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 ...
Bulletin of the Atomic Scientists

CRISPR therapies can treat disease but cost millions. An equity-based approach could bring them to more people

For some diseases, gene therapies offer the potential for lifelong disease amelioration and even cure. And these immensely important novel biotechnologies may be on the cusp of a boom. That is in part ...

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 ...
Purdue University

Blueprints for a molecular machine more powerful than CRISPR

WEST LAFAYETTE, Ind. — More than a decade ago, scientists harnessed a bacterial molecular machine that identifies and cuts specific sections of DNA, revolutionizing the ability to edit genes and ...

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 ...