S. Florida-Based Organization and Its Supporters Honor 
Those Who Have Helped Drive Therapy Breakthroughs Together

Boca Raton, FL, April 16, 2019 –The Mathew Forbes Romer Foundation (MFRF), a non-profit organization dedicated to the education and research of children’s genetic diseases of the brain, marked its 21st year with a celebration of Gene Therapy, which included fundraising over $90,000 for the cause.

During the Foundation’s annual “Sweetness & Laughter” Gala, which was appropriately themed a CELEBRATION of GENE THERAPY, over 150 guests were treated to dinner and dessert, an update on Gene Therapy advancements and progress, the “See the Light” Awards presentation, and to highlight the “Gene Therapy” theme, guests wore Jeans and enjoyed the hilarious entertainment by legendary performer Gene Merola.

The update on Gene Therapy advancements into clinical trials and MFRF progress are featured the following presentation:

https://www.youtube.com/watch?v=n84FUa7EMYM&t=298s

During the See the Light Awards presentation, two extraordinary leaders were honored:

  • Jeffrey Castelli, Ph.D., Chief Portfolio Officer and Head of Gene Therapy for Amicus Therapeutics. Castelli was honored for his Company’s long-term commitment to people living with rare diseases, as well as its recent acquisition of an extensive gene therapy portfolio of 10 clinical and preclinical AAV Programs in neurologic lysosomal storage disorders, including interests in Tay-Sachs.
  • Terence Flotte, MD, Dean of University of Massachusetts School of Medicine & Former Director of the University of Florida’s Powell Gene Therapy Center. Dr. Flotte was honored for his pioneering work in Gene Therapy and his critical role as the principal investigator leading the team into clinical trials for Tay-Sachs gene therapy and related diseases.

“This year was truly special for the MFR Foundation as we made real progress in gene therapy and were able to share the news with our supporters and impacted families,” said Kevin Romer, President of the Mathew Forbes Romer Foundation and past President of the NTSAD. “Our gala was a wonderful opportunity to show our appreciation to leaders in our field. We are thankful for both Dr. Castelli of Amicus and Dr. Flotte of UMass and confident in their abilities to keep pushing forward for advancements in Gene Therapy to help so many of our affected families.”

The Mathew Forbes Romer Foundation has been supporting research in Gene Therapy for over 15 years. Through the National Tay-Sachs and Allied Diseases Research Initiative that it co-founded as well as the Tay-Sachs Gene Therapy Consortium to drive to clinical trials. With the leadership of honoree Dr. Flotte, this has led to the first patient in clinical trials. The work was so compelling that In December 2018, Axovant Sciences, a clinical-stage company focused on innovative gene therapies for neurological and neuromuscular diseases, announced it licensed exclusive worldwide rights for the development and commercialization of two novel gene therapy programs to address GM1 gangliosidosis and GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases) from the University of Massachusetts Medical School.

To help keep the momentum going, The Mathew Forbes Romer Foundation hosted a “Celebration of Gene Therapy” Roundtable session at Florida Atlantic University College of Medicine in Boca Raton the day after the gala. The exclusive meeting brought together the honorees and leaders of industry, academia, and science who attended the MFRF Sweetness & Laughter gala to discuss how each can continue to drive results and success with Gene Therapy and the related family of diseases. Takeaways included: Sharing expertise and tools; Maintaining a dialogue between all participants; Understanding newborn screening opportunities; and more.

The MFRF has raised over $2 million itself to fund projects spanning its mission and vision across three pillars: Research of Treatments and Cures, Education and Genetic Screening, and care through Nursing School Educational programs.

  • Research: Conceived of and grew the National Research Initiative to 61 projects, $3.9 million in grants and many promising therapies with NTSAD. This has led to over $20 million dollars in funding from the National Institutes of Health (NIH) and similar institutions. Helped establish the Tay-Sachs Gene Therapy Consortium to accelerate pre-clinical and clinical trials. Sponsored the Medical and Scientific Sessions at the annual NTSAD Family Conference
  • Screening: Hosted 21 South Florida Genetic Testing and Education Fairs; screened over 1,550 potential parents with as high as 50 percent detection. Produced the training video of the International Tay-Sachs Quality Control Laboratory
  • Nursing: Funded more than 35 nursing scholarships with Florida Atlantic University, Florida International University, and Nova Southeastern University; created nursing school training programs including simulating in-home family care

About The Mathew Forbes Romer Foundation
Inspired by the strength of Mathew Romer, who lost his eight-year battle with Tay-Sachs disease in 2003, The Mathew Forbes Romer Foundation (MFRF) was founded in South Florida 21 years ago with a mission to promote and lead critical awareness, testing, counseling, and research initiatives that hold promise for the prevention and eventual cure of fatal children’s genetic diseases of the brain.

The MFRF is the South Florida affiliate of National Tay-Sachs and Allied Diseases Association (NTSAD), celebrating its own 61st year anniversary, and one of the oldest patient advocacy groups in the country, and a member of the Jewish Genetic Disease Consortium. The president of the MFRF (Kevin Romer) is also past president of NTSAD. The Mathew Forbes Romer Foundation was recognized in 2014 with the Best of Boca Raton Award – Education & Research Foundation. For more information, visit http://www.mfrfoundation.org/.

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Melissa Perlman
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