CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

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

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

Becoming a qualified treatment center can take six to 18 months, according to Mahmoudjafari. Some institutions begin ...

For most of his 20 years, a New Jersey man knew pain as a daily reality. Now, after a one-time CRISPR gene-editing treatment at Children's Hospital of Philadelphia, Austin Louis says he is finally ...

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

As cell and gene therapies move into more hospitals and cancer centers, health system leaders are learning that clinical success is only part of the equation. These treatments may offer life-changing ...

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and ...

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...

Since scientists first discovered that human immune cells could be modified to become cancer-fighting agents, they've been trying to engineer a cell that's effective against solid tumors, which ...