Technology
SCIENTIFIC PRESENTATIONS
NACFC 2022
Update on AAV-mediated CFTR gene delivery programme (4D-710)
Jennifer L. Taylor-Cousar, et al. Presented at the the North American Cystic Fibrosis Conference (NACFC), November 3, 2022.
Plenary 2: Novel CF Therapeutics & Clinical Trial Strategy to Accelerate Our Mission
Deepika Polineni, MD, MPH, and Nicole Mayer-Hamblett, PhD Presented at the the North American Cystic Fibrosis Conference (NACFC), November 4, 2022.
WORLDSymposium 2022
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males with Fabry Disease
Jerry Vockley, et al. Presented at the 18th Annual WORLDSymposium Meeting, February 9, 2022.
ASGCT 2021
A Targeted AAV Gene Therapy Product Candidate, 4D-310, for the Treatment of Fabry Disease: Intravenous Biodistribution, Transgene Expression & Safety in Non-Human Primates
Kevin Wittlesey, et al. Presented at the 24th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 11-14, 2021.
A Multi-Mechanistic Anti-Angiogenic AAV Gene Therapy Product Candidate, 4D-150, for the Treatment of Wet Age-Related Macular Degeneration (Wet AMD) and Diabetic Macular Edema (DME): Intravitreal Biodistribution, Transgene Expression, Safety and Efficacy in Non-Human Primates
Peter Francis, et al. Presented at the 24th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 11-14, 2021.
ATS 2021
Identification and Characterization of a Novel AAV Capsid & Product for the Treatment of Cystic Fibrosis Lung Disease
Melissa A. Calton, et al. Presented at the American Thoracic Society (ATS) 2021 Virtual Conference, May 14-19, 2021.
ASGCT 2020
Directed Evolution of AAV Targeting Lung Epithelia Using Aerosol Delivery Identifies 4D-A101, a Variant Demonstrating Robust Gene Delivery in Non-Human Primates
Melissa Kotterman, et al. Presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.
In Vitro Fabry Disease Correction in Patient iPSC-Derived Cardiomyocytes and Endothelial Cells Using an Evolved and Optimized AAV Gene Therapeutic (4D-310)
Chris Schmitt, et al. Poster presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.
Highly-Evolved Novel AAV Gene Therapy Directly Addresses Fabry Disease Pathology In Vivo by Cell Autonomous Expression in the Heart and Other Target Organs
Kevin Wittlesey, et al. Poster presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.
4D-C102, a Novel Muscle-Tropic AAV Variant Demonstrates Superior Gene Delivery in Cardiac and Skeletal Muscle Tissues Versus Wild-Type AAV in Human Cells and Non-Human Primates
Melissa Kotterman, et al. Poster presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.
