news & meetings - latest announcements & happenings | oneAMYLOIDOSISvoice
CRISPR Gene Editing Reduces Disease-Causing Protein In Hereditary Transthyretin Amyloidosis
Clinical Trials and Research

CRISPR Gene Editing Reduces Disease-Causing Protein in Hereditary Transthyretin Amyloidosis

The results of the phase 1 study open-label trial showed the therapy was safe, raising the possibility of a more effective and more appealing treatment for the progressive and fatal disease, in which amyloid builds up in the body's organs and tissues, where it can cause loss of sensation, heart problems, or other symptoms. Life expectancy after diagnosis is about seven to 12 years. Current treatment involves long-term therapy to stabilize the protein and slow the formation of amyloid or inhibit TTR protein production by degrading its mRNA, thereby slowing the formation of amyloid.

The new therapy, called NTLA-2001, was given once to six patients at 0.1 mg/kg to half and 0.3 mg/kg to the other half. After 28 days, the concentration of TTR protein in the serum was reduced by 52 percent in those receiving the lower dose and by 87 percent in those receiving the higher dose.


Oct 7, 2021
Alnylam Reports Positive Topline 18-Month Results From HELIOS-A Phase 3 Study Of Vutrisiran In Patients With HATTR Amyloidosis With Polyneuropathy
Clinical Trials and Research

Alnylam Reports Positive Topline 18-Month Results From HELIOS-A Phase 3 Study of Vutrisiran in Patients With hATTR Amyloidosis With Polyneuropathy

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, met all secondary endpoints measured at 18 months, including statistically significant improvements in neuropathy as measured by the modified Neuropathy Impairment Score (mNIS+7), quality of life (QOL), gait speed, nutritional status and overall disability, relative to external placebo data from the APOLLO Phase 3 study of patisiran.
The final secondary endpoint, reduction in serum TTR levels with vutrisiran, demonstrated non-inferiority relative to the within-study patisiran arm, as expected. In addition, patients treated with vutrisiran showed improvements in exploratory endpoints, including the biomarker NT-proBNP and certain echocardiographic parameters, relative to placebo, and an improvement in technetium uptake relative to baseline in a majority of patients in a planned cohort, providing potential evidence for reduced cardiac amyloid burden.
Oct 27, 2021

To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences.
More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.