Health systems learn why cell and gene therapy success depends on workflows, staffing, and finances—not just science—and how to build them.

A cell-specific gene therapy has been shown to silence pain encoding neurons in mice, offering a potential alternative to ...

For most of his 20 years, a New Jersey man knew pain as a daily reality. Now, after a one-time CRISPR gene-editing treatment at Children's Hospital of Philadelphia, Austin Louis says he is finally ...

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

What you need to know: Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease A biotechnology company in Uganda has developed a new ...

Recent therapies like hydroxyurea (Droxia, Bristol-Myers Squibb), the first FDA-approved drug for SCD in 1994, increase fetal hemoglobin (HbF), which, through unknown pathways, improves SCD symptoms ...

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 ...

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene ...

One of the biggest challenges to the cell and gene therapy (CGT) landscape over the last couple of years has been the reduction in venture financing from $35.9bn in 2021 to $23.3bn in 2024 (GlobalData ...

Sickle cell anemia (HbSS) is the homozygous dominant variant and the most common and severe form of the disease. Whereas patients who only inherited one gene encoding for abnormal HbS and a different ...

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