The agency's decision is based on results from a clinical trial in which the treatment improved hearing in 80 percent of the ...

Intellia Therapeutics, a leading biotechnology company, announced that its CRISPR-based treatment for a rare swelling ...

Astellas Pharma has done some spring cleaning of its pipeline, dropping two phase 1 candidates and slamming the brakes on a ...

MPR spoke with Dr Daniela Carvalho, an investigator in the CHORD trial, the results form which formed the basis for the approval of Otarmeni.

Eli Lilly’s latest attempt to strengthen its genetic medicine offering has seen the Big Pharma team up with Profluent to ...

The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical ...

In a historic leap for medical science, the U.S. Food and Drug Administration (FDA) has greenlit the first-ever gene therapy ...

In vitro data demonstratedself-complementary AAV9 (scAAV9) enabled ~30-fold higher MeCP2 protein expression compared to single-stranded AAV9 ...

Topline results from the pivotal HAELO trial demonstrate that a single infusion of lonvo-z significantly reduces HAE attacks and the need for chronic prophylactic therapy.

Gene editing has emerged as a powerful approach for targeting the genetic causes of disease, but getting the editing ...

Researchers say a gene therapy allowed deaf children and adults as old as 32 to hear for the first time. The benefits have ...