Videos

The associate professor of medicine, Medical College of Wisconsin, discussed advantages of afami-cel and data from the pivotal SPEARHEAD-1 trial.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed emphasized topics at this year's ASGCT meeting.

The director of Cell Therapy and Transplant at Penn Medicine discussed important considerations with administering CAR-T in a new field.

The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed the rapid advancements in RNA-based treatments in the past 2 decades and potential advancements that remain on the horizon.

The chief of cardiology at Weill Cornell Medical College discussed gene therapy research into Friedreich ataxia and more.

The professor from Cedars Sinai discussed how the cell therapy stands to revolutionize the treatment landscape.

The head of the early neurodevelopment clinic at Monash Children's Hospital discussed preclinical work in the context of neonatal cell therapy research.

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

The chief scientific officer of CureDuchenne discussed challenges to tackle in the field.

The lead scientist at Percheron Therapeutics discussed the design of the study assessing proteomics of ATL1102.

The Assistant Professor of Medicine at Weill Cornell Medical College discussed Orca-T cell therapy and next steps assessing it.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed his research career with muscular dystrophies.

The assistant attending physician and bone marrow transplant specialist at Memorial Sloan Kettering Cancer Center discussed updated data on GDA-201 plus rituximab.

The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the latest results announced from the phase 2 portion of the PRISM study.

The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the need to set standard guidelines regarding potential fertility issues for CAR-T treatment.

The professor of microbiology, immunology, and molecular genetics at UCLA discussed how a better understanding of treatment impact on a cellular level could help improve future gene therapy approaches.

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.

The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed talks from a session on dysfunction and trafficking at the 2024 MDA Conference.

The lead of preclinical research at Amylyx Pharmaceuticals discussed the literature supporting AMX0114’s target and its upcoming trial design.

The postdoctoral scholar at University of California – Irvine discussed his research looking at TDP-43 and alternative polyadenylation in neuronal cells.

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed investigations into liver toxicity and deaths after AT132 gene therapy.

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.

The lead scientist at Percheron Therapeutics discussed how ATL1102 could target inflammation as a secondary cause of muscle damage in DMD.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the difference between developing targeted therapies for well-understood diseases like SCD and less well-understood diseases like MS.

The director of Cell Therapy and Transplant at Penn Medicine discussed how experience with CAR T in oncology has enabled the use of the technology in autoimmune diseases.

The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed past, present, and potential future applications of siRNA in the treatment of various diseases.

The Assistant Professor of Medicine at Weill Cornell Medical College shared the background rationale of her retrospective analysis presented at Tandem 2024.

In observance of Rare Disease Day 2024, the vice president of public policy and advocacy at the Muscular Dystrophy Association discussed advocacy and policy progress regarding rare neuromuscular diseases.