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Gene targeting therapies – what does the future hold for neurological disorders?

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An overview of antisense oligonucleotide therapies in development for neurodegenerative diseases.

There are no effective disease modifying therapies for neurodegenerative diseases such as Alzheimer’s (AD), Parkinson’s (PD), amyotrophic lateral sclerosis (ALS) or Huntington’s disease (HD). Huntington’s disease (HD) is a devastating autosomal dominantly inherited neurodegenerative disease and the genetic predictability of HD provides an opportunity for early therapeutic intervention many years before overt symptom onset and at a time when reversal or prevention of neural dysfunction may still be possible. As HD is monogenetic, fully penetrant, and characterised by a long premanifest phase, it is emerging as a potential model for studying therapeutic intervention in other neurodegenerative conditions such as Alzheimer’s or Parkinson’s disease where no preclinical diagnostic tests exist. In addition, HD manifests with a broad range of clinical symptoms and signs, many of them common to these other diseases, and involves widespread pathology throughout most of the brain involving similar protein misfolding. Understanding of HD pathogenesis is evolving, and I will present an overview of important approaches in development for targeting mutant HTT DNA and RNA , the cause of HD pathogenesis, and in particular I will present our recent successful phase 1b/2a clinical trial testing the effects of antisense oligonucleotide therapy (ASO) with RG6042 (formerly known as IONIS HTT Rx) in patients with early Huntington’s Disease and present the results of the first successful HTT -lowering drug trial (Tabrizi et al New England Journal of Medicine 2019). This study is the first to demonstrate antisense-mediated protein suppression in patients with a neurodegenerative disease. While this particular ASO holds great promise for HD, our findings have broader implications. These data suggest that antisense technology has the potential to provide disease-modifying benefits in other neurodegenerative diseases associated with aberrant production of proteins, including ALS , Alzheimer’s disease and many other diseases that currently lack adequate treatments. In my talk I will review ASO approaches in development for CNS diseases.

All welcome – free for CSAR members, £2 for undergraduates, postgraduates and recent PhDs, £5 for non-members.

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