A groundbreaking new approach in cancer treatment has emerged, utilizing light to disable the mitochondria, the energy-producing...

Hypophosphatasia (HPP), or "soft bone disease" is a rare genetic disorder that impacts bone and tooth development, leading to weak bones...

Several clinical trials in 2025 are pushing the boundaries of gene and cell therapy, aiming to address a range of complex diseases. Below...

Novartis's gene therapy, Zolgensma, is already approved in the U.S. for children under the age of two with spinal muscular atrophy (SMA),...

A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide...

As CRISPR-based therapies edge closer to becoming mainstream, groundbreaking treatments for blood disorders and cancer are making their...

Sickle cell disease (SCD), which affects over 100,000 people in the U.S., is about to see a major shift in treatment accessibility. With...

Gene therapy has shown promise in treating many genetic diseases, but some conditions, like muscular dystrophy, involve very large genes...

Imagine controlling genes with the precision of a light switch— controlling specific parts of the body as you will. That’s what the...

A recent 6-year checkup on boys treated with gene therapy for cerebral adrenoleukodystrophy (CALD) reveals lasting benefits for most...

In exciting news for Parkinson’s disease research, MeiraGTx has announced positive results from a Phase 2 clinical bridging study of...

Imagine switching brain circuits on and off using only magnetic fields. Researchers have just developed a cutting-edge gene therapy that...

A team from the Broad Institute of MIT and Harvard, along with Harvard University and the Howard Hughes Medical Institute, has made...

The fight against diabetes is being revolutionized with an innovative approach from Genprex, with its gene therapy candidate, GPX-002....

On August 2nd, 2024, the FDA granted accelerated approval for afami-cel -- the first gene-engineered T-cell Therapy for advanced...

In a project funded by the National Institute of Mental Health (NIMH) and the National Institute of Neurological Disorders and Stroke...

On June 20th, 2024, the FDA expanded its approval of ELEVIDYS , an innovative AAV-based gene therapy developed by Sarepta Therapeutics ,...

Gene therapy is transforming the landscape of treatment options, and the recent FDA approval of BEQVEZ marks a significant milestone for...

New Approach for Liver Cancer Treatment and Possible Prevention: Gene Therapy Targets Biomarker for Hepatocellular Carcinoma (HCC)...