The FDA granted orphan drug designation to Sineugene Therapeutics' SNUG01, a first-in-class experimental gene therapy for ...

The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.

A recent study involving researchers from the University of Basel reveals that slowing down the intracellular transport of ...

Gabriella Ngang, of Farmington Hills, is one of three in Michigan to take part in a clinical trial of a new sickle cell ...

Using an AI-driven drug discovery process, the drug known as vorinostat was identified as a promising treatment for Rett ...

DCC gives nod to amend Rules related to licensing of stem cell derived & gene therapy products: Gireesh Babu, New Delhi Monday, July 7, 2025, 08:00 Hrs [IST] The Drugs Consultativ ...

The newly licensed drug, leniolisib, is the first targeted treatment for her condition, Activated PI3-kinase Delta Syndrome ...

Scientists discovered a cancer-fighting compound in mold from ancient tombs, revealing a powerful new drug against leukemia.

Approved eight months ago for a certain class of patients with both hemophilia A or B, Pfizer’s Hympavzi has now shown its ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Equipped with a new CEO, Vor Bio is transitioning into an autoimmune player, leaving cell and gene therapy behind.

Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with genetic diseases?