Videos

In observance of World Amyloidosis Day, the cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University discussed the changing landscape of care in this field.

In observance of Epidermolysis Bullosa Awareness Week, the debra of America representatives discussed unmet needs for the patient population and the approval of the first EB gene therapy.

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed preclinical research on overcoming limitations of Treg cells through genetic engineering.

The professor in residence of pediatrics at University of California San Francisco discussed a potential long-term consequence of gene therapy and myeloablative conditioning.

The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed the design of the MyPeak-1 clinical trial for TN-201 and the associated MyCLIMB natural history study.

The associate professor of ophthalmology at Harvard Medical School discussed investigational corneal transplant therapy.

The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed TN-201, an investigational gene therapy for MYBPC3-associated HCM.

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.

The chief research officer of the Muscular Dystrophy Association discussed research needs that remain in the field in light of recent progress and approvals.

The associate professor of clinical ophthalmology at University of Miami discussed his experience using the gene therapy in a compassionate use program.

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.

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.

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.

The senior partner at Retina Vitreous Associates Medical Group discussed efficacy and safety data from the phase 2 AAVIATE trial.

The professor of medicine and pediatrics at Washington University in St. Louis discussed future challenges and exciting research in the sarcoma field.

The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.

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.

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.

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.

The chairman, chief executive officer, and cofounder of Ocugen discussed unmet needs within retinitis pigmentosa and the potential of OCU400 gene therapy.

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.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed recent and upcoming milestones in the field.

The chief scientific officer of CureDuchenne discussed progress in the field so far and upcoming milestones.

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.

The chief scientific officer at Omega Therapeutics discussed the issues with other modes of therapies that OEC therapy may address.

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.

The chief research officer, Muscular Dystrophy Association, shared a message for Duchenne Action Month.

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.