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

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

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

Researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch applied to the skin. One day, researchers want to use switches of this kind to ...

Aug. 29, 2024 — For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

From life-altering genetic conditions to promising new treatments, here's what you need to know about 7 inherited diseases ...

Qure's AMT-130 shows 80% Huntington's progress reduction, eyeing FDA approval. Click here to read why I rate QURE stock a Buy ...

Experts discuss the challenges and breakthroughs in iPSC-based therapies, including scalability, safety, and regulatory ...

According to research by nova one advisor, the global cell and gene therapy market size was valued at $18.13 billion in 2023 ...

The introduction of anti-VEGF agents has revolutionized the field of medical retina care by reducing rates of vision loss and ...

Rockville’s MaxCyte has made its first acquisition in its more than quarter-century of operation. It’s keeping its eyes out ...