Alcyone Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders

• Alcyone's proprietary CNS precision delivery and dosing platform enables unprecedented control and versatility for CNS biodistribution and gene therapy development
• RTW Investments, LP contributes $23 million in funding and will join Alcyone's Board of Directors
• Alcyone's pipeline currently utilizes 4 gene therapy platforms and 12 AAV gene therapy programs targeting severe CNS disorders; lead program based upon novel "Pipeline in a Product" X-Reactivation platform, with Rett syndrome as initial indication
• Collaboration with Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI) leverages Alcyone's delivery platform and AWRI's research and technologies to advance broad pipeline

LOWELL, Mass., June 9, 2021 /PRNewswire/ -- Alcyone Therapeutics ("Alcyone"), a biotechnology company pioneering next-generation precision central nervous system (CNS) therapies to improve the lives of patients and families impacted by severe neurological conditions, today announced its launch and $23 million in funding from funds affiliated with RTW Investments, LP ("RTW"). Piratip Pratumsuwan, Managing Director at RTW, will join Alcyone's Board of Directors.

Alcyone's optioned pipeline currently includes 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone's four gene therapy platform technologies include X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation.

Through a collaboration, Alcyone's proprietary, next-generation CNS delivery platform and product development, manufacturing and commercialization capabilities will unite with four gene therapy technologies and discovery, research and early development capabilities developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI). At AWRI, the collaborations are led by Kathrin Meyer, Ph.D., and Nicolas Wein, Ph.D., both Principal Investigators in the Center for Gene Therapy at AWRI. They are investigating and employing the use of gene therapies for the prevention and treatment of certain human diseases. Dr. Meyer is Chair of Alcyone's Scientific Advisory Board.

"Our mission at Alcyone Therapeutics is to provide life-changing therapies for children and their families impacted by severe neurological conditions," said PJ Anand, Founder, President and Chief Executive Officer of Alcyone Therapeutics. "Our next-generation precision delivery platform shows strong potential in overcoming the fundamental challenge in CNS therapy development. Coupled with multiple cutting-edge gene therapy technologies and research approaches at the Abigail Wexner Research Institute at Nationwide Children's Hospital, we aim to transform the efficiency of CNS therapeutics. As we advance our broad pipeline towards the clinic, we are grateful to the investors who have supported us and pleased to have the financial and strategic support of RTW."

Alcyone's precision CNS delivery technology platform allows for unprecedented control and versatility for biodistribution of genetic medicines to CNS regions of interest, including deep brain regions and spare off-target areas. Alcyone's CNS precision delivery can improve the efficiency of CNS gene therapy significantly impacting safety and efficacy, and the number of vectors needed, for an effective gene therapy.

Mr. Pratumsuwan commented, "We are excited by the potential for Alcyone's CNS precision delivery platform to generate a pipeline of drug candidates aimed at improving the lives of patients affected by neurological disease. I look forward to working with Alcyone's Board and team of experienced gene therapy leaders as they advance towards the clinic."

About Alcyone Therapeutics
Alcyone Therapeutics is a biotechnology company pioneering next-generation precision central nervous system (CNS) therapies to improve the lives of patients and families impacted by severe neurological conditions. Alcyone Therapeutics is advancing a pipeline of adeno-associated virus (AAV) gene therapy programs that leverage its proprietary, next-generation CNS delivery technology platform. Alcyone Therapeutics' lead programs target the treatment of Rett syndrome and spinal muscular atrophy with respiratory distress type 1 (SMARD1). For more information, please visit https://alcyonetx.com/.

About RTW Investments, LP (RTW)
RTW Investments, LP ("RTW") is a New York-based, global, full life-cycle investment firm that focuses on identifying transformational and disruptive innovations across the biopharmaceutical and medical technologies sectors. As a leading partner of industry and academia, RTW combines deep scientific expertise with a solution-oriented investment approach to support emerging medical therapies and the companies and/or academics developing them.

For further information about RTW, please visit www.RTWfunds.com.

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SOURCE Alcyone Therapeutics