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 ...
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.

FDA proposes framework to speed rare disease gene therapy approvals

The plan could provide patients earlier access to treatments that would otherwise stall because only limited data can be gathered.
KLCC

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 ...
WUFT

Where patients live matters for access to gene therapy

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 ...
The Lancet

Impact of adding hormone therapy to postoperative radiotherapy in prostate cancer

The addition of hormone therapy to radiotherapy improves overall survival in men with high-risk prostate cancer, over radiotherapy alone.1 Mechanisms proposed to explain this effect include ...