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 ...
Science Daily

New CRISPR tool enables more seamless gene editing -- and improved disease modeling

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

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 ...

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 ...