Alcyone Lifesciences and Lysogene Partner to Develop Treatment for Childhood Neurological Disease

Lowell, Mass.-based Alcyone Lifesciences Inc., which makes neural intervention systems for neurological conditions and targeted drug delivery, and Parisan company Lysogene, a privately held biotechnology company focused on recombinant adeno-associated virus (rAAV) based gene therapy for the central nervous system (CNS), have begun a collaboration.  The companies plan to join forces to evaluate the intraparenchymal delivery of Lysogene’s proprietary rAAV to treat patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo A, using the Alcyone MEMS cannula (AMC) targeted delivery platform. MEMS stands for microelectromechanical systems.

MPS IIIA presents in early childhood, causing progressive neurodegeneration associated with intractable behavioral problems and developmental regression. Life span is shortened, with death usually occurring in the mid teen years. There is currently no treatment. Lysogene’s gene therapy is delivered directly to the CNS in one neurosurgical procedure. It is hoped that the delivery of the missing hSGSH (human N-sulfoglucosamine sulfohydrolase) gene will provide a permanent source of functional enzyme in the brain that reverses phenotypic abnormalities of CNS neural cells.

“Alcyone and Lysogene represent synergy.  This is a great opportunity for both companies to forward research in delivering gene therapy to the CNS” said Karen Aiach, founder and CEO of Lysogene.

“We are excited about this collaboration.  Lysogene is a unique company with incredible science and a strong culture,” said P.J. Anand, founder and CEO of Alcyone Lifesciences. “Karen Aiach and her team are truly impressive.  We hope to be part of their success in treating patients with these debilitating conditions.”

“Effective therapeutic and optimized delivery is an absolute requirement for successful therapy,” said Olivier Danos, co-founder of Lysogene and scientific advisor to Alcyone Lifesciences.  This is an excellent match and I look forward to the outcome of the collaboration.”

Lysogene’s most advanced product candidate is a, rAAV vector that carries the hSGSH for the treatment of MPS IIIA. According to the company, the recently completed Phase I/II study in four MPS IIIA children demonstrated that the gene therapy and neurosurgical procedure is safe, well tolerated and exploratory efficacy profiles are encouraging.

The Alcyone MEMS cannula targeted delivery system was developed using the company’s proprietary MEMS technology platform.  Without burdening the neurosurgery community with unnecessary additional capital equipment, the AMC can be utilized with any existing commercial imaging and stereotactic system in conjunction with the work-flow friendly clinical use guideline designed by the company’s scientist and neurosurgery advisors.  Neurosurgeons can select a target, navigate the AMC precisely to the target, and observe in real-time the precision delivery of the therapeutic agent, all under intra-procedural MRI (magnetic resonance imaging) guidance.  In addition to the MEMS tip that has dual micro-channels, the AMC features a unique patented distal end design that helps prevent reflux or back flow along the cannula shaft, which can be a significant drawback with current devices. The AMC platform device is designed for optimal targeted bio-distribution and neurosurgeon’s ease of use.