FDA Approves Tisa-Cel Expanded Indication in R/R Follicular Lymphoma
The approval was based on data from the phase 2 ELARA clinical trial, in which a complete response of more than 65% was observed.
Expediting the Development of Patient-Specific iSPC Cell Therapies
Stefan Braam, PhD, chief executive officer of Ncardia and Cellistic discussed the companies’ work in the cell therapy space.
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.
Noopur Raje, MD, on Unmet Needs With CAR T-Cell Therapy in Multiple Myeloma
The director of the Center for Multiple Myeloma and Mass Gen discussed unmet needs with CAR T-cell therapy in multiple myeloma.
CGTLive’s Weekly Rewind – May 27, 2022
Review top news and interview highlights from the week ending May 27, 2022.
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.
Around the Helix: Cell and Gene Therapy Company Updates – May 25, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Gene Therapy for Knee Cartilage Lesions Receives RMAT Designation
NeoCart was originally developed by the company Histogenics.
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.
EtranaDez Accepted for Priority Review in Hemophilia B
A final analysis of the pivotal HOPE-B study was presented at ASGCT 2022.
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.
Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion
The positive opinion was based on findings from studies conducted in Taiwan.
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.
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.
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.
