pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

DECEMBER 20, 2023

In 2023, Dr. Varshney notes that the US Food and Drug Administration (FDA) issued a request for industry comment on the integration of AI in drug and biologics development. CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119