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

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.

Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...

Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

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

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.

Press release Genethon and Eukarÿs announce a strategic partnership to develop a breakthrough technology to reduce the ...

The partners will adapt Eukarÿs' C3P3 engineered enzyme tech to work with adeno-associated virus vectors to boost biomanufacturing yields.

Financial writer recommends Voyager Therapeutics, Inc. for high-risk investors due to promising preclinical Alzheimer's ...

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.