Cautious Optimism for GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.
Patient-Informed Study to Evaluate Hunter Syndrome Gene Therapy
The trial design was informed by both patient and physician perspectives.
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.
The Potential Impact of Gene and Cell Therapy in Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.
First In-Human Trial to Assess Krabbe Disease Gene Therapy
Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.
Gene Therapy Well-Tolerated in Late-Onset Pompe Disease
Interim safety data from the FORTIS study were presented at WORLDSympsoium.
Achieving Developmental Milestones With GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed new data from the IMAGINE-1 trial presented at WORLDSymposium.
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.
Gene Therapies for Mucopolysaccharidosis Show Safety, Efficacy
Interim data from trials of RGX-121 and RGX-111 were presented at WORLDSymposium 2022.
Study to Evaluate Gene-Edited Cell Therapy for MPSII
The LV-HSCGT therapy has demonstrated efficacy in preclinical studies in mouse models.
Orchard Plans OTL-200 European Launch, FDA BLA Strategy
Orchard Therapeutics released its plans for 2021, which included the launch of OTL-200 (Libmeldy) in Europe and development of an FDA submission strategy in the United States.
AT-GTX-502 Could Provide JNCL Patient Stabilization
Descriptive findings from the phase 1/2 study allude to promise for the gene therapy in the rare pediatric disorder.
LYS-SAF302 Linked to MPS-IIIA Biologic Response in AAVance Trial
New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.
Transpher B Findings: ABO-101 Benefits CNS Biomarkers in Patients with MPS-IIB
The AAV-based gene therapy was well-tolerated and showed enough promising effect to warrant further investigation, researchers reported.
Transpher A Findings: ABO-102 Provides Continuous MPS-IIIA Neurocognition Gain
Investigators observed sustained, dose-dependent benefits for young patients at 24 months.
FLT201 Shows Promise as a Gene Therapy for Gaucher Disease
FLT201 is a combination of AAVS3 capsid and a liver-specific promotor to drive the expression of GCasevar85.
Fabry Disease Gene Therapy AVR-RD-01 Completely Reduces Toxic Kidney Substrate
New WORLDSymposium findings show a treated patient had 100% reduction in the accumulating fatty substrate associated with the burdensome rare disease after 1 year.
