When even one tiny section of a person’s genetic code becomes mutated or modified, cells can begin to act out of ...

Gene therapy is an area of intensive research and development in the retina space, with promising prospects and significant challenges. The benefits and pitfalls of gene therapy were discussed by two ...

For decades, treatment options for rare diseases have been limited, often focusing on symptom management rather than ...

Qure's AMT-130 receives FDA Breakthrough Therapy designation for Huntington's disease, adding to Fast Track, RMAT, and Orphan ...

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

AMT-130 is administered through magnetic resonance imaging-guided, convection-enhanced stereotactic neurosurgical delivery into the striatum.

Additionally, improving CAR-T biological efficiency could reduce dosing needs, further cutting costs and expanding access.

Lexeo Therapeutics’ gene therapy provides a healthy version of the frataxin gene, which is critical for energy production in ...