Pioneering
Next-Generation
Precision CNS
Genetic Therapies

Unlocking New Possibilities for CNS Therapeutics​

changing the future of
CNS therapeutics

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.

pipeline

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. 

cpu neuroscience

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.

strategic collaborations

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.

ROCHE

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. 

partners

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.

PJ Anand
PJ Anand
Founder, President &
Chief Executive Officer
Bio
Ravi Mehrotra
Ravi Mehrotra, Ph.D.
Acting CFO &
Head of Strategy
Bio
Ottavio Vitolo
Ottavio Vitolo, M.D., Ph.D.
CMO & Global Head of R&D
Bio
Susan D'Costa
Susan D'Costa, Ph.D.
EVP Technology Development
Bio
Christopher Coletta
Chris Coletta, Ph.D.
Head of Development, Precision Delivery
Bio
Bryn Martin
Bryn Martin, Ph.D.
VP Research, Drug Delivery,
& CSF Sciences
Bio
deep pic 2 circle
Deep Singh
Head of R&D, Precision Delivery
Bio
Andrew East
Andrew East
VP Product Development
Bio
Rachel
Rachel Salzman, D.V.M
EVP, Portfolio, External Affairs & Development
Bio

Chief Advisors

katherin
Kathrin Meyer, Ph.D.
Chief Scientific Advisor,
Principal Investigator,
Nationwide Children's Hospital
Bio
nicolas
Nicolas Wein, Ph.D.
Core Scientific Advisor,
Principal Investigator,
Nationwide Children's Hospital
Bio
Scientific Advisors

Kathrin Meyer, Ph.D.

Chief Scientific Advisor, Chair of the Scientific Advisor Board, Principal Investigator, Abigail Wexner Research Institute, Nationwide Children’s Hospital

Maurizio Pellechia, Ph.D. 

Director, Center for Molecular and Translational Medicine, University of California, Riverside

Isabelle Aubert, Ph.D.

Senior Scientist, Sunnybrook Research Institute, University of Toronto

Sanchita Bhatnagar, Ph.D. 

Assistant Professor, Biochemistry & Molecular Genetics, University of Virginia

Nicolas Wein, Ph.D.  

Principal Investigator, Abigail Wexner Research Institute, Nationwide
Children’s Hospital

Laura Ferraiuolo, Ph.D. 

Lecturer in Translational Neurobiology, Department of Neuroscience, University of Sheffield

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

Timothy Benke, M.D., Ph.D.  

Professor, Pediatrics-Neurology, School of Medicine, University of Colorado Anschutz Medical Campus

Jeffrey L. Neul, M.D., Ph.D. 

Professor of Pediatrics, Division of Neurology, Vanderbilt University Medical Center

Roxana O Carare, M.D., Ph.D. 

Professor of Neuroanatomy, University of Southampton  

Mimoun Azzouz., Ph.D

Chair of Translational Neuroscience, Director of Gene Therapy Innovation & Manufacturing Centre (GTIMC), IMI ARDAT Coordinator, ERC Advanced Investigator, Director of MSc Course in Cell & Gene Therapies

Board of Directors

PJ Anand

Founder, Chief Executive Officer, Alcyone Therapeutics, Inc.

Richard Upton 

Partner, Harbor Light Capital Partners

Cameron Durrant, MD, DRCOG, DIPCH, MRCGP, MBA

Chairman & CEO, Humanigen

Piratip Pratumsuwan

Managing Director, RTW Investments, LP

Stephen Oesterle, MD

Health Care Industry Consultant, former Senior VP, Medicine & Technology Medtronic

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

Edgar D. Jannotta, Jr.

Former Managing Principle and Co-Manager of Healthcare Group. GTCR GOLDER RAUNER, LLC.

David Donabedian, PhD, MBA 

Co-founder, CEO and Director, Axial Biotherapeutics & Venture Partner, Longwood Fund

Dennis Durbin, M.D., M.S.C.E (Observer)

Board Observer, CSO, Nationwide Children’s Hospital & Professor of Pediatrics at Ohio State University

news and events

get in touch

Info@alcyonetx.com

Alcyone Therapeutics, Inc.
116 John St.
Suite 300,
Lowell, MA, 01852
(978) 709-1946

careers

Coming soon

Linkedin-in Twitter
Copyright © 2021 Alcyone Therapeutics, Inc
PJ Anand
PJ Anand
Founder, President, & CEO

PJ is the founder of Alcyone Therapeutics and its predecessor Alcyone Lifesciences, and serves as the CEO. PJ sets the vision for the company including corporate strategy, research & development, business devlopment and commercialization.

 PJ has 25+ years of experience in the life science industry with a lab to clinic translational track-record of next-generation technologies for use in treatment of end-stage kidney disease, peripheral vascular disease, orthopedic and CNS disorders including brain cancer and diagnostics. He is a recognized expert in translating academic research into clinical value proposition, and in the area of convergence of drugs and devices. He also has over 100 families of patents and patent applications and several scientific publications to his credit. PJ is also the founder of Anuncia, Inc., a company focused on treatment and management of cerebrospinal fluid dysfunction including hydrocephalus and the co-founder and Chairman of Arthromeda, Inc., a company focused on developing an A.I. based patient-specific navigation platform for orthopedic applications. Before founding Alcyone Lifesciences and Arthromeda, PJ Anand was a founding executive member of WMR Biomedical (now Arsenal Medical & Lyra Therapeutics) where he was responsible for key strategic funding transactions.

Early in his career, PJ served as Vice President & General Manager of Spire Biomedical, Inc., a division of Spire Corporation, where he was responsible for developing a convergence product for the treatment of ESRD that resulted M&A transaction.

PJ Anand
PJ Anand
Founder, President, & CEO

PJ is the founder of Alcyone Therapeutics and its predecessor Alcyone Lifesciences, and serves as the CEO. PJ sets the vision for the company including corporate strategy, research & development, business devlopment and commercialization.

PJ has 25+ years of experience in the life science industry with a lab to clinic translational track-record of next-generation technologies for use in treatment of end-stage kidney disease, peripheral vascular disease, orthopedic and CNS disorders including brain cancer and diagnostics. He is a recognized expert in translating academic research into clinical value proposition, and in the area of convergence of drugs and devices. He also has over 100 families of patents and patent applications and several scientific publications to his credit. PJ is also the founder of Anuncia, Inc., a company focused on treatment and management of cerebrospinal fluid dysfunction including hydrocephalus and the co-founder and Chairman of Arthromeda, Inc., a company focused on developing an A.I. based patient-specific navigation platform for orthopedic applications. Before founding Alcyone Lifesciences and Arthromeda, PJ Anand was a founding executive member of WMR Biomedical (now Arsenal Medical & Lyra Therapeutics) where he was responsible for key strategic funding transactions.

Early in his career, PJ served as Vice President & General Manager of Spire Biomedical, Inc., a division of Spire Corporation, where he was responsible for developing a convergence product for the treatment of ESRD that resulted M&A transaction.

Ravi Mehrotra
Ravi Mehrotra, Ph.D.
Acting CFO & Head of Strategy

Dr. Mehrotra has over 25 years of biotechnology equity research, investment banking, strategy and executive team operational experience. He previously held leadership roles at Evercore ISI, MTS Health Partners, Ovascience, Credit Suisse and Cowen. Dr. Mehrotra holds a B.S. and a Ph.D. in biochemistry and molecular biology from The Wellcome Trust Center.

Ottavio Vitolo
Ottavio Vitolo, M.D., Ph.D.
CMO & Global Head of R&D

Dr. Vitolo is a neuropsychiatrist at Massachusetts General Hospital and an experienced industry executive in CNS drug development. Dr. Vitolo has held roles at Vitam Therapeutics, Relmada Therapeutics (Nasdaq: RLMD), Homology Medicines, Pfizer and Shire (Takeda). Dr. Vitolo holds an M.S.S.C in translational medicine from Harvard Medical School and an M.D. from University of Rome La Sapienza.

Susan D'Costa
Susan D'Costa, Ph.D.
EVP Technology Development

Dr. D’Costa is a molecular virologist with over 25 years of experience in virology and viral vector analytics, development and manufacturing. She has held various leadership roles at Thermo Fisher Scientific, Viral Vector Services and its predecessor companies – Brammer Bio and Florida Biologix. Dr. D’Costa holds a Ph.D. in Biology from Texas Tech University, a M.S. in Biochemistry from Mumbai University (Grant Medical College), and a B.S. in Microbiology/Biochemistry also from Mumbai University (St. Xavier’s College).

Christopher Coletta
Christopher Coletta, Ph.D.
Head of Development, Precision Delivery
Dr. Coletta leads business operations and strategic planning for Alcyone Therapeutics. Previously, Dr. Coletta was a member of the executive leadership team at Coactuate, a strategic design consultancy, and the Drug Delivery Innovation and Strategy Lead at Amgen. Before joining Amgen, Dr. Coletta was an Engagement Manager with McKinsey & Company, advising pharmaceutical and medical device clients on strategy development, organizational structure redesign, efficiency program design and implementation, and due diligence of acquisition targets. He started his career as a Drug Discovery Chemist at Pfizer’s Groton site. Dr. Coletta received his Ph.D. in organic chemistry from Harvard University and his B.S. in chemistry from the University of Illinois.
Bryn Martin
Bryn Martin, Ph.D.
VP Research, Drug Delivery,
& CSF Sciences

Dr. Martin serves as Vice President of Research, Precision Delivery and Cerebrospinal Fluid Sciences at Alcyone Therapeutics. In this role, he is responsible for overseeing a multidisciplinary research team focused on the advancement of the Alcyone Precision Delivery portfolio. This team specializes in the application of engineering principles to transform targeted CNS drug delivery and interacts across Alcyone Therapeutics’ early pre-clinical to clinical programs.

Dr. Martin has 20 years of experience in cerebrospinal fluid mechanics and physiology, has co-authored/authored over 60 peer-reviewed journal publications and book chapters in the field, and currently serves as Associate Editor of Fluids and Barriers of the CNS (Springer/Nature). Prior to joining Alcyone Therapeutics, Dr. Martin earned the rank of Associate Professor of Chemical and Biological Engineering at University of Idaho and served as Principal Investigator for research funded by National Institute of Health (NIH), National Aeronautics and Space Administration (NASA), National Science Foundation (NSF), industry (such as Genentech, Biogen, Voyager Therapeutics and others) and foundations (such as the American Syringomyelia and Chiari Alliance Project). Dr. Martin has also served as a consultant for 10+ years to the medtech and pharma sectors in the areas of medical devices and CNS drug delivery. He completed his postdoctoral fellowship in bioengineering and biomechanical engineering at the Swiss Federal Institute of Technology under Professor N. Stergiopulos and served as the first Director of the Conquer Chiari Research Center at The University of Akron. Dr. Martin earned a Ph.D. in mechanical engineering from the University of Illinois at Chicago.

deep pic 2 circle
Deep Singh
Head of R&D, Precision Delivery

Deep leads innovation, business and strategy for Alcyone’s Precision Drug Delivery. Deep was part of the founding team at Alcyone, and has a broad leadership experience in precision delivery technology innovation and translation, CSF and CNS transport sciences, R&D and manufacturing, regulatory, clinical deployment, business and corporate development, and alliance management. Deep holds several patents and publications in precision delivery and CSF management. Previously, Deep worked at Accellent (now Integer) in medical technology R&D, manufacturing, and global program management. Deep has also worked at Abbott Bioresearch (now AbbVie) in manufacturing process development, as well as Heamonetics in R&D. Deep has a Bachelor of Science degree in Biomedical Engineering with a minor in Material Sciences from Worcester Polytechnic Institute, and a Certificate in Project Management from Boston University.

Andrew East
Andrew East
VP of Product
Development

Andrew serves as Vice President of Product Development, responsible for overseeing an engineering development team and coordinating cross functionally across the organization, focused towards advancement of the Alcyone Therapeutics Precision Delivery portfolio. Andrew holds a B.S. in mechanical engineering and has over 18 years of experience bringing innovative technologies to the market, from concept, through regulatory clearance and product launch. Andrew also holds certificates in Medical Device Regulatory Affairs from Northeastern University and in Medical Device Management from Worcester Polytechnic Institute. Andrew first joined Alcyone to lead the development of a Hydrocephalus shunt technology, known as ReFlow, which was spun out to form Anuncia Inc. in 2018. Prior to joining Alcyone Therapeutics, Andrew held positions at Accellent Inc., now Integer (which later became Lake Region Medical) and Ranfac Corporation. Andrew holds numerous patents over a wide range of product families spanning over 18 years of cutting-edge technology development.

Rachel Salzman, D.V.M
EVP, Portfolio, External Affairs & Development

Dr. Rachel Salzman serves as the EVP of Portfolio, External Affairs & Development at Alcyone Therapeutics. Prior to joining Alcyone, Rachel co-founded SwanBio Therapeutics in 2017, and served as CEO through 2019, followed by heading up Portfolio Development until the end of 2020. SwanBio is an AAV gene therapy company focused on developing treatments for patients with serious neurological diseases, with a lead program in adrenomyeloneuropathy, the adult manifestation of X-linked adrenoleukodystrophy (ALD). There, she co-led over $75 million in fundraising, established the Company’s initial operational structure and oversaw its R&D footprint. As Chief of Portfolio Development, she grew the pipeline, including through leading program nominations, and introduced external innovation while ensuring a value-driven enterprise.  

Prior, Dr. Salzman was Chief Science Officer of The Stop ALD Foundation (SALD) since 2001. SALD is dedicated to employing entrepreneurial approaches and innovative methodology for advancing effective therapies, cures and prevention of ALD, an often-fatal neurodegenerative disease. As CSO at SALD, she made critical contributions in driving forward the world’s first ex-vivo lentiviral gene therapy clinical trial conducted in non-HIV infected patients. This program enabled the subsequent successful establishment of a public gene therapy company, and an EC (European Commission)-approved genetic medicine for boys diagnosed with early cerebral ALD. This marketing authorization is currently valid in all 27 member states of the EU.

Dr. Salzman has been an active member of the American Society of Gene & Cell Therapy (ASGCT) for 20 years and served in a leadership capacity on multiple committees and task forces designed to grow and strengthen the science and status of gene and cell therapy in both the U.S. and EU. Her work includes early exploration into patient access and sustainable commercialization models, while advancing policy initiatives during her tenure as inaugural Chair of the Government Affairs Committee. She received the Sonia Skarlatos Public Service Award in 2015. She currently represents ASGCT at the National Academies of Science, Engineering and Medicine’s Forum on Regenerative Medicine. She is also a long-time member of the American Academy of Neurology’s Animal Science Rapid Response Team. In 2021, Dr. Salzman was selected to be a Henri Termeer Fellow by the Termeer Foundation. 

Dr. Salzman earned her Doctor of Veterinary Medicine Degree from Oklahoma State University and was in large animal practice until ALD manifested in her own family. This seismic event was what catalyzed her career shift from horses to humans.

Kathrin Meyer, Ph.D.
Alcyone Chief Scientific Advisor,
Chair of the Scientific Advisory Board, Alcyone Board Observer and PI,
Abigail Wexner Research Institute, Nationwide Children’s Hospital

Kathrin C. Meyer, Ph.D., studied cellular and biomedical Sciences at the Institute of Cell Biology in Berne, Switzerland. Her postdoctoral research was performed in Brian Kaspar’s laboratory at the Center for Gene Therapy in Columbus, Ohio. During that time, Dr. Meyer established a new and fast reprogramming method for in vitro modeling of neurodegenerative diseases using patient skin cells. Moreover, Dr. Meyer developed intrathecal gene therapy programs for several neurodegenerative diseases, including Spinal Muscular Atrophy and Batten Disease. Multiple clinical trials that are based on this work are currently ongoing at Nationwide Children’s Hospital. In 2017, Dr. Meyer became a Principal Investigator at Nationwide Children’s Hospital. She is also an Assistant Professor in the Department of Pediatrics at The Ohio State University, Columbus, Ohio.

Dr. Nicolas Wein, Ph.D.
Alcyone Scientific Advisor
PI, Abigail Wexner Research Institute, Nationwide Children’s Hospital

Dr. Nicolas Wein received his Ph.D. in molecular biology in the laboratory of Nicolas Levy, M.D., Ph.D., at the Marseille Medical School in France, where he worked on the development of new diagnosis tools for neuromuscular diseases, the discovery of the Minidysferline and its use for gene transfer, and investigation of the use of exon-skipping for dysferlinopathies. In 2011, he joined the team of Kevin Flanigan, M.D., principal investigator in The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, as a postdoctoral scientist. Dr. Wein worked on the identification of a novel IRES in the dystrophin gene and on an out-of-frame exon-skipping approach that can benefit patients with 5’ mutations in the DMD gene.

Gregory Stewart, Ph.D.

Head of R&D, Gene Therapy
Dr. Stewart is a neuroscientist with more than 30 years of bench, R&D and leadership industry experience. Previously, Dr. Stewart worked at Axovant Sciences (Sio Gene Therapies), Pairnomix, Alchemy Neuroscience, Voyager Therapeutics, Medtronic Genzyme (Sanofi Genzyme), ALZA and Roche Bioscience. Dr. Stewart holds a Ph.D. in neural biology from Washington University in St. Louis and a B.S. in neuroscience from Texas Christian University.