LOWELL, Mass., June 29, 2017 /PRNewswire/ — Alcyone Lifesciences, Inc., a leader in precision advanced central nervous system (CNS) delivery systems announced that it has entered into a feasibility study agreement with Pfizer Inc. (NYSE: PFE) in the area of genetic therapy targeting rare and orphan neurological disorders. The feasibility study combines Alcyone’s proprietary PulsarTM Advanced Intrathecal Precision Delivery Platform Technology and Pfizer’s Recombinant Adeno-associated Viral Vector (rAAV) gene therapy technology in an effort to develop a method of administering genetic therapies that may address neurological disorders more effectively.
Gene therapy is a potentially transformational therapeutic approach for patients suffering from certain rare and orphan neurological disorders, which focuses on the use of one-time treatments that may address the fundamental cause of the disease caused by genetic mutations. The very potential of this therapy is dependent on the utilization of highly effective and reliable delivery technology, and biodistribution of the viral vectors, such as rAAV, that carry the corrective genetic material to the targets of interest in the CNS. This is particularly critical in CNS gene therapy, because there is one opportunity to get the appropriate concentration of active molecule to the right targets associated with the disease in order to achieve a successful procedure.
Alcyone’s proprietary PulsarTM advanced intrathecal precision delivery platform is an auto-intrathecal injector that utilizes cerebrospinal fluid dynamics along with convection-dispersion enhancements to deliver and biodistribute the viral vectors to targets of interest in the CNS. This platform has been demonstrated to increase the concentration of the vector to the targets of interest consistently and reliably. The platform is being designed to enable it to be easily deployed in the clinical setting and to allow for ease of use with custom infusion algorithms for treating CNS diseases.
“We are excited about this collaboration with the talented scientists and gene therapy experts in Pfizer’s Rare Disease Research Unit,” said PJ Anand, Alcyone’s chief executive officer and president of Alcyone Lifesciences. “Highly targeted and effective delivery to the CNS is going to be critical in any disease modifying therapies. Alcyone will be at the forefront of this effort working closely with our biopharma partners with the goal of ultimately making a positive impact in the lives of many patients worldwide.”
“We look forward to leveraging our collective expertise and technologies to develop therapies with the potential to truly make a difference for patients with neurodegenerative diseases,” said Gregory LaRosa, senior vice president and chief scientific officer, Rare Disease Research Unit, Pfizer. “Pfizer continues to actively build capabilities in vector design, delivery and manufacturing so that we can enable comprehensive development and commercialization of gene therapies for patients who have few or no options for treatment. Working with Alcyone, we hope to develop therapies that can potentially advance treatments for CNS disorders.”
About Alcyone Lifesciences, Inc.
Alcyone Lifesciences, based in Lowell, Massachusetts, is a privately-held therapeutic device company focused on development of novel treatment modalities for chronic neurological conditions including advanced precision delivery technologies for the central nervous system. The Company’s patented technology platform is based on a uniquely engineered amalgamation of microfabrication technologies, along with advanced biomedical engineering, with a biologically inspired core product focus on targeted drug therapy and hydrocephalus. Alcyone’s team of scientists, physicians and advisers includes recognized leaders in the field of neurology and neurosurgery. For more information, please visit www.alcyonels.com.