Here in New Zealand, clinicians and advocates are pushing our country’s government to update its genetic editing and modification legislation. Historically, New Zealand has maintained some of the strictest gene technology regulations globally. Currently, genetic modification is heavily regulated under the Hazardous Substances and New Organisms Act…
A single infusion of YOLT-201, Yoltech Therapeutics’ investigational gene-editing therapy, appears to be safe and to effectively reduce blood levels of the disease-driving TTR protein in people with familial amyloid polyneuropathy (FAP). That’s according to preliminary data from six FAP patients given the therapy in the initial,…
One year after a single dose of Intellia Therapeutics’ experimental gene-editing therapy NTLA-2001, blood levels of the disease-driving transthyretin (TTR) protein dropped by a mean of 91% in people with familial amyloid polyneuropathy (FAP). Available data at two years showed sustained TTR level reductions, which were accompanied…
The first patient has been dosed in a China-based Phase 1/2a clinical trial testing YOLT-201, Yoltech Therapeutics’ one-time gene-editing therapy, in people with hereditary transthyretin amyloidosis (hATTR), a group of diseases that includes familial amyloid polyneuropathy (FAP). Two weeks after receiving the investigational therapy, which was given as…
Regulatory authorities in China have authorized YolTech Therapeutics to launch a Phase 1 clinical trial testing YOLT-201, a one-time gene-editing therapy, in people with hereditary transthyretin (hATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The approval from the National Medical Products Administration’s Center for…
A single infusion of NTLA-2001, an experimental gene-editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). These updated, interim data cover 65 of 72 patients…
Intellia Therapeutics is planning to launch pivotal clinical trials of its gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). A pivotal trial is one where the data are expected to support an application for a therapy’s regulatory approval. Intellia is…
One-time treatment with the experimental gene-editing therapy NTLA-2001 led to substantial reductions in the levels of toxic transthyretin protein that were sustained for several months in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). That is according to data shared by NTLA-2001’s co-developer, Intellia Therapeutics, at the American Heart Association…
A one-time treatment with NTLA-2001, Intellia Therapeutics’ experimental gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other types of ATTR amyloidosis, continued to demonstrate reduced levels of the disease-driving transthyretin (TTR) protein for at least six months, new data show. “Based on the interim data shared today,…
The first patient has been dosed in the second part of a Phase 1 trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis. The first part of the study exposed patients to single ascending doses of…