Gene Therapy on the Horizon for Hunter Syndrome

Hunter Syndrome, is a heartbreaking twist of genetic fate, and we celebrate that the FDA has granted Rare Pediatric Disease Designation to REGENXBIO’s RGX-121 Gene Therapy.

Hunter Syndrome causes seemingly healthy boys to begin experiencing developmental delays, sometimes as early as 18 months, and actually regress by age six and a half.  Research on a treatment or cure has been agonizingly slow.

Regenxbio, located in Rockville, MD, has been making some inroads, through their gene vector delivery system called NAV Technology Platform. You can see a graphic video of this here:

Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. …we plan to file Investigational New Drug applications for RGX-111 and RGX-121 in the first half of 2017. We look forward to working with the FDA to advance this platform of gene therapies for MPS to patients in need.” –Biospace

The National MPS Society is a resource for families, dealing with Hunter Syndrome located in Durham, NC, but available on-line. One of their most important initiatives is the creation of a data base in conjunction with PatientCrossroads. Such patient data bases help speed research initiatives because a scientist with an idea, does not have to spend precious time trying to locate rare patients. They also help understanding the natural history of a disease, so that potential therapeutic agents can be properly evaluated, and clinical trials structured.

Another important program of the National MPS Society is their annual family conference which this year begins in Columbus, Ohio, and runs through August 6th. Patient conferences afford the opportunity to meet other families struggling with the same challenges, and to get updates on research and potential therapeutics.

Given the exciting developments by Regenxbio, and other pharmaceutical and bio firms, how wonderful that gene therapy and emerging technologies will be highlighted at this year’s program.


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