Farah Sheikh, PhD, on Continuing Gene Therapy Research Into Arrhythmias, Cardiac Dysfunction
The professor of medicine at University of California San Diego discussed the advent of gene therapy into the field of cardiac medicine.
Thomas McCauley, PhD, on the Potential of Epigenetics for Therapy Development
The chief scientific officer at Omega Therapeutics discussed the importance of understanding more about and researching epigenetics.
J. Andrew Livingston, MD, and Brian Van Tine, MD, PhD, on Sarcoma Awareness Month 2023
The clinicians from MD Anderson Cancer Center and Washington University – St. Louis shared messages on sarcoma awareness.
Rebecca Cottman, PhD, on Enhancing Cell Therapy Cytotoxicity With a Regulated Gene Circuit
The scientist at Senti Biosciences discussed preclinical research presented at ASGCT 2023.
Fiona Freeman, PhD, on Benefits of miR-29b in Osteosarcoma Models
The assistant professor at University College Dublin discussed identifying miR-29b as a target for research in osteosarcoma.
Thomas Povsic, MD, PhD, on EXACT Clinical Trial: Investigating Gene Therapy for Refractory Angina
The interventional cardiologist and professor, Duke University School of Medicine, discussed trends of new data on XC001.
Thomas Crawford, MD, on Confirming Long-Term Efficacy of Nusinersen for SMA
The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed updated data from the NURTURE study.
John Leonard, PhD, on RNA-Targeting Gene Therapy for ALS, DMD
The chief scientific officer of LocanaBio discussed preclinical research presented at ASGCT 2023.
Maria Escolar, MD, on Treating Krabbe Disease With Investigational Gene Therapy FBX-101
The chief medical officer of Forge Biologics discussed updated data from the phase 1/2 RESKUE clinical trial that she presented at ASGCT’s 2023 conference.
Joseph Fraietta, PhD, on Achieving a Deeper Understanding of CAR T-Cell Therapy
The assistant professor of microbiology at Penn Medicine discussed further research to be done with EGR2 and type 1 interferon.
Olivier Danos, PhD, on Enhancing Gene Therapy for DMD
The chief scientific officer at REGENXBIO discussed RGX-202 and the ongoing clinical trial.
Magdalena Cichewicz, PhD, on Improving AAV Gene Therapy With Synthetic Promoters
The scientist II at Senti Biosciences discussed the potential of synthetic promoters to improve target specificity in AAV vector-based gene therapies in retinal diseases and beyond.
Paul Harmatz, MD, on Assessing D2S6 in Trials for MPS Type 2
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed biomarker findings from the phase 1/2 CAMPSIITE trial.
Gary Owens, MS, on the Potential of ARCUS–Mediated Gene Editing in Duchenne Muscular Dystrophy
The associate director for gene therapy discovery at Precision Biosciences discussed preclinical research he presented at ASGCT’s 2023 conference.
Carol Miao, PhD, on the Importance of Continuing Preclinical Research in Gene Therapy
The principal investigator at Seattle Children’s Research Institute discussed the continuing need to share preclinical research at conferences even as more gene therapies enter the clinic.
Francesca Barone, MD, PhD, on Viral Immunotherapy for Glioma, Prostate Cancer
The chief scientific officer at Candel Therapeutics discussed progress in investigations with CAN-3110 and CAN-2409.
Carol Miao, PhD, on Bringing Together Novel Gene Editing Tools and Delivery Methods
The principal investigator at Seattle Children’s Research Institute discussed the potential of new delivery methods and gene editing to overcome the limitations of AAV vector-based gene therapies.
Sung-Yun Pai, MD, on Further Research With Lentiviral Gene Therapy for X-SCID
The senior investigator at the National Cancer Institute Center for Cancer Research discussed unmet needs that remain and further research to be done.
Jacob Appelbaum, MD, PhD, on Investigating SC-DARIC33 and IL-15 for AML
The senior research fellow at Fred Hutch Cancer Center discussed research trends at the 2023 ASGCT meeting.
Mary Jane "MJ" Mulcahey, PhD, OTR/L, CPPC, CLCP, FASIA, on Reevaluating Outcome Measures for Spinal Cord Injury Clinical Trials
The director of the Center for Outcomes and Measurement in the Jefferson College of Rehabilitation Sciences at Thomas Jefferson University discussed the research she presented at the first Annual SCI Investor Symposium.
Farah Sheikh, PhD, on Developing Gene Therapy for Arrhythmogenic Right Ventricular Cardiomyopathy
The professor of medicine at University of California San Diego discussed preclinical data on the LX2020 gene therapy.
Krystof Bankiewicz, MD, PhD, on GDNF Gene Therapy for Parkinson’s and AADC Deficiency
The professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.
Jonathan Yen, PhD, on Continuing Research With Prime Editing for Sickle Cell Disease
The director of therapeutic genome engineering, St. Jude Children’s Research Hospital discussed challenges with performing prime editing in cells.
Brian Culley, MS, on Sharing Knowledge From the Development of Spinal Cord Injury Cell Therapy
The chief executive officer of Lineage Cell Therapeutics discussed findings related to its cell therapy OPC1 that the company presented at the first Annual SCI Investor Symposium.
Raphaël Ognar on Adding New Options to the Oncologic Cell Therapy Arsenal
The cofounder and chief executive officer of NKILT Therapeutics discussed unique advantages of the CIR platform.
Steven Katz, MD, on Assessing New Delivery Methods for CAR T-Cell Therapy
The chief medical officer of Trisalus Life Sciences discussed studies evaluating the company’s PEDD system.
Bella Neufeld, PhD, on Addressing Pain Points in AAV Gene Therapy Development
The director of research and development at Teknova discussed the company’s recent announcement of a new line of products intended to streamline process development in AAV gene therapy manufacturing.
Dan Shelly, PhD, on Developing an Allogeneic, Adaptable CAR Platform
The vice president of business development and alliances at Prescient Therapeutics discussed advantages of the OmniCAR, SpyTag, and SpyCatcher technologies.
Bella Neufeld, PhD, on Streamlining Buffer Selection for AAV Gene Therapy Manufacturing Process Development
The director of research and development at Teknova discussed the company’s new screening kit and its potential to speed up AAV gene therapy development.
Frank Borriello, MD, PhD, on Next Steps for SUPLEXA-101 for Solid Tumors
The scientific cofounder and chief executive officer of Alloplex Biotherapeutics discussed progress of the phase 1 trial and possible future evaluations of the therapy.
