Sophia’s Cure Announces Open Enrollment in Gene Therapy Clinical Trial


Sophia’s Cure Foundation is a nonprofit 501(c)3 public charity which was formed shortly after the daughter of Catherine and Vincent Gaynor was diagnosed with Spinal Muscular Atrophy. The foundation was created to assist in funding for clinical research to help find a cure for SMA and to offer support to families affected by this disease by providing advocacy, awareness, education, and support.

The organization was proud to announce enrollment for their gene therapy clinical trial is now open.

Phase I Clinical Trial of Systemic SMN Gene Therapy for Spinal Muscular Atrophy is set to begin at Nationwide Children’s Hospital in May. The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of self-complementary scAAV9.CB.SMN as a treatment of Spinal Muscular Atrophy type 1 (SMN1).

This was a program Sophia’s Cure Foundation lead the way in with a multi-million dollar investment. They made their first donation in 2010 and continued financial support every year since.

“Our proudest accomplishment was funding all of the product that will treat SMA babies for the very first time. We are very proud to have spearheaded the effort to insure that this program was fully funded. There were many doubts that a Mom and Pop 100% volunteer nonprofit could support a multi-million dollar program through the clinical trial process. The message started here, but traveled throughout the world. We could not have done it without your help– each and every donation, and fundraiser you did, made a difference. We are very thankful for the enormous support from Avery’s Bucket List. The Little Aviator Avery was monumental in pushing this program forward. She moved the world and made sure this would get to trial. Every parent involved in the 200 campaign helped push this forward. Together we brought the giant to its knees. We did this together and against all odds. Thank you for believing in us and in SOPHIA!!! We love you all,” read the announcement on the foundation’s website.

Click here for information on enrolling in the clinical trial.

Filed Under: Innovation, Research

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