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
