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.
The Potential of Gene Therapy for AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the mechanism of gene therapy in AADC deficiency.
FDA Grant Supports Recessive Dystrophic Epidermolysis Bullosa Gene Therapy Study
Dabocemagene autoficel has previously received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations.
Encapsulated Cell Therapy for Retinal Diseases
John Pollack, MD, discusses gene therapy advancements for retinal diseases.
Developing Personalized Immunotherapies for Solid Tumors
Jessica Baker Flechtner, PhD, chief scientific officer, Genocea, discussed the company’s focus on solid tumors and manufacturing cell therapies.
Gene Editing Clinical Trial for PKU Announced
Homology is also developing a gene therapy, HMI-102, being evaluated in the phase 1/2 pheNIX trial.
Improving Practicality of Cell and Gene Therapies
Josh Ludwig, global director, commercial operations, ScaleReady, discussed making cell and gene therapy widely practical and viable for patients with cancer.
Gene Therapy Promotes Wound Closure of Diabetic Foot Ulcers
VM202 was found to be particularly effective in treating neuroischemic ulcers.
Collaborating to Develop Oncologic Cell Therapies
André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s partnerships and science.
Eli-cel Gene Therapy for Early CALD to Be Withdrawn From EU, UK
bluebird bio is also planning to withdraw the marketing authorization for their β-thalassemia therapy, beti-cel, from the EU and UK.
Mantle Cell Lymphoma Treatment Landscape 1 Year After CAR T Approval
Michael Wang, MD, discussed the evolution of treatment in the MCL space specifically with the emergence of chimeric antigen receptor T cells.
The Potential of ALLO-715 in Relapsed/Refractory Multiple Myeloma: Doris Hansen, MD
The hematologist from Moffitt Cancer Center discussed the promise of ALLO-715 in relapsed/refractory multiple myeloma.
Next Steps With CAR T-Cell Therapy in Multiple Myeloma: Omar Castaneda Puglianini, MD
The hematologist and medical oncologist discussed next steps with CAR T-cell therapy in penta-refractory multiple myeloma.
In-Vivo Genome Editing Therapy Shows Efficacy in Children With MMA
The phase 1/2 SUNRISE trial is now enrolling patients as young as 6 months old with methylmalonic acidemia after positive review under a DSMB.
Potential of mRNA-Based Gene Therapies
Jake Becraft, PhD, chief executive officer and cofounder, Strand Therapeutics, discussed the company’s future research and plans.
GeneTherapyLive’s Weekly Rewind – October 22, 2021
Review top news and interview highlights from the week ending October 22, 2021.
Developing Customizable CAR T-Cell Therapies
Rick Fair, president and chief executive officer, Bellicum Pharmaceuticals, discussed the company’s GoCAR, iMC, and caspaCIDe technologies.
