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.
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.
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.
Continuing Innovation on Modality-Specific Technology for Cardiovascular-Targeted Genomic Medicines
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s research on capsids, promoters, and manufacturing improvements.
Cabaletta Bio’s CAR-T CABA-201 Shows Initial Signs of Efficacy in Myositis and Lupus Trials
The data, from the first patients dosed in each trial, also continues to show safety.
Gene-Edited Exa-Cel Continues to Show Long-Term Benefit in Sickle Cell, Thalassemia
Data from up to 5 and 6 years of follow-up were presented at the 2024 EHA Congress.
Daniel Hart, PhD, on CRISPR-Mediated In Vivo Epigenomic Activation
The Senior Director and Head of Technology Development at Epic Bio discussed potential applications of the new technology.
Mustang Bio’s CAR-T MB-106 Achieves Responses in 9 of 10 Patients With Waldenstrom Macroglobulinemia
The responses seen in the 9 patients included 3 complete responses (CRs), 2 very good partial responses, and 4 partial responses.
Luke Roberts, MBBS, PhD, on Developing Gene Therapy for Congestive Heart Failure
The medical director of clinical development at AskBio discussed the progress of an early clinical trial of AB-1002.