American Society for Gene & Cell Therapy (ASGCT)

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.

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.

The vice president of cell & gene therapy product development and the vice president of genetic medicine platforms at Pacira discussed the company’s symposium at ASGCT 2025.

The vice president of cell & gene therapy product development at Pacira BioSciences discussed findings related to the immunogenicity of the therapy’s high-capacity adenovirus vector.

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, also discussed the Institute’s presentations at ASGCT’s 2025 conference.

Nicholas Giovannone, PhD, a senior principal scientist at Regeneron, also discussed the problem of preexisting immunity caused by natural exposure to AAVs.

Nicholas Giovannone, PhD, a senior principal scientist at Regeneron, discussed an advanced approach to enabling gene therapy redosing.

Kiran Musunuru, MD, PhD, and Rebecca Ahrens-Nicklas, MD, PhD, physician-scientists at CHOP, discussed long-term future expectations for personalized gene editing therapy.

The CEO of Mission Bio also discussed the company’s plans for collaboration.

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, physician-scientists at CHOP, discussed what lies on the horizon after KJ’s treatment with a CRISPR-based gene editing therapy for CPS1 deficiency.

Kiran Musunuru, MD, PhD, and Rebecca Ahrens-Nicklas, MD, PhD, physician-scientists at CHOP, discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

The senior research scientist at Seattle Children’s Research Institute discussed preclinical work on using dimerizing agent-regulated immune-receptor complex T-cells to target plasma cells.

Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.

The senior principal scientist at Regeneron also discussed the problem of preexisting immunity caused by natural exposure to AAVs.

Nathan Yozwiak, PhD, the head of research at the Gene and Cell Therapy Institute at Mass General Brigham, discussed how innovative research can be held back by financial considerations.