Genome Editing/Gene Editing

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

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

With CRISPR Gene Editing, Unique Treatments Begin to Take off for Rare Diseases

In February last year, Doherty - now about age 65 - began to experience the same early breathing symptoms his father had had. As an avid hiker who has trekked the…

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

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics leveraging CRISPR-based…

He Inherited A Devastating Disease. A CRISPR Gene-Editing Breakthrough Stopped It

Patrick Doherty had always been very active. He trekked the Himalayas and hiked trails in Spain. But about a year and a half ago, he noticed pins and needles in his…

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Expert and Community Perspectives on Attruby®

In this special session, Dr. Barry Trachtenberg and members of the ATTR amyloidosis community came together to share medical insights and lived experiences related to Attruby™ (acoramidis). This expert-led discussion explores the clinical data, the patient perspective, and what lies ahead for those considering or currently taking Attruby.