Juan Francisco Cabello, MD, on How Gene Therapy Fits Into the Global Initiative for Newborn Screening
The head of the Pediatric Neurology Fellowship Program at the University of Valparaiso in Chile discussed how a lack of cost-effectiveness may hold back the potential of newborn screening in gene therapy.
Sharon Hesterlee, PhD, on Unprecedented Progress in Muscular Dystrophies
The chief research officer, Muscular Dystrophy Association, shared a message for Duchenne Action Month.
Juan Francisco Cabello, MD, on the History and Current State of Newborn Screening
The head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile discussed positive impact of newborn screening so far and work that still needs to be done.
Pamela Garzone, PhD, on Exploring Unknowns in CAR-T for Solid Tumors
The chief development officer of Anixa Biosciences discussed the company’s trial of a CAR-T in ovarian cancer that is looking at the effect of regional administration and lymphodepletion on efficacy.
Fiona Freeman, PhD, on Investigating miRNA-29b in Osteosarcoma Models
The assistant professor at University College Dublin discussed challenges and different approaches to using microRNA in preclinical models of osteosarcoma.
Pamela Garzone, PhD, on Tackling Challenges of Treating Solid Tumors With CAR-T
The chief development officer of Anixa Biosciences discussed how the company is tackling 2 major challenges presented by solid tumors in its ovarian cancer clinical trial.
Robert J. Hariri, MD, PhD, on the Unique Potential of Chimeric Therapy
The founder, chairman, and CEO of Celularity shared his view on the concept of “chimeric vigor” and the potential benefits arising from it in the cell therapy field.
Thomas Povsic, MD, PhD, on Addressing Unmet Needs in Refractory Angina With Gene Therapy
The interventional cardiologist and professor, Duke University School of Medicine, discussed how XC001 could improve outcomes for patients with refractory angina.
Robert J. Hariri, MD, PhD, on Cord Blood Awareness Month 2023
The founder, chairman, and CEO of Celularity shared his view on the importance of cord blood-derived cells for the medical and clinical community.
Robert J. Hariri, MD, PhD, on the Importance of Placental-Derived Stem Cells in Cell Therapy
The founder, chairman, and CEO of Celularity discussed the advantages of cells derived from umbilical cord blood versus other sources of cells for cell therapies.
Thomas Crawford, MD, on Bridging the Gap Between Screening and Treatment for Infants With SMA
The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the importance of connecting infants with spinal muscular atrophy to a therapy as soon as possible.
Richard Fessler, MD, on OPC1 Improving Quality of Life With Spinal Cord Injury
The professor of neurosurgery at Rush University Medical School discussed follow-up in the phase 1/2 trial of LCTOPC1 and further research to be done.
David Boyer, MD, on Potential Advantages of Suprachoroidal Gene Therapy for Neovascular AMD
The senior partner at Retina Vitreous Associates Medical Group discussed new findings from the AAVIATE study of RGX-314.
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.
Dilsher Dhoot, MD, On Reducing Treatment Burden in Diabetic Retinopathy With Gene Therapy
The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed unmet needs with the current treatment landscape for DR.
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.
Brian Van Tine, MD, PhD, on Progress and Challenges With Cell Therapy for Sarcoma
The professor of medicine and pediatrics at Washington University in St. Louis discussed the current investigative landscape for cell therapy in treating sarcomas.
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.
J. Andrew Livingston, MD, on Potential Advantages of TCR NK Therapy for Synovial Sarcoma and MRCL
The associate professor at MD Anderson Cancer Center discussed challenges with developing cell therapy for sarcoma and how TCR NK therapy's unique characteristics may prove useful.
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.
