Barry Paul, MD, on Managing CRS and ICANS in Patients Receiving CAR-T for Cancer
Paul discussed common symptoms and typical treatments for the 2 adverse events.
Amod Sarnaik, MD, on New Frontiers in Cell Therapy for Advanced Melanoma
Sarnaik discussed several potential areas of innovation on the horizon.
Targeting Hematological Malignancies With Autologous and Allogeneic Cell Therapies
Thomas Willemsen, president and chief executive officer, Tessa Therapeutics, discussed the company’s technologies and 2023 milestones.
Terese Hammond, MD, on Investigating iNKT Cells in COVID-Associated ARDS
The director of the CCU/ICU at Saint John’s Health Center discussed unmet respiratory treatment needs in the beginning of the COVID-19 pandemic.
Amod Sarnaik, MD, on Lifileucel’s Place in the Treatment Lanscape for Advanced Melanoma
Sarnaik discussed the investigational TIL therapy’s potential as an additional option for patients.
Omid Hamid, MD, on Improving Duration of Response in Refractory Melanoma With Lifileucel
The chief of research and immunotherapy at Cedars-Sinai The Angeles Clinic and Research Institute discussed updated data from the phase 2 C-144-01 study of the lifileucel TIL therapy.
Susan Foy, PhD, on Evaluating Personalized, Neoantigen-Specific T Cell Therapy in Solid Tumors
The CRIPSR-edited neoantigen-specific T cell therapy demonstrated safety and feasibility in the first-in-human PACT-0101 study presented at SITC 2022.
Samir Parekh, MD, on Surprising Efficacy of Sequential T-Cell Redirection Therapy in Multiple Myeloma
The director of translational research in myeloma at the Tisch Cancer Institute discussed the relevance of the new research as more CAR T therapies come to market.
Barry Paul, MD, on Improving CAR T-Cell Therapy Efficacy in Multiple Myeloma
The assistant professor of Hematologic Oncology and Blood Disorders at Atrium Health discussed integrating CAR T therapy into treatment paradigms.
Paul Melmeyer on Addressing Gene Therapy Regulatory Bottlenecks
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.
Paul Melmeyer on Regulatory Advocacy for Patients With Neuromuscular Diseases
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.
Robert Wenham, MD, MS, FACS, On Advantages of FSHR-CER T for Ovarian Cancer
The chair of gynecologic oncology at Moffitt Cancer Center discussed advantages of targeting follicle stimulating hormone receptor with cell therapy for ovarian cancer.
Alice Zhou, MD, PhD, and Omar Butt, MD, PhD, on Surprising Associations With Neurotoxicity and Neurofilament Light
The medical oncologists at Washington University School of Medicine in St. Louis discussed surprising findings from their retrospective study.
Benjamin L. Schlechter, MD, on TAC01-HER2 in HER2-Positive Solid Tumors
Schlechter discussed the potential advantages of TAC01-HER2 over current standards of care.
Benjamin L. Schlechter, MD, on Addressing Unmet Needs in HER2 Positive Solid Tumors
Schlechter discussed the design of the TACTIC-2 clinical trial for patients with HER2-positive solid tumors.
Bijal Shah, MD, on AUTO1 in R/R B-Cell ALL and the FELIX Trial
Shah explained that the trial is unique due to its inclusion of patients with MRD–positive B-ALL.
Aimee Donald, MBChB, PhD, on Making Progress With Gene Therapy in Lysosomal Storage Diseases
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.
Vlad Hogenhuis, MD, MBA, on Developing Controllable CAR Ts for Solid Tumors
The chief executive officer of Chimera Bioengineering discussed CBIO-007 and its planned IND submission for colorectal and triple negative breast cancer.
TJ Cradick, PhD, on A Potential Cure for HIV With EBT-101
The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy.
Aimee Donald, MBChB, PhD, on Assessing PR001 in Children With Type 2 Gaucher Disease
The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.
Joseph Rossano, MD, on the Potential of Gene Therapy in Danon Disease
Rossano discussed unmet needs for patients with Danon disease and the potential of investigational gene therapy RP-A501.
Vinaya Murthy, MS, MPH, on Genetic Counseling Services for Rare Disorders
As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.
Chris Bond, PhD, on Allogeneic and Induced Pluripotent Stem Cell Approaches to Cell Therapy
The senior vice president of Research & Early Development at Notch Therapeutics discussed the company’s approach to overcoming the drawbacks of autologous cell therapies.
Vishal Patel, MD, on the Potential Advantages of a Gene Therapy Approach for Polycystic Kidney Disease
Patel discussed the potential advantages of his team’s gene therapy approach compared to an investigational PKD drug.
Vishal Patel, MD, on Unmet Needs in Polycystic Kidney Disease
Patel discussed the current standard of care for PKD and the potential advantages of a new gene therapy approach.
Alice Zhou, MD, PhD, and Omar Butt, MD, PhD, on Investigating Associations With CAR T Toxicities
The medical oncologists at Washington University School of Medicine in St. Louis discussed background to their research on associations between neurofilament light and neurotoxicity.
Improving Access to Rare Disease Care
Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health Care
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
Clive Svendsen, PhD, on Unlocking the Potential of Astrocyte Cell Therapy
The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
Samir Parekh, MD, on Improving Survival in Multiple Myeloma With Sequential T-Cell Redirection Therapies
The director of translational research in myeloma at the Tisch Cancer Institute discussed positive findings, including a 30-month PFS in study participants.
