Jacob Appelbaum, MD, PhD, on Controlling DARIC CAR Therapy With Rapamycin for AML
The senior research fellow at Fred Hutch Cancer Center discussed findings from the PLAT-08 trial presented at ASGCT 2023.
BCMA-directed CAR-T HBI0101 Demonstrates Safety and Efficacy in Patients With Light Chain Amyloidosis
All 8 patients with light chain amyloidosis who received HBI0101 responded.
CSF Infusion of Gene Therapy Transduces Cochlear Cells
The most robust transduction in nonhuman primates was seen with ICV delivery of pooled PM4.AAV.mRFP and AAV2.mNG gene therapy,
Farah Sheikh, PhD, on Modeling Arrhythmogenic Right Ventricular Cardiomyopathy
The professor of medicine at University of California San Diego discussed new research from her lab presented at the 2023 ASGCT meeting.
Jason Fontenot, PhD, on Addressing Unmet Needs in Neuropathic Pain and Prion Disease With Zinc Finger Genome Regulation
The chief scientific officer of Sangamo Therapeutics discussed data from 2 of the company’s programs that were presented at ASGCT’s 2023 conference.
Kyle Garland, PhD, on the Potential of mRNA-engineered iPSC-derived Cell Therapies for Solid Tumors
The senior scientist at Eterna Therapeutics discussed the company’s numerous presentations at ASGCT’s 2023 conference.
Dual Injections of 2-Vector Gene Therapy May Stabilize Disease in GM2 Gangliosidosis
Data trends suggesting stabilization of disease were seen in patients with Tay-Sachs, but more research is needed, especially for those with Sandoff disease.
Multi-Characteristic Opsin Gene Therapy for Retinitis Pigmentosa Improves Multiple Measures of Vision
Updated data from the RESTORE study on MCO-010, Nanoscope Therapeutics' investigational gene therapy, were presented at the 2023 ASGCT meeting.
Jennifer Gori, PhD, on the Potential of a Prime Editing Approach in Treating Chronic Granulomatous Disease
The vice president of research at Prime Medicine discussed a preclinical study on a novel autologous HSCT treatment that was presented at ASGCT’s 2023 annual meeting.
LCAT-GMAC Cell Therapy Improves Symptoms in Patient with Familial LCAT Deficiency
Following implantation with CellGenTech’s LCAT-GMAC, the patient’s serum LCAT activity increased by approximately 50% of baseline.
Trial to Assess Selecta’s Gene Therapy/Immunomodulatory Combo in Patients With MMA
ImmTOR induces immune tolerance and may reduce or eliminate Nab formation, allowing potential redosing of gene therapy.
Sleeping Beauty Engineered CAR-T Therapy CARCIK-CD19 Demonstrates Safety and Anti-Leukemia Activity in R/R B-ALL
Among 27 evaluated patients with relapsed/refractory B-cell acute lymphoblastic leukemia, 18 achieved a complete response.
Krystof Bankiewicz, MD, PhD, on Dialing Back Progression of Parkinson Disease With GDNF Gene Therapy
The professor at Ohio State University and University of California – San Francisco discussed updated data from a phase 1 study of GDNF gene therapy in patients with PD.
Intraarticular Gene Therapy Well-Tolerated, Improves Pain in Knee Osteoarthritis
Updated data from a phase 1 study of PCXR201, formerly known as Flexion’s FX201, were presented at the 2023 ASCGT Annual Meeting.
Bietti Crystalline Dystrophy Gene Therapy Demonstrates Safety in Early Phase 1 Trial
Vitalgen’s gene therapy was generally well-tolerated with no serious adverse events reported.
Re-Dosing With Gene Therapies in Rare Diseases
Takashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.
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.
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.
