Taking a Second Look at Placental-Derived Mesenchymal-Like Adherent Stromal Cell Therapy for Crohn Disease
Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed a new analysis of gene and protein signatures from patients treated in the company’s legacy clinical trials.
Paul Harmatz, MD, on Challenges With Assessing Neurocognitive Outcomes
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges with clinical trials in neurocognitive diseases including MPS2.
mRNA-engineered iPSC-derived Cell Therapies Hold Potential for Tackling Solid Tumors
Kyle Garland, PhD, a senior scientist at Eterna Therapeutics, discussed the company’s preclinical work with partner Factor Bioscience that was presented at ASGCT’s 2023 conference.
Adrian Kilcoyne, MD, MPH, MBA, on Adjusting Expectations for Remission Rates in Non–CAR-T Cell Therapy Trials
The chief medical officer of Cellularity discussed the big picture implications of a new analysis of patient samples from legacy studies evaluating MLASC therapy in Crohn disease.
Adrian Kilcoyne, MD, MPH, MBA, on Reevaluating Placental-Derived Mesenchymal-Like Adherent Stromal Cell Therapy for Crohn Disease
The chief medical officer of Celularity discussed MLASC and a new analysis of gene and protein signatures from patients treated in legacy clinical trials that he presented at ASGCT’s 2023 conference.
Stem Cell Prime Editing May Provide Novel Treatment Approach for p47phox Chronic Granulomatous Disease
Jennifer Gori, PhD, vice president of research at Prime Medicine, discussed what distinguishes prime editing from other forms of gene editing.
Maria Pia Morelli, MD, PhD, on Promising Safety of Sleeping Beauty TCR-T Therapy in Solid Tumors
The assistant professor at MD Anderson Cancer Center discussed progress of the phase 1/2 trial being conducted at the center.
Binod Dhakal, MD, on Benefit of Cilta-Cel in Earlier Lines of Multiple Myeloma Treatment
The associate professor at Medical College of Wisconsin discussed current studies evaluating cilta-cel and ongoing research.
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.
AAV Immuno-Gene Therapy May Hold Untapped Potential in Oncology Indications
Nicole Paulk, PhD, the CEO, founder, and president of Siren Biotechnology, discussed research she presented at ASGCT’s 2023 conference.
Gene Therapy After Umbilical Cord Blood Transplantation May Help Prevent Immune Response in Patients With Krabbe Disease
Maria Escolar, MD, the chief medical officer of Forge Biologics, discussed the atypical approach being used in the company’s trial for AAV gene therapy FBX-101.
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.
John Leonard, PhD, on RNA-Targeting Gene Therapy for ALS, DMD
The chief scientific officer of LocanaBio discussed preclinical research presented at ASGCT 2023.
Synthetic Promoters May Help Reduce Off-Target Expression in AAV Gene Therapy
Magdalena Cichewicz, PhD, a scientist II at Senti Biosciences, discussed preclinical research she presented at ASGCT’s 2023 conference.
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.
ARCUS–Mediated Gene Editing Shows Promise for Duchenne Muscular Dystrophy In Preclinical Work
Gary Owens, MS, the associate director for gene therapy discovery at Precision Biosciences, discussed data he presented at ASGCT’s 2023 conference.
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.
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.
Hope Remains for CAR-T Approaches in Ovarian Cancer
Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, discussed past and current clinical trials for investigational CAR-T therapies in ovarian cancer.
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.
