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

causing heart cells and proteins to become better organized. The research team is working with pharma company TikkunLev Therapeutics to prepare the gene therapy for use in humans. They intend to ...

Scientists looked at multiple techniques used to measure ... standardization. In gene therapy, a person's faulty genes are either replaced or modified to treat or prevent disease.

Dyno Therapeutics announced that Roche has exercised its option to license a novel capsid for use in a gene therapy program for an undisclosed neurological disease. The capsid license follows an ...

AMT-130 consists of an adeno-associated virus (AAV) vector carrying ... Vertex's gene-editing therapy for sickle cell disease, Casgevy, has been given the nod for use by the NHS in England after ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

Astellas paves backloaded biobucks deal to test 4DMT's eye disease gene therapy vector The reorganization ... Those funds will be used mostly to fuel pre-commercialization activities and collect ...

has exercised its option to license a novel capsid for use in a gene therapy program for an undisclosed neurological disease indication. The capsid license is pursuant to the agreement between ...