French contract services firm Novasep has signed an agreement with compatriot biopharma Lysogene to develop and manufacture LYS-GM101, the latter’s Adeno-Associated Virus (AAV)rh10-based gene therapy drug candidate to treat GM1 gangliosidosis, a rare and inherited neuronopathic lysosomal storage disorder that progressively destroys nerve cells in the brain and spinal cord.

The agreement consolidates an existing and long-lasting partnership between the two companies that started four years ago with the development and manufacture of Lysogene’s lead gene therapy product LYS-SAF203, which is currently in clinical phase 2/3.

“By extending our collaboration, we secure the clinical production of our experimental treatment for GM1 gangliosidosis and take an option for a smooth and effective technical transfer to a future commercial process,” said Lysogene’s chief technical officer Mark Plavsic.

Lysogene is focused on treating orphan diseases of the central nervous system (CNS), including lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial for LYS-SAF203 to treat mucopolysaccharidosis (MPS) type IIIA is currently ongoing with US-based partner Sarepta Therapeutics.

The companies previously signed an exclusive license agreement in October 2018 for LYS-SAF203, under which Sarepta will hold commercial rights to the therapy in the US and in markets outside Europe, while Lysogene will maintain commercial exclusivity in Europe.

The Paris-based biopharma is also collaborating with an unnamed academic partner to define the strategy of development for treating Fragile X syndrome, a genetic disease related to autism.