ODAC Supports MRD Endpoint in Accelerated Approval for Multiple Myeloma
The ODAC unanimously voted in favor of the use of minimal residual disease negativity.
NIH Strategizes to Streamline Gene Therapy Development
PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed ongoing initiatives.
Parkinson Gene Therapy Shows Stability, Some Improvements in ON/OFF Time Disease Measures
Based on these data, the phase 2 Regenerate PD trial of AB-1005 will begin enrolling later this year.
Looking Forward to A Potential First Cell Therapy Approval for Sarcomas
John A. Charlson, MD, associate professor of medicine, Medical College of Wisconsin, discussed advantages of afami-cel for synovial sarcoma and other solid tumors.
ASO Fast Tracked for Myotonic Dystrophy
PGN-EDODM1 is being evaluated in the US and Canada in a phase 1 trial.
TMZ γδ T-Cell Regimen May Have Potential in Treating Glioblastoma
A phase 1 trial evaluating INB-200 has shown a PFS benefit in treated participants.
Taysha Transfers Deprioritized Gene Therapy Programs Back to Partners
The company’s TSHA-201 AAV gene therapy for Rett syndrome is its current priority.
Taking Lessons Learned With CAR-T in Oncology to the Autoimmune Space
David Porter, MD, director of Cell Therapy and Transplant, Penn Medicine discussed how experience in oncology enabled the logical expansion.
First Patient With Diabetic Foot Ulcers Screened for SkinTE Pivotal Trial
Another recent program in the investigational landscape was Helixmith’s deprioritized Engensis diabetic neuropathy gene therapy.
iNKT Cells Safe, Shows Anti-Inflammatory Response in Patients With SARS-CoV-2
The cells may have potential to modulate disease in other cases of respiratory disease and critical illness.
Lineage Cell to Evaluate Novel Delivery of OPC1 Cell Therapy for Spinal Cord Injury
The company expects to open the first clinical site in the DOSED study in the second quarter of 2024.
First Patient With Gastric Cancer Dosed in iNKT/CTLA-4 Inhibitor/Anti-PD-L1 Trial
A case study previously described successful immune modulation with AgenT-797 in a patient with high-risk gastric cancer leading to a partial response.
One Big Step Away From BLA Submission for Sangamo’s Fabry Gene Therapy
The FDA is allowing a small, single-arm study to support a BLA submission of ST-290, if Sangamo can find help to continue developing the therapy.
Improving Quality of Life in Patients With Fabry With Gene Therapy
Robert Hopkin, MD, Cincinnati Children’s Hospital Medical Center, discussed unmet needs and patient-reported outcomes from the STAAR trial.
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.