Gene therapy uses a delivery vehicle, referred to as a vector, to introduce a gene encoding a therapeutic protein into cells to replace the defective or missing mutated gene responsible for the patients’ disease.
Yet, unlike traditional, monogenic gene therapies which target a specific gene, our AAV-based gene therapy products work independently of the causative gene mutation to restore physiological mechanisms in the retina and preserve vision.
Building upon our world-leading expertise in ocular gene therapy, SparingVision is working alongside its partner Intellia Therapeutics to develop a new generation of novel ocular therapies using Intellia’s leading CRISPR/Cas9 technologies.
We believe the application of genome editing in the ocular space will allow us to address more inherited retinal diseases which remain inadequately treated with existing medicines.
What is Genome Editing?
Genome editing is a technique by which DNA is inserted, replaced, removed, or modified at particular locations in the human genome in order to treat a disease.
CRISPR/Cas9 is a specific genome editing technique, which can make precisely targeted and permanent changes in patients’ DNA to repair the harmful genetic mutation responsible for patients’ disease.
There are two main components to CRISPR/Cas9
- The Cas9 protein, a type of enzyme which acts like small “molecular scissors” to precisely cut the target DNA sequence.
- The guide RNA, which is designed to match the DNA and guides the Cas9 scissors to the exact location and activate the scissors.
How will genome editing be used in the retina?
SparingVision intends to use its unique expertise of self-inactivating AAVs in the ocular space to deliver CRISPR/Cas9 directly to the photoreceptors, where mutations of the genes identified with RP are located.
In Vivo Genome Editing
Illustration: Excision genome editing strategy in the retina.