Connecting Patient Advocacy and Industry for Gene Therapy Development
Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the panel discussion she participated in at BIO 2022.
Pursuing Slow but Steady Gene Therapy Development
Michael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.
David Weinstein, MD, on Delivering Gene Therapy Directly to the Brain
The senior vice president of clinical development at Passage Bio discussed intracisternal magna administration of PBGM01.
Nilanjan Ghosh, MD, PhD, on Real-World Efficacy of CAR T-Cell Therapies
The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed data from real-world experience studies.
Steven W. Pipe, MD, on Challenges in Gene Therapy Trials for Hemophilia
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed common challenges with gene therapy clinical trials.
Thomas Martin, MD, on Exciting Cell Therapy Options in Multiple Myeloma
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed cilta-cel and upcoming cell therapies.
Massimo Trucco, MD, on the Potential of Islet Cell Transplant
The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed possible applications of the procedure.
Keith Fargo, PhD, on Potential Treatment Avenues for Charcot-Marie-Tooth
The chief scientific officer of the CMT Research Foundation discussed investigative cell and gene therapy approaches for treating CMT.
Adriana Rossi, MD and Luciano J. Costa, MD, PhD on Earlier CAR T in Multiple Myeloma
Oncologist discuss the possibility of earlier-line CAR T-cell therapies in MM.
Noopur Raje, MD, on Current CAR T Options in Multiple Myeloma
The director of the Center for Multiple Myeloma at Mass Gen discussed current CAR T-cell therapies in multiple myeloma.
Developing Controllable Tumor Infiltrating Lymphocyte Therapies
Jan ter Meulen, MD, PhD, chief scientific officer, Obsidian Therapeutics, discussed the potential of the cytoDRIVE platform.
Frederick Locke, MD, on Further CAR T Research
The vice-chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center discussed more work to be done with CAR T-cell therapies.
Re-Dosing With Gene Therapies in Rare Diseases
Takashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.
Massimo Trucco, MD, on Improving Access to Islet Cell Transplant
The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed islet cell transplant for the treatment of chronic pancreatitis.
Axi-Cel Superior to SOC in Elderly Patients with Diffuse Large B-Cell Lymphoma
Jason Westin, MD, FACP, leader, DLBCL research team, MD Anderson Cancer Center, discussed results from the ZUMA-7 trial at ASCO 2022.
Cilta-Cel Yields 100% ORR in Patients With Early Relapse of Multiple Myeloma
Niels van de Donk, MD, PhD, professor, Amsterdam University Medical Centers, discussed positive findings from cohort B of the CARTITUDE-2 study.
Kim A. Reiss Binder, MD, on the Potential of CAR Macrophages in Solid Tumors
The assistant professor of medicine at University of Pennsylvania Hospital stressed the importance of tumor sequencing.
Binod Dhakal, MD, on Developing Controllable CAR T-Cell Therapies for Multiple Myeloma
The associate professor of medicine at Medical College of Wisconsin discussed advantages of the ARC-SparX platform.
Tim Miller, PhD, on Supporting Gene Therapy Development
The cofounder, president, and chief executive officer of Forge Biologics discussed the company’s approach to gene therapy manufacturing.
Donald Kohn, MD, on the Trajectory of Gene Therapy
The renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.
Expediting the Development of Patient-Specific iSPC Cell Therapies
Stefan Braam, PhD, chief executive officer of Ncardia and Cellistic discussed the companies’ work in the cell therapy space.
Noopur Raje, MD, on Unmet Needs With CAR T-Cell Therapy in Multiple Myeloma
The director of the Center for Multiple Myeloma and Mass Gen discussed unmet needs with CAR T-cell therapy in multiple myeloma.
Amy Pooler, PhD, on Improving Gene Delivery to the CNS
The vice president of neuroscience at Sangamo Therapeutics discussed identifying novel capsids with improved neuronal transduction to the CNS.
Jason Fontenot, PhD, on Reducing the Need for Immunosuppression in Renal Transplantation
The chief scientific officer of Sangamo Therapeutics discussed the first-in-human STEADFAST trial.
Andras Heczey, MD, on Antitumor Activity of CAR NKTs in Solid Tumors
The director of the Liver Tumor Program at Texas Children’s Hospital discussed a phase 1 study of CAR NK T-cells in neuroblastoma.
Carsten Bönnemann, MD, on Identifying At-Risk Genotypes for DMD Gene Therapy
The senior investigator from NINDS discussed findings investigating serious AEs across different trials.
Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene-Edited Cell Therapy in PKD
The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.
Florian Eichler, MD, on Promising Efficacy of AXO-AAV-GM2 in GM2 Gangliosidosis
The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.
Yara Abdou, MD, on the Advantages of CAR Macrophages in Solid Tumors
The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.
Florian Eichler, MD, on How Novel Gene Therapy Delivery Improved Targeting in GM2 Gangliosidosis
The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.
