The Howard Hughes Investigator at the University of Iowa discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.
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The research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.
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The senior vice president of genetic medicines at Syneos Health discussed the importance of end-to-end thinking and collaboration when bringing gene therapies from development to the market.
The medical doctor and scientist at Bambino Gesù Chidren’s Hospital discussed phase 1/2 clinical trial data she presented at ASGCT’s 2024 Meeting.
The medical director of clinical development at AskBio discussed safety and efficacy data from a phase 1 trial of AB-1002.
The chair of the Department of Otolaryngology—Head and Neck Surgery at Columbia University College of Physicians discussed the phase 1/2 CHORD clinical trial evaluating DB-OTO.
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The associate investigator of neurology at Massachusetts General Hospital discussed research on extracellular vesicle-associated AAVs presented at ASGCT.
The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.
The senior research scientist at RTI International discussed how patient preference studies can help patients with rare diseases have their voices heard.
The professor of medicine at Baylor College of Medicine discussed research with NK-T cells and alternatives to αβ T-cells.
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The pediatric neurologist at Nemours Children’s Health offered advice and discussed his experiences delivering pediatric gene therapy.
The senior research scientist at RTI International discussed the progress that has taken place in recent years with regard to patient preference research.
The vice president of ASGCT discussed important research that will be represented at the upcoming conference and challenges for the field to address in the future.
The director of the Mount Sinai Fuster Heart Hospital discussed trends in research and challenges with adopting new therapies.
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington gave a background on ASGCT, its founding, and how far the field has come since.
The medical director of the Center for Inherited Blood Disorders discussed barriers to the widespread adoption of val-rox by the hemophilia A community.
The chief of cardiology at Weill Cornell Medical College shared his outlook on the future of gene therapy research in the field.
The medical director of the Center for Inherited Blood Disorders discussed the entrance of BioMarin’s val-rox into his center’s practice.
The professor from Cedars Sinai discussed data he is anticipating from further investigations into lifileucel.
The President Elect of ASGCT and a distinguished professor of microbiology at Keck School of Medicine of USC also discussed recent milestones in gene therapy.
The lead scientist at Percheron Therapeutics discussed a phase 2 blinded study of ATL1102 being conducted in the Europe and Australia.
The Assistant Professor of Medicine at Weill Cornell Medical College discussed the changing cell transplant landscape.
The principal investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center discussed innovative uses of gene editing technology.
The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital also discussed hurdles to accessibility for SCD gene therapy.
The associate professor of dermatology at Stanford University discussed how the May 2023 approval of B-VEC may shift the treatment field for RDEB.
The medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center discussed results from a phase 1b trial for allo-HCT alternative Orca-T that he presented at Tandem’s 2024 conference.
The postdoctoral scholar at University of California – Irvine discussed research aiming to link genetic variants and neurodegeneration.
The infectious diseases specialist at Memorial Sloan Kettering Cancer Center discussed the session she chaired for the Infectious Diseases Track at Tandem’s 2024 Meeting.
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed highlights from the meeting.
The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from an MDA session on rare diseases.
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