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These gene mutations cause other retinal disorders, including Leber Congenital Amaurosis and some Retinitis Pigmentosa types.
The treatment is working by sending the billions of engineering virus to a person’s retinal cells with a correct copy of the RPE65 gene. Directly injected with a quick procedure.
In a clinical study, gene therapy improved the viewing rate of 29 participants treated with 29 percent of participants – or approximately 93 percent of scientists have tested the visions of the participants by passing through a barrier under the low light. A year after treatment, 21 (72 percent) of treated (72 percent) were successfully passed.
Spark thinks that 6,000 people around the world will be suitable for gene treatment in the United States for 1,000 to 2,000 people. Most of these patients will lose little vision in time and eventually lead to the completion of blindness. There is no approved drug for people with genetic mutation.
Finally, Jean Bennsylvania University of Pennsylvania, who will be luxturna, is a doctor and scientist, Jean Bennett, says that the waiting approval of Spark is a long time.
Bennett and their colleagues began to research on gene treatment for hereditary retinal diseases in the 1990s. We started trying to understand how to deliver DNA in the laboratory to the retinities of the research dogs. In July 2000, Bennett reminded his team with an early gene treatment of his team on three dogs.
Bennett was “extremely cowardly and frightened because they couldn’t see it.” “Then the behavior has changed after the correct copy of an eye gene has changed. They started running and play. Indeed it was clear that it has a positive impact.”
Bennett and his friends were very excited. However, these early promising results were mitigated by the tragedy. In 1999, a 18-year-old patient called Jesse Gelsinger, was dead in a gene therapy experiment aiming to treat genetic liver disease. Then, at the beginning of the 2000s, the children in gene treatment trials developed leukemia as a result of their treatment. The trial has been stopped. Gene therapy companies are closed. Bennett has not received resources to continue investigating.
Finally, in 2005, the team and his team partnership with Philadelphia Children’s Hospital researchers led by Katherine High, President of Spark Therapeutics and the Chief Science official. Bennet stated that he was convinced to invest in gene therapy investigations at a time that the hospital is cooled. The hospital rented researchers from companies that are folded and started to clinical research.
Scientists have solved the basic issues that have been treated against rare hereditary diseases. Then, we will see that the same approach can handle cancer, heart discomfort and other common diseases.
Security research has come to better news after the trial participants who have an eye therapy have very few side effects. In 2013, Spark Therapeutics was launched with $ 50 million from Children’s Hospital.
“Since the beginning, we are all right since the beginning will work and it will be safe. It is very pleasing to this point.” Says Bennett.
Spark’s success contributed to re-interest in gene therapy. According to the Alliance of Cell and Gene Therapy, a Industrial Group, Gene Therapy Companies provided an increase of $ 1.2 billion in the second quarter of 2017 and increased by 56 percent compared to the same quarter in 2016. And 504 gene therapy clinical trial is driving according to the organization, the worldwide, including 34 tests in the most advanced stages. Large Pharma companies are now interested in gene treatment.
The head research of the foundation of the foundation of the Foundation Carriage of the Foundation Carriage, which supports the early research on gene treatments for retinal diseases, tells Stephen Rose, the approval of Spark’s treatment will reflect the “fruits of a long history”. Explained that the other gene has opened the door, the treatments that can end over 225 genetic mutations known to cause blindness.
Two gene therapies for other hereditary diseases are already approved in Europe: a rare metabolic disease, Glybera and immune insufficiency type strimvelis. Glybera was pulled out of the market due to low demand (see “the most expensive medicine of the world”). When the treatment is approved in Europe, the manufacturer Uniqure adjusted the price to $ 1 million.
The Strimvelis marketed by Glaxosmithkline is 594,000 euros or approximately $ 700,000, but when patients are not improved comes with a refund guarantee.
After confirming the first gene treatment of FDA, Kymriah producer Novartis in November has set the price as $ 475,000. The company has justified the price of Kymriah saying that it is highly personalized and complicated for production. To create therapy that treats a childhood leukemia that is often fatal