John Finn, PhD, on Using Programmable Genomic Integration to Make Edits Big and Small
The chief scientific officer of Tome Biosciences also discussed the company’s pipeline for its new technology.
David Dimmock, MBBS, on a Promising Case Study of Ultra-Rare, AI-Guided, ASO Development
The chief medical officer at Creyon Bio discussed findings from a treated patient with a TNP02 missense mutation.
Scott Jeffers, PhD, on The Importance of Precise Reproducibility of AAVs
The chief technology officer at GenSight Biologics discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.
Michael Severino on In Vivo Gene Editing With RNA Gene Writers
The chief executive officer of Tessera Therapeutics discussed ongoing research into developing its platform for sickle cell disease, CAR T-cell therapy, and more.
Chris Wright, MD, PhD, on Anelloviruses, a Potential Alternative to AAV for Gene Therapy
The chief medical officer and head of translational research at Ring Therapeutics discussed research presented at ASGCT 2024.
John Finn, PhD, on the Potential of Programmable Genomic Integration
The chief scientific officer of Tome Biosciences discussed the company’s technologies in the context of integrative gene therapy and cell therapy.
Caribou Biosciences' chRDNA Technology May Enable New Treatments for ATTR and Familial Hypercholesterolemia
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences, discussed results from preclinical research evaluating the gene editing approach.
Assessing CAR T-Cell Therapy With Novel Targets for R/R Multiple Myeloma
Omar Nadeem, MD, clinical director, Myeloma Cellular Therapies, Dana-Farber Cancer Institute, discussed GPRC5D-targeted CAR-T, BMS-986393.
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.
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.
Caribou Biosciences chRDNA Technology Provides a Unique Approach to CRISPR-Based Gene Editing
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences, discussed the company’s platform for genome editing.
Stephanie Tagliatela on Researching AAV for Lennox-Gastaut, Alzheimer Disease, SCN9a Pain
The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.
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.
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.
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.
Interest in Genomic Medicine Research for Heart Disease Continues to Grow
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed trends in precision medicine for cardiovascular indications.
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.
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.
Eque-cel Demonstrates High PFS in Newly Diagnosed, High-Risk Multiple Myeloma
Patients in FUMANBA-2 received eque-cel after being ineligible for ASCT after 4 cycles of induction therapy.
Gene Transfer Versus Gene Editing Approaches in Cardiology
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.
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.
