Rbd-638

Key attributes:

RBD-638 is a type of gene therapy that utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the SMN1 gene to patients' cells. The SMN1 gene is responsible for producing a protein essential for the survival of motor neurons, which are responsible for controlling voluntary movements. Individuals with spinal muscular atrophy (SMA), a debilitating genetic disorder, often possess one or more mutated copies of the SMN1 gene, leading to a reduction in the production of this vital protein. rbd-638