Videos

The assistant professor of Hematologic Oncology and Blood Disorders at Atrium Health discussed integrating CAR T therapy into treatment paradigms.

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval.

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.

The chair of gynecologic oncology at Moffitt Cancer Center discussed advantages of targeting follicle stimulating hormone receptor with cell therapy for ovarian cancer.

The medical oncologists at Washington University School of Medicine in St. Louis discussed surprising findings from their retrospective study.

Schlechter discussed the potential advantages of TAC01-HER2 over current standards of care.

Schlechter discussed the design of the TACTIC-2 clinical trial for patients with HER2-positive solid tumors.

Shah explained that the trial is unique due to its inclusion of patients with MRD–positive B-ALL.

The pediatrician from Royal Manchester Children’s Hospital discussed how further research in Gaucher disease can benefit the field of rare lysosomal diseases as a whole.

The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy.

The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.

Rossano discussed unmet needs for patients with Danon disease and the potential of investigational gene therapy RP-A501.

As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.

The senior vice president of Research & Early Development at Notch Therapeutics discussed the company’s approach to overcoming the drawbacks of autologous cell therapies.

Patel discussed the potential advantages of his team’s gene therapy approach compared to an investigational PKD drug.

Patel discussed the current standard of care for PKD and the potential advantages of a new gene therapy approach.

The medical oncologists at Washington University School of Medicine in St. Louis discussed background to their research on associations between neurofilament light and neurotoxicity.

Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.

The director of translational research in myeloma at the Tisch Cancer Institute discussed positive findings, including a 30-month PFS in study participants.

The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.

The chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.

The chief medical officer of Triumvira Immunologics discussed the company’s future plans.

The professor of genetics at Federal University of Rio Grande do Sul discussed how RGX-121 has the potential to change the treatment landscape of MPSII.

The chief executive officer of Abintus Bio discussed the company’s in vivo approach and how it could help expand patient access to cell therapies.

The director of the Regenerative Medicine Institute at Cedars Sinai discussed results from a phase 1/2a study of CNS10-NPC-GDNF.

The senior scientist at Thermo Fisher Scientific discussed the company’s approach to scaling and strategic partnerships.