Determining Order of CAR T Therapy: Elias Jabbour, MD; Jae Park, MD
The oncologists from MD Anderson and Memorial Sloan Kettering Cancer Centers discuss sequencing CAR T-cell therapies and other key therapies in patients with B-cell acute lymphoblastic leukemia.
Assessing Efficacy of Fibroblast Cell Therapy in MS: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.
A Positive Outlook for Rare Disease Research: Michael Singer, MD, PhD
The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.
Improving Rare Disease Awareness: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.
Developing Therapies for Rare Diseases: Kinnari Patel, PharmD
The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.
Improving Rare Disease Awareness: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.
Ongoing Gene Therapy Trials in Stargardt Disease : Prof. Carel Hoyng
The professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.
Launching PDGENEration to Advance Genetic Testing in Parkinson Disease: James Beck, PhD
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the organization’s initiative to advance genetic testing.
Managing Safety in Fitusiran Treatments of Hemophilia: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.
Unmet Needs in Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.
Assessing Safety of Fibroblast Cell Therapy in Multiple Sclerosis: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology in MS.
Risk Alleles in Age-Related Macular Degeneration: Steffen Schmitz-Valckenberg, MD
The ophthalmologist from John A. Moran Eye Center discussed the close association between disease progression and uncovered risk alleles.
Targeting Unmet Needs in Rare Dermatologic Diseases With Gene Therapy
Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.
Elucidating Associations Between EBV and MS: Jakob Dupont, MD
The global head of research and development at Atara Biotherapeutics discussed the link between EBV and MS.
Improving CAR T-Cell Therapies for Multiple Myeloma: Thomas G. Martin, MD
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed CAR T-cell therapies and safety signals of cilta-cel.
The Need for Analysis in Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.
The Potential Impact of Gene and Cell Therapy in Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.
Achieving Remission in Chronic Lymphocytic Leukemia With CD4+ CAR T-Cells: Jan Joseph Melenhorst, PhD
The translational immunologist and research professor at Perelman School of Medicine, University of Pennsylvania, discussed persistence of CD4+ CAR T-cells in CLL.
Latest Updates on Geographic Atrophy Gene Therapy FOCUS Trial: Nadia K. Waheed, MD
The associate professor from Tufts University School of Medicine discussed the latest updates on the FOCUS trial.
Achieving Developmental Milestones With GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed new data from the IMAGINE-1 trial presented at WORLDSymposium.
Fibroblast Cell Therapy or Stem Cell Therapy for MS: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed the advantages of fibroblast cell therapy over stem cell therapy.
Treating Solid Tumors With Personalized Immunotherapy: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed targeting HLA loss in solid tumors.
The Importance of Collaboration in Developing Next Generation Cell Therapies
Dolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed the company’s collaboration with 2seventy bio to develop TCR T-cell therapies.
Assessing EtranaDez for Hemophilia B in HOPE-B: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed results of the final analysis of the HOPE-B study.
Assessing Fitusiran for Hemophilia in ATLAS-INH: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed the ATLAS-INH trial.
Challenges in Managing Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed challenges in hemophilia A and B.
Assessing Gene Therapy in Recessive Dystrophic Epidermolysis Bullosa
Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed the company and its pipeline.
Tumor-Infiltrating Lymphocyte Therapy After Immunotherapy in Advanced Melanoma: Omid Hamid, MD
The director of the Melanoma Program at Cedars-Sinai Medical Center discussed the efficacy of TIL therapy following immunotherapy in advanced melanoma.
Brexucabtagene Autoleucel in R/R Mantle Cell Lymphoma
Experts discussed the ZUMA-2 clinical trial of brexucabtagene autoleucel.
Evaluation Criteria for TIL Therapy in Melanoma: Omid Hamid, MD
The director of the Melanoma Program at Cedars-Sinai Medical Center discussed evaluation criteria for TIL therapy in melanoma.
