Advantages of mRNA-Based Gene Therapies
Jake Becraft, PhD, chief executive officer and cofounder, Strand Therapeutics, discussed the company’s technology and focus on mRNA therapies.
TCR-T Cell Therapy Cleared for Phase 1/2 Study in Acute Myeloid Leukemia
Intellia Therapeutics is also initiating a phase 1/2 study of a CRISPR therapy for hereditary angioedema in New Zealand.
FDA Approval of Brexucabtagene Autoleucel for B-Cell ALL: Bijal Shah, MD, MS
The hematologist from Moffitt Cancer Center discussed the FDA approval of brexucabtagne autoleucel in relapsed/refractory B-cell acute lymphoblastic leukemia.
Investigating CAR T-Cell Therapies in Indolent Lymphoma
Longer follow-up is needed to determine the potential of CAR T-cell therapies in difficult-to-treat malignancies.
Challenges With Conducting Clinical Trials in Gaucher Disease: Neil Weinreb, MD
The chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.
Posoleucel for Virus-Associated Hemorrhagic Cystitis Receives Orphan Drug Designation
ALVR109, another of AlloVir’s T-cell therapies, also recently showed efficacy in treating COVID-19 in high-risk patients.
NK Cell Therapy Potential in Treating COVID-19 and Other Viruses
Allen Feng, PhD, chief scientific officer, HebeCell, discussed the company’s NK cell technologies.
KRd-Autologous Stem Cell Transplant Improves PF Survival in Multiple Myeloma
Data from the FORTE trial were presented at the 18th International Myeloma Workshop.
Unmet Needs and Management in Multiple Myeloma: Peter Voorhees, MD
The hematologist/oncologist discussed unmet needs and novel therapies in multiple myeloma and give practical advice for community oncologists.
GeneTherapyLive’s Weekly Rewind – October 8, 2021
Review top news and interview highlights from the week ending October 8, 2021.
AlloCAR T Programs Halted Following Chromosomal Abnormality
Allogene continues to investigate and characterize the chromosomal abnormality and its relationship to the gene editing.
Safety Concerns With Gene Therapies: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed adverse events in gene therapies.
Natural Killer Cell Therapy Well Tolerated in Solid Tumors
Thorsten Graef, MD, PhD, chief medical officer, Acepodia, discussed data on ACE1702 presented at ESMO 2021.
Krabbe Disease Gene Therapy Receives Second Orphan Drug Designation
Forge Biologics and Solid Biosciences have also partnered to help develop a gene therapy for Duchenne muscular dystrophy.
Regulating Cell and Gene Therapy Activity
Paul Wotton, PhD, chief executive officer, Obsidian Therapeutics, discussed the company’s pipeline and technologies.
Gavo-Cel Efficacious in Refractory Mesothelin-Expressing Solid Tumors
Investigators have identified the maximum tolerated dose and continue to refine the recommended phase 2 dose in the phase 1 part of the study.
Adverse Events in CAR T-Cell Therapies: Joseph A. Fraietta, PhD
The director of the Solid Tumor Immunotherapy Lab at the University of Pennsylvania discussed safety concerns with CAR T-cell therapies.
SGT-001 Improves Pulmonary Function in Duchenne Muscular Dystrophy
Improvements in peak expiratory flow, forced expiratory volume, and forced vital capacity were observed in all evaluable treated patients in the IGNITE-DMD trial.
Challenges in Developing Gene Therapies for Gaucher Disease: Neil Weinreb, MD
The chair of the International Collaborative Gaucher Group Registry discussed the place of gene therapies in the Gaucher treatment landscape.
Ide-Cel Continues to Show Durable Efficacy in Multiple Myeloma
Overall response rate was 73% in all patients treated with ide-cel at a median follow-up of 24.8 months (range, 1.7-33.6).
Future Research With Stem Cell Therapies in Hematologic Malignancies
The chief executive and chief medical officers of Vor Biopharma discussed the company’s future research and plans.
Suprachoroidal Delivery of RGX-314 Well-Tolerated in Wet AMD
Patients treated with RGX-314 also had stable BCVA and central retinal thickness.
Brexucabtagene Autoleucel Approved for Refractory B-Cell Acute Lymphoblastic Leukemia
ZUMA-3 showed that a single infusion of the CAR T-cell therapy elicited a high and durable response rate in heavily pretreated patients with relapsed/refractory B-ALL.
ASC Transplant Eligibility in Multiple Myeloma: Elisabet E. Manasanch, MD
The associate professor from The University of Texas MD Anderson Cancer Center discussed eligibility for autologous stem cell transplant in patients with multiple myeloma.
Angelman Syndrome Gene Therapy Trial Resumes Following Clinical Hold
A clinical hold was previously placed after patients treated with the highest dose developed lower extremity weakness.
Exposing the Advantages of iPSC-Natural Killer Cell Therapies
Allen Feng, PhD, chief scientific officer, HebeCell, discussed advantages of induced pluripotent over hematopoietic stem cell therapies.
Gene Therapy for AADC Deficiency Yields Durable Developmental Improvements
Improvements persisted through up to 10 years of follow-up.
Cellular, Tissue, and Gene Therapies AdComm Meeting Takeaways: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed the recent meeting of the FDA Cellular, Tissue, and Gene Therapies Advisory Committee.
GeneTherapyLive’s Weekly Rewind – October 1, 2021
Review top news and interview highlights from the week ending October 1, 2021.
CAR T-Cell Therapy Axi-Cel Seeks Expanded Indication for Second-Line LBC Lymphoma
Treatment with the CAR T-cell therapy yielded a 60% reduction in the risk of EFS events compared with SOC with a median follow-up of 2 years.
