A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene therapy for severe hemophilia A.

Cell and gene therapies , or CGT , have come a long way since they were first introduced. In the last few decades, both cell therapy -- the transplantation of living cells -- and gene therapy -- the ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

Get a closer look at the biotech ophthalmology landscape to discover exactly how companies are approaching the treatment of eye diseases.

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For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology. It uses electronics to step in as a replacement for the biological ear that ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

A patient has died in a clinical trial evaluating a Rocket Pharmaceuticals gene therapy shortly after complications emerged that led the FDA to pause the study. An inquiry into the fatality is ...

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...