Antisense oligonucleotide therapy and biomarker assay for C9ORF72 hexanucleotide expansion disorders

Case ID:
C12195

Therapy to Treat and Assay to Monitor Neurodegenerative Disease  


JHU REF: [C12195]


Invention novelty: This technology is the use of antisense oligonucleotides (ASO) as a therapeutic for specific neurodegenerative diseases. The technology could also be used in development of a biomarker assay to monitor the efficacy of the ASO therapy.   


Value Proposition: Current therapeutic efforts for both frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) have proven costly and relatively ineffective. More recent advances in therapy aimed at these neurodegenerative diseases, however, have revealed some promising agents designed for better disease control. Implementation of this technology – both through use of antisense oligonucleotides (ASO) therapy, as well as development of monitoring assays of ASO treatment – could produce more effective delivery strategies, higher stability in administration, and improved clinical outcomes. 


Technical Details

Johns Hopkins and Ionis Pharmaceuticals' researchers have found that C9ORF72 hexanucleotide repeat expansion, located on chromosome 9p21, is a highly frequent pathological mutation in diseases. This technology, through designed antisense oligonucleotides (ASO), effectively targets this mutation. The therapy is targeted at specific neurodegenerative diseases which are characterized this particular RNA repeat expansion. It is based on the discovery that C9ORF72 hexanucleotide repeat expansion, located on chromosome 9p21, is a highly frequent pathological mutation in diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). 



Looking for Partners: To develop & commercialize this technology to generate biomarker assays and antisense oligonucleotide therapies.


Stage of Development: Discovery/Preclinical


Data Availability: Under NDA / CDA


Inventors: Jeffery Rothstein (JHU), Rita Sattler (JHU), Christopher Donnelly (JHU). Frank Bennett (Ionis), and Susan Freier (Ionis)


Patent Status: Pending


Publication(s)/Associated Cases: Not at this time

Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
Methods for Monitoring C9ORF72 Expression PCT: Patent Cooperation Treaty United States 14/436,039 10,577,604 4/15/2015 3/3/2020 10/15/2033 Granted
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For Information, Contact:
Nakisha Holder
nickki@jhu.edu
410-614-0300
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