April 20th, 2017
Preclinical Brain Distribution Data Drives Selection
(FR0013233475 – LYS), a
PARIS, France, and CAMBRIDGE, MA, US – April 20th, 2017 – Lysogene
leading biopharmaceutical company, pioneering in gene therapy technology focused on central nervous system (CNS) diseases, today announced that it has selected the MRI Interventions’ SmartFlow® cannula to be used in the planned Phase II/III clinical trial in Mucopolysaccharidosis Type IIIA (also called MPS IIIA or Sanfilippo A). The clinical study is scheduled to begin in early 2018.
Selection of the cannula was based on results of a preclinical research study that evaluated the performance characteristics of the cannula, including ease of use, while testing various injection parameters for optimal brain distribution of Lysogene’s clinical candidate, LYS-SAF302.
“Several commercially-available cannulas were considered for use in our upcoming Phase II/III trial,” said Kimberley S. Gannon, Chief Scientific Officer of Lysogene. “The SmartFlow cannula performed extremely well in our preclinical evaluation study. We found no indication of reflux up the injection track and the study resulted in widespread distribution of LYS-SAF302 in the brain.” Lysogene plans to present the results of the preclinical study at a scientific conference later this year.
“The SmartFlow cannula has been selected by several drug delivery partners, and we are particularly pleased to see the selection of the SmartFlow cannula for this important clinical trial,” said Frank Grillo, CEO of MRI Interventions, Inc. “The extensive pre-clinical testing completed by Lysogene has been very encouraging, and we believe this cannula is ideally suited to provide widespread brain distribution of LYS- SAF302. We look forward to supporting Lysogene in their clinical trial next year.”
Lysogene is Targeting Treatment for the Neurological Symptoms of MPS IIIA:
Lysogene’s gene therapy candidate for MPS IIIA is a rAAV vector serotype rh.10 carrying the gene coding for SGSH. This in vivo gene therapy offers the possibility of a one-time treatment by inserting a healthy copy of the SGSH gene and allowing the body to start making the missing enzyme, therefore slowing or halting disease progression. Lysogene’s gene therapy is delivered directly to the CNS during a neurosurgical procedure. By delivering the missing SGSH gene, Lysogene believes MPS IIIA patients will be
provided a permanent source of functional enzyme in the brain that reverses phenotypic abnormalities of CNS cells.
About the MRI Interventions SmartFlow Cannula
The SmartFlow cannula is an MRI-compatible injection and aspiration cannula for delivery of therapeutic agents into the brain. For more information, refer to http://www.mriinterventions.com.
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 gene therapy platform and network, with lead products in MPS IIIA and GM1 Gangliosidosis, to become a global leader in orphan CNS diseases. Lysogene has obtained orphan drug designation from the EMA and FDA, and rare pediatric designation from the FDA for both programs.
For more information, visit www.lysogene.com.
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