AVXS
gene therapy, unico candidato en ph1, el AVXS-101
AVXS-101: granted Orphan Drug Designation for the treatment of all types of spinal muscular atrophy (SMA) and Breakthrough Therapy Designation, as well as Fast Track Designation for the treatment of SMA Type 1 (type the co. has set focus on). A one-time, intravenous treatment. Gene therapy should do good against SMA due to the monogenic nature of the disease—meaning it's caused by the deletion of or mutations in a single gene.
y sobre la SMA: severe neuromuscular disease caused by a genetic defect in the SMN1 gene—leading to the loss of motor neurons and resulting in progressive muscle weakness and paralysis. SMA is divided into sub-categories—SMA Types 1, 2, 3, and 4—based on disease onset and severity.
60% of SMA patients have Type 1, no FDA o EMA approved treatment exists.
la comentamos con igonber. hay competencia bastante avanzada, IONS y BII tenían una ph3 superada para la misma indicación y estaban por iniciar trámites para comercialización. pero si no recuerdo mal se comentó que igualmente AVXS tenía potencial. pero igonber es quién sabe jeje, yo me la dejé de lado por el momento.
edito: tu edit llegó tarde jeje