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People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually builds up in the heart and nerves. Symptoms include numbness in the hands and feet, loss of control of the bowel and bladder and immobility. The condition gets progressively worse and is …

Gene Editing Therapy Paves Way for Revolution in Treatment of Genetic Disorders Read More »

The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an enormous hurdle: getting the molecular scissors into the body and having it slice DNA in the tissues where it’s needed. Now, in a medical first, researchers have injected a CRISPR …

CRISPR Injected Into the Blood Treats a Genetic Disease for First Time Read More »

Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and efficacy of in vivo CRISPR genome editing in humans. The team of researchers based in the U.S., the U.K., and New Zealand showed in a study that the companies’ lead in vivo genome editing candidate …

New Era of Medicine: Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans Read More »

Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene editing can be deployed directly into the body to treat disease. The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components — in this case targeting a protein that is made mainly in the liver …

Landmark CRISPR Trial Shows Promise Against Deadly Disease Read More »

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 …

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

Cardiac amyloidosis is not nearly as rare as one might think, and with effective treatments finally emerging in recent years, there’s a need to increase awareness of the condition to allow for earlier diagnosis and better patient outcomes. So say the editors behind an amyloidosis-focused special issue of JACC: CardioOncology, published Tuesday, and other researchers …

As Amyloidosis Field Explodes, Physicians Need to Spot and Treat Read More »

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 …

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

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics leveraging CRISPR-based technologies, today announced that the company is scheduled to present virtually at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022 at 2:15 p.m. ET. A live audio webcast of Intellia’s presentation can be accessed …

Intellia Therapeutics to Present at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022 Read More »

Intellia Therapeutics, Inc. and Regeneron Pharmaceuticals, Inc. announced positive interim data from an ongoing Phase 1 clinical study of their lead in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. The interim data released today include 15 hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) patients treated across …

Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-Causing Protein Read More »

The first team to disable a disease gene directly in a person through an infusion of the genome editor CRISPR reported yesterday that levels of the toxic protein made by the gene dropped as much as 93% for up to 1 year. The researchers hope the long-term reduction means patients in the clinical trial will …

First Gene-Editing Treatment Injected Into the Blood Reduces Toxic Protein for up to 1 Year Read More »