28 Nov 2011

Gene therapy becomes a reality in 2014!

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Gene Therapy for Sanfilippo A and B

 A consortium of family foundations from around the world have come together in an effort to fund clinical trials in 2014 for Sanfilippo A and B using gene therapy. The trials will be conducted at Nationwide Children’s Hospital in Columbus, Ohio.  We urge everyone to get involved by hosting a small fundraiser this summer to raise needed funds to make this a reality.  We have all worked hard through The Pepsi Challenge and other fundraisers to fund Sanfilippo A animal studies for gene therapy.  Researchers and clinicians are now ready to bring potential treatments for both Sanfilippo A and B to human clinical trials together, side by side.  We are all in this fight together and we need everyone’s participation.  Even if it is as small as a change box at work that raises $100 its one step closer. Together we can move mountains.

The two lead products, ABX-A and ABX-B, uniquely deliver the therapeutic product to the CNS with the aim of preventing and/or reversing the effects of the genetic errors that cause the disease. ABX-A and ABX-B induce cells in the CNS and digestive tract to produce the missing enzymes and help repair and prevent damage caused to the cells. Safety studies conducted in large animal models have demonstrated that delivery of ABX-A and ABX-B are well-tolerated with minimal side effects. Importantly, efficacy studies in animals with Sanfilippo syndrome have demonstrated unprecedented therapeutic benefit months after treatment. A single dose of ABX-A or ABX-B significantly restored normal cell and organ function and increased the lifespan of animals with SF over 100% a year after treatment compared to untreated control animals. Similarly, animals treated with ABX-A and ABX-B demonstrated corrections of cognitive defects that remained months after drug administration. These results are consistent with studies from several laboratories suggesting ABX-A and ABX-B treatment could potentially benefit patients with Sanfilippo Syndrome Type A and B, respectively.

ABX-A and ABX-B will be administered systemically for efficient drug delivery to the whole body and CNS – a significant advantage over current therapy options.