Tim Miller, PhD, on Supporting Gene Therapy Development
The cofounder, president, and chief executive officer of Forge Biologics discussed the company’s approach to gene therapy manufacturing.
Donald Kohn, MD, on the Trajectory of Gene Therapy
The renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.
Liso-Cel Yields High Response Rates and Positive PROs in LBCL
Treated patients had significant improvements in fatigue and lymphoma symptoms.
Lentiviral Cell Therapy Normalizes Hemoglobin in Pyruvate Kinase Deficiency
RP-L301 also improved hemolysis and eliminated the need for red blood cell transfusions for up to 1year after therapy.
Developing Safer Viral Vectors for Gene Therapy
The chief executive officer of Ring Therapeutics, Tuyen Ong, MD, discussed the potential of anellovirus vectors for use in gene therapy.
Amy Pooler, PhD, on Improving Gene Delivery to the CNS
The vice president of neuroscience at Sangamo Therapeutics discussed identifying novel capsids with improved neuronal transduction to the CNS.
Jason Fontenot, PhD, on Reducing the Need for Immunosuppression in Renal Transplantation
The chief scientific officer of Sangamo Therapeutics discussed the first-in-human STEADFAST trial.
Gene Therapy May Eliminate Need for ERT in Late-Onset Pompe Disease
All study participants met criteria to remain off enzyme replacement therapy through 104 weeks after treatment.
Andras Heczey, MD, on Antitumor Activity of CAR NKTs in Solid Tumors
The director of the Liver Tumor Program at Texas Children’s Hospital discussed a phase 1 study of CAR NK T-cells in neuroblastoma.
Cell Therapy Modulates Tumor Microenvironment in Glioblastoma
The cell therapy was tolerated although there was a SUSAR of GGT elevations.
Carsten Bönnemann, MD, on Identifying At-Risk Genotypes for DMD Gene Therapy
The senior investigator from NINDS discussed findings investigating serious AEs across different trials.
Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene-Edited Cell Therapy in PKD
The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.
Gene Therapy for LAD-I Posts Impressive Survival Data
In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.
CGTLive’s Weekly Rewind – May 20, 2022
Review top news and interview highlights from the week ending May 20, 2022.
Florian Eichler, MD, on Promising Efficacy of AXO-AAV-GM2 in GM2 Gangliosidosis
The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.
DMD Gene Therapy Shows Continued Microdystrophin Expression
Motor and pulmonary function have stabilized or improved in participants treated with SGT-001.
Hematopoietic Recovery After Myeloablative Transplantation May Be Entirely Driven by Hematopoietic Stem Cells
Findings from a preclinical study, which are in contrast with the current biphasic recovery model, have implications for both HSC transplantation and gene therapy.
Yara Abdou, MD, on the Advantages of CAR Macrophages in Solid Tumors
The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.
Homology-Directed Repair-Developed TCR Therapy Shows Potential in Type 1 Diabetes
Investigators from Seattle Children’s Hospital found that engineered T-cell regulatory products demonstrated significant suppression of effector T cells.
Florian Eichler, MD, on How Novel Gene Therapy Delivery Improved Targeting in GM2 Gangliosidosis
The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.
Final Analysis of Phase 3 HOPE-B Trial: Durability of Etranacogene Dezaparvovec and QoL Improvements
The trial met its primary efficacy end point, and participants reported improvements in quality-of-life factors such as outlook on the future and feelings about having hemophilia.
Lentiviral Gene Therapy for Infants With X-Linked SCID Helps Build ‘Functional’ Immune System
The interim study results presented at ASGCT 2022 likely represent the longest follow-up data to date for the largest cohort of infants with XSCID treated with lentiviral vector gene therapy.
Gene Therapy for Refractory Angina Promotes Increased Exercise Tolerance
Safety and efficacy data suggest this to be a feasible and clinically meaningful treatment for a patient population with high disease burden.
Around the Helix at ASGCT: Cell and Gene Therapy Company Updates – May 18, 2022
Catch up on the latest news and announcements in cell and gene therapies presented at the annual ASGCT meeting.
Analysis Elucidates Lentiviral Vector-Mediated Myelodysplastic Syndrome Cases
Multiple insertions were observed in affected participants, including insertions in the MECOM and PRDM16 genes.
Adenovirus/Chemotherapy Combination Well-Tolerated, Surprising OS Benefit Observed in mPC
One patient with metastatic pancreatic cancer remains alive on maintenance chemotherapy 3 years after treatment.
Improvements in Developmental Milestones Observed With GM1 Gangliosidosis Gene Therapy
Data from 2 patients dosed in the Imagine-1 clinical trial were presented in a late breaking session at ASGCT 2022.
CAR Macrophage Therapy for HER2 Solid Tumors Continues Through First in Human Trial
Investigators are evaluating the safety, tolerability and preliminary efficacy of CT-0508 in patients with solid tumors and HER2 overexpression.
Autoantigen CAR T Cell Therapy Well-Tolerated in Mucosal-Dominant Pemphigus Vulgaris
Investigators found a dose-dependent increase in CAART cell persistence.
At-Risk Genotypes for DMD Gene Therapy Identified
Trial sponsors Pfizer, Sarepta, Genethon, and Solid Biosciences organized a group of experts to investigate shared serious AEs.
