Zollie Yavarow, PhD, MA’s Post

Our latest #ScienceSimplified highlights the differences between a gene therapy clinical trial and a cure. 🧬💜 Read the latest article authored by Zollie Yavarow, PhD, MA here: https://bit.ly/3xiF152 #SLC13A5Epilepsy #TESSResearch

Did you miss our latest Masterclass session? Watch the recording to learn more about the implications of gene therapy and how you can prepare. Special thanks to the Global Alliance of Sickle Cell Disease Organizations (GASCDO) and our esteemed panelists. View more:

So exciting to see this Phase I/IIa data on wet AMD NAV® AAV8-based therapy. Manufacturability of gene therapies is key for serving patient populations, and process understanding can help ensure successful manufacturing runs through clear understanding of design space, trajectory correction and cost control. #AAV #genetherapy #processdevelopment #processoptimization

Muscular Dystrophy Association Chief Research Officer, Sharon Hesterlee, PhD, shared what we have to look forward to at the upcoming 2024 MDA Clinical and Scientific Conference, March 3-6 in Orlando, FL. Our focus includes an ongoing series on practical considerations for #GeneTherapy, exploring organizational practices, lessons learned, and pathways to success. Learn more about what to except in this CGTLive feature, and register today at MDAconference.org #MDAconference

At #ISCT2024 today? Check out poster #1140 to learn how Pin-point™ base editing technology is advancing gene therapy. Learn more about Pin-point http://ms.spr.ly/6041Y9U7L

Writing about severely sick infants is always heart breaking. Therefore it's a pleasure to share the story of a patient with SMA, who got the chance to receive gene therapy. Read my newest paper! https://lnkd.in/g4-Kgqh7

For World Hemophilia Day, CGTLive took a look at how the #hemophilia field has adapted to the introduction of gene therapy to the treatment landscape. Read more about what's changed - and what hasn't - since. https://lnkd.in/e3pfrkS2

Gene therapy in Lipid metabolism and CVD lecture .

The future of gene therapy is now.

Interested in the current state of FDA approvals for cell and gene therapies, CMC-related challenges and the specifics of AAV-based gene therapy? Parexel Regulatory Strategy's Rajiv Gangurde discusses these topics and more in a two-part series with Shurig Solutions, Inc. Check out both episodes: https://lnkd.in/gEC54AJ2 https://lnkd.in/gHdyxMD4