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 ...

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 Globe and Mail

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

CAMBRIDGE, Mass., Oct. 27, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based ...