Thanks for being a part of our FAST community, Cure. We’re really excited to be sharing regular updates with the FAST community, from family spotlights, to research updates, to upcoming events and fundraisers. 

If you have updates you would like to be included in next week’s newsletter, please email us at info@cureangelman.org. Thanks and happy reading!

Last week, the FDA approved Zynteglo, the first gene therapy for individuals with beta-thalassemia. This is a monumental step forward for the entire rare disease community.

Zynteglo is a hematopoietic progenitor cell gene therapy (HSC-GT) to treat a rare disease called beta-thalassemia. This is an elegant type of gene therapy using a patient's own bone marrow stem cells that come from their own blood, which are then personally modified to produce the gene that they are naturally missing. Without treatment these patients become blood transfusion dependent, leading to lifelong challenges. This is a one-time gene therapy that is personalized to each patient’s own bone marrow cells. In this study, after treatment, 89% of patients achieved transfusion independence, ultimately resulting in a curative intent gene therapy. This is the first cell-based gene therapy of this type approved for a rare disease by the FDA. There are many programs similar to this that are currently in clinical trials showing just as promising results, including those for neurologic genetic disorders like metachromatic leukodystrophy (MLD), which has been approved in Europe.

This is relevant for our AS community because FAST has been robustly supporting a very similar program to accelerate a therapeutic for all individuals living with Angelman syndrome since 2016 through University of California Davis. These HSCs are programmed to carry the non-functional gene impacted in Angelman syndrome (UBE3A) and can cross from the blood into the brain (the blood brain barrier). A recent publication here showed that this gene therapy was able to correct all of the behaviors tested in the AS mouse model, in both the newborn and adult age groups. Here is a video by Drs. Abedi and Anderson at the 2021 Annual FAST Science Summit sharing their excitement about this program. We look forward to sharing more at the 2022 Annual FAST Summit and Gala!

Zynteglo’s exciting announcement further paves the way for future advancements for the Angelman community and more broadly for the entire rare disease community where this approach is applicable.

Making Up Las Vegas will host the Beauty Room at the 2022 FAST Gala. Lisa Strawther, founder of Making Up Las Vegas, will return to this year's event to provide hair and make-up services for attendees. Lisa offers affordable rates for attendees to relax in a great atmosphere and enjoy an afternoon of pampering and swag before an evening of glitz, glamour and dancing. To sign up for hair and/or make-up services, join FAST Gala Hair & Makeup by Making Up Las Vegas on Facebook:

We are so grateful to those who help support our mission by participating in our annual fundraising program— Cure Angelman Now (CAN). Every donation made to a CAN campaign will fund important research grants to develop meaningful therapeutics for our loved ones with Angelman syndrome.

This year’s top CAN fundraisers will win a day with leading Angelman syndrome therapeutics researcher Dr. Albert Keung and his team in North Carolina State University’s lab! Our special guests will have the opportunity to tour Dr. Keung’s laboratory, socialize and engage with his entire team, and see firsthand the significant contributions his laboratory is making in the effort to understand and test therapeutic options for Angelman syndrome.

Watch the Grand Prize video to see what you could win:

If you haven’t started your own CAN fundraiser yet, it’s not too late! Learn more and start a fundraiser today for your chance to win:

FAST has earned a 2022 Platinum Seal of Transparency from Candid’s Guidestar, the world’s largest database of nonprofit organizations. Only 0.01% of all nonprofits receive this exceptional designation, which signifies that FAST shares clear and vital information about goals, strategies, capabilities, achievements, and progress indicators with the public.

On July 30th, Brenda Leclercq hosted the 10th Annual Michigan Golf Tournament. For the past 10 years, Brenda’s Michigan Golf Tournament has been held to support FAST. On behalf of FAST and the Angelman syndrome community, a special thank you to Brenda Leclercq for her continued dedication to support the FAST mission of accelerating research to bring effective therapeutics to all individuals living with Angelman Syndrome.

You can get started with your own fundraiser here:

PTC Therapeutics is offering a new free webinar as part of their Insightful Moments series. This next webinar, on August 31st, will focus on how to navigate local, state, and federal resources while advocating on behalf of your rare disease journey. See the image above for additional details and registration/contact info.

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