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.
Michel Michaelides, MD, FACP, on Gene Therapy's Potential in Retinitis Pigmentosa
The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.
Devon J. Shedlock, PhD, on Shifting from Autologous to Allogeneic Therapies
The chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.
Deyaa Adib, MD, on Optimizing Manufacturing of Solid Tumor Cell Therapies
The chief medical officer of Triumvira Immunologics discussed the company’s future plans.
Roberto Giugliani, MD, PhD, on Advantages of Gene Therapy in MPS Type 2
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.
Nick Boyle, PhD, on Advantages of In Vivo CAR Therapies
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.
Clive Svendsen, PhD, on Astrocyte Progenitor Cell Therapy in ALS
The director of the Regenerative Medicine Institute at Cedars Sinai discussed results from a phase 1/2a study of CNS10-NPC-GDNF.
Evan Zynda, PhD, on Optimizing Manufacturing to Increase Access to Cell Therapy
The senior scientist at Thermo Fisher Scientific discussed the company’s approach to scaling and strategic partnerships.
Samir Parekh, MD, on Investigating Treatment Options for Relapsed Myeloma
The director of translational research in myeloma at the Tisch Cancer Institute discussed investigating sequential T cell redirection therapies.
Treating Autoimmune Diseases With Smarter Cell Therapies
Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.
Christine Coughlin, MD, PhD, on TRACK-NK Technology in Non-Small Cell Lung Cancer
The chief executive officer of Cytoimmune discussed the company’s technology platforms and programs.
