Spark Therapeutics Pioneering Gene Therapy Robert F Higgins 2018
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In 2018, Spark Therapeutics pioneered gene therapy that had the potential to transform the treatment of genetic diseases. This article explores Spark’s groundbreaking gene therapy, as well as its impact on the global gene therapy market. Background: Spark Therapeutics is a biopharmaceutical company based in California that develops therapies for genetic diseases. It was founded in 2004 by Dr. Robert F. Higgins, an oncologist and
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I’ve always thought that a cure for inherited retinal degeneration (IRD) was possible, as it’s a genetic disease that’s typically diagnosed at birth. So, when I learned about Spark Therapeutics’ pioneering gene therapy (GT) for an inherited form of retinal degeneration that is currently in clinical trials (NCT03342075), my pulse quickened. Spark has a unique approach: developing gene therapies to treat diseases where the genetic component is not
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“In the late 1990s, there were no drugs on the market that could cure AIDS. In 2010, however, the FDA approved the world’s first gene therapy to treat AIDS. Spark Therapeutics revolutionized the HIV/AIDS treatment field. Their first product, GM-CSF-P, was the first product to address the root cause of the disease—immune activation. GM-CSF-P is a protein that stimulates the
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Spark Therapeutics is a San Francisco, California-based biotechnology company focused on gene therapy to treat genetic disorders. In January 2018, Spark announced the successful completion of a phase 1/2a clinical trial for STI571 (a gene therapy candidate for spinal muscular atrophy (SMA)). This groundbreaking trial was the first successful clinical outcome for gene therapy in SMA. The potential of this treatment lies in its ability to correct the underlying genetic mut
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Case Study: I’m writing about Spark Therapeutics’ revolutionary gene therapy (called Lentiviral Vector-based AAV1-SCGFγ2 Gene Therapy for Sindactyl Syndrome) — which is the very first humanized gene therapy, administered as an IV to children at the age of two. news The results were truly transformative, and we’ve had fantastic results. The children were given a dose of 15 mg/kg, given every four weeks for 6 months. The ther
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