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

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

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

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

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

Specialists selected patients from overseas for the initial procedures, as the condition is so rare. | ITV National News ...

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

Jace’s mum DJ, 35, told the PA news agency: “Pre-surgery, at around two years old, you could have held up any object, even a ...

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 ...

Regenerative Medicine Advanced Therapy (RMAT) designation follows Phase Ib 36-month data Study demonstrated favorable safety profile and continued positive trends in assessed clinical outcome measures ...

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 ...

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...