Videos

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.

The chief scientific officer at Avidity Biosciences discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

In Episode 2 of ImmunoLogic, Bruce Levine, PhD, discussed the current the risk-benefit-ratio for CAR-T therapy.

The pediatric neurologist at Children’s Hospital of Philadelphia offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.

The chief executive officer of Eledon Pharmaceuticals discussed the company’s collaboration with EGenesis.

The chief executive officer of ASGCT broke down the highlights of the organization’s final report for 2024.

The director of the Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program at the Hospital of the University of Pennsylvania discussed data from the phase 2 BMT CTN 1902 trial.

The adult metabolic consultant at Salford Royal Hospital discussed areas of interest for future study for the Gaucher disease gene therapy product.

The associate professor of medicine at Medical College of Wisconsin discussed efficacy data presented at the 2025 Tandem Meetings.

The associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center discussed results from a phase 1 clinical trial.

The adult metabolic consultant at Salford Royal Hospital discussed results she presented at the 21st Annual WORLDSymposium.

The hematology-oncology and BMT cell therapy fellow at Stanford University discussed implications of his institution’s findings on treatment-related secondary malignancies.

In Episode 1 of ImmunoLogic, Stephen J. Schuster, MD, discussed the potential of treating lymphoma without chemotherapy.

The chief medical advisor of the Muscular Dystrophy Association also shared his thoughts on the 75th anniversary of the organization.

The chief medical advisor of the Muscular Dystrophy Association discussed what attendees can expect at the 2025 MDA Meeting.

The medical director of the Immune Effector Cell Therapy Program at UCLA discussed initial data from a phase 1/2 trial.

The director of cell therapy and transplant at Penn Medicine also summed up his main message regarding the current state of the field in general.