Carol Miao, PhD, on Using UMGD to Target Liver Sinusoidal Endothelial Cells
The principal investigator at Seattle Children’s Research Institute discussed her lab’s work on using ultrasound mediated gene delivery to target hemophilia A.
Jacques Galipeau, MD, on Exponential Progress With Cell and Gene Therapy
The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison discussed the state of cell and gene therapy development.
Carol Miao, PhD, on the Potential of Ultrasound Mediated Gene Delivery
The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research with UMGD for the treatment of hemophilia.
Lucas Harrington, PhD, on Using an Ultracompact CRISPR System to Target Cells Outside the Liver
The cofounder and chief scientific officer of Mammoth Biosciences discussed the company’s mouse model research on treating hypertriglyceridemia.
Miloš Miljković, MD, on mRNA-CAR-T Descartes-08's Potential for Treating Myasthenia Gravis
The chief medical officer of Cartesian Therapeutics discussed data presented at ASGCT 2024 from a phase 2a study.
Manali Kamdar, MD, on Liso-Cel's Ongoing Benefit in the Treatment Lanscape for LBCL
The associate professor of medicine at University of Colorado discussed data presented at ASCO 2024.
Steve Kanner, PhD, on Evaluating chRDNA Technology for ATTR and Familial Hypercholesterolemia
The chief scientific officer of Caribou Biosciences discussed results from preclinical research evaluating the gene editing approach.
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.
Manali Kamdar, MD, on The Importance of Bringing Liso-Cel to Earlier Lines of Lymphoma Treatment
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
The senior researcher at Seattle Children’s discussed the development and validation of A2-CAR-CISC EngTreg cells.
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
The medical director of clinical development at AskBio discussed setbacks in the phase 1 trial of AB-1002 gene therapy.
Steve Kanner, PhD, on Caribou’s chRDNA Technology
The chief scientific officer of Caribou Biosciences discussed the company’s platform for genome editing.
Paul Y. Song, MD, on Bringing SNK01 to Clinical Trials for Parkinson Disease
The chairman and chief executive officer of NKGen discussed the new clinical study design for evaluating the NK cell therapy in PD.
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
The PhD candidate from Lieden University Medical Center and Breakefield Lab at Mass Gen discussed her work presented at the ASGCT 2024 meeting.
Manali Kamdar, MD, on Acclimating to Routine CAR T Practice in the Field
The associate professor of medicine at University of Colorado discussed how the field has shifted to support the growing use of CAR Ts and progress to come.
Paul Y. Song, MD, on Using Autologous Natural Killer Cells to Treat Parkinson Disease
The chairman and chief executive officer of NKGen discussed the mechanism behind the company’s NK cell therapy SNK01 and promising early results in patients with PD.
Jennifer Taylor-Cousar, MD, MSCS, on Early Signals of Efficacy With Cystic Fibrosis Gene Therapy
The professor of internal medicine and pediatrics at National Jewish Health discussed updated data from the phase 1/2 AEROW trial of 4D-710.
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.
Paul Y. Song, MD, on Taking a Holistic Approach to Treating Parkinson Disease
The chairman and chief executive officer of NKGen discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.
Manali Kamdar, MD, on Evaluating Liso-Cel in Mantle Cell Lymphoma by Lines of Therapy, Prior BTKi
The associate professor of medicine at University of Colorado discussed a post hoc analysis study on subgroup analyses of liso-cel response.
Ignacio Mata, PhD, on the Research Needed to Bring Gene Therapy to PD
The associate professor of neurology at the Cleveland Clinic Lerner Institute discussed the challenges that will need to be overcome to apply gene therapy to a complex neurological disorder like PD.
Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell Therapy
The senior researcher at Seattle Children’s discussed advantages of engineered Tregs compared with natural Tregs.
Ignacio Mata, PhD, on the Challenges of Developing New Therapies in Parkinson Disease
The associate professor of neurology at the Cleveland Clinic Lerner Institute pointed out that genetic forms of PD may be the best place to start for new therapeutic research.
Mark Walters, MD, on the Future of Sickle Cell Disease’s Landscape of Care
The professor in residence for pediatrics at the Sickle Cell Center of Excellence at the University of California discussed gene therapy’s ongoing transformation of the treatment landscape.
Jacques Galipeau, MD, on International Insights Into Cell and Gene Therapy Development
The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison emphasized similar variables in the way of therapy uptake regardless of country.
Sharif Tabebordbar, PhD, on Improving In Vivo Gene Editing for DMD
The cofounder and chief executive officer of Kate Therapeutics discussed research that awarded him an Outstanding New Investigator at the ASGCT 2024 meeting.
Manali Kamdar, MD, on Bringing Liso-Cel to Earlier Lines of Treatment
The associate professor of medicine at University of Colorado discussed data in the primary relapsed LBCL setting.
Deborah Phippard, PhD, on the Challenges of Evaluating Gene Therapy for Parkinson Disease
The chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.
Omid Hamid, MD, on Assessing TIL Combination Therapies, Expanding Past Melanoma
The professor from Cedars Sinai discussed further research being conducted in the field after lifileucel’s approval.
Nathan Yozwiak, PhD, on Collaboration for Cell and Gene Therapy Development
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed work the center is engaged in with cell and gene therapy.