Gene Therapy to the Rescue

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 ››

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 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 ››

Cargo size is key Duchenne Muscular Dystrophy (DMD) is a genetic disease that affects ~1 in every 5,000 people. Read More ››