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).

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 ...

With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...

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 ...

Despite groundbreaking 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 ...

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 ...

UAE patient with heart failure, thalassemia treated with gene therapy after transplant ...

Groundbreaking advancements in gene therapy at UTMB represent new possibilities for patients living with rare genetic conditions.

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...

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 ...