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Exciting Angelman Syndrome Development

Dear Friends,

I have exciting news to share. 

As many of you know, in 2017 FAST launched its own biotechnology company called GeneTx Biotherapeutics to accelerate the development of an investigational antisense oligonucleotide drug, GTX-102, for the potential treatment of AS. 

Very quickly, GeneTx attracted investment by Ultragenyx Pharmaceuticals, a company involved in the research and development of novel products for the treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and a high unmet medical need. Together, GeneTx and Ultragenyx pioneered a Phase 1/2 clinical study of GTX-102 in the U.S, the United Kingdom and Canada—the first potential disease-modifying therapy in human clinical trials for Angelman syndrome. 

This afternoon, Ultragenyx Pharmaceuticals announced it had closed on the acquisition of GeneTx, and will now take over the GTX-102 clinical program. As you can see in Ultragenyx's press release from today, the interim data is promising. Having Ultragenyx in the driver’s seat for this clinical trial makes us very proud, and confident that it is excellent hands.
Watch The Video To Learn More

You may have questions about what this means for your loved ones. As the news unfolds, we promise to work with Ultragenyx to ensure that the community is given updates as soon as possible. We feel confident that Ultragenyx exercising its option to buy GeneTx suggests that the company sees great potential for this program.

Indeed, what happened today is exactly why FAST was founded, why we started GeneTx, and why we donate and fundraise: To discover and accelerate promising therapies for AS. 

FAST was founded in 2008 by incredible visionaries, led by Paula Evans and a group of highly motivated parents, who were driven by a singular mission: to cure Angelman syndrome. This vision laid the groundwork for incredible progress—driven by the development of an innovative “Roadmap to a Cure,” an effort to bring together lead scientists from around the world to further their technology to move from bench to bedside.

With the proceeds from this sale FAST is now able to pledge millions of dollars to supercharge numerous pathways and promising programs, and in the process enrich the entire landscape of AS-related therapeutic research. Over the past few months, numerous programs in biotech have been paused, shelved, or cancelled due re-prioritization of programs and tumultuous financial markets—a trend that could derail potentially promising Angelman programs from being advanced. With FAST now able to focus on acceleration and progress, we can lend support to help advance a host of promising opportunities. The platforms currently being pursued and advanced through your donations to FAST include AAV gene therapy, hematopoietic stem cell gene therapy, enzyme replacement therapy, CRISPR technology, miRNA, and many more. FAST has not stopped pushing the timelines to help these programs get closer to human clinical trials in order to ensure that no stone is left unturned. 

Since starting this journey with you, FAST has gained much ground and our momentum is growing: clinical studies are underway and innovative partnerships have been forged. None of this would have been possible without your contributions: your time, your persistence and your dollars have brought us to this exciting moment, and will continue to drive us forward.  There are no words to fully express our deep gratitude to you and to the entire AS community, except “thank you.” So, thank you, from the depth of our hearts. 

Today we all celebrate a milestone, but we will not linger here because we still have a long journey ahead. Our next stop is tomorrow: Join FAST and Ultragenyx Pharmaceuticals for a zoom discussion on Tuesday, July 19 at 5:00pm EDT. To register, please click here

We have the wind at our backs! As we’ve been saying, #BetterDaysAreComing. Join us as we make that a reality—FAST. 

John Schlueter

Board Chair, FAST

Click here for FAST press release, ULTRAGENYX press release and FAQs.