Researchers Submit Patent Application, "Compositions And Methods For Modulation Of Smn2 Splicing In A Subject", for Approval (USPTO 20240238326).

Researchers from Biogen MA Inc. have submitted a patent application for a method of modulating splicing in a subject's SMN2 gene. This method aims to treat proximal spinal muscular atrophy (SMA), a neurodegenerative disorder caused by the loss of the SMN1 gene. The researchers propose administering an antisense compound into the cerebrospinal fluid of the subject to target intron 7 of the SMN2 gene and increase the inclusion of exon 7 in the mRNA. The patent application discusses various methods of administering the compound, including bolus injection or infusion with a delivery pump, and mentions the possibility of combining the antisense compound with other therapies. [Extracted from the article]