Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

Spark Therapeutics’ and Novartis Luxturna gene therapy for a rare inherited eye disease has been okayed by European regulators, paving the way for an EU licence in the coming months. A marketing ...

He said the company was ‘locked and loaded’ to start manufacturing the complex gene therapy product, but was a little bit more circumspect about whether Luxturna could make money.

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

The lab of The Wistar Institute's Jessie Villanueva, Ph.D., has identified a new strategy for attacking treatment-resistant melanoma: inhibiting the gene S6K2. .

NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE), a $4.08 billion market cap biotechnology company with 27% revenue growth in the past year, today unveiled new data indicating ...

It seeks to understand the process of trait inheritance from parents to offspring, including the molecular structure and function of genes, gene behaviour in the context of a cell or organism (e.g ...