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 ...

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 ...

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 United Kingdom say they have successfully trialed what could become the world's first gene therapy for Huntington's disease – a fatal neurodegenerative disorder that is typically ...

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

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 ...

Sales of Casgevy, a gene-edited treatment developed with CRISPR Therapeutics, more than tripled compared to the third quarter ...

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

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

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 ...