Title:
COMBINATION THERAPY FOR SPINAL MUSCULAR ATROPHY
Document Type and Number:
WIPO Patent Application WO/2021/030766
Kind Code:
A8
Abstract:
Aspects of the application relate to compositions and methods for treating spinal muscular atrophy in a subject. In particular, this application provides therapeutic combinations of a small molecule that promotes SMN function and/or a recombinant nucleic acid that encodes the survival of motor neuron 1 (SMN1) protein (e.g., in a viral vector), and/or an antisense oligonucleotide (ASO) that increases full-length survival of motor neuron 2 (SMN2) mRNA (e.g., that is targeted to a nucleic acid molecule encoding the survival of motor neuron 2 (SMN2) and promotes the inclusion of exon 7 in SMN2 mRNA).
Inventors:
MCCAMPBELL ALEXANDER (US)
Application Number:
PCT/US2020/046546
Publication Date:
March 18, 2021
Filing Date:
August 14, 2020
Export Citation:
Assignee:
BIOGEN MA INC (US)
International Classes:
A61K9/00; A61K31/7088; A61K31/7125; A61K48/00; A61P21/00; A61P21/02
Attorney, Agent or Firm:
GE, Lingyin et al. (US)
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