Alcyone Presents Preclinical Data on Gene Therapy Programs With the Center for Gene Therapy at the Abigail Wexner Research Institute (AWRI) at ASGCT 2022

Sid Martin Client Alcyone Therapeutics Inc. (“Alcyone”), a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions, presented preclinical data showing the Company’s partnered novel adeno associated virus serotype 9 (AAV9) gene therapy vector ACTX-101 safely and effectively reactivates the inactive X chromosome in a Rett syndrome mouse model and is safe in a preliminary nonhuman primate study at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting.

Alcyone also presented data from its partnered gene therapy program for ACTX-401, a gene replacement therapy currently in a Phase 1/2 clinical study for the treatment of IGHMBP2-related disorders (IRDs), and on the Company’s internal AAV manufacturing process and platform.

“X-reactivation is a novel gene therapy approach to correcting mutations in X-linked dominant disorders such as Rett syndrome,” said Ottavio Vitolo, M.D., M.M.Sc., Chief Medical Officer and Global Head of R&D at Alcyone. “The Rett mouse model preclinical data produced thus far by Dr. Kathrin Meyer’s lab at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and by Dr. Sanchita Bhatnagar, formerly with the University of Virginia, currently at the University of California Davis School of Medicine, demonstrate that activation of the silenced X chromosome has the potential to be a safe and effective approach, which mitigates the risk of MECP2 overexpression typical of gene replacement therapies.”

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