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

With the FDA expecting to approve 10–20 Cell and Gene Therapies (CGTs) annually by 2025, it is evident that the field will continue to hold immense commercial and medical opportunities. To harness ...

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

Andelyn Biosciences has been selected by Drake Rayden Foundation to apply the AAV Curator Platform to manufacture clinical grade AAV for the Treatment of Nonketotic Hyperglycinemia (NKH).

Ori Biotech has lassoed a significant milestone for its space- and cost-efficient cell and gene therapy manufacturing platform. The FDA has granted the company’s IRO platform an Advanced Manufacturing ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

This story is the first in an occasional series following Louisiana’s first patients to receive gene therapy for sickle cell disease. On the third floor of Manning Family Children’s Hospital in New ...

What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...

Forbes contributors publish independent expert analyses and insights. Bruce Japsen writes about healthcare business and policy. More than 70% of employers and health plans expect affordability of gene ...

The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.

Just before the Covid-19 pandemic, it seemed gene therapies were a few years away from curing all sorts of diseases with a quick, simple infusion. The idea was that drugmakers could stuff some genetic ...