Penn and CHOP will test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...

CRISPR therapy setback: Intellia pauses Phase 3 ATTR trials as patient develops serious liver reaction to nex-z

Intellia Therapeutics has paused dosing in its Phase 3 MAGNITUDE trials after a patient experienced severe liver toxicity, as the company investigates and works with regulators on its CRISPR-based ...
The Motley Fool

Where Will CRISPR Therapeutics Be in 5 Years?

CRISPR Therapeutics now has an approved product in its portfolio in Casgevy. The treatment has the potential to be a game changer for the company's business. Even with more treatments in its portfolio ...
STAT

Biotech investors: Stop expecting CRISPR science to make big leaps

Banas is associate director of research at the Gene Editing Institute at ChristianaCare, where Kmiec is founder and executive director. The medical promise of CRISPR gene editing can be seen most ...

Intellia Therapeutics Provides Update on MAGNITUDE Clinical Trials of Nexiguran Ziclumeran (nex-z)

Conference call today at 8:30 a.m. ETCAMBRIDGE, Mass., Oct. 27, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on ...

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...