Ula V. Jurkunas, MD, on Developing CALEC Transplant for Limbal Stem Cell Deficiency
The associate professor of ophthalmology at Harvard Medical School discussed investigational corneal transplant therapy.
Milind Desai, MD, MBA, on Addressing Unmet Needs in MYBPC3-associated Hypertrophic Cardiomyopathy
The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed TN-201, an investigational gene therapy for MYBPC3-associated HCM.
David Rawlings, MD, on Research That Supports the Development of Engineered B-cell Therapies
The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed findings from several preclinical studies that could help bring engineered B-cell therapies to clinical trials.
Sharon Hesterlee, PhD, on Further Research Needs With Muscular Dystrophies
The chief research officer of the Muscular Dystrophy Association discussed research needs that remain in the field in light of recent progress and approvals.
Richard James, PhD, on the Potential of Engineered Plasma Cells in Cancer
The associate professor at the University of Washington, and principal investigator at Seattle Children's Research Institute discussed results from a leukemia mouse model.
Alfonso Sabater, MD, PhD, on Treating Ocular DEB With B-VEC
The associate professor of clinical ophthalmology at University of Miami discussed his experience using the gene therapy in a compassionate use program.
Nadezhda Omelchenko, MD, on Treating Rare Sarcomas With Autologous Natural Killer Cell Therapy
The research associate at Cancer Center of Southern California in Santa Monica discussed findings from 3 patients treated with a combination therapy that included NK cell therapy SNK01.
Thomas Povsic, MD, PhD, on Ongoing Research With XC001 Angina Gene Therapy
The interventional cardiologist and professor, Duke University School of Medicine, discussed other populations that may benefit from XC001 and the challenges and opportunities with a surgical delivery of treatment.
Thomas Crawford, MD, on Taking a Closer Look at Nusinersen Data
The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the updated analyses of the NURTURE study that affected the big picture of the data.
David Boyer, MD, on Promising Efficacy of Suprachoroidal Gene Therapy in nAMD
The senior partner at Retina Vitreous Associates Medical Group discussed efficacy and safety data from the phase 2 AAVIATE trial.
Brian Van Tine, MD, PhD, on Looking Ahead on Cell Therapy for Sarcomas
The professor of medicine and pediatrics at Washington University in St. Louis discussed future challenges and exciting research in the sarcoma field.
Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314
The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.
Deborah Phippard, PhD, on the Future of Gene Therapy in Neurology
The chief scientific officer of Precision for Medicine discussed what lies on the horizon for gene therapies directed at neurological indications, such as Alzheimer disease and Parkinson disease.
J. Andrew Livingston, MD, on Forging Forward With Novel Sarcoma Trials
The associate professor at MD Anderson Cancer Center discussed the upcoming trial of NY-ESO-1 TCR/IL-15 NK cell therapy for SS and MRCLS.
Deborah Phippard, PhD, on Designing Clinical Trials for Gene Therapies in Neurological Indications
The chief scientific officer of Precision for Medicine discussed the unique aspects of clinical trials for gene therapies that need to be considered before initiation.
Shankar Musunuri, PhD, on Gene Modifier Therapy vs Gene Replacement Therapy for IRDs
The chairman, chief executive officer, and cofounder of Ocugen discussed unmet needs within retinitis pigmentosa and the potential of OCU400 gene therapy.
Deborah Phippard, PhD, on the History and Current State of Gene Therapy for Neurological Indications
The chief scientific officer of Precision for Medicine discussed the previous and current delivery methods used for gene therapies and new innovations on the horizon.
Pat Furlong, BSN, RN, on the State of Gene Therapy in Muscular Dystrophy
The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed recent and upcoming milestones in the field.
Michael Kelly, PhD, on Continuing Progress With Gene Therapy in Muscular Dystrophy
The chief scientific officer of CureDuchenne discussed progress in the field so far and upcoming milestones.
Sebastian Michels, MD, on the Role of Alternative Polyadenylation in ALS Disease Pathogenesis
The postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed findings he presented at ANA’s 2023 conference that may have implications for future RNA therapy development.
Thomas McCauley, PhD, on Potential Advantages of Epigenetic Therapy Over Small Molecule, Gene Therapy
The chief scientific officer at Omega Therapeutics discussed the issues with other modes of therapies that OEC therapy may address.
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.
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