Gene Therapy Initiative at UC San Diego

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This breakthrough gene therapy clinical trial for the rare disease cystinosis that is the subject of this California Institute for Regenerative Medicine (CIRM) blog was developed and conducted here at the University of California San Diego under the guidance of Stephanie Cherqui. The Gene Therapy Initiative at UC San Diego recognizes that courageous patients like Kurt are crucial for understanding the potential of gene therapies.

As Porter Gale, a renowned public speaker, networker, and entrepreneur, once said, "Your network is your net worth." That's why we're excited to offer registered attendees of our Pharma-Ready workshop on March 20 a unique networking opportunity following the presentations. The social hour is hosted by the Gene Therapy Initiative at UC San Diego and supported by Avid Bioservices. Don't worry if you missed out on this sold out event - we'll be announcing future GTI Workshops soon, and we look forward to helping you build a strong local network that can propel your drug development journey forward. Stay tuned for updates and get ready to tap into the power of your professional community! Sign up for the GTI Newsletter to be among the first to receive announcements for these popular events. https://lnkd.in/gVeQDA3j #genetherapy #gtiUCSD #networking Stephanie Cherqui Alysson Muotri Kenny Smart, MS, MBA Elie H.

#InternationalWomensDay ?? Join the Gene Therapy Initiative at UC San Diego in celebrating the talented clinicians, researchers, and innovators in our community who are pushing the boundaries of genetic therapy and transforming lives.? Their work is a shining example of the impact that women can have when given the opportunity to pursue their passions and make a difference in the world. We encourage you to give a shout out in the comments to women in the San Diego region who deserve recognition for their outstanding achievements Farah Sheikh , Stephanie Cherqui, Ester J. Kwon, Angels Almenar-Queralt, Vivian Hook, Ana Maria Moreno, Olivia Kim-McManus, Chamindra Laverty, Nicole Coufal, Annette von Drygalski #genetherapy #empowerment

On Rare Disease Day, the Gene Therapy Initiative at UC San Diego (GTI) wishes to recognize the San Diego Rare community that organized #SDRareDiseaseDay. The powerful statement by Rare Dad and advocate Paul Purdy, 'We may be fighting different battles, but we're fighting the same war,' resonates deeply with gene therapy researchers. Although they focus on various rare diseases, their shared goal is to harness the potential of gene therapy to alleviate the devastating impact of genetic disorders. The GTI is dedicated to arming scientists with the necessary expertise and resources to tackle this collective challenge and bring hope to those affected by rare diseases. Thanks to the Effie Parks (Once Upon A Gene), E. Gay Grossman & Steve Grossman (ADCY5.org), Jane Maine, CMP Meetings & Events for organizing this event. We met so many inspiring people this evening!

LaunchBio Inc. organized a fantastic rare disease impact investing forum in collaboration with UC San Diego Office of Innovation and Commercialization today. The Gene Therapy Initiative at UC San Diego was honored to be included among the innovative strategies that bolster rare disease research. The opening remarks from UCSD Vice Chancellor for Research, Corinne Peek-Asa, highlighted what a unique and collaborative environment the GTI is fortunate to work in at UCSD. The breaking news that our university is now ranked #8 in the WORLD for INNOVATION speaks volumes. #innovation #genetherapy #raredisease #diseasefoundations Stephanie Cherqui Alysson Muotri Betty Cabrera Rebecca Beattie

LaunchBio Inc. organized a fantastic rare disease impact investing forum in collaboration with UC San Diego Office of Innovation and Commercialization today. The Gene Therapy Initiative at UC San Diego was honored to be included among the innovative strategies that bolster rare disease research. The opening remarks from UCSD Vice Chancellor for Research, Corinne Peek-Asa, highlighted what a unique and collaborative environment the GTI is fortunate to work in at UCSD. The breaking news that our university is now ranked #8 in the WORLD for INNOVATION speaks volumes. #innovation #genetherapy #raredisease #diseasefoundations Stephanie Cherqui Alysson Muotri Betty Cabrera Rebecca Beattie

LaunchBio Inc. organized a fantastic rare disease impact investing forum in collaboration with UC San Diego Office of Innovation and Commercialization today. The Gene Therapy Initiative at UC San Diego was honored to be included among the innovative strategies that bolster rare disease research. The opening remarks from UCSD Vice Chancellor for Research, Corinne Peek-Asa, highlighted what a unique and collaborative environment the GTI is fortunate to work in at UCSD. The breaking news that our university is now ranked #8 in the WORLD for INNOVATION speaks volumes. #innovation #genetherapy #raredisease #diseasefoundations Stephanie Cherqui Alysson Muotri Betty Cabrera Rebecca Beattie

Happy Valentines Day! ?? If topics in gene therapy make your heart flutter, and you wish to meet others who share your passion, don't miss the March 20th workshop and networking hour hosted by the Gene Therapy Initiative at UC San Diego. https://lnkd.in/ghiapB25 #genetherapy #gtiucsd #workshop #vectormanufacturing #commercialization #intellectualproperty #benchtobedside

The Gene Therapy Initiative at UC San Diego (GTI) celebrates the announcement that came out earlier this year that UC San Diego Health is among the first in the western U.S. to offer a one-time infusion of gene therapy, Hemgenix, to hemophilia B patients. It takes immense dedication from clinicians, pharmacists and hospital support staff to coordinate this advanced care. UC San Diego Health also has the required infrastructure and institutional support to make this available to patients. The GTI works with scientists and clinician scientists on the UC San Diego campus to improve the odds that novel ideas?? for safe and effective gene therapies make it out from the lab and to patients. We look forward to the day that announcements like these are more commonplace and gene therapies are available for a variety of diseases with limited treatment options. #GeneTherapy #gtiUCSD https://lnkd.in/gNh3D-B3