Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic ...

Many Indians laid low by disease have turned to biohacking. Others are healthy but want to live longer. Aiding them in their ...

Cell and gene therapy tout promising results, but manufacturing challenges around personalized treatments require industry ...

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their ...

The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through innovative gene editing approaches.

A new inhalable gene therapy for cystic fibrosis (CF) has entered Phase I clinical trials, a major milestone on the journey ...

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe.

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 ...

Researchers developed a new gene-editing strategy that dramatically boosts the effectiveness of gene therapies in the liver, a breakthrough that could lead to new treatments for about 700 genetic ...