Lysogene Selects Brammer Bio to produce GM1 Gangliosidosis Gene Therapy Product

Lysogene Selects Brammer Bio to produce GM1 Gangliosidosis Gene Therapy Product 150 150 Lysogene

Strategic manufacturing agreement covers Lysogene’s AAVrh10-based gene therapy treatment

PARIS, France, and CAMBRIDGE, MA, US – May 24th 2017 – Lysogene (FR0013233475 – LYS), a leading, biopharmaceutical company pioneering gene therapy technologies to treat central nervous system diseases, today 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.

We are pleased to have secured Brammer Bio, a leading manufacturer with proven expertise in the development of robust industrial-scale manufacturing of AAV-based products, to provide the highest quality LYS-GM101 product for clinical testing,” said Mark Plavsic, Chief Technical Officer at Lysogene.

This agreement ensures Lysogene has established commercial-ready gene therapy manufacturing in line with our need for the product”.

Pre-clinical data in animal models of GM1 show that LYS-GM101 treatment delivers a functional gene encoding the βgal 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.

We are delighted to partner with Lysogene to manufacture this commercial-ready AAVrh10 gene therapy product,” stated, Mark Bamforth, President and CEO of Brammer Bio. “We embrace this opportunity to help patients in need and re-affirm Brammer Bio’s leadership position as the manufacturer of choice in the gene therapy space.

About GM1

GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-balactosidase (ßgal) enzyme. The resulting enzymatic 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. It is a devastating disease for patients and families. There is currently no disease modifying treatment available.

About Brammer Bio

Brammer Bio provides clinical and commercial supply of vectors for in vivo gene therapy and ex vivo modified-cell based therapy, along with process and analytical development, and regulatory support, enabling large pharma and biotech clients to accelerate the delivery of novel medicines to improve patients’ health. Brammer is owned by Ampersand Capital Partners, the only institutional investor in the company, and its founders. For more information, please visit

About Lysogene

Lysogene is a clinical stage biotechnology company pioneering the basic research and clinical development of AAV gene therapy for CNS disorders with a high unmet medical need. Since 2009, Lysogene has established a solid platform and network, with lead products in Mucopolysaccharidosis type IIIA and GM1 Gangliosidosis, to become a global leader in orphan CNS diseases. Lysogene is listed on the Euronext regulated market in Paris (ISIN code: FR0013233475). For more information visit

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