David Suhy, PhD, on Using DNA Nanoplasmids to Detect Cancer
The cofounder and chief scientific officer at Earli discussed the company’s unique approach to cancer diagnosis.
Jeffrey Chamberlain, PhD, on Helping Progress Cell and Gene Therapy Development
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed the role the ASGCT plays in the field.
Verdot's FlexiPro Chrom Platform Helps Address Bioprocessing Needs for Gene Therapy Manufacturing
Sebastien Lefebvre, MSc, the vice president of R&D at Verdot, discussed the company's unique platform.
Viral Immunotherapy Combo Improves Survival in Patients With NSCLC
Candel also recently received orphan drug designation for CAN-3110 for treating recurrent, high-grade glioma.
Vanee Pho, PhD, on Detecting Translocations in Gene Edited Cells
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
Steven W. Pipe, MD, on Confirming Efficacy, Safety of Hemgenix Gene Therapy in Hemophilia B Populations
The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed a sub analysis of the HOPE-B trial.
Lete-cel Study in Synovial Sarcoma, MRCLS Meets Primary Endpoint With 40% ORR
Nine responses were ongoing as of the March 2023 cutoff date.
Rawan Faramand, MD, on Evaluating Orca-T in Patients With Acute Myeloid Leukemia
The assistant professor at Moffit Cancer Center discussed the subgroup analysis she presented at ASCO’s 2024 Annual Meeting.
Metastatic Colorectal Cancer CAR-T Therapy GCC19CART Demonstrates Safety and Clinical Activity in US Patients
GCC19CART targets both guanylate cyclase 2C and CD19.
First Patient Receives TCR-T Therapy in Multiplexed Study Screening Patients With Melanoma, Cervical Cancer and More
Of 300 screened participants, 68 have been matched by target antigen and HLA expression to a TCR-T in the ImmunoBank.
Liso-cel Sustains Disease Improvement in Primary Refractory or Early Relapsed LBCL
Patients with large B-cell lymphoma in the TRANSFORM trial showed improvements over 3-year period compared with standard of care.
Manali Kamdar, MD, on Liso-Cel's Continued Efficacy in Second-Line LBCL at 3-Year Follow-up
The associate professor of medicine at University of Colorado discussed updated follow-up data from the phase 3 TRANSFORM trial presented at the 2024 ASCO meeting.
No GvHD With Allogeneic CRISPR-edited CAR T for LBCL, PFS Associated With Partial HLA Match
PFS jumped from under 3 months to over 14 when participants received CB-010 from a donor with at least 4 matched HLA alleles.
Jeffrey Chamberlain, PhD, on Continuing the Marathon of Muscular Dystrophy Research
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington shared his outlook on the trajectory of research in the field.
AIC100 Safe, Effective for Advanced Thyroid Cancer
The CAR T-cell therapy AIC100 demonstrated promising responses and a low level of toxicity in patients with advanced thyroid cancer.
N. Nora Bennani, MD, on Diving Deeper Into T-Cell Lymphomas
The assistant professor at Mayo Clinic School of Medicine shared her outlook and predictions on research with T-cell lymphomas.
Safe Handling Guidelines Needed for Gene Therapies in the Clinic
Current handling guidelines do not align between the laboratory and clinical settings.
Anjali Pradhan, MS, on Using Single Cell Sequencing for Gene Editing Quality Control
Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy
The deputy director, clinical research, San Raffaele Telethon Institute for gene therapy, discussed long-term follow-up data of up to 12 years.
LCA2 Gene Therapy Shows Visual, Retinal Improvements at Low Doses
No serious adverse events or significant retinal atrophy occurred.
Brian Kim, MBA, on the Importance of Single Cell Analysis to Cell and Gene Therapy
The CEO of Mission Bio discussed the company’s Tapestri platform for single cell sequencing.
Ethical Framework Needed for Gene Therapy Clinical Trials
An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.
Xandra Breakefield, PhD, on Trying New Approaches to AAV Therapy for Glioblastoma
The investigator of neurology at Mass General Research Institute discussed research from her grad student, Lisa Nieland, presented at the ASGCT 2024 meeting.
Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed research from the center presented at ASGCT 2024.
Cabaletta Bio’s CAR-T CABA-201 Shows Safety for First Patients Dosed in Myositis and Lupus Trials
The data comes from the phase 1/2 RESET-Myositis clinical trial (NCT06154252) and the phase 1/2 RESET-SLE clinical trial (NCT06121297).
Janice Chen, PhD, on Novel Approaches to Gene Editing
The cofounder and chief technology officer of Mammoth Biosciences discussed the importance of diverse approaches to gene editing to address a variety of indications.
Manipulation of the FOXO1 Gene Holds Potential to Enhance CAR T-Cell Fitness
Evan Weber, PhD, an assistant professor of pediatrics at Children's Hospital of Philadelphia, discussed his work on the role of the FOXO1 gene in T-cell persistence and exhaustion.
Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe Disease
The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her talk on preparing for gene therapy administration.
John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1
The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.
