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 Non Proliferative Diabetic Retinopathy (NPDR), has achieved promising results in a Phase Ib/IIa trial, and is now moving into Phase IIb.
How Our Drugs Work
Diseases of the retina are one of the leading causes of blindness worldwide, with the most common being Wet Age-Related Macular Degeneration (wAMD) and Diabetic Macular Oedema (DMO). Both wAMD and DMO are caused by over-proliferation of blood vessels in the retina, largely driven by VEGF.
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.
VEGF inhibitors and VEGF receptor inhibitors are already the standard of care for a number of diseases, and current therapies for retinal vascular diseases involve the inhibition of VEGF-A. These therapies, however, target all isoforms of VEGF-A, regardless of whether they are pro- or anti- angiogenic, and are therefore associated with limited efficacy, resistance, off-target effects and toxicity.
Additionally, these existing VEGF therapeutics are administered via intraocular injections, which carry their own risks, including ocular hypertension, endophthalmitis, and retinal detachment. Both patients and clinicians are averse to this form of administration, but current attempts to improve this course of treatment only focus on reducing the injection frequency through the development of depot delivery systems.
Our novel compounds not only increase the ratio of anti- to pro- angiogenic VEGF-A isoforms, they are designed to be delivered as an eye drop. We therefore have the potential to develop safer, more efficacious therapies that can vastly improve patient wellbeing.
We are developing a minimally invasive therapy to prevent vision loss in patients with retinal vascular diseases.
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?
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