A new gene therapy, capable of treating an inherited eye disability that causes blindness developed by Spark Therapeutics, is closer to get approved as it succeeded in its late stages of clinical trial.
Spark Therapeutics announced that people involved on the trials were able to increase their ability to handle themselves in spaces with poor lightning. This therapy is one of the few that could manage to avoid the safety issues involving gene treatments before.
“This is an important moment for the field of gene therapy, and demonstrates Spark’s ability to carefully and precisely integrate technologies and approaches across a range of disciplines to move the concept of gene therapy towards a therapeutic reality for patients,” said Kathy High, MD, co-founder, president and chief scientific officer of Spark, according to the press release.
The company said that the results were measured and compared to a control group, basing their analysis on mobility. They reported that no adverse events or dangerous immune responses were observed during the trials. Doctors separated an initial 31 subject group with the condition in two groups: a 21 subject group who received the treatment, and the other 10 people control group.
In order to study the results, scientists recorded videos so they could track the patients as they sorted a series of obstacles using their vision, showing important progress.
“We saw a substantial restoration of vision in patients who were progressing toward complete blindness,” said Albert M. Maguire, MD, lead investigator in the trial. “The majority of the subjects given the treatment derived the maximum possible benefit that we could measure on the primary visual function test, and this impressive effect was confirmed by a parallel improvement in retinal sensitivity”.
Scientists insist on the great benefits that this gene therapy would provide to people suffering from the disease, saying that “if approved, the therapy should have a positive, meaningful impact on the lives of patients with this debilitating condition”.
Researchers attribute the positive results to more than a decade of hard work and innovation, designing all phases of the therapy and taking care of the factors involved.
This kind of genetic therapy works based on the injection of genetic material into the person cells, being able to treat or prevent various diseases. They expect to be the exception on the field of genetic treatment, as other attempts have failed. Several hundreds of these trials have taken place since 1990, and none has succeeded to get the FDA approvement.
The company offered some few details of the therapy, announcing that more information will be presented at a medical meeting in Paris, on October 10th. Also, they announced that they will be ready to present the first commerce and marketing strategy to the FDA on 2016, hoping they get the approvement seal.
“We wish to thank the trial participants and families as well as the investigators and all who contributed to this groundbreaking trial and successful result,” concluded Kathy High.
Source: Spark Therapeutics