Scientists have developed a method to reprogram cancer-fighting immune cells directly inside the body, potentially eliminating the need for complex lab-based manufacturing. Early results show rapid ...

Etranacogene dezaparvovec (Hemgenix), the only commercially available gene therapy for hemophilia B, is temporarily ...

Scientists at the University of Science and Technology of China have identified a signaling chain that starts with gut bacteria, triggers serotonin release from intestinal cells, and ends with liver ...

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

The firm noted that they find the company to be an upcoming leader in gene therapy for blinding ocular disorders.

The 23-year-old man received a gene therapy transfusion Wednesday at Manning Family Children's Hospital that doctors say should cure his sickle cell disease. It’s a condition he has lived with his ...

A new study has for the first time elucidated the gut-liver immune regulatory axis jointly maintained by intestinal commensal bacteria and the intestinal endocrine system, and uncovered the ...

A New Berlin mother is hoping the FDA approves a new drug from Denali to save her kindergarten-aged son from a fatal disease called Hunter Syndrome.

Mike Francisco was an athletic 35-year-old Phoenix medical professional when he was diagnosed with late-stage colon cancer. His story is not unusual.

A breakthrough in CAR-T therapy may allow cancer treatment with a single injection, cutting costs and making care more available.

In a letter to European patients, Roche said its decision to end development of “emugrobart” was based on the drug “not consistently” improving muscle growth and motor function in a key study.

The new Andelyn Foundation plans to financially support one patient- or family-founded rare disease charity every year.