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 glimpses of forward momentum as Vertex and Genetix Bio provide updates.

Hospitals and cancer centers that are launching cell and gene therapy programs need more than scientific breakthroughs to succeed. Delivering these therapies safely and efficientl ...

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

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

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teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

A new, highly sensitive cell therapy was able to wipe out three different kinds of solid tumors in mice, an important advance in an area where CAR-T cells have long struggled. But significant ...

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

Cell and gene therapies , or CGT , have come a long way since they were first introduced. In the last few decades, both cell therapy -- the transplantation of living cells -- and gene therapy -- the ...

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and clinicians in Lund have now presented a new model for cooperation that will ...

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