Videos

The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.

The associate chief science officer of the Kennedy Krieger Institute discussed an aspect of clinical trial design highly relevant to gene therapy development for rare diseases.

The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.

The associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.

The chief executive officer of Tenaya Therapeutics discussed the growing interest in genomic medicines in cardiology.

The chief executive officer of Tenaya Therapeutics discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.

The assistant professor of pediatrics at Children's Hospital of Philadelphia discussed the role of the FOXO1 gene in T-cell persistence and exhaustion.

The chief executive officer of Tenaya Therapeutics discussed the company’s research on capsids, promoters, and manufacturing improvements.

The cofounder, chairman, and CEO of Kriya Therapeutics discussed the company’s goal of bringing gene therapy to a much broader population of patients.

The research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.

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.

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.

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.