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
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
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
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
Barb Bailus, PhD It may seem odd that FAST has added an HIV expert to the SAB, but Dr. Joseph Anderson also specializes in stem cells and unique delivery methods for biological therapeutics. Dr. Anderson received his PhD from Colorado State University, Fort Collins. Dr. Anderson currently has a laboratory at University of California, Davis
Barb Bailus, PhD As FAST funded science moves toward clinical trials, it has become essential to add SAB members that are familiar with the process of human clinical trials and FDA regulations. As this need became more apparent, Dr. Allyson Berent reached out to Dr. Lauren Black who had advised Dr. Timothy Yu on the Milasen Batten Disease
Barb Bailus, PhD With the introduction of the newest AS animal model, the FAST funded LEGEND-rat, it was time to add “rat whisperer” Dr. Jill Silverman to the SAB. This new rat model, which was described in detail at the 2018 FAST gala, was behaviorally characterized by Dr. Jill Silverman, Dr. Ed Weeber and Dr.
Barb Bailus, PhD Dr. Cristina Alberini is an international addition to the FAST SAB, hailing from Italy. Dr. Alberini received her PhD from University of Genoa, Italy. She did her postdoctoral work at Columbia University focusing on learning and memory, using sea slugs as her model organism. Since sea slugs are not a common disease
Barb Bailus, PhD FAST is always on the lookout for excellent physicians who interact with AS patients and can help design and explain what is feasible in a clinical trial. Dr. Jessica Duis is one such physician, and is currently the medical director of the Angelman Syndrome comprehensive clinic at Vanderbilt Children’s Hospital in Tennessee. Dr.
Barb Bailus, PhD Dr. Mark Zylka is so new to FAST that he hasn’t yet joined us at a FAST Gala, but that is something that should change this year. He did listen to all 11 hours of the FAST Science Summit in 2018, which inspired him to work more closely with the mission of