half of his/her children will also develop the disease in the future. There are an estimated 50,000 patients with FAP worldwide and 100 in New Zealand. Present Treatment in NZ Up until now the only effective treatment for FAP has been liver transplantation, which replaces the liver which makes the abnormal TTR with one which does not. Unfortunately liver transplantation is very limited and to-date only 9 New Zealanders with FAP have been transplanted. CRISPR/Cas9

CRISPR/Cas9 Genome Editing Technology

HOW CRISPR/ Cas9 Works Video “CRISPR/Cas9 genome editing can make permanent, precisely targeted changes in patients’ chromosomes and repair the underlying genetic mutation, whereas more traditional

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