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Pfizer and Sangamo voluntarily paused the trial after participants experienced FVIII activity levels of over 150%.

The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency.

The designation follows durable responses seen in a phase 1 study of the agent.

The hematologist/oncologist from Mayo Clinic discussed the role of HLA loss in a variety of cancers.

The investigational gene therapy, which will receive priority review, has a PDUFA date of May 20, 2022.

From baseline, all participants showed improvements on CGI-Improvement scale scores while all but 1 patient demonstrated improvement on modified Hoehn and Yahr scores.

The recommended phase 2 dose of cilta-cel elicited deep and durable responses, along with a tolerable safety profile.

Positive data has been seen with CAR T-cell therapy in patients with indolent lymphoma.

The professor from University of Pittsburgh School of Medicine discussed the combination of an optogenetics device and gene therapy in treatment of RP.

A 1-stage factor VIII assay revealed no apparent decrease in factor VIII activity over time.

Review top news and interview highlights from the week ending November 19, 2021.

The gene-edited approach hopes to overcome with the shortcomings of autologous hematopoietic stem-cell transplantation.

The chief executive officer of California Retina Consultants discussed the positive results of RGX-314 gene therapy in wet AMD.

Experts discussed using CD19-targeted agents in patients with various subtypes of diffuse large B-cell lymphoma.

Following updated safety data, Rocket Pharmaceuticals has decided to discontinue the high-dose cohort.

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.

The phase 1 trial is the first in-human trial to assess a gene therapy for CMT.

The cell therapy is set to be evaluated in a 2-part, phase 1/2 trial.

AJ Joshi, MD, chief medical officer, Atara Biotherapeutics, discussed data from the ALLELE study to be presented at ASH 2021.

The Doheny Image Reading and Research Lab is leading a sub study to analyze OCT images with their proprietary 3D-OCTOR software.

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.

Dan Oliver, cofounder and chief executive officer, Rejuvenate Bio, discussed the company’s ultimate goal of reversing aging.

The NK cell therapy is also being evaluated in a phase 1 study for acute myeloid leukemia.

The professor of medicine at Duke University School of Medicine previously served as commissioner in 2016.

The hematologist from Ohio State University Comprehensive Cancer Center–The James discussed the impact of socioeconomic factors on completion of autologous stem cell transplant in patients with mantle cell lymphoma.

Overall survival was 72% at 12 months but dropped to 54% at 14.6 months.

The request aims to ensure the comparability of omidubicel manufactured at different sites.

The director of Lymphoma Clinical Research at the University of Texas MD Anderson Cancer Center, discussed the future of DLBCL treatment.