The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current ...
As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” ...
IDT played a pivotal role in manufacturing the personalized gene editing therapy given to baby KJ Muldoon to treat his rare ...
Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale ...
More than a year after receiving the first personalized CRISPR therapy, Baby KJ is thriving, offering proof-of-concept for ...
While researchers have long appreciated the enormous potential of genome editing to treat disease, applications of these technologies in humans have historically been limited by safety concerns. Even ...
A phase 3 trial of Intellia Therapeutics’ in vivo gene-editing therapy lonvoguran ziclumeran (lonvo-z) has hit its primary ...
A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelial cells to achieve therapeutic correction. Gene editing is a method to permanently ...
In late April 2026, analysts highlighted expectations for higher year-over-year earnings and revenue at CRISPR Therapeutics ahead of its March-quarter report, alongside strong launch metrics for ...
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