Dual-Targeted CAR T Therapy Efficacious in Relapsed/Refractory B-Cell Malignancies
The phase 1 study met its primary endpoints, with 97% of the CAR T-cell therapies reaching the protocol-specified dose and no dose-limiting toxicities during dose escalation.
Gene Therapy for LAD-1 Demonstrates Efficacy
Rocket Pharmaceuticals announced positive data on RP-L201 in leukocyte adhesion deficiency-I, as well as RP-A501 for Danon disease.
Gene Therapy Yields Improvements in AADC-Deficiency: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed 3 studies of PTC-AADC for AADC deficiency.
Challenges in Treating Relapsed/ Refractory DL B-Cell Lymphoma
Experts discussed challenges with treating relapsed or refractory diffuse large B-cell lymphoma.
CAR T-Cell Therapy and COVID-19 Vaccinations: Muhammad Bilal Abid, MD, MRCP
The assistant professor of medicine from Medical College of Wisconsin discussed the potential impact of CAR T-cell therapy on the efficacy of COVID-19 vaccinations.
Fabry and Gaucher Gene Therapies Well-Tolerated
AVROBIO shared interim safety data from ongoing phase 1 and 2 trials.
The Future of Frontline CAR T in Mantle Cell Lymphoma: Brad S. Kahl, MD
The professor of medicine at Washington University School of Medicine in St. Louis discussed the potential utility of frontline CAR T-cell therapy in mantle cell lymphoma.
Tofersen Fails to Meet Endpoint in Familial ALS Despite Favorable Trends
Tofersen failed to meet its primary end point in change from baseline in ALSFRS-R but differences in total cerebrospinal fluid SOD1 protein and neurofilament light chain were observed.
Diabetes Cell Therapy Improves Insulin Production in First Patient Dosed
The patient’s daily insulin requirement went from 34 units a day at baseline to 2.9 units a day 90 days after treatment.
Access to Gene Therapies for Rare Diseases: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed improving access to gene therapies for patients with rare diseases worldwide.
CLL Cells Edited With CRISPR and mRNA-Based Editing
More than 70% to 80% of homozygous knockout of a given gene can be achieved by electroporating CLL cells with ribonucleoprotein complexes that harbor recombinant Cas9 nuclease and synthetic guides.
CAR T-Cell Therapy for Non-Hodgkin Lymphoma
The chief of oncology and hematology at University of Nebraska Medical Center discussed the use of CAR T-cell therapy in non-Hodgkin lymphoma.
Solid Tumors Targeted in Next-Generation CAR T Collaboration
The collaboration combines AdAlta’s i-bodies and Carina Biotech’s chemokine receptor platform.
Cilta-Cel Outcomes in R/R Multiple Myeloma: Omar Castaneda Puglianini, MD
The hematology and medical oncologist at Moffitt Cancer Center discussed results from the phase 1b/2 CARTITUDE-1 trial of cita-cel in R/R MM.
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.
