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- | LYS-SAF302 / Lysogene, Sarepta Therap
Journal, Gene therapy: Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA. (Pubmed Central) - Jun 19, 2023
This alters extracellular matrix composition, depletes heparan sulfate, impairs astrocyte and oligodendrocyte function, and causes cystic white matter degeneration at the site of highest gene expression. The AAVance trial results will reveal the potential benefit-risk ratio of this therapy.
- LYS-SAF302 / Lysogene, Sarepta Therap
Update on AAVance, a Phase 2/3 Clinical Trial of LYS-SAF302 Gene Therapy in Children with MPS IIIA () - Sep 25, 2022 - Abstract #ESGCT2022ESGCT_441;
- LYS-SAF302 / Lysogene, Sarepta Therap
An Update on LYS-SAF302 Gene Therapy Study (AAVance) in Children with Mucopolysaccharidosis Type IIIA (Poster Board Number: Tu-312; Hall D) - Apr 20, 2022 - Abstract #ASGCT2022ASGCT_873;
P2/3
Safety, tolerability, biomarkers, effect on behavior, sleep and quality of life are secondary endpoints. An update on the status of the study will be presented.
- |||| LYS-SAF302 / Lysogene, Sarepta Therap
Biomarker: Lysogene Reports Positive Biomarker Data With LYS-SAF302 https://t.co/6LNxZ7uF8Y (Twitter) - Dec 27, 2020
- || LYS-SAF302 / Lysogene, Sarepta Therap
Enrollment closed, Gene therapy: AAVance: Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (clinicaltrials.gov) - Jun 23, 2020
P2/3, N=20, Active, not recruiting, Sponsor: LYSOGENE
An update on the status of the study will be presented. Recruiting --> Active, not recruiting
- | SAF-301 / Lysogene, Alcyone, LYS-SAF302 / Lysogene, Sarepta Therap
Preclinical, Journal: An improved AAV vector for neurological correction of the mouse model of Mucopolysaccharidosis IIIA. (Pubmed Central) - Mar 14, 2020
Biodistribution of SAF302 was further assessed using GFP (SAF302GFP), indicating that vector spread was limited to the area around the injection tract. Further modification of the injection strategy to a single depth with higher injection volume increased vector distribution leading to more widespread GFP distribution and sustained expression, suggesting this approach should be adopted in future trials.
- |||| LYS-SAF302 / Lysogene, Sarepta Therap
Fast track designation: Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA https://t.co/BsrsZKF1wp (Twitter) - Feb 24, 2020
- | LYS-SAF302 / Lysogene, Sarepta Therap
Preclinical, Journal: AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains. (Pubmed Central) - Jan 10, 2020
The ability of the vector adeno-associated virus (AAV)rh.10-CAG-SGSH (LYS-SAF302) to correct disease pathology was evaluated in a mouse model for MPS IIIA...Increases of SGSH enzyme activity of at least 20% above endogenous levels were detected in 78% (dogs 4 weeks after injection) and 97% (monkeys 6 weeks after injection) of the total brain volume. Taken together, these data validate intraparenchymal AAV administration as a promising method to achieve widespread enzyme distribution and correction of disease pathology in MPS IIIA.
- || LYS-SAF302 / Lysogene, Sarepta Therap
Enrollment open, Gene therapy: AAVance: Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (clinicaltrials.gov) - Dec 21, 2018
P2/3, N=20, Recruiting, Sponsor: LYSOGENE
Taken together, these data validate intraparenchymal AAV administration as a promising method to achieve widespread enzyme distribution and correction of disease pathology in MPS IIIA. Not yet recruiting --> Recruiting
- | LYS-SAF302 / Lysogene, Sarepta Therap
New P2/3 trial, Gene therapy: AAVance: Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (clinicaltrials.gov) - Aug 1, 2018
P2/3, N=20, Not yet recruiting, Sponsor: LYSOGENE