onasemnogene abeparvovec-xio (Zolgensma)

Indications

• gene therapy medication used to treat spinal muscular atrophy

Dosage

• administered intravenously or intrathecally Mechanism:
• biologic drug consisting of AAV9 virus capsids deprived original viral DNA, instead contain a SMN1 transgene along with promoters
• the SMN1 transgene is delivered to neuronal cell nuclei by the AAV9 viral vector
• transcription of SMN1 gene begins with translation of resultant mRNA to produce a functional SMN protein
• the procedure addresses the root cause of spinal muscular atrophy, lack of a functional SMN protein
• motor neurons do not divide, thus a single dose of Zolgensma is expected to have a lifelong effect

More general terms

• neurologic agent