The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

According to research by nova one advisor, the global cell and gene therapy market size was valued at $18.13 billion in 2023 ...

A groundbreaking sickle cell disease treatment has been approved for use in the UK's National Health Service (NHS). The treatment offers patients with severe forms of the condition hope for a ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Sangamo (SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate ST-920 in the treatment of Fabry disease. Read more here.

As part of The Wistar Institute's Melanoma Research Center, the Villanueva lab pursues new ways to overcome the challenge of melanoma drug resistance. In this paper, the group took aim at melanomas ...

Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy ...

The American Cancer Society estimates that 83,000 new cases of bladder cancer will be diagnosed this year, with nearly 17,000 ...

The lab of The Wistar Institute's Jessie Villanueva, Ph.D., has identified a new strategy for attacking treatment-resistant melanoma: inhibiting the gene S6K2. .

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing ...

In honor of Rare Disease Day on February 28, 2025, Morgan Lewis is publishing a series of posts on As Prescribed and Health ...