The FDA has offered positive feedback on possible paths forward for accelerated or traditional approval of FLT201 using data from a single-arm study.

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 ...

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 ...

The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.

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

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

How cells respond to mitochondrial DNA (mtDNA) damage is not fully understood. This study uncovers a mechanism of mtDNA ...

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

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