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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 ,

Hope for patients as new sickle cell therapy is approved


Casgevy and Lyfgenia are now available for sickle cell patients aged 12 years and above.

Casgevy- , Ickle-cell , Lyfgenia , Us-food-and-drug-administration- , White-blood-cells , Ed-blood-cells , Haemoglobin ,

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-