GB2603454A — Novel therapeutics for the treatment of neurodegenerative disorders
Assigned to UCL Business Ltd · Expires 2022-08-10 · 4y expired
What this patent protects
An anti-sense oligonucleotide (ASO) including a nucleotide sequence of 13-30 nucleotides, wherein the nucleotide sequence has sequence complementarity with SEQ ID NO 1 and is capable of modulating splicing by preventing inclusion of an UNC13A cryptic exon into an UNC13A mature mR…
USPTO Abstract
An anti-sense oligonucleotide (ASO) including a nucleotide sequence of 13-30 nucleotides, wherein the nucleotide sequence has sequence complementarity with SEQ ID NO 1 and is capable of modulating splicing by preventing inclusion of an UNC13A cryptic exon into an UNC13A mature mRNA. Guide RNAs including the ASO for use in CRISPR, CAs13 nuclease mediated gene regulation, and viral vectors expressing the ASO are also described. The ASOs and guide RNAs may be used as a medicament, for example, to treat neurodegenerative disorders, particularly those associated with TDP-43 pathology including ALS, fronto temporal dementia(FTD),Alzheimer’s disease, Parkinson’s disease, FOSMN, Perry Syndrome, or any combination thereof.
Drugs covered by this patent
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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