Blog

GeneTx Announces Launch of KIK-AS Clinical Trial

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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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

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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

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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

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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

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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

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