Data Roundup: February 2024 Features Updates in Blood Cancers, Lysosomal Storage Disorders at WorldSymposium, Tandem Meetings
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
First Patient With Esophageal Cancer Receives TAC101-CLDN18.2 Cell Therapy
The patient is doing well so far and may receive a booster dose of the TAC cell therapy.
UX111 Reduces Heparin Sulfate, Correlating With Benefit in Cognitive Function in Pediatric MPSIIIA
Investigators concluded that the data support the use of HS as a predictive biomarker in Sanfilippo syndrome.
mHA-Specific Donor Tregs Show Potential in Preventing GvHD in Donor HCT
The expanded donor Treg clones maintained lineage fidelity and were persistent through 1 year post-HCT.
Supporting CAR T-Cell Therapy Treatments With Manufacturing Efficiency and Capacity
Chris McDonald, senior vice president and global head of technical operations, Kite Pharma, discussed the recent axi-cel manufacturing change approval.
LV20.19 CAR T-Cells Show Efficacy in CLL, Richter, But Unusual Rates of IEC-HS
Going forward, the dose for patients with CLL has been reduced to 1x106 cells/kg.
Multi-Respiratory Virus T-Cell Therapy Well-Tolerated after HCT
Beleaguered company AlloVir has shut down the program before moving on to phase 2.
Immune Effector Cell Therapy Associated With Longer PFS After Ide-Cel Relapse in R/R MM
Differences between IEC and non-IEC post-relapse outcomes showed some significance in PFS but not in ORR.
IL1RAP, CAR, TGFβi, CXCR2 Modified NK Cells Enhances In Vitro Efficacy in Ewing Sarcoma
The researchers are continuing to investigate in vivo efficacy to pave the way to IND-enabling studies.
Analysis Informs Need for New Staging, Therapeutic Evaluations for EMD in B-ALL Prior to CAR T-Cell Therapy
Patients with high bone marrow burden and CNS/non-CNS EMD were found to experience the worst OS and EFS outcomes.
Around the Helix: Cell and Gene Therapy Company Updates – February 21, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
IND Cleared for Solid Tumor Trial of ULBP6-Targeting Natural Killer (NK) Cell Activator
The ULBP6 target was discovered through 23andMe’s proprietary research platform of de-identified human genetic and phenotypic information.
Oricell’s GPRC5D-Targeted CAR Cleared for US Evaluation in Patients With R/R Multiple Myeloma
Promising data on OriCAR-017 were previously published in the Lancet Haematology in 2023.
Reducing Turnaround Time of Axi-Cel CAR T-Cells
Chris McDonald, senior vice president and global head of technical operations, Kite Pharma, discussed axi-cel's FDA approved manufacturing changes.
IND Cleared for First RNA Exon Editor Therapy, Trial to Initiate in Stargardt Disease
Ascidian Therapeutics presented positive preclinical data at the 2023 ASGCT meeting.
European Commission Approves Exa-Cel for SCD and TDT
The approval follows the separate US approvals in December 2023 and January 2024.
FDA Pushes Rocket’s LAD-I Gene Therapy PDUFA for CMC Review
Rocket submitted additional CMC data in response to FDA information requests.
Sylentis’ siRNA Therapy Fails to Improve Symptoms of Dry Eye Disease Due to Sjögren’s Syndrome
The phase 3 trial of tivanisiran failed its primary endpoint.
CR in First Patient With B-ALL Treated With PMB-CT01 CAR T-Cell Therapy
The BAFFR-CAR T-cell therapy is being investigated in a phase 1 single-center and phase 1 multicenter trial in B-ALL and B-NHL.
Data Roundup: January 2024 Features Updates in GI Cancer, AMD, Neurology
Discontinued GM1 Gangliosidosis Gene Therapy Shows no Evidence of Clinical Benefit
Data from defunct company Lysogene’s discontinued trial of LYS-GM101 were presented at WORLDSymposium.
Arsa-Cel Continues to Show Durable Benefit in MLD Outcomes Over Natural History
Orchard Therapeutics has also randomized the first patient with Hurler Syndrome in the phase 3, registrational HURCULES trial of OTL-203 gene therapy.
MAGE-A4 T-Cell Engager Cleared for Phase 1 Trial in Solid Tumors
CDR-Life also recently presented positive preclinical data at the November 2023 SITC Annual Meeting.
First Patient With AML Receives CD33-CAR T Cells From Previous HCT Donor
Vor Bio recently also announced data from the phase 1/2 trial of trem-cel.
FDA Approves Kite’s Axi-Cel Manufacturing Change to Reduce Turnaround Time
Kyverna has also announced data on its proprietary CAR-T manufacturing process, Ingenui-T.
Indapta Treats First Patients With Multiple Myeloma, NHL, With NK Cell Therapy
The company also shared that it has manufactured sufficient IDP-023 to supply the Phase 1 clinical trial through the second half of 2024.
Fate and Ono Initiate Enrollment for HER2-CAR T Trial
FT825 is an iPSC-derived multiplexed-engineered, CAR T-cell therapy targeting HER2 with a novel antigen binding domain.
Allogeneic CD22 CAR Cleared for First-in-Human Trial in B-Cell Malignancies
Sana also recently announced data from the first patient treated with its CD19-targeted CAR.
Genascence Initiates Knee Osteoarthritis Gene Therapy Trial
An investigator-initiated trial presented positive data at the ESGCT annual meeting in October 2023.
Bria-IMT Cell Immunotherapy Shows 71% Intracranial ORR in Breast Cancer With CNS Metastases
BriaCell also shared specific positive data from one patient with proptosis and ocular pain.