A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...

Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...

Gene therapy (introducing genetic material into living cells to fix, replace, enhance, or block a faulty gene) is rapidly gaining traction as a strategy for the treatment of genetic diseases. The ...

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

When Genenta Science listed on the Nasdaq at the tail end of 2021, the Milan-based biotech was squarely focused on its pipeline of cell-based gene therapies. | Four years after listing on the Nasdaq, ...

This sponsored article was provided by a partner and is not editorial content from Los Angeles Magazine or the Engine Vision Media Network. Every cell in your body holds a unique genetic code within ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...

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 ...

A research team at the University of California San Diego has discovered a novel and promising method of treating arrhythmogenic cardiomyopathy (ACM), a rare inherited heart disease that can strike ...

Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...