Understanding EYS Gene Mutations is Key to Retinitis Pigmentosa Treatment
Improved knowledge of the effects of mutations in the EYS gene may help inform new treatments.
GeneTherapyLive’s Weekly Rewind – December 17, 2021
Review top news and interview highlights from the week ending December 17, 2021.
CYAD-211 Induces Favorable Safety, Supports Proof of Concept in Patients With Relapsed/Refractory Myeloma
Safety and the recommended dose of CYAD-211 and the lymphodepletion regimen served as the primary end point of the study.
Cilta-Cel Improves Survival in R/R Multiple Myeloma Over Physician Choice of Treatment
Patients treated with cilta-cel had a 76% reduction in the risk of death compared to patients treated with physician's choice of treatment.
Assessing Axi-Cel in R/R LBC Lymphoma in ZUMA-7: Frederick Locke, MD
The vice chair, Blood and Marrow Transplant and Cellular Immunotherapy Program, and co-leader, Immuno-Oncology, Moffitt Cancer Center, discussed the results of the phase 3 ZUMA-7 trial.
Safety Advantages of Tab-Cel Over Other T-Cell Therapies in EBV+ PTLD
AJ Joshi, MD, chief medical officer, Atara Biotherapeutics, discussed safety findings from the phase 3 ALLELE study.
Anti-Thrombin siRNA Reduces Bleeding in Severe Hemophilia Without Inhibitors
Annualized bleeding rate, spontaneous bleeding, and joint bleeding rates were all reduced in patients dosed with fitusiran compared to those receiving on-demand treatment.
Anti-Thrombin siRNA Therapeutic Reduces Bleeding in Hemophilia
Over half of patients in the fitusiran arm of the ATLAS-INH study had 0 treated bleeding events.
Optogenetics Device With Gene Therapy is Safe, Efficacious in Retinitis Pigmentosa
Following GS030 optogenetic therapy, the first treated patient was able to locate and count objects on a table and could identify crosswalks in the street.
Beti-Cel Induces Transfusion Independence in Patients With β-thalassemia
The findings, which were simultaneously reported in the New England Journal of Medicine, support beti-cel as a potentially curative, one-time treatment option for these patients.
ADI-001 Highly Effective, Safe for Heavily Pretreated NHL in Preliminary Phase 1 Data
ADI-001 does not require genetic engineering to remove TCRs to avoid GvHD, making them ideal for an off-the-shelf cell therapy.
Navigating CAR T-Cell Therapy Referrals: Bijal Shah, MD; Michael Wang, MD
The hematologists from Moffitt Cancer Center and University of Texas MD Anderson Cancer Center discussed navigating the referral process for CAR T-cell therapy in hematologic malignancies.
Axi-Cel Superior to SoC in Second-Line Large B-Cell Lymphoma Treatment
Patients treated with the CAR T-cell therapy had an over 4-fold increase in event-free survival.
GeneTherapyLive’s Weekly Rewind – December 9, 2021
Review top news and interview highlights from the week ending December 9, 2021.
Gene Edited Cell Therapy Improves Fetal Hemoglobin Expression in Patients With Sickle Cell Disease
All evaluable treated participants experienced a reduction in vaso-occlusive events after treatment with ARU-1801.
Hemophilia B Gene Therapy Meets Primary End Points in HOPE-B Trial
CSL Behring plans to submit regulatory applications in the US and EU in the first half of 2022 for the AAV gene therapy etranacogene dezaparvovec.
Using HLA LOH as a Biomarker in Targeting Cancers: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed the role of HLA loss in common cancers and its use as a biomarker.
Improving Personalized Cell Therapies for Solid Tumors
Cedrik Britten, MD, chief medical officer, Immatics discussed data on IMA203 presented at SITC 2021.
Autologous Adipose Stem Cells Safe in Alzheimer Disease, More Efficacy Data Needed
Autologous adipose stem cell data in a small cohort provides a feasibility for future validation studies.
Managing CAR T-Associated Neurotoxicity and CRS: Bijal Shah, MD, MS; Michael Wang, MD
The hematologists from Moffitt Cancer Center and MD Anderson discussed managing CAR T-cell therapy-associated toxicities.
Using Polygenic Risk Score to Better Outcomes in Primary Open-Angle Glaucoma
Current studies are using a polygenic risk score to evaluate the risk and clinical outcomes in primary open-angle glaucoma (POAG).
CAR T-Cell Therapy Shows Efficacy and Safety in B-Cell Malignancies
Adicet Bio reported positive interim data from the phase 1 GLEAN study.
Reprogramming the Immune System for Kidney Transplant and Scleroderma
Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the company’s goal to reprogram the immune system.
Celularity, Imugene Collaboration to Focus on CAR T in Solid Tumors
The companies will combine Imugene’s CF33-CD19 oncolytic virus and Celularity’s investigational placental-derived CAR T-cell therapy.
Gene Therapy for Diabetic Macular Edema Shows Mixed Efficacy, Safety
Findings from the phase 2 INFINITY trial identified dose-dependent safety outcomes of ADVM-022 in patients with diabetic macular edema.
More Research Needed to Understand COVID-19 Vaccine Effects in CAR T Recipients
Further understanding of how COVID-19 vaccinations affect CAR T-cell therapy recipients is needed.
Gene Therapy for Sanfilippo Syndrome: Latest Updates
Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed LYS-SAF302, its mechanism of action, trial data, and further research.
Challenges in R/R LBC Lymphoma After CAR T Therapy
Julio Chavez, MD, MD, discussed treatment options for LBCL after relapsing on CAR T-cell therapy.
Importance of Screening and Early Treatment in Genetic Disorders: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed gene therapy studies in AADC deficiency, Parkinson disease, SMA, and Pompe disease.
GeneTherapyLive’s Weekly Rewind – December 3, 2021
Review top news and interview highlights from the week ending December 3, 2021.
