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Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis


Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
Ongoing enrollment of a total of 16 patients in the UK, US and France
Regulatory News:
Lysogene (Paris:LYS) (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient, at Royal Manchester Children s Hospital, part of Manchester University NHS Foundation Trust, in the global adaptative-design clinical trial with LYS-GM101 (NCT04273269), a gene therapy for the treatment of GM1 gangliosidosis.
This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human ß-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients wi ....

United Kingdom , United States , Simon Jones , Karen Aiach , Manchester University , European Union , University Of Manchester , University Dental Hospital Of Manchester , Sarepta Therapeutics Inc , Foundation Trust , Second Lysogene , Royal Manchester Children , Founder Chairman , Chief Executive Officer , Chief Investigator , Paediatric Inherited Metabolic Disease , Senior Lecturer , Rare Pediatric Disease , Casimir Trials , Sarepta Therapeutics , Manchester Royal Infirmary , Saint Mary , Manchester Royal Eye Hospital , University Dental Hospital , Trafford General , Altrincham Hospital ,

Lysogene Reports its Cash Position as of 31 March 2021


Lysogene Reports its Cash Position as of 31 March 2021
1
Regulatory News:
Lysogene (Paris:LYS) (FR0013233475 LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, today reports its cash position as of 31 March 2021.
As of 31 March 2021, cash and cash equivalents amounted to €20.0 million
1 (compared to €18.8 million as of 30 December 2020) notably reflecting the state-guaranteed loan ( PGE ) received in February 2021.
About Lysogene
Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable a delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing. An adaptive clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed ....

United States , Sarepta Therapeutics Inc , Sarepta Therapeutics , French Autorit , Des March , French Markets Authorities , Durant Des Aulnois , Financial Officer , ஒன்றுபட்டது மாநிலங்களில் , சாறெப்டா சிகிச்சை இன்க் , சாறெப்டா சிகிச்சை , தேஸ் அணிவகுப்பு , பிரஞ்சு சந்தைகள் அதிகாரிகள் , நிதி அதிகாரி ,