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

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

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 ...

The California Institute for Regenerative Medicine (CIRM) has approved a new funding program to accelerate therapies for rare diseases, which sets aside $100 million over two years to create a ...

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 ...

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 ...

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

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 ...

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 ...

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, ...