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Gene Therapy to the Rescue

Driving CAR-T Therapy in the Clinic and with CRISPR
CAR-T cell therapy is rapidly becoming a clinically viable option for cancer treatment, and innovations in the field are on the rise, with 6 FDA-approved CAR-T cell therapies currently available. Read More ››
No SMAll Feat
Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder characterized by the loss of motor neurons in the spinal cord and brainstem, leading to progressive muscle weakness and atrophy. Read More ››
Beta Late Than Never
Beta thalassemia is a genetic blood disorder that affects the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. Read More ››
GLAd to Be Treating DMD
Cargo size is key Duchenne Muscular Dystrophy (DMD) is a genetic disease that affects ~1 in every 5,000 people. Read More ››
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