Gene Therapy Well-Tolerated in Fabry Disease
Primary efficacy data released by Sangamo Therapeutics showed that above normal α-Gal A activity was maintained for up to a year after treatment.
Socioeconomic Factors Affect Stem Cell Transplant in Mantle Cell Lymphoma
Yazeed Sawalha, MD, hematologist, Ohio State University Comprehensive Cancer Center, discussed how socioeconomic factors can affect use of ASCT in mantle cell lymphoma.
Challenges in Developing Gene Therapy for AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the challenges of working with a rare disease.
Autologous HSCT's Role in the Mantle Cell Lymphoma Treatment Landscape
Autologous hematopoietic stem-cell transplantation maintained its long-term efficacy over interferon alfa in patients with mantle cell lymphoma.
Developing Gene Therapies for Rare Diseases
The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s lead programs and future plans.
New Project Aims to Increase CAR T Safety, Availability
A new project is manufacturing a “purified” version of CAR-T-cells currently used to treat types of lymphoma and leukemia.
Real-World Efficacy of Brexucabtagene Autoleucel in Mantle Cell Lymphoma: Luhua Wang, MD
The professor from University of Texas MD Anderson Cancer Center discussed the real-world efficacy of the first CAR T therapy for the treatment of mantle cell lymphoma.
GeneTherapyLive’s Weekly Rewind – November 5, 2021
Review top news and interview highlights from the week ending November 5, 2021.
CAR T Therapy as a New Option in Non-Hodgkin Lymphoma: Michael R. Bishop, MD
The hematological oncologist from University of Chicago Medicine discussed chimeric antigen receptor T cells therapy in non-Hodgkin lymphoma.
Novel Therapies Reduce the Need for Chemotherapy in Leukemias and Lymphomas
Dipenkumar Modi, MD, discussed novel treatment options in leukemias and lymphomas.
Gene Therapies Show Efficacy in Fabry Disease, Choroideremia
4D Molecular Therapeutics announced positive data from trials assessing 2 of their gene therapies.
Future Research With NK and CAR T Therapy in Multiple Myeloma: Irene Ghobrial, MD
The director at Dana-Farber Cancer Institute discussed future efforts to prevent the development of multiple myeloma.
Tisagenlecleucel Granted Priority Review for R/R Follicular Lymphoma
The FDA also accepted a Type II Variation for the use of tisagenlecleucel in patients with R/R FL following 2 prior lines of treatment.
Improving Cell Therapy Manufacturing Through Strategic Partnerships
Paul Lammers, MD, MSc, president and chief executive officer, Triumvira Immunologics, discussed the challenges of manufacturing cell therapies.
FDA Extends Cilta-Cel BLA PDUFA Date for R/R Multiple Myeloma
The FDA has pushed cilta-cel's BLA PDUFA date back by almost 4 months.
Improving Outcomes in AADC Deficiency With Gene Therapy: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed further research he would like to do with the gene therapy PTC-AADC.
Cell Therapy Cancer Vaccine Shows Promise for Progression-Free Survival in Metastatic Pancreatic Cancer
The vaccination therapy improved progression-free survival by 3.1 months over standard care in 1 patient treated.
Personalized Vaccine-Based Therapy for Multiple Myeloma: Nina Shah, MD
The hematologist/oncologist from UCSF Helen Diller Family Comprehensive Cancer Center discussed the potential of vaccine-based therapies in multiple myeloma.
Rare Disease Consortium to Focus on Optimizing AAV Gene Therapies
Partners in the Bespoke Gene Therapy Consortium include the NIH, FDA, private companies such as Pfizer and Biogen, and non-profits.
Efficient Gene Editing in Monogenic Diseases
André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s expansion into gene therapies.
Cell Therapy May Yield Deep Remissions in Multiple Myeloma
Ide-cel's March 2021 FDA approval marks a significant advance in relapsed/refractory multiple myeloma treatment.
Developing Safer, Next-Generation CAR T-Cell Therapies
Rick Fair, president and chief executive officer, Bellicum Pharmaceuticals, discussed the company’s programs and future plans.
The Roles of Stem Cell Transplant and CAR T-Cell Therapy in B-Cell Lymphoma
Doctors debated the roles of allogeneic hematopoietic stem cell transplant and CAR T-cell therapy for the treatment of aggressive B-cell lymphoma.
CRISPR Therapy for ATTR Amyloidosis Granted Orphan Drug Designation
The phase 1 study of NTLA-2001 previously showed positive interim data, including serum TTR reductions, which were reported in June 2021.
Optimizing COVID-19 Vaccines in Patients Receiving CAR T Therapy: Muhammad Bilal Abid, MD, MRCP
The assistant professor of medicine at the Medical College of Wisconsin discussed immune-compromising factors that are indigenous to CAR T-cell therapy recipients.
Stem Cell-Derived Eye Drops Improve Outcomes in Dry Eye Disease
Researchers have developed a corneal epithelial stem cell-derived eye drop therapy for dry eye disease.
GeneTherapyLive’s Weekly Rewind – October 29, 2021
Review top news and interview highlights from the week ending October 29, 2021.
MPS 2 Systemic Gene Therapy Evaluated in New Trial
The FDA cleared the IND application for HMI-203 in October 2021.
Targeting CD19 With CAR T in R/R DL B-Cell Lymphoma: Brian T. Hill, MD, PhD; Mazyar Shadman, MD, MPH
Doctors from University of Washington and Cleveland Clinic discussed targeting CD19 in CAR T-cell therapy for relapsed/refractory DLBCL.
Cell Therapy, Other New Strategies for Chronic Lymphocytic Leukemia
Liso-cel is at the forefront of clinical development for patients with chronic lymphocytic leukemia.
