Fierce Pharma

NOVEMBER 7, 2023

Vertex and CRISPR Therapeutics are up first with a Dec. | But on Tuesday bluebird said that Vertex’s price tag will not factor into how the Massachusetts company will price its treatment. Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month.

FDA 212

FDA 212

Pharmaceutical Technology

APRIL 13, 2023

Released on April 12, the report focuses on bluebird bio’s lovotibeglogene autotemcel and Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel or exa-cel and their potential use in treating sickle cell disease. If approved, exa-cel would be the first FDA-approved gene therapy based on CRISPR editing.

Healthcare 59

Healthcare Healthcare Pharmaceutical FDA 59

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. This includes the first potential approval of a CRISPR-based gene therapy called exa-cel , which is developed by CRISPR Therapeutics and Vertex Pharmaceuticals. There has been extremely rapid progress.

Food and Drug Administration 59

Food and Drug Administration Engineering Patients Education 59

European Pharmaceutical Review

NOVEMBER 10, 2022

Figure 2 : Super-resolution imaging of CRISPR/Cas9 reporter cell lines enables visualisation of cellular uptake of complex medicines. BT-474 cell line was gene-edited to incorporate a green fluorescent protein (GFP) tag into late endosomes/lysosomes (green). Figure 2 ). During her PhD at St.

Medicine 96

Medicine Distribution Engineering Electronics 96