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Two new gene therapies could present cure for sickle cell disease, but access is an obstacle

High costs and the complexity of administering the drugs may keep them out of reach of patients in need

Canada , Saskatchewan , Manitoba , Saskatoon , United-kingdom , Toronto , Ontario , Calgary , Alberta , United-states , British , Canadians

New CRISPR Gene-Editing Treatment Approved In Europe

The decision comes after Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy was approved in the UK and US for sickle cell disease and beta thalassemia.


Rome , Lazio , Italy , United-kingdom , Franco-locatelli , Drug-administration , European-commission , European-medicines-agency , European-union-on , European-union , Catholic-university-of-the-sacred-heart , Catholic-university

FDA Approves New CRISPR Gene-Editing Treatment

The Food and Drug Administration approved the use of Casgevy, a CRISPR gene-editing therapy, for treating the serious blood disorder transfusion-dependent beta thalassemia—the second major approval for the emerging therapy.

Reshma-kewalramani , Emmanuelle-charpentier , Jennifer-doudna , Vertex-pharmaceuticals , Drug-administration , Nobel-prize , Fda , Casgevy , Crispr-therapeutics , Us- ,

Next-generation genome editing tools surpass CRISPR milestone

Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease (SCD). But science does not stop.

Jennifer-doudna , Emmanuelle-charpentier , Vertex-pharmaceuticals-inc , Crispr , Casgevy , Lyfgenia , Sickle-cell-disease , Spinal-muscular-atrophy , Phenylketonuria , Base-editing , Prime-editing ,

ICYMI: AMCP Nexus Conference Highlights

From the 2023 Academy of Managed Care Pharmacy (AMCP) Nexus meeting, held in October, expert interviews and insightful updates on the Inflation Reduction Act, gene therapy affordability and access, and the 340B Drug Pricing Program comprise the conference highlights.


Florida , United-states , Orlando , American , Aimee-tharaldson , Ben-urick , B-drug-discount-pricing-program , Public-health-services , B-drug-pricing-program , Academy-of-managed-care-pharmacy , Vertex-pharmaceuticals , Managed-care-pharmacy

US FDA approves first CRISPR-based gene therapy Casgevy for sickle cell; Lyfgenia gets nod, too

Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.

United-states , American , Bluebird-bio-inc , Vertex-pharmaceuticals-inc , Both-vertex-pharmaceuticals , Bluebird-bio , Vertex-pharmaceuticals-inc- , Casgevy , Exagamglogene-autotemcel , Exa-cel , Bluebird-bio-inc-