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To date, FAST has funded over $50 million in research related to Angelman syndrome, and is poised to fund much more. How? By finding the gaps in the research landscape, advancing promising programs, and creating our own future by developing potential therapeutics for those living with Angelman syndrome as methodically, carefully, expeditiously as possible.

Rather than sit on the sidelines of the research and development game, FAST adopted a business model that enabled it to assess the landscape of potential research areas related to AS; seed that landscape through generous grants; have clear go-and-no-go decisions for funding; and, if and when lightning struck, shepherd new insights or technologies from the academic space to the clinical one—all the while maintaining input and control over the direction of the research. (You can read about our early days and how we came to this strategy here.)

We have had some very exciting early success, as you can see here. And visit here for a view on our whole current pipeline.

But in our effort to cure Angelman syndrome, we are up against two big challenges: drug development is very expensive, and we need many shots on goal to actually find meaningful options for our loved ones. We cannot stop at the first or the second idea when it’s our job to chase down every possible option to find  transformative treatments.

Our model of venture philanthropy involves recruiting a superior in-house team to work in partnership with leading scientists on ambitious, high-risk/high-reward ideas. Unlike other organizations, our grant process does not have a research deadline. Instead, we maintain a rolling application process—enabling us to support or scoop up promising ideas as soon as they surface, while being proactive and personally recruiting the best in the world with novel ideas to work on Angelman syndrome when they may not have otherwise.

We then engage aggressively in the biotech space to ensure potential therapeutics do not languish in the laboratory, on a shelf, or beyond—particularly in the “valley of death” of drug development, the period during which innovative medical research discoveries are made, generally in an academic lab, and yet somehow often never making it to clinical trials.

And sometimes, as we’ve learned, the best way to avoid losing a promising therapeutic—especially one we’ve funded from its inception, via one of our grants—is to build our own company around it. This allows us to guardrail the potential treatment through some of the toughest terrain of the drug development pipeline, namely from a human candidate drug, through INDenabling studies, and into early phase clinical trials. If it succeeds through all of these, it might then be an attractive asset for a biotech or pharmaceutical company that is big enough to take it through the most expensive and complicated final stages of testing, pivotal trials, all the way through to possible regulatory approvals and global commercialization. Human data is more powerful than any cellular or animal data, and that gives Angelman syndrome the closest guarantee to attract the best partners to advance a future therapy for all patients.

Drug research and development is a universe where risks, and challenges, abound. FAST gets new research opportunities  nearly every day, and when the time is right and the scientific data supports it, FAST will work to take the rights to a license, or option to take the license, to these promising technologies. This is the best way to ensure that each program is  advanced as a priority program, ensuring robust safety studies are performed, while acting expeditiously with aggressive timelines.

This also means some will be successful, and some will fail. Go and no-go decisions will always have to be made, and if we must pivot away from one program it will be with the utmost care, scientific rigor and responsibility. We spend enormous amounts of time and money to ensure that programs are safe, their results are solid. We are not afraid to invest more to ensure that we believe in the results we are seeing. This is what our loved ones living with AS deserve.

To top it all off, we also invest in a myriad of projects related to supporting all of the work that has to happen around drug development, both before and after, to ensure that it moves safely and quickly—from creating animal models and cell lines for each genotype to achieving alignment on endpoints and biomarkers, which are required to measure the effect a therapeutic has on patients in clinical trials. To read about FAST Ventures in detail, see below. To inquire about investing with FAST, please write to FAST President Alana Newhouse at

Angelman Syndrome News