Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

Will consider data that includes "the experience of individuals," FDA commissioner says ...

Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

Durable national strategy will require alignment across five domains: manufacturer negotiations, provider networks, clinical criteria, financing mechanisms, and data infrastructure. No single public ...

Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Explore how community blood centers are shaping the future of cell and gene therapies, from customized collections to ...