GeneTx Biotherapeutics, in partnership with Ultragenyx Pharmaceutical, recently announced that the Food and Drug Administration (FDA) has cleared GeneTx’s Investigational New Drug (IND) application for clinical trial. What does this mean? The United States Food and Drug Administration’s Investigational New Drug (IND) program is the means by which a Sponsor obtains permission to start human
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Live Stream of the Educational Tracks of the Global Science Summit
Join in today to watch the live stream of the educational tracks of the FAST Global Science Summit! Session A: AAC and Behavior Session B: Physical and Sensory Development
Live Stream of Global Science Summit
Join in today to watch the live stream of the FAST Global Science Summit!
An interview with Dr. David Segal on the UC Davis center and the future of gene editing as therapy
Barb Bailus, PhD David Gurzick, PhD Hello Everyone! Today we have a very special guest with us, Dr. Dave Segal! He is joining us to discuss the amazing new nine million dollar grant the University of California Davis just received to study the safety of gene editing tools. This grant is part of a much
Part II: CRISPR for Angelman syndrome
David Gurzick, PhD Barb Bailus, PhD In a recent post, we introduced gene editing technologies with a focus on CRISPR (haven’t seen it? – it’s available on the FAST blog). In this post we will focus on the applications of CRISPR in the gene editing field with specific attention on how this technology can help
FAST 2019 Ticket and Scholarship Winners
If you did NOT receive an email, but your name is listed below, please email kelly@cureangelman.org to claim your tickets for the 2019 FAST Gala. Names are alphabetical order by first name. If you are a CAN page winner, those tickets will replace the Giveaway tickets. Ticket Giveaway Winners Abbey Dunn Abigail Nite Alexandre Boucher
A message to the Angelman community from GeneTx Biotherapeutics
Two press releases were issued recently with one announcing an Exclusive Option Agreement between GeneTx Biotherapeutics, LLC (“GeneTx”) and Ultragenyx Pharmaceutical, Inc. (“Ultragenyx”), and the other announcing GeneTx’s lead candidate receiving Orphan Drug Designation (ODD) and Rare Pediatric Drug Designation (RPD). GeneTx would like to take this opportunity to provide additional information to our community about this
Part I: Introducing CRISPR, A Promising Gene Editing Technology
Barb Bailus, PhD David Gurzick, PhD Hello Everyone, Over the next few weeks, we are going to be looking at the technology behind many of the gene therapy techniques that are showing tremendous potential for application in Angelman syndrome. We’ll start with some of the background science and then dive in to talk about how
Following the Yellow Brick Road: The Path through Clinical Trials
Barb Bailus, PhD Hello Everyone! Today we are going to spend some time reviewing the various phases of drug discovery and clinical trials, which can allow for advancement of promising therapeutics into the clinic for patients. There are many variations on this scheme but this is the traditional approach. The very first phase of treatment
Congratulations Dr. Anne Anderson: Creating a Standard of Care Guideline for Angelman Syndrome
Barb Bailus,PhD Hello Everyone! Today we want to congratulate our FIRE team member Dr. Anne Anderson for being awarded a highly competitive Million Dollar Bike Ride Grant in 2018 from the Orphan Disease Center . Dr. Anderson was awarded a $45,000 grant available for Angelman Syndrome (AS) in the 2018 edition of the MDBR event. The Million
