Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

Miriya Nurse, 16, spent months in the hospital undergoing a groundbreaking treatment that cured her rare blood disorder.

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...

FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.

Findings from two clinical trials suggest Novartis' Itvisma is safe and effective for SMA patients over two years old.

Gene therapy is now being used for certain cancers and sickle cell anemia.A South Florida teen was among the first to get ...

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...