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teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

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 ...

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

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

As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm behind its approved Duchenne muscular dystrophy (DMD) treatment. The FDA, for ...

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 ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

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 ...

Venture capital investment in gene therapies fell to $1.4 billion in 2024 from 2021 peak of $8.2 billion Total global biopharma venture funding rose to $27 billion in 2024 from $23.2 billion in 2023 ...

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 ...