EGFR-Targeting γδ2T-Cell Cleared for Solid Tumor Trial
Acepodia is also evaluating a CD20-targeted γδ2T-cell therapy in patients with non-Hodgkin lymphoma.
FDA Requires Boxed Warnings for Secondary Cancer for Approved CAR-T Therapies
The FDA has requested that black box warnings related to secondary cancer risks be added to all 6 CAR-T therapies currently on the market.
MCO-010 Phase 2b Data Supports BLA Submission for Retinitis Pigmentosa
Nanoscope Therapeutics received feedback from the FDA endorsing significant change in BCVA as a primary endpoint supporting approval.
Gene Therapy Improves Vision Recovery in LHON Over Idebenone, Natural History
Data from a meta-analysis of 3 groups of patients were presented at AAN 2024.
A Slow Embrace: Hemophilia's Gradual Adoption of Gene Therapy
For World Hemophilia Day, CGTLive takes a look at how the hemophilia field has adapted to the introduction of gene therapy to the treatment landscape.
AskBio Evaluates ACTUS-101 Gene Therapy for Late-Onset Pompe Disease
The more recent data were presented at the 2022 ASGCT Meeting.
4DMT Aligns on Path Forward for Cystic Fibrosis Gene Therapy
The company plans to initiate a pivotal trial of 4D-710 in the second half of 2025.
RGX-314 Reduces nAMD Treatment Burden, Shows Disease Improvements
One participant experienced a possibly related serious AE of pigmentary changes in the macula with severe vision reduction.
Allogeneic CAR-T Yields Some Responses in Multiple Myeloma After Anti-BCMA Therapy
The best response was very good partial response in 3 of 5 participants.
Gene Therapy Stabilizes Neurocognition, Reduces CSF D2S6 in MPSII
Paul Harmatz, MD, professor, UCSF, discussed benefits in neurocognitive outcomes and ERT therapy usage after RGX-121 gene therapy for Hunter syndrome.
ASC Therapy Does Not Show Significant Difference Over Placebo in Treating Xerostomia
Adipose-derived MSCs did significantly increase salivary flow rate from baseline in patients with previous head and neck cancer.
CAR TEAM Cells Show Promise in Multiple Preclinical Models of Pancreatic Cancer
The cells showed activity toward PDAC cells as well as cancer-associated fibroblasts, which may address challenges with the tumor microenvironment.
Data Roundup: March 2024 Features Updates in Muscular Dystrophies at MDA 2024
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
FDA Approves Ide-Cel's Expanded Indication to Second-Line, Triple-Class Refractory Multiple Myeloma
The approval comes a couple weeks after ODAC voted in favor of Abecma’s benefit-risk profile and sBLA.
MD Anderson’s CAR/NK Therapy Shows Improved Safety Profile in B-Cell Malignancies
Investigators observed no notable toxicities including cytokine release syndrome, neurotoxicity or graft-versus-host disease.
Kyverna’s CAR T Seems Feasible in Multiple Sclerosis
KYV-101 is also being evaluated in lupus nephritis, myasthenia gravis, and systemic sclerosis.
First US Patient With Hemophilia A Receives Gene Therapy
Val-rox was approved as Roctavian in June 2023.
Verve Moves on to Second Gen Cardiovascular CRISPR Therapy After Adverse Events
The sixth participant dosed experienced grade 3 treatment-induced ALT increases and thrombocytopenia.
NG-01 Cell Therapy Reduces NfL, GFAP in Patients With Progressive MS
Data from the OLE of a phase 2 clinical trial at Hadassah Medical Center were presented at the 2024 ACTRIMS meeting.
Nusinersen Improves Some Suboptimal Motor Functions Post-SMA Gene Therapy
Updated data from the RESPOND study evaluating Spinraza in patients with SMA after onasemnogene abeparvovec were presented.
Patients With Hemophilia Biding Their Time for Gene Therapy
Many surveyed participants were content with current treatments or wary of gene therapy’s adverse events and durability.
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.