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Safety studies nearing completion in gene therapy drug trial


The safety studies needed to begin the gene therapy drug trial for a promising cure for Hunter syndrome are nearing completion, according to Project Alive President Dr. Kim Stephens.

Stephens updated the Hunter syndrome community on the status of the work behind the scenes on the research for a cure on MPS Awareness Day May 15. She said the safety study is a necessary first step in the process of a gene therapy drug trial.

“What this means is we’ve gone through checks to make sure that the vector is safe in the animal models,” Stephens said referring to vehicle by which the desired gene is delivered to the cells during the therapy being tested. “After the safety study is complete, a report of the findings will be submitted to the FDA. This will bring us another step closer to moving forward with the actual study of the gene therapy drug.”

Project Alive is a grassroots organization formed by parents whose children suffer from Hunter Syndrome, a rare degenerative disease that affects mainly boys and limits life expectancy to the early teen years in the most severe cases. The organization works to raise awareness for Hunters Syndrome, provides support for Hunter Syndrome families and raises funds for research to find a cure. The non-profit has raised a total of $2.35 million and funded the preclinical work and vector manufacturing for the trial set to be conducted at Nationwide Children’s Hospital in Columbus, Ohio. That is just $150,000 shy of the funding needed for the full clinical trial.

“We needed a cure yesterday, but we’re optimistic we will be able to move things along to the next step very soon,” Stephens said, whose son Cole was diagnosed with Hunter syndrome in 2012 at two and a half years old. The founders of Project Alive, the world’s leading Hunter syndrome research and advocacy foundation, initiated their own gene therapy research for the disease when there was none. They contacted and collaborated with researchers and have been funding this trial from its preclinical stages to its IND approval from the FDA to its current stage. They will continue to advocate for families affected by Hunter Syndrome as well as act as a resource for the rare disease community once the trial begins.

Project Alive is poised to serve the Hunter syndrome community by continuing to fund and design research, assist industry in the space, and bring new insights into the scientific community about this rare disease. Project Alive continues to work closely with the National MPS Society and the other Hunter syndrome groups with the goal of saving and improving the lives of those affected by the disease. Plans are in the works to start a rare disease registry for Mucopolysaccharidosis or MPS. Hunter syndrome is a form of Mucopolysaccharidosis known as MPSII.

Project Alive is a powerful voice for children and adults with Hunter syndrome, bringing together families and advocates with researchers, industry, and regulators. Project Alive funds promising curative research, assists researchers and industry with designing research studies for our community, and advocates for the most effective and efficient system of clinical research, evaluation, and approval. Through its innovative campaigns and grassroots efforts, Project Alive has made significant advances in public awareness about Hunter Syndrome and its symptoms, the need for early diagnosis, and available treatments and clinical trials.

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