Study Aims to Gain Insights into Genetics of Glaucoma
The study is aiming to gain insight into glaucoma in African American populations.
CRISPR/Cas9-Edited CAR and NK Cell Therapies Show Preclinical Efficacy
CRISPR Therapeutics presented positive data on 2 preclinical programs at the 2021 SITC meeting.
Utility of CAR T-Cell Therapy in Relapsed Multiple Myeloma: Thomas G. Martin, MD
The co-leader of Cancer Immunology & Immunotherapy Program, UCSF Helen Diller Family Comprehensive Cancer Center discussed he utility of CAR T-cell therapy in late relapsed multiple myeloma.
GeneTherapyLive’s Weekly Rewind – November 26, 2021
Review top news and interview highlights from the week ending November 26, 2021.
Developing Gene Therapies for Indications With Unmet Needs
The chief executive and scientific officers of Lysogene discussed Lysogene’s patient-focused therapy development.
CD19+ B-Cell Malignancy Cell Therapy Receives RMAT Designation
In 26 patients with LBCL followed for at least 28 days, the objective response rate (ORR) was 58% with complete responses (CRs) in 38% of patients.
Advantages of Developing Platform-Agnostic Gene Therapies
The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.
Hemophilia A Gene Therapy Placed on Clinical Hold
Pfizer and Sangamo voluntarily paused the trial after participants experienced FVIII activity levels of over 150%.
Targeting the Putamen in AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency.
FasTCAR Cell Therapy Granted Orphan Drug Designation for Multiple Myeloma
The designation follows durable responses seen in a phase 1 study of the agent.
Overcoming Challenges With CAR T Therapies in Solid Tumors: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed the role of HLA loss in a variety of cancers.
FDA Accepts Beti-Cel BLA for β-thalassemia
The investigational gene therapy, which will receive priority review, has a PDUFA date of May 20, 2022.
Gene Therapy for Parkinson Disease Demonstrates Safety, Improved Motor Function
From baseline, all participants showed improvements on CGI-Improvement scale scores while all but 1 patient demonstrated improvement on modified Hoehn and Yahr scores.
Cilta-Cel is Well-Tolerated with Durable Efficacy in R/R Multiple Myeloma
The recommended phase 2 dose of cilta-cel elicited deep and durable responses, along with a tolerable safety profile.
CAR T-Cell Therapy's Continued Benefit in B-Cell Non-Hodgkin Lymphoma
Positive data has been seen with CAR T-cell therapy in patients with indolent lymphoma.
Gene Therapy Optogenetics Combination Efficacious in Retinitis Pigmentosa: José-Alain Sahel, MD
The professor from University of Pittsburgh School of Medicine discussed the combination of an optogenetics device and gene therapy in treatment of RP.
CAR T or Transplant in DLBC Lymphoma: John M. Burke, MD; Marin F. Xavier, MD
Experts discussed using newer targeted treatment approaches as frontline therapy instead of transplant in patients with relapsed/refractory diffuse large B-cell lymphoma.
Hemophilia A Gene Therapy Shows Sustained Factor VIII Expression
A 1-stage factor VIII assay revealed no apparent decrease in factor VIII activity over time.
GeneTherapyLive’s Weekly Rewind – November 19, 2021
Review top news and interview highlights from the week ending November 19, 2021.
Gene-Edited, Autologous HSP Cells Correct Metabolic Activity in Hurler Syndrome
The gene-edited approach hopes to overcome with the shortcomings of autologous hematopoietic stem-cell transplantation.
RGX-314 Gene Therapy Efficacious in Wet AMD: Robert L. Avery, MD
The chief executive officer of California Retina Consultants discussed the positive results of RGX-314 gene therapy in wet AMD.
CD19 CAR T and Therapies in LBC Lymphoma: Marin F. Xavier, MD; John M. Burke, MD
Experts discussed using CD19-targeted agents in patients with various subtypes of diffuse large B-cell lymphoma.
Danon Disease Gene Therapy Yields Sustained Clinical Benefit
Following updated safety data, Rocket Pharmaceuticals has decided to discontinue the high-dose cohort.
ASCT Utilization Rates in Mantle Cell Lymphoma: Yazeed Sawalha, MD
The hematologist at Ohio State University Comprehensive Cancer Center–The James discussed utilization rates of autologous stem cell transplant in patients with mantle cell lymphoma.
Charcot-Marie-Tooth Gene Therapy Yields Improvements in Disability and Muscle Loss
The phase 1 trial is the first in-human trial to assess a gene therapy for CMT.
IND Cleared for Focal Epilepsy Cell Therapy Phase 1/2 Trial
The cell therapy is set to be evaluated in a 2-part, phase 1/2 trial.
Assessing Tab-Cel for EBV+ Post-Transplant Lymphoproliferative Disease
AJ Joshi, MD, chief medical officer, Atara Biotherapeutics, discussed data from the ALLELE study to be presented at ASH 2021.
OpRegen Shows Durable Efficacy in Dry AMD Geographic Atrophy
The Doheny Image Reading and Research Lab is leading a sub study to analyze OCT images with their proprietary 3D-OCTOR software.
Late Mortality Has Decreased in Younger Patients Undergoing Allogeneic Blood or Marrow Transplantation
A reduction in late mortality among patients who received allogeneic blood or marrow transplantation in the last 40 years was limited to those who received treatment at a younger age.
A New Indication for Gene Therapy: Aging
Dan Oliver, cofounder and chief executive officer, Rejuvenate Bio, discussed the company’s ultimate goal of reversing aging.
