Cure Sanfilippo funds $380,000 for first MPS IIIC gene therapy

May 15, 2020

Cure Sanfilippo Foundation funds $380,000 to Phoenix Nest Inc. to support path to first-ever MPS IIIC Gene Therapy

COLUMBIA, SC (May 15, 2020) — Cure Sanfilippo Foundation is thrilled to announce funding of $380,000 to the New York-based biotechnology company Phoenix Nest, Inc. The funding will help support creation of plasmid (a critical component of gene therapy drug production), as well as the required in-vivo efficacy study in the MPS IIIC mouse model. These and other steps will enable the project to be evaluated by the U.S. Food and Drug Administration (FDA) for approval to proceed with a clinical trial. The goal is to bring forward the first-ever gene therapy clinical trial for Sanfilippo syndrome (MPS III) type C with the hope of providing relief to children suffering from Sanfilippo syndrome.

Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is a rare genetic disease that has devastating effects on the central nervous system. Approximately 1 in 70,000 children are born with this inherited, yet initially invisible, condition. Those with the disease lack an enzyme that breaks down large sugar molecules. Those molecules accumulate in their cells, causing a cascade of damaging effects in the brain and body. What follows is the loss of critical brain tissue, vision and hearing impairment and ultimately dementia. The effects of Sanfilippo syndrome typically become apparent in the pre-school years of life. Tragically, there is no FDA-approved therapy available today to stop the rapid neurological deterioration, suffering, and death for these helpless children. Sanfilippo is sometimes referred to as “childhood Alzheimer’s.”

Jill Wood, President of Phoenix Nest, stated, “Fundraising for an ultra-rare disease is difficult, made even more so during these uncertain times. We can’t thank the generous supporters of Cure Sanfilippo Foundation enough. Not only will this funding contribute to our Investigational New Drug (IND) enabling studies but hopefully will act as seed funding for future investors.”

Jill Wood knows the journey to this point well, having rallied much of the support by working in advocacy herself for the past 10 years, and now fully focusing on her role leading the biotech. Jill is also mother to her 12-year-old son Jonah with Sanfilippo syndrome type C.

“Today is a very uplifting and inspiring day. It is because of the incredibly hard work and grassroot efforts of the parents and supporters of children with Sanfilippo syndrome type C from around the world that we are able to help move this research forward,” commented Cure Sanfilippo Foundation President Glenn O’Neill. “I have seen firsthand how families do everything possible to save the children they love. We have more work ahead, and we must continue the momentum to help bring this to a clinical trial for children with Sanfilippo syndrome type C, as soon as possible.”

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Cure Sanfilippo Foundation is a 501c3 nonprofit whose mission is to advocate and fund research to bring about treatments and/or a cure for Sanfilippo Syndrome. Cure Sanfilippo has funded 25 research grants around the world which has helped three clinical trials get underway, to date, treating children with Sanfilippo Syndrome. The Foundation creates and explores innovative and breakthrough research to ensure multiple promising paths are being explored in the effort to save children and better their quality of life. In addition to funding research, the Foundation has a focus on: 1) working with the U.S. Food and Drug Administration and biotechs regarding appropriate clinical trial endpoints and ensuring the caregiver voice is being incorporated every step of the way; 2) earlier diagnosis, diagnostic testing, and newborn screening; and 3) providing guidance and support to families for navigating the landscape of clinical trials, insurance and physician support. Learn more about Sanfilippo syndrome and the Foundation.

Phoenix Nest, Inc., is a biotechnology company focused on Sanfilippo syndrome type C and D. The company collaborates with various academics to develop treatments for Sanfilippo syndrome. Phoenix Nest has been the recipient of five grants from the National Institutes of Health to date.

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