pharmaphorum

Intellia files CRISPR drug after historic phase 3 readout

Intellia Therapeutics has reported the first-ever phase 3 results with an in vivo gene-editing therapy, hereditary angioedema ...

Intellia seeks FDA approval for in vivo CRISPR therapy

The company's treatment for hereditary angioedema reduced attacks by 87% vs. placebo in a Phase 3 trial, clearing the way for ...
Morning Overview on MSN

Compact CRISPR tool boosts in-body gene editing to 90% in lab tests

For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...
Healio

Advanced gene editing ‘promising’ for sickle cell disease, but access challenges remain

Two papers published in The New England Journal of Medicine highlight the potential of gene editing for treatment of sickle ...
The Peninsula Qatar

Sidra Medicine introduces Qatar’s first advanced gene-editing therapy for sickle cell disease and thalassemia

Sidra Medicine, a member of Qatar Foundation, has been qualified by Vertex Pharmaceuticals as one of a of a limited number of ...

Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for Lonvoguran Ziclumeran (lonvo-z) as a One-Time Treatment for Hereditary Angioedema

Intellia also separately announced positive topline data today from the Phase 3 HAELO clinical trial of lonvo-z in HAE. The trial met its primary and all key secondary endpoints, demonstrating that a ...
News Medical

CRISPR-Cas3: A safer gene-editing tool shows promise for transthyretin amyloidosis treatment

Clustered regularly interspaced short palindromic repeats (CRISPR) refer to the small fragments of viral DNA that are stored by the bacteria as a part of their defense mechanism. CRISPR–Cas9 is a ...