REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO’s AAV Therapeutic platform, including Novartis’ ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people.
REGENXBIO的外部链接
9804 Medical Center Dr
US，Maryland，Rockville，20850
Join REGENXBIO’s Chief Medical Officer, Steve Pakola, MD, and EVP of Commercial Strategy & Operations, Ram Palanki at #OISXIV as they discuss suprachoroidal drug delivery and innovation in eye care.
?????????? ?????? ?????? ????????! The countdown is on for 2 incredible days of innovation, insights, and networking—kicking off this Friday! We can’t wait to see you there. Let’s make it unforgettable! https://ois.net/ois-xiv/ #OIS #OISXIV #Ophthalmology #Innovation
Congratulations, Dr. Muenzer and team, for the grand opening of the UNC Muenzer MPS Research & Treatment Center. We applaud your achievements and your commitment to the MPS community!?
Our new UNC Muenzer MPS Research and Treatment Center in Chapel Hill aims to help more children like Cole, who is featured in this video. He has a form of mucopolysaccharidosis (MPS), a rare genetic disorder that can cause physical and neurological symptoms. His mom, Kim Stephens, drove 5.5 hours for treatment from Dr. Joseph Muenzer, MD, PhD, one of the leading MPS experts and the Bryson Distinguished Professor of Pediatrics. Thanks to a $10 million gift from Vaughn and Nancy Bryson, the Muenzer MPS Research and Treatment Center is now open. The center will focus on providing expert care and advancing research to develop new therapies. More info: https://lnkd.in/e7FVPHkd https://lnkd.in/eMSeEMgW
Our REGENXBIO Manufacturing Innovation Center was designed to accelerate product development and bring life-changing medicines to patients as quickly as possible. With full, end-to-end capabilities in-house, we’re realizing this vision—reducing supply lead times, and streamlining drug development. A big thank you to the OPTIMA packaging group team for partnering with us to deliver this industry-leading technology for gene therapies!
At REGENXBIO, we often talk about how “what we do matters” - to patients, to their families, and to their communities. Hear from Marianne Kreiner, Sr. Director, HR Business Partner, as she reflects on what that means, not only for the communities we serve, but also for our people.
Join Jahannaz Dastgir and Parent Project Muscular Dystrophy to hear more about our recent updates from our AFFINITY DUCHENNE trial.
Please join PPMD and REGENXBIO on Wednesday, November 20th at 1 PM ET for a community webinar, “RGX-202: AFFINITY DUCHENNE Pivotal Program and Functional Data.” REGENXBIO will provide an update on its AFFINITY DUCHENNE trial and share interim clinical data for RGX-202, a next-generation microdystrophin gene therapy. PPMD's VP of Clinical Care & Education, Rachel Schrader, MS, APRN, CPNP-PC, and REGENXBIO’s Dr. Naz Dastgir will discuss the company’s pivotal trial plans, explore the first functional data for RGX-202, and offer the Duchenne community an opportunity to ask questions. Register today and submit your questions in advance: https://lnkd.in/effJ_vGH
Today, we announced that AFFINITY DUCHENNE, our potential best-in-class gene therapy for Duchenne, has advanced to pivotal stage and reported positive functional data. Learn more: https://lnkd.in/efSsHHws
Today, we announced that we will be hosting a webcast to discuss our AFFINITY DUCHENNE pivotal program and new clinical data, including the first functional data from the ongoing Phase I/II study of RGX-202 for the treatment of Duchenne. Learn more: https://lnkd.in/eJNrBekb
This Veteran’s Day, we pause to honor the brave men and women who have served in the armed forces. Your courage, dedication, and sacrifice have protected our freedom and shaped our nation. To our veterans and their families - thank you for your service and for the countless ways you continue to inspire us.?
REGENXBIO reports third?quarter 2024 financial results and recent operational highlights. Learn more: https://lnkd.in/eq3Pszxs
REGENXBIO will participate in upcoming investor conferences. Learn more: https://lnkd.in/eVgWM7yH