FDA Approval, Genetic Disease, In-Vivo and Life Science

Search:

DAY

WEEK

MONTH

YEAR

Select your country:
Sign up | Log in

FDA Approval

Genetic Disease

In-Vivo

Life Science

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

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

Gene Therapy FDA Approval Gene In-Vivo

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Clinical Research Informer

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Stay Connected