Gene Therapy Research and Treatment
Gene therapies offer the possibility of long-lasting treatment for challenging and untreatable diseases, including genetic and non-genetic ones.
Adeno-associated viruses (AAV) are delivery vehicles widely used in gene therapies, given their ability to deliver genes efficiently and safely to a broad range of cells. In fact, the first AAV-mediated gene therapy was approved by the FDA in 2017 and by the TGA in 2020, which consisted of AAV carrying a healthy version of a gene to replace the non-functioning one causing a genetic retinal disease.
After that, many clinical trials with AAV have been conducted and more products have been approved, this time for haemophilia B and spinal muscular atrophy. Eye diseases, particularly, can highly benefit from gene therapies with AAV. They enable long-term control of complex and sight-threatening conditions after a single injection, offering millions of patients the possibility of slowing down vision loss or even restoring their sight, in some cases.
That is why the Layton Vision Foundation (LVF) research team has been studying AAV-mediated gene therapies for the treatment of eye diseases in the past years and developed promising candidates for age-related macular degeneration and intraocular inflammation. These diseases affect a high percentage of the world population and rank among the leading causes of blindness and visual loss worldwide.
In addition, their current treatments are burdensome for patients and clinicians, failing to achieve good outcomes in many cases. Our gene therapy candidates contain a unique type of AAV, discovered here at LVF, which allow optimal delivery to retinal cells, outperforming other vectors currently tested in clinical trials. We are also developing controllable features in our gene therapies to allow a more individualised treatment, regarding how much of the therapeutic substance is needed and when is the best time to release it, essential characteristics when managing such complex and chronic diseases.
This feature also offers additional safety for our candidates. Our therapies have been extensively tested in cell models and have shown promising and encouraging results. We are additionally testing our gene therapies in human tissues provided by the Queensland Eye Bank, that besides resembling a very close-to-reality environment, reduce the use of experimental animals.
Written by: Dr Brenda Castro, Scientific Fellow, LVF Ophthalmology Research Centre