It was noted that the CA19-9 reductions in the 5 patients observed after infusion of satri-cel ranged from 51.3% to 96.1%.
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The primary end point of the study was met.
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Jainu Jogani, the cofounder of Child’s Cure Genetic Research, discussed his daughter’s rare genetic disease and the need for new treatment options.
The cofounder of Child’s Cure Genetic Research discussed his daughter’s rare genetic disease and the need for new treatment options.
Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, discussed a trend of physicians and scientists working on models to make gene therapy products commercially viable.
The director of the Mass General Brigham Gene and Cell Therapy Institute discussed a trend of doctors and scientists working on models to make gene therapy products financially viable.
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The director of the Mass General Brigham Gene and Cell Therapy Institute discussed innovations in viral vectors, CAR-T, and more.
The trial, which is taking place in the United States, is expected to enroll 40 patients in total.
Daniel Galbraith, PhD, the chief scientific officer of Solvias, discussed potential uses for the company’s platform.
The chief scientific officer of Solvias discussed potential uses for the company’s platform.
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Robert Alexander Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, expressed optimism for RNA therapy's future.
The director of RNA therapeutics at MGB’s Gene and Cell Therapy Institute expressed optimism for RNA’s potential role in CAR T-cell therapy, gene editing, and more.
Alex Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, discussed the Institute's work in exploring the new modality.
The director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute discussed the Institute's work in exploring the new modality.
John Murphy, PhD, and Tia DiTommaso, PhD, of Arbor Biotechnologies, discussed ABO-101, the company’s gene therapy for primary hyperoxaluria type 1.
The chief scientific officer and senior director of preclinical pharmacology Arbor Biotechnologies discussed ABO-101, the company’s gene therapy for primary hyperoxaluria type 1.
Annaiz Grimm, BS, a research scientist at Seattle Children's Research Institute, discussed the potential use of EngTregs to treat autoimmune disease.
The research scientist at Seattle Children's Research Institute discussed a potential alternative to standard of care immunosuppressive therapy.
Damien Maura, PhD, a senior scientist at Voyager, discussed the company’s work on developing AAV capsids capable of overcoming patients’ preexisting immunity.
The senior scientist at Voyager Therapeutics discussed the company’s work on developing AAV capsids capable of overcoming patients’ preexisting immunity.
The postdoctoral research fellow at Brigham and Women's Hospital discussed research on focused ultrasound aimed at enhancing AAV delivery across the blood-brain barrier.
Rajeev Sivasankaran, PhD, vice president of neuroscience at Voyager Therapeutics, discussed preclinical data on the company’s AAV-delivered RNA interference therapy, VY-1706.
AAV2-BDNF gene therapy shows promise in preventing neuronal loss and cognitive decline in early Alzheimer disease, according to trial results.
The vice president of neuroscience at Voyager Therapeutics shared preclinical data on the company’s AAV-delivered RNA interference therapy, VY-1706.
Sven Moller-Tank, PhD, a senior director at Regeneron, also discussed his thoughts on trends in the gene therapy field in general.
Sven Moller-Tank, PhD, a senior director at Regeneron discussed the company’s receptor-driven approach to AAV targeting, leveraging antibody engineering to enhance tissue specificity and reduce off-target effects.
The senior director at Regeneron discussed the company’s receptor-driven approach to AAV targeting, leveraging antibody engineering to enhance tissue specificity and reduce off-target effects.
Nathan Yozwiak, PhD, the head of research at the Gene and Cell Therapy Institute at Mass General Brigham, discussed the Institute’s efforts to bring about clinical translation of preclinical work.
The head of research at Mass General Brigham’s Gene and Cell Therapy Institute discussed the Institute’s efforts to bring about clinical translation of preclinical work.
Derek Jackson, BS, MA, and Kilian Guse, PhD, of Pacira Biosciences, discussed the company’s symposium at ASGCT 2025.
