[PRNewswire] FOUNDATION FOR ANGELMAN SYNDROME THERAPEUTICS (FAST) ANNOUNCES $5

MILLION GIFT TO ADVANCE CLINICAL TRIALS IN RARE NEURODEVELOPMENTAL DISORDERS

-- The Center at Rush University Will Be Directed By Dr. Elizabeth Berry-Kravis

(MIAMI, Dec. 2, 2022 PRNewswire=연합뉴스) The Foundation for Angelman Syndrome Therapeutics (FAST) announced today a $5 million gift to establish the new clinical trial and translational research effort for rare neurodevelopmental disorders, a first-of-its-kind flagship center to be directed by Dr. Elizabeth Berry-Kravis. Named the Rush F.A.S.T. Center for Translational Research, it will be the global headquarters for training individuals in how to run neurogenetic clinical trials and deliver innovative interventional therapies that require novel delivery methods and specialized care.

Many of the doctors working on neurogenetic disorders are running excellent general and specialized care clinics for specific subsets of disorders, but are not trained in the intricacies of setting up, running, and reporting on clinical trials-which means the absence of specific training to expeditiously execute trials is significantly limiting trial enrollment capabilities.

"This is the dawn of a new era. Today there are over 25 therapeutic programs in the development pipeline for Angelman syndrome, with a majority robustly funded by FAST," said Dr. Allyson Berent, chief science officer at FAST. "We are at a turning point, where we are ready for many of these programs to reach human patients for early stage first-in-human clinical trials, but most hospital centers don't have the bandwidth to keep up with this exploding need."

To answer this need, the new center-under the leadership of Dr. Berry-Kravis, who has been leading clinical trials for over 20 years-will be the first organized training program for individuals focusing on clinical trial execution and novel drug delivery for rare neurogenetic disorders. By establishing a formal fellowship program for both national and international candidates, physicians will be trained in: understanding the steps it takes to onboard a prospective clinical trial; the regulatory hurdles required to get through contracts and ethics approvals; challenges in setting up infrastructure for patient testing, and putting together the specialized clinical teams required for these trials (e.g. anesthesia, trial coordinators, trained neuropsychologists, neurologists/epileptologists, neurosurgeons, and any other specialists relevant to a specific trial), and more. In addition, this program will support the infrastructure and buildout needed to create a state-of-the-art facility at Rush, with clinical trial space built around the needs of this unique patient population.

This FAST trials center will put all these pieces under one roof as a model of how to most efficiently and effectively execute a clinical trial-enabling Dr. Berry-Kravis to formally share her expertise with others, and to help to grow the capabilities at dozens of additional centers globally. When a FAST fellow completes their training, they will be able to bring this expertise to other institutions around the world. To support this, FAST is committing to financially support the upgrading of current, and the establishment of new, clinical trial centers, to arborize this expertise to ensure the capabilities of numerous centers can meet the huge demand.

FAST fellows will have hands-on training and be integrated into the Center for a full year, focusing entirely on running clinical trials for NDDs. In addition to the fellows, the Rush F.A.S.T. Center will also train externs in shorter stints to learn about drug delivery, advance their own skill sets, and train in efficiencies to bring back to their own institutions to better deliver interventional therapeutics to more patients in the safest and most efficient ways.

"We are on the cusp of so many vital breakthroughs, which is why it is an honor to be named director of the Rush F.A.S.T. Center at such a critical time," Dr. Elizabeth Berry-Kravis. "This is going to help so many people, and I am looking forward to working with the brightest minds in the field to generate the medical breakthroughs of tomorrow."

Media Contact:

Eric Koch

617-733-6891

ericdkoch@gmail.com

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Source: Foundation for Angelman Syndrome Therapeutics

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