Two New AAV Gene Therapy Clinical Trials for Infantile and Late Infantile GM1 to Begin in Q1/Q2 2021
Cure GM1 is pleased to share the tremendous news that both Passage Bio and Lysogene have received regulatory approvals…
A Newborn Screening Assay for GM1 Gangliosidosis, Gelb Laboratory, University of Washington
Tandem Mass Spectrometry Assay of Beta-Galactosidase for Multiplex Newborn Screening of GM1 Gangliosidosis Michael H….
First Gene Therapy Clinical Trial Initiated May 2019!
The first gene therapy clinical trial for GM1 Gangliosidosis is a beacon of hope for…
Passage Bio and UPenn Orphan Disease Center AAV Gene Therapy and Natural History Study
Passage Bio was recently launched with the vision to be a first in class fully-integrated…
2019 The Jackson Laboratory – Natural History Studies of Two GM1 Mouse Models
Scientific researchers Cat Lutz and Aamir Zuberi at the Rare Disease Center of the Jackson…
Axovant Intravenous AAV9 Gene Therapy 2019 Clinical Trial Announced!
GM1 Intravenous AAV9 Gene Therapy Clinical Trial to Begin in the First Half of 2019…
2018-2020 UCSF Preclinical development of a gene therapy for GM1 Gangliosidosis
Widespread vector distribution in the brain and vector serotype selection have presented significant challenges to…
2018 Jackson Laboratory – Publicly Available Mouse Models
Dr. Cat Lutz and Dr. Aamir Zuberi are scientists at The Jackson Laboratory. Dr. Lutz…
2017 Dorphan Small Molecule Research and Mouse Study
Cure GM1 granted funds for a drug repurposing project and mouse study to study a…
2017 UC Davis Lentiviral Gene Therapy Research
Cure GM1 recently granted funds towards the development of a lentiviral gene therapy approach using…