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Antisense oligonucleotides for ALS

Novel treatment of neurodegenerative diseases with accompanying TDP-43 proteinopathy
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Targeted antisense oligonucleotide treatment of a range of neurodegenerative diseases involving compromised biology of the protein, TDP-43


Treatment of neurodegenerative diseases with associated TDP-43 proteinopathy, such as ALS and Parkinson’s.

Development Status

Proof-of-principle correction achieved at the molecular level.

IP Status

Protected by priority patent application.


University of Edinburgh researchers have developed a new approach for the potential treatment and prevention of a number of neurodegenerative diseases. The treatment corrects specific gene targets that control the biology of the protein, TDP-43, which is shown to be compromised in patients with frontotemporal dementia, Alzheimer’s, Parkinson’s and >98% of amyotrophic lateral sclerosis (ALS) cases.

A priority patent application was filed on this invention.


One of the major health problems faced by an ageing population is the development of any number of neurodegenerative diseases, such as ALS, Alzheimer's or Parkinson's. Existing therapies for these currently incurable diseases are focused on managing symptoms rather than curing the illness; there are no effective therapies available to treat ALS on the market at this time. Other treatments currently in development have not been shown to be effective or target genetic variants only present in a small number of cases.

This technology provides an avenue to treat or prevent a range of neurodegenerative diseases that present with accompanying TDP-43 proteinopathy. Specifically, it uses modified oligonucleotides as a strategy to modulate the gene expression of multiple important targets that are hypothesised to fuel the progressive TDP-43 abnormalities seen in a range of neurodegenerative diseases. Indeed, TDP-43 aggregation is a pathological feature in several common neurodegenerative diseases, as well as the overwhelming majority of ALS cases, which allows for the range of applicability of this technology. The modified oligonucleotides – antisense oligonucleotides (AON) – are active against specific targets whose activity is hypothesised to drive this disease-relevant TDP-43 aggregation.


  • Applicable to potential treatment of several major neurodegenerative diseases which involve TDP-43 protein misfunctions, such as ALS, Alzheimer's, Parkinson's and others
  • Works against a protein misfunction present in >98% of ALS cases.
  • Targeted therapy - helps reduce non-target effects

Quote: TEC1104564

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Neringa Barmute

Technology Transfer Manager