Videos

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

The CEO of Mission Bio discussed the company’s single cell assay platform in the context of the broader developing field cell and gene therapy field.

The physician-scientists at CHOP discussed long-term future expectations for personalized gene editing therapy.

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 physician-scientists at CHOP discussed what lies on the horizon after KJ’s treatment with a CRISPR-based gene editing therapy for CPS1 deficiency.

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

The physician-scientists at CHOP discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

The senior principal scientist at Regeneron discussed an advanced approach to enabling gene therapy redosing.

The head of research at Mass General Brigham’s Gene and Cell Therapy Institute discussed how innovative research can be held back by financial considerations.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed treatment decisions in newly diagnosed SMA.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed the evolving SMA treatment landscape.

The chief scientific officer of the Parkinson’s Foundation discussed how broad genetic testing through PD GENEration is uncovering valuable prognostic and diagnostic insights in Parkinson disease.

The chief scientific officer of the Parkinson’s Foundation discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.

The CEO of Capricor Therapeutics discussed future plans for the product after the promising data presented at MDA’s 2025 conference.

The chief scientific officer at Precision Biosciences discussed the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy.

The chief scientific officer at Precision Biosciences discussed the ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy.

The CEO of Capricor Therapeutics discussed clinical data presented at MDA’s 2025 meeting.

The professor of neurology at the University of Texas Health Science Center San Antonio discussed discussed challenges of therapeutic development for OPMD.

The professor of neurology at the University of Texas Health Science Center San Antonio discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.

The chair of the department of medicine at Roswell Park Comprehensive Cancer Center discussed the need to bring more innovations from nonclinical research to patients.

The chair of the department of medicine at Roswell Park Comprehensive Cancer Center discussed his views on toxicities and costs associated with CAR-T, and when and how the field should address them.

The chair of the department of medicine at Roswell Park Comprehensive Cancer Center discussed his view on how the cell therapy field should proceed with regard to innovation.

In Episode 3 of ImmunoLogic, Marcela Maus, MD, PhD, discussed her research on CAR-T for treating glioblastoma.

The chair of the department of medicine at Roswell Park Comprehensive Cancer Center discussed the innovations necessary to make CAR-T therapy effective in solid tumor indications.

The chief executive officer of Eledon Pharmaceuticals also discussed other future plans for the company.

The former FDA commissioner discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.

The codirector of pediatric neuromuscular clinical research at Washington University in St. Louis discussed the session she chaired at MDA’s 2025 conference.

The chief executive officer of Eledon Pharmaceuticals discussed the company’s investigational monoclonal antibody and its use along with EGenesis porcine kidney transplant product.