Vertex Pharmaceuticals demonstrates resilience with robust CF franchise, new approvals, and diversification efforts. Read why ...

CRISPR Therapeutics AG ( NASDAQ: CRSP) TD Cowen 45th Annual Healthcare Conference March 3, 2025 10:30 AM ET ...

Biotech companies can skyrocket in a short period because of excellent clinical or regulatory progress. And if we take the ...

The FDA approval and launch of CASGEVY, a gene therapy for treating sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older, have created ...

But the voucher was never granted by the U.S. regulator despite providing one for Vertex and CRISPR Therapeutics’ development of their sickle cell disease gene therapy Casgevy. With the pricing ...

Second, CF isn't Vertex's only growth opportunity. The company is still in the early stages of the commercial launch of Casgevy, a gene-editing therapy that is a one-time treatment for sickle cell ...

What Casgevy does is turn a patient’s own cells, already a perfect match, into a transplant. Doctors harvest stem cells from the patient’s bone marrow and then send them off to a lab ...

Especially for therapies, hurdles remain. One is cost. Casgevy, the CRISPR cure for sickle-cell disease and beta-thalassemia, is expensive to make and to buy. Health-care systems in America and ...

In a closely competitive race, the therapy secured FDA approval ahead of time in December 2023 on the same day as Vertex/CRISPR’s Casgevy (exagamglogene autotemcel), approved for the same indication.

In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long endeavor reached fruition in the form of a new treatment, CASGEVY, approved by ...

The FDA approval and launch of CASGEVY, a gene therapy for treating sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older, have created ...