US FDA proposes framework to speed rare disease gene therapy approvals

The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for ...
WISN 12 News on MSN

Rare Disease Day highlights advances in gene therapy

With 300 million people living with rare diseases, experts say new gene and cell therapies are giving families in Wisconsin hope for lasting treatment options.
Fierce Biotech

After gene therapy exit, Pfizer locks in global license for Beam gene editing candidate

After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.
The Public's Radio

The FDA creates a quicker path for gene therapies

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would ...

Gene therapy is transforming lives, but for many Americans it's hard to reach

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

After a baby became the world's first CRISPR gene editing therapy patient, CHOP wants to expand treatment to others

One year after receiving CRISPR gene editing therapy at CHOP, a Delaware County baby is thriving, and doctors want to expand ...

New 'navigator' model aims to speed cell and gene therapies to patients

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 ...