Pipeline
We believe that we have a cutting edge technology with the potential to revolutionise treatment using mRNA targeted therapies
Exonate’s science targets mRNA through innovation of SRPK1 altering it so that an isoform of VEGF is produced to cause inhibition of new blood vessel growth.
Pipeline Overview
Exonate is leveraging our expertise in our revolutionary, mRNA targeted, small molecule compounds. Our lead programme, for the treatment of Diabetic Macular Oedema (DMO), has entered Phase I clinical trials and we are actively developing targeted therapies across a range of diseases.
How Our Drugs Work
Drugs which inhibit VEGF and its receptors are already the standard of care for a number of diseases including wAMD and DMO and is a mainstay of cancer therapy. VEGF is alternatively spliced into two classes of protein: pro-angiogenic VEGF-Axxxa isoforms which are responsible for disease progression, and anti-angiogenic VEGF-Axxxb isoforms which mitigate these effects through competitive binding.
Current therapies, however, target all isoforms of VEGF, are associated with limited efficacy, resistance, off-target effects and toxicity. Modulation of VEGF splicing using our novel compounds aims to reduces pathological VEGF, angiogenesis and disease progression without inhibiting non-pathological, protective forms of VEGF, and so has the potential to offer safer, more efficacious therapies.
Target Indication & Ophthalmology
Wet Age-Related Macular Degeneration (wAMD) is a neovascular disease of the retina, which means blood vessel growth is upregulated in the back of the eye, in a process largely driven by VEGF. In wAMD, the growth of new vasculature through from the choroid ruptures Bruch’s membrane and causes disruption to the usually uniform layer of photoreceptive cells responsible for vision.
How Our Technology Works
By exploiting the alternative splicing of vascular endothelial growth factor (VEGF) we aim to use our cutting edge technology to introduce an Eye Drop to treat wet AMD and DMO. This has the potential to become the patients treatment of choice in an area of high medical unmet need. Our novel small molecule inhibitors have the ability to alter VEGF splicing in a number of indications in addition to our lead ophthalmology programme.
What is happening at Exonate?
Learn more about Exonate and discover our latest company news, achievements and discoveries. Don’t miss a thing and get updated on our latest news.
- In the press Exonate Shortlisted for Biotech Company of the Year by The Cambridge Independent Science and Technology Awards.
- In the press Exploristics and Exonate announce successful collaboration to enhance clinical trial study design using cloud-based simulation
- In the press Exonate first-in-class eye drop Phase Ib/IIa trial data demonstrate safety and biological activity in treatment of diabetic retinopathy and diabetic macular oedema