Liso-Cel Plus Ibrutinib Well-Tolerated in Relapsed/Refractory CLL/SLL
Updated data from the phase 1 TRANSCEND-CLL-004 trial were presented at the International Workshop on CLL.
IND Accepted for Wet AMD Gene Therapy
4D Molecular Therapeuitcs plans to initiate a phase 1/2 trial of 4D-150 before the end of the year.
Multiple Myeloma KarMMA Trial Methodology: Larry Anderson, MD
The associate professor from Harold C. Simmons Comprehensive Cancer Center discussed the methodology of the phase 2 KarMMa trial.
Optogenetic Therapy Shows Efficacy in Autosomal Recessive Retinitis Pigmentosa
Results from a phase 1/2 study from Nanoscope Therapeutics were presented at the 2021 ASRS meeting.
Ide-Cel's Benefit on PF Survival: Doris Hansen, MD
The hematologist from Moffitt Cancer Center discussed the survival benefit of ide-cel in the phase 2 KarMMa trial.
GeneTherapyLive’s Weekly Rewind – October 15, 2021
Review top news and interview highlights from the week ending October 15, 2021.
Identifying Patient-Specific Tumor Targets
Jessica Baker Flechtner, PhD, chief scientific officer, Genocea, discussed the ATLAS platform and the TiTAN trial.
Study Investigates New CAR T Therapy for T-Cell Lymphoma
The study follows Legend Biotech’s initial success with cilta-cel, whose PDUFA date is set for November 2021.
CAR T Therapies for Hematologic Malignancies and Solid Tumors
André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed therapies the company is developing.
Cell Therapy a Promising Option in Corneal Endothelial Disease
The cell therapy yielded improvements in visual acuity and central corneal thickness.
Brexucabtagene Autoleucel's Impact on MCL Treatment Landscape: Luhua Wang, MD
The professor from The University of Texas MD Anderson Cancer discussed the impact that brexucabtagene autoleucel's approval has had on mantle cell lymphoma.
CAR T-Cell Therapy From CRISPR Therapeutics Efficacious in CD19+ B-Cell Malignancies
Enrollment in the CARBON trial has so far focused on aggressive disease such as diffuse B-cell lymphoma.
Gene Editing's Potential for COVID-19 and Other Indications
New human gene editing therapies, drug discoveries, targets, and CRISPR technology holds the potential to usher in a new age in medicine.
Current Limitations of CAR T-Cell Therapy in Multiple Myeloma: Muhamed Baljevic, MD
The assistant professor from University of Nebraska Medical Center discussed the current limitations of CAR T-cell therapy in multiple myeloma.
Ocular Gene Therapy RGX-314 Expands Efficacy to Diabetic Retinopathy
REGENXBIO previously announced positive data in patients with wet AMD who were treated with the same gene therapy.
Sarepta’s Duchenne Gene Therapy Shows Efficacy in Multiple Studies
Sarepta Therapeutics has also initiated the pivotal phase 3 EMBARK study in pediatric patients with DMD.
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.
