Vutrisiran Approved for hATTR Amyloidosis
The approval came earlier than the recently extended PDUFA date of July 24, 2022.
CAR T-Cell Therapy Yields High Response Rates in Follicular Lymphoma
Mustang Bio presented data on MB-106 at the 2022 EHA Congress.
Tisa-Cel Shows Durable Efficacy in Pediatric and Young Adult Patients With R/R B-Cell Acute Lymphoblastic Leukemia
Among treated patients, 42% achieved 5-year event free survival.
Around the Helix: Cell and Gene Therapy Company Updates – June 15, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Re-Dosing With Gene Therapies in Rare Diseases
Takashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.
Dosing Continues in Hemophilia B Gene Therapy Trial
Updated date from the B-LIEVE study is expected in the second half of 2022 and first half of 2023.
Clinical Hold Lifted on Homology’s pheNIX Trial for Phenylketonuria
The trial was placed on hold after elevated liver function test scores.
Massimo Trucco, MD, on Improving Access to Islet Cell Transplant
The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed islet cell transplant for the treatment of chronic pancreatitis.
Dual-Targeted CAR T-Cell Therapy Continues to Show Efficacy in R/R Multiple Myeloma
GC012F also showed efficacy in B-cell non-Hodgkin lymphoma.
Anbal-Cel Shows Promising Responses in Large B-Cell Lymphoma
The CD-19 CAR T-cell therapy is developed using Curocell’s OVISTM platform.
Exa-Cel Eliminates Transfusions in TD Thalassemia and VOCs in Sickle Cell Disease
Results from the CLIMB THAL-111 and CLIMB SCD-121 trials were presented at the EHA 2022 Congress.
Axi-Cel More Efficacious With Higher Toxicity Than Tisa-Cel in Diffuse Large B-Cell Lymphoma
Response rates were significantly higher but patients had more cases of ICANS and CRS with axi-cel treatment compared to tisa-cel treatment.
Lentiviral CAR T Therapy Delivers Good Efficacy, Safety in R/R Multiple Myeloma
Participants in the trial achieved a sustained response and experienced low-grade toxicity.
CD30-Targeted CAR T Cell Therapy Promising in Heavily Pretreated Hodgkin Lymphoma
A phase 2 clinical trial evaluating a T-cell memory enriched anti-CD30 CAR-T is now underway.
CD33 CAR NK Cell Therapy Shows Improved Targeting in Acute Myeloid Leukemia
As of the data cutoff, 3 of 5 treated participants remain in complete response.
Ide-Cel Treatment Associated With Greater Quality of Life Than Belantamab Mafodotin in R/R MM
Statistically significant differences were seen in global health status, pain, and fatigue.
Bluebird Bio's Beti-Cel Gains Unanimous Support of Benefit in β-thalassemia
The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee again voted in favor of the risk-benefit of bluebird bio's gene therapy candidate.
CGTLive’s Weekly Rewind – June 10, 2022
Review top news and interview highlights from the week ending June 10, 2022.
Allogeneic CAR T-Cell Therapy Yields 100% CR Rate in B-NHL
Three of 6 patients have an ongoing response.
Eli-Cel Earns Unanimous FDA AdComm Vote Supporting Risk-Benefit in CALD
The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted in favor of the benefits of bluebird bio's eli-cel in patients with cerebral andrenoleukodystrophy despite substantial safety concerns.
Off the Shelf CD19 Cell Therapy for CAR T Relapse Patients Shows Promise
Data from a phase 1/2a study showed notable response rates in patients who had received a median of 5 prior lines of therapy.
Around the Helix: Cell and Gene Therapy Company Updates – June 8, 2022
CARsgen Therapeutics’ CT041 Shows Promise in Advanced Gastric/Gastroesophageal Junction Adenocarcinoma
The chimeric antigen receptor-modified autologous T cells targeting CLDN18.2 had a manageable safety/tolerability profile and promising efficacy.
Gamma Delta CAR T-Cell Therapy Shows Promising Responses in B-Cell Malignancies
100% overall response and complete response rates were seen in patients treated with prior CD19 CAR T-cell therapy.
Tevogen Initiates Dosing in Final Cohort of COVID Cell Therapy Trial
The fourth and final cohort is dosing participants with the highest investigated dose of 3 x 106 cells/kg.
NY-ESO-1 TCR T-Cell Therapy Shows Clinical Activity in Soft Tissue Sarcoma
Overall response rate was 41.7% and median progression free survival was 7.2 months.
Axi-Cel Superior to SOC in Elderly Patients with Diffuse Large B-Cell Lymphoma
Jason Westin, MD, FACP, leader, DLBCL research team, MD Anderson Cancer Center, discussed results from the ZUMA-7 trial at ASCO 2022.
Islet Cell Therapy Reduces Insulin Dependence in Type 1 Diabetes
The phase 1/2 trial of VX880 remains on clinical hold in the US.
Poverty, Opportunity May Impact CAR T-Cell Therapy Access, Outcomes
The findings suggest that less disadvantaged patients with high disease burden may have greater ability to advocate for CAR T-cell therapy.
Synthetic Binding Domain CAR T Therapy Yields 100% ORR in R/R Multiple Myeloma
69% of patients followed up for at least 1 year continue to have an ongoing response.
