A gene-editing therapy for sickle cell disease, with a price tag of £1.65m, is to be offered to patients on the NHS in ...

The lab of The Wistar Institute's Jessie Villanueva, Ph.D., has identified a new strategy for attacking treatment-resistant ...

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 ...

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

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.

A new gene editing treatment has been approved for use in the NHS, which will help treat sickle cell disease patients ...

In 2019, the first CRISPR clinical trials were conducted to treat sickle cell diseases and in 2020, this treatment was ...

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...

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 ...