Phase 3 Trial Seeks to Continue Supporting Arsa-Cel Gene Therapy for MLD
The gene-edited cell therapy has been approved as Lenmeldy by the FDA.
ODAC Meeting: Cilta-Cel Gets Unanimous Vote, Ide-Cel Wins Majority Vote for Benefit-Risk Profile
Cita-cel, approved as Carvykti, is currently under review for an indication expansion as an earlier line of therapy, which the FDA will decide on by April 5, 2024.
FDA Approves Liso-Cel (Breyanzi) as First CAR-T for CLL, SLL
Breyanzi was approved for the expanded indication under the accelerated approval pathway.
Gene Therapy Improves Functional Measures in XLRP
Investigators found AGTC-501 to have a favorable benefit-risk profile.
Gene Editing Investigations Mature With heart-1 Trial for Familial Hypercholesterolemia
Verve Therapeutics plans to expand evaluations into the US after an IND clearance was delayed by a clinical hold.
CK0803 Treg Therapy Seems Safe for ALS, Trial Set to Treat Second Cohort
The positive DSMB recommendation came after no participants were found to experience any serious adverse events from the therapy.
ODAC Meeting to Discuss Ide-Cel for Earlier Lines of Triple-Class Exposed R/R Multiple Myeloma Treatment
The committee is set to discuss idecabtagene vicleucel’s sBLA on March 15, 2024.
Alan Beggs, PhD, on Digging Dipper Into Unexpected Toxicities With Neuromuscular Gene Therapy
The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed investigations into liver toxicity and deaths after AT132 gene therapy.
RGX-202 Gene Therapy Reduces CK, Yields Detectable Microdystrophin in Patients With DMD
RGX-202 delivers a slightly larger form of microdystrophin than other gene therapies, including the CT domain of dystrophin.
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.