An antisense drug targeting the SRY gene in men with Parkinson’s disease (PD)

Market Size

Global PD market is valued at US$3.2bn; more than 10m patients with PD, 80% men.

Investment Landscape

Cynapsus Therapeutics late stage PD symptom treatment acquired by Dainippon Pharma Co. for US$820m (2016)
Acorda Therapeutics acquired Biotie Therapies Phase 2 PD symptom treatment for US$363m (2016)


  • Parkinson’s Disease is 3 times more common in men than women. Dysregulation of SRY expression in PD is the proposed mechanism for this male bias.
  • In various PD animal models, SRY levels are abnormally high in the substantia nigra. Reducing the level of SRY produced reduced the damage to the brain of these animals.
  • Antisense oligonucleotides have been used clinically to stop the production of genes and resulting proteins by interacting directly with a patients DNA.
  • Antisense-based inhibition of nigral SRY may be a novel neuroprotective strategy for PD in males


  • Broad patent covering SRY for all neurological diseases; including antisense oligonucleotide therapy
  • Compelling animal model data supporting the role of SRY in Parkinson's Disease
  • Current product reduces or halts the cell death, motor symptoms and disease progression.
  • Intention to develop a new patented compound following success of proof-of-concept trial.

Competitive Advantage

  • No available therapies to halt progression of PD
  • Unique drug target, animal models, mechanism of action with a with a demonstrated effect on disease.
  • Team and Laboratory expertise; 25 years work on SRY; developing unique models and human and rodent SRY antibodies
  • Antisense downregulation has been used successfully in other related neurological diseases including Alzheimer's disease.
  • The team has full access to HMRI laboratory and clinical facilities to sponsor research through to Phase 1.

Lab Notes

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