Children in the verge of losing their eyesight, has found a new light in Sparks Therapeutics gene therapy. Sparks Therapeutics is a young biotech company formed out of research in the Children’s Hospital of Philadelphia. The company is currently conducting a clinical trial on the potential of genetic therapy in treating rare diseases that can cause blindness.
Jeff Marrazzo, CEO of Sparks Therapeutics explained how the idea of gene therapy works in a recent press release statement. The idea is to make a copy of the functional gene with the correct DNA sequence and add it on the missing or dysfunctional gene. In this way, the cells would create a new copy of the gene having the correct DNA sequence.
Exploring the Unexplored Realm of Gene Therapy
Gene therapy is relatively a new field in Medicine. There are a lot of untapped areas in this field. Although, scientists have been working in this field for decades, they haven’t had concrete evidence on how this procedure really works.
Basically, gene therapy uses a modified virus and used it to deliver the copy of the gene to the specific area that causes the disease.
It has been a long and dreary journey for the scientists who are working on this field. Gene Therapy almost collapsed when a patient died in a clinical trial in 1999. But it managed to take resurgence because scientists are gaining a better understanding of modern science. And also, a lot of companies are now exploring and investing in this field.
Gene Therapy Approaching the US Market Soon
In the next few years, Sparks Therapeutics is expecting that new gene therapies will hit the US Market soon. Among the various companies that are working on gene therapy, Sparks Therapeutics has the best and most advanced modern facilities.
Their latest gene therapy project aims to help more blind patients. The procedure will create a healthy gene which will be delivered through an eye surgery. Once the modified gene is delivered, it is designed to work like a normal functioning gene.
The great thing about this procedure is that patient will have to undergo only one treatment. Marrazzo shared that what they are working on is actually different from the other gene therapy procedures. They are delivering a blueprint, and then the cells will be the one to complete the last step.
The modified gene will stimulate the cells to create a protein or missing enzymes that is lacking in the gene sequence. For that reason, Marrazzo hopes that this procedure will provide a long-term effect.
Sparks Therapeutics is committed in the thought of providing a single treatment that will provide a long-lasting effect. This promising new treatment may influence more investors to put on their assets to this company.
Gene Therapy Giving Hope to the Blind
Though, the field is relatively new, gene therapy has a lot of potential and benefits for the patients. First, it helps target rare diseases so it can help solve complicated cases that have no treatment yet. Second, it promises a single treatment procedure which is beneficial for the patient because of reduce costs and shorter hospital stay. Third, this procedure will help young patients who are in the brink of losing their eyesight. Gene therapy will help these young children to live normal and productive lives.