T-Cell Lymphoma CAR T Trial Placed on Hold
Only 1 patient has been dosed in the trial so far, which initiated in October 2021.
Gene Therapy for Friedreich Ataxia Cardiomyopathy Set to Move Forward
The FDA has approved LEXEO Therapeutics’ investigational new drug application.
Improving CAR T-Cell Therapies for Multiple Myeloma: Thomas G. Martin, MD
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed CAR T-cell therapies and safety signals of cilta-cel.
NK Cell Therapy CYNK-101 Wins Orphan Drug Designation for HER2+ Gastric/GEJ Cancers
This designation follows a fast track designation and IND application approval for the agent.
Lysogene Gene Therapies Show Promise in GM1 Gangliosidosis and MPSIIIA
Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.
The Need for Analysis in Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.
Stem Cell-Derived Retinal Implants Demonstrate Long-Term Viability
Investigators did not find any clinical signs of rejection of the mismatched donor cells.
First In-Human Trial to Assess Krabbe Disease Gene Therapy
Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.
Achieving Remission in Chronic Lymphocytic Leukemia With CD4+ CAR T-Cells: Jan Joseph Melenhorst, PhD
The translational immunologist and research professor at Perelman School of Medicine, University of Pennsylvania, discussed persistence of CD4+ CAR T-cells in CLL.
Gene Therapy Well-Tolerated in Late-Onset Pompe Disease
Interim safety data from the FORTIS study were presented at WORLDSympsoium.
Latest Updates on Geographic Atrophy Gene Therapy FOCUS Trial: Nadia K. Waheed, MD
The associate professor from Tufts University School of Medicine discussed the latest updates on the FOCUS trial.
GeneTherapyLive’s Weekly Rewind – February 11, 2022
Review top news and interview highlights from the week ending February 11, 2022.
Molecular Analysis Elucidates Cell Therapy Destiny and Distribution
AVROBIO presented data on their analytical pipeline and exploratory studies of molecular follow-up from their lentiviral cell therapy products.
Gene Therapy Stabilizes Disease Progression in GM1 Gangliosidosis
Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.
Fabry Disease Gene Therapy Shows Encouraging Effect on Cardiac End Points
The dual-mechanism of the therapy helps address both systemic and organ-specific deficits.
CNGB3 Achromatopsia Gene Therapy Improves Visual Sensitivity
Clinical development will be stopped on the CNGA3 achromatopsia program as no clinical improvements were seen.
Around the Helix: Cell and Gene Therapy Company Updates - February 9, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapies for Mucopolysaccharidosis Show Safety, Efficacy
Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.
Treating Solid Tumors With Personalized Immunotherapy: Julian Molina, MD, PhD
The hematologist/oncologist from Mayo Clinic discussed targeting HLA loss in solid tumors.
Study to Evaluate Gene-Edited Cell Therapy for MPSII
The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.
Beti-Cel Yields Durable Transfusion Independence in β-Thalassemia
The gene therapy is currently under FDA review for the treatment of β-thalassemia across genotypes.
The Importance of Collaboration in Developing Next Generation Cell Therapies
Dolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed the company’s collaboration with 2seventy bio to develop TCR T-cell therapies.
Etranacogene Dezaparvovec Superior to FIX Prophylaxis in Hemophilia B
Results from the final analysis of the HOPE-B trial were presented at the 2022 EAHAD meeting.
X-Linked Retinitis Pigmentosa Trial Exceeds Enrollment
Applied Genetic Technologies is also developing AGTC-401 and AGTC-402 for the treatment of achromatopsia.
Assessing EtranaDez for Hemophilia B in HOPE-B: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed results of the final analysis of the HOPE-B study.
GeneTherapyLive’s Weekly Rewind – February 4, 2022
Review top news and interview highlights from the week ending February 4, 2022.
COVID-19 Cell Therapy Trial Completes Dosing
No dose-limiting toxicities or treatment-emergent adverse events have been reported.
Stargardt Disease Gene Therapy IND Cleared to Proceed
The investigational therapy has received orphan drug designation from the FDA for Stargardt disease as well as retinitis pigmentosa.
CAR T-Cell Therapy Efficacious at 10 Years in Chronic Lymphocytic Leukemia
Carl June, MD, discussed the longest-term follow-up data on CAR T-cell therapy so far.
Bispecific TCR Cell Therapy Approved for Unresectable or Metastatic Uveal Melanoma
Kimmtrak is now the first approved therapy for unresectable or metastatic uveal melanoma.
