Date: 2017-05-24
Type of information: Production agreement
Compound:
- LYS-GM101 - adeno-associated viral vector serotype rh.10 expressing beta-galactosidase
Company: Lysogene (France) Brammer Bio (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Technology - Services
Type agreement: manufacturing - coproduction
Action mechanism:
- gene therapy. GM1 gangliosidosis is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-balactosidase (?gal) enzyme. The resulting deficiency leads to accumulation of GM1-ganglioside in cells. Clinical presentation is mainly neurological with rapidly progressive impairment (motor, cognitive and behavioral) leading to premature death, mostly in early childhood.
- LYS-GM101 is made of an adeno-associated virus that contains the gene for beta-galactosidase, the enzyme that is missing in patients with GM1 gangliosidosis. It is designed to replace this defective gene in the cells of GM1 patients, in order to allow for production of a functional lysosomal acid beta-balactosidase and to prevent the progressive nature of the neurological damage caused by GM1 in humans.
Disease: GM1 gangliosidosis
Details:
- • On May 24, 2017, Lysogene announced that it has entered into a strategic manufacturing agreement with Brammer
Bio, a best-in-class viral gene and cell therapy manufacturer.
Brammer Bio will produce LYS-GM101, an AAVrh10-based gene therapy, for clinical testing of the therapeutic candidate in patients with GM1 Gangliosidosis, a rare neuronopathic lysosomal storage disorder. Pre-clinical data in animal models of GM1 show that LYS-GM101 treatment delivers a functional gene encoding the ?gal enzyme resulting in a reduction of GM1 gangliosides and transforms the animal phenotype. These studies will support an Investigational New Drug application and the launch of the Phase I clinical trial, expected to launch in 2019.
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