A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

CINCINNATI ( Cincinnati Business Courier) - Backed by big money and aggressive plans to manufacture cutting-edge gene and cell therapies, a biomanufacturer made big promises about a revolution in its ...

Spacecraft unveils 1st-ever images of sun's south pole: 'Beginning of a new era' ...

The FDA’s accelerated approval pathway plays a vital role, and scrapping it or reversing course on already-approved therapies ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

According to a new study in Haemophilia, up to 60% of patients with severe HA have no known family history, making early ...

A combination therapy that adds a recently approved drug to the current standard of care for newly diagnosed acute myeloid ...

Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...

The benefits of drug warranties extend beyond risk management. They can also serve as market differentiators in crowded ...

CRISPR-Cas13, a powerful RNA-targeting technology is gaining increasing attention as a next-generation gene therapy platform due to its precision and reduced side effects.