Axi-Cel Shows Favorable Outcomes in Large B-Cell Lymphoma Regardless of Race, Ethnicity
No differences were found in overall survival and progression free survival across races.
CGTLive’s Weekly Rewind – June 3, 2022
Review top news and interview highlights from the week ending June 3, 2022.
Allogeneic CAR T-Cell Therapy Well-Tolerated and Effective Across B-Cell Malignancies
A recent meta-analysis reviewed data across 146 patients in 9 clinical trials.
CAR T-Cell Therapy Yields High Response Rates in B-NHL
Relma-cel was previously approved in China in September 2021 for the treatment of B-cell non-Hodgkin lymphoma.
ICER Finds Beti-Cel Superior to Standard-of-Care for β-Thalassemia
Beti-cel was rated a B+ for lingering questions about durability and unknown risks.
NK Cell Therapy Avelumab Combo Well Tolerated With Some Clinical Activity in Sarcoma
A phase 1 trial evaluated the combination in patients regardless of PD-L1 status and no differences were seen between those negative or positive.
Tim Miller, PhD, on Supporting Gene Therapy Development
The cofounder, president, and chief executive officer of Forge Biologics discussed the company’s approach to gene therapy manufacturing.
Around the Helix: Cell and Gene Therapy Company Updates – June 1, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Val-Rox BLA for Hemophilia A Delayed Again
BioMarin now expects to file in September 2022 instead of June.
Donald Kohn, MD, on the Trajectory of Gene Therapy
The renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.
Liso-Cel Yields High Response Rates and Positive PROs in LBCL
Treated patients had significant improvements in fatigue and lymphoma symptoms.
Lifileucel Shows Promising Data in Advanced Melanoma
A lower objective response rate in cohort 4 reflected a higher disease burden with a greater number of tumors.
FDA Approves Tisa-Cel Expanded Indication in R/R Follicular Lymphoma
The approval was based on data from the phase 2 ELARA clinical trial, in which a complete response of more than 65% was observed.
Expediting the Development of Patient-Specific iSPC Cell Therapies
Stefan Braam, PhD, chief executive officer of Ncardia and Cellistic discussed the companies’ work in the cell therapy space.
Lentiviral Cell Therapy Normalizes Hemoglobin in Pyruvate Kinase Deficiency
RP-L301 also improved hemolysis and eliminated the need for red blood cell transfusions for up to 1year after therapy.
Noopur Raje, MD, on Unmet Needs With CAR T-Cell Therapy in Multiple Myeloma
The director of the Center for Multiple Myeloma and Mass Gen discussed unmet needs with CAR T-cell therapy in multiple myeloma.
CGTLive’s Weekly Rewind – May 27, 2022
Review top news and interview highlights from the week ending May 27, 2022.
Developing Safer Viral Vectors for Gene Therapy
The chief executive officer of Ring Therapeutics, Tuyen Ong, MD, discussed the potential of anellovirus vectors for use in gene therapy.
Amy Pooler, PhD, on Improving Gene Delivery to the CNS
The vice president of neuroscience at Sangamo Therapeutics discussed identifying novel capsids with improved neuronal transduction to the CNS.
Around the Helix: Cell and Gene Therapy Company Updates – May 25, 2022
Jason Fontenot, PhD, on Reducing the Need for Immunosuppression in Renal Transplantation
The chief scientific officer of Sangamo Therapeutics discussed the first-in-human STEADFAST trial.
Gene Therapy May Eliminate Need for ERT in Late-Onset Pompe Disease
All study participants met criteria to remain off enzyme replacement therapy through 104 weeks after treatment.
Gene Therapy for Knee Cartilage Lesions Receives RMAT Designation
NeoCart was originally developed by the company Histogenics.
Andras Heczey, MD, on Antitumor Activity of CAR NKTs in Solid Tumors
The director of the Liver Tumor Program at Texas Children’s Hospital discussed a phase 1 study of CAR NK T-cells in neuroblastoma.
EtranaDez Accepted for Priority Review in Hemophilia B
A final analysis of the pivotal HOPE-B study was presented at ASGCT 2022.
Cell Therapy Modulates Tumor Microenvironment in Glioblastoma
The cell therapy was tolerated although there was a SUSAR of GGT elevations.
Carsten Bönnemann, MD, on Identifying At-Risk Genotypes for DMD Gene Therapy
The senior investigator from NINDS discussed findings investigating serious AEs across different trials.
Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion
The positive opinion was based on findings from studies conducted in Taiwan.
Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene-Edited Cell Therapy in PKD
The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.
Gene Therapy for LAD-I Posts Impressive Survival Data
In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.
