Gene Therapy Market to Experience boom as Researchers Introduce a Platform that can narrow down Effective Therapies for Curing Genetic Blindness

Posted On December 30, 2021     

Vision loss is one of the most brutal conditions faced by humankind other than cancer and Alzheimer’s disease. People are incredibly fearful of becoming blind as it denotes that they would be forever dependent on other people for care. Blinding hereditary diseases of the retina are thought to be highly infrequent. Nonetheless, approximately 1 in every 3000 people globally carries one or more copies of genes that can cause vision loss and retinal degradation. For years, people suffering from inherited blindness have been forced to live a significant portion of their life without the ability to see.
A new study has made a giant leap in solving this problem. Researchers have unveiled a platform that can successfully identify best-performing viral vectors. Such prediction would ensure that gene therapies are appropriately delivered to the retina with efficiency and precision. The development provides a great application of gene therapy and may bring boom within Gene Therapy Market as it would help find the best gene therapy approaches that could treat genetic blind disorders while saving time and resources.
Several gene therapies have already been introduced in the market of the U.S. and Europe, and there are dozes in clinical trials. However, the critical obstacle is guaranteeing that sectors or inactivated viruses’ having the therapeutic genetic code enters the specific cells envisaged by the scientists. Since the retina is made of millions of cells, forming a series of layers, it is challenging to target the vector at a particular location.
To approach this problem, the team created a computational platform known as scAAVengr. It uses single-cell RNA sequencing and determines which AVV (Adeno-Associated Virus Vector) would best deliver gene therapy to the required location. The platform ensures the process identified is quick and quantitative while considering several options available.
Generally, dogs are known to develop blinding disorders that are akin to conditions of human diseases. In addition, the canine eye anatomy is similar to the human eye. Thus, the team used the directed evolution method to identify which variants would aim at the outer retina. This is because RPE (Retinal Pigment Epithelial) and photoreceptors (Rods and Cones) are found in the outer retina. Researchers discovered that their technique successfully targeted the outer retina – an essential task for performing a successful vision restoring gene therapy.
The vision restoration sector is currently going through a revolution wherein several patients are receiving adequate treatment for the first time. Due to these circumstances, the potential of the platform and its relativity increases even more. This is because it can help translate emerging therapies already working for patients to bring much more quickly and efficiently to the clinics.

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