Genetic engineering in non-human primates has long been limited by the need for virus-based gene delivery methods. Recently, ...

This infographic explores the role of gene therapy in treating haemophilia, focusing specifically on haemophilia A and B. It ...

The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...

The goal of genome editing technology is to develop strategies for the correction of those mutations that are responsible of ...

It's early days for xenotransplantation, but eGenesis, Eledon, United Therapeutics and more are working to develop solutions ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. Roche described the restructuring in ...

“This is the beginning of a new era of medicine,” said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania’s Perelman School of Medicine. He added that the method ...

Protein therapies for the eye must be dosed as frequent injections. Gene therapy offers one-time treatment, but must also be administered by injection. Neurotech Pharmaceuticals’ gene therapy ...