dm+d

Unassigned

New Medicines

GM1 gangliosidoses

Information

New molecular entity
Lysogene
Lysogene

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes
Dec 21MHRA awards LYS-GM101 an Innovation Passport for the treatment of GM1 gangliosidosis under the UK Innovative Licensing and Access Pathway (ILAP). The Innovation Passport is the entry point to ILAP [5].
Jul 21Granted fast track status in US [3].
Feb 17Has orphan drug status in EU and US [3].
Jan 17FDA grants Rare Paediatric Disease status to LYS GM101 for the treatment of GM1 gangliosidoses [3].

Category

An AAVrh10 gene therapy product, utilising an adeno-associated viral vector (AAV), that replaces the defective gene in the cells of GM1 patients, which will allow production of functional beta-galactosidase-1 enzyme. Given by intracisternal injection.
GM1 gangliosidosis is an inherited lysosomal storage disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. The condition may be classified into three major types based on the general age that signs and symptoms first appear: classic infantile (type 1); juvenile (type 2); and adult onset or chronic (type 3). GM1 gangliosidosis is caused by mutations in the GLB1 gene [1]. GM1 gangliosidosis is estimated to occur in 1 in 100,000 to 200,000 newborns [2].
GM1 gangliosidoses
Intracerebral

Trial or other data

Feb 22Lysogene doses the first patient in France with LYS-GM101 gene therapy at the Armand Trousseau Hospital (AP-HP). This is the third patient treated with LYS-GM101 in the global adaptative-design clinical trial (NCT04273269). Enrolment of the 4th patient of the safety cohort is ongoing, following which Lysogene will initiate the treatment of the 12 patients of the efficacy confirmatory cohort [6].
May 21PI/II trial to investigate the efficacy and safety of intracisternal injections of LYS GM101, for the treatment of infantile GM1 gangliosidosis starts (P1-GM-101; NCT04273269). The trial includes patients population of early infantile, late infantile and juvenile disease (age up to 3 years). The single-arm, open-label trial is intended to enrol 16 patients. and has a 2-stage adaptive design. In the first safety and proof-of-concept stage of the study, 4 subjects with infantile GM1 gangliosidosis will receive a single dose of LYS-GM101 by intracisternal injection. The second stage of the study is confirmatory and will include additional patients with GM1 gangliosidosis. Enrolment is underway in the US, in the UK (at Manchester University NHS Foundation Trust) and in France. Primary outcomes are numerous; collection of these data is due to complete Jun 25 [4].