changing the future of
We are mission-driven to overcome the challenges in CNS therapy development through applying precision CNS delivery & dosing technologies to novel gene therapy technologies with a team of gene therapy and industry leaders.
Alcyone’s pipeline currently includes 12 AAV (adeno-associated virus) gene therapy programs and four gene therapy platform technologies targeting severe CNS disorders. Lead programs include ACTX-101, a gene therapy for Rett syndrome using X-reactivation technology and currently in pre-IND enabling studies, and ACTX-401, a gene therapy for spinal muscular atrophy with respiratory distress type 1 (SMARD1) currently in IND stage.
Alcyone’s four gene therapy platform technologies include X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation. The X-reactivation platform, which is used in our lead candidate, ACTX-101 for Rett syndrome, has the potential to become the gene therapy industry’s first “pipeline within a product” gene therapy, where the same gene product can be applied to potentially treating other X-related syndromes.
precision CNS delivery platform
Precise and optimal delivery has a profound impact on both efficacy and safety of gene therapy. Alcyone has developed a transformational CNS delivery technology platform that provides control of biodistribution to the targets of interest. This has the potential to move the frontier of the field by impacting safety, efficacy and potentially the amount of vectors needed for an effective therapy.
NATIONWIDE CHILDREN’S HOSPITAL
In August 2020, Alcyone Lifesciences and the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) entered into a first-of-its-kind collaboration. The collaboration unites Alcyone’s proprietary, next-generation CNS delivery platform and product development, manufacturing and commercialization capabilities, with four gene therapy technologies and discovery, research and early development capabilities developed at AWRI. Alcyone Lifesciences, which was founded in 2010, pioneered the development if a next-generation precision CNS delivery platform.
In December 2018, Alcyone and Roche entered into a broad strategic collaboration to optimize intrathecal therapies and develop novel antisense oligonucleotide delivery treatment options for patients with neurological disorders.
Our precision delivery and genetic therapy platforms combine to form a range of therapeutics programs. We are continuously exploring strategic relationships that extend the reach and capabilities of our technology platform. Contact us below to connect.
leadership and advisors
Our team holds deep scientific expertise and a passion to meaningfully change the lives of patients. Our number one priority is improving patient outcomes, and we are steadfast in our collaboration, innovation and ambition to deliver.
Kathrin Meyer, Ph.D.
Chief Scientific Advisor, Chair of the Scientific Advisor Board, Principal Investigator, Abigail Wexner Research Institute, Nationwide Children’s Hospital
Sanchita Bhatnagar, Ph.D.
Assistant Professor, Biochemistry & Molecular Genetics, University of Virginia
Dave Arnold, M.D.Associate Professor, Division of Neuromuscular Disease, Department of Neurology, Department of PM&R, Department of Neuroscience, The Ohio State Wexner Medical Center
Roxana O Carare, M.D., Ph.D.
Professor of Neuroanatomy, University of Southampton
Maurizio Pellechia, Ph.D.
Director, Center for Molecular and Translational Medicine, University of California, Riverside
Nicolas Wein, Ph.D.
Principal Investigator, Abigail Wexner Research Institute, Nationwide
Timothy Benke, M.D., Ph.D.
Professor, Pediatrics-Neurology, School of Medicine, University of Colorado Anschutz Medical Campus
Isabelle Aubert, Ph.D.
Senior Scientist, Sunnybrook Research Institute, University of Toronto
Laura Ferraiuolo, Ph.D.
Lecturer in Translational Neurobiology, Department of Neuroscience, University of Sheffield
Jeffrey L. Neul, M.D., Ph.D.
Professor of Pediatrics, Division of Neurology, Vanderbilt University Medical Center
Founder, Chief Executive Officer, Alcyone Therapeutics, Inc.
Managing Director, RTW Investments, LP
Edgar D. Jannotta, Jr.
Former Managing Principle and Co-Manager of Healthcare Group. GTCR GOLDER RAUNER, LLC.
Partner, Harbor Light Capital Partners
Stephen Oesterle, MD
Health Care Industry Consultant, former Senior VP, Medicine & Technology Medtronic
David Donabedian, PhD, MBA
Co-founder, CEO and Director, Axial Biotherapeutics & Venture Partner, Longwood Fund
Cameron Durrant, MD, DRCOG, DIPCH, MRCGP, MBA
Chairman & CEO, Humanigen
Kathrin Meyer, Ph.D. (Observer)
Board Observer, Chief Scientific Advisor,
Chair of the Scientific Advisory Board PI, Abigail Wexner Research Institute, Nationwide Children’s Hospital
Dennis Durbin, M.D., M.S.C.E (Observer)
Board Observer, CSO, Nationwide Children’s Hospital & Professor of Pediatrics at Ohio State University
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Lowell, MA, 01852