StemCell Therapy

Abstract on Rett syndrome treatment through X-reactivation receives Excellence in Research Award

Updated preclinical safety and efficacy data will be presented for ACTX-401, a gene replacement therapy currently in a Phase 1/2 study for the treatment of IGHMBP2-related disorders

LOWELL, Mass., May 2, 2022 /PRNewswire/ -- Alcyone Therapeutics Inc. ("Alcyone"), a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions, today announced the acceptance of four abstracts at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, an event being held May 16-19, 2022, at the Walter E. Washington Convention Center in Washington, D.C. and virtually.

Accepted abstracts include preclinical data from its X-reactivation gene therapy platform, an approach to correct X-linked dominant genetic disorders by reactivating the silenced X chromosome; preclinical safety and efficacy data from its gene replacement platform, which utilizes viral vectors to deliver a functional gene to compensate for a cell's missing or mutated gene; and improved efficiency of AAV gene therapy manufacturing through its platform upstream process, which is scalable and easily transferrable between multiple systems.

"The oral presentations will highlight the potential of Alcyone's discrete gene therapy platforms, X-reactivation for treatment of Rett syndrome and gene replacement for IGHMBP2-related disorders, spinal muscular atrophy with respiratory distress type 1 and Charcot Marie Tooth disease type 2S, which we are advancing in partnership with the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children's Hospital," said PJ Anand, Chief Executive Officer of Alcyone Therapeutics. "We also will have a poster presentation demonstrating the scalability and multi-system transferability of our AAV production process, which is a critical piece in our gene therapy platform development."

Anandcontinued, "Alcyone's multidisciplinaryapproach to addressing complex neurological conditionsis comprised of our partnered novel gene-based therapeutic platform combined with our proprietary CNS precision drug delivery and dosing technology platform, FalconTM,and leverages a scalable CMC process optimized to produce high-quality clinical material. Our three-pronged approach is designed to better address the current challenges of treating people living with severe neurological disorders."

The presentations are listed below, and the full preliminary program is available online on the ASGCT website.

Oral Presentation: A Novel Gene Therapy for Rett Syndrome through Reactivation of the Silent X Chromosome

Oral Presentation Details:

Presenting Author: Kathrin Meyer, Ph.D., Principal Investigator, Nationwide Children's Hospital and Chief Scientific Advisor, AlcyoneSession Title: Novel Therapeutic Targets to Treat CNS DisordersSession Date/Time: Wednesday, May 18, 3:45 5:30 p.m. ETPresentation Time: 4:45 5:00 p.m. ETRoom: Room 202Abstract #: 837

Samantha Powers, Ph.D., from the Center for Gene Therapy at Nationwide Children's Hospital, received the Excellence in Research Award in recognition for presenting one of the top 18 abstracts submitted for the ASGCT 25th Annual Meeting by a postdoctoral fellow or student.

Oral Presentation: Multicenter AAV Gene Therapy Studies for SMARD1/CMT2S Establish Safety and Efficacy in Multiple Animal Models and Pave the Way for Initiation of a Phase I/II Clinical Trial

Oral Presentation Details:Presenting Author: Kathrin Meyer, Ph.D., Principal Investigator, Nationwide Children's Hospital and Chief Scientific Advisor, AlcyoneSession Title: Musculo-skeletal DiseasesSession Date/Time: Monday, May 16, 10:15 a.m. 12:00 p.m. ETPresentation Time: 11:15 11:30 a.m. ETRoom: Salon GAbstract #: 33

Poster Presentation: Development of an Upstream Process and Analytics for AAV Manufacturing

Poster Presentation Details:Presenting Author: Desyree Jesus, Ph.D., Associate Director, CMC Analytics, AlcyoneSession Title: Vector Product Engineering, Development or Manufacturing IIISession date/time: Wednesday, May 18, 5:30 6:30 p.m. ETRoom: Hall DPoster Board #: W-286Abstract #: 1160

Poster Presentation: Evaluation of AAV9 Gene Therapy for SMARD1/CMT2S in Different Mouse Models Reveal Differences in Efficacy Dependent on Promoter Choice

Poster Presentation Details:Presenting Author: J. Andrea Sierra Delgado, M.D., M.Sc., Chief Research Associate in Dr. Kathrin Meyer's Lab, Nationwide Children's HospitalSession Title: Musculo-skeletal DiseasesSession Date/Time: Wednesday, May 18, 5:30 6:30 p.m. ETRoom: Hall DPoster Board #: W-198Abstract #: 1072

About Alcyone TherapeuticsAlcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The Company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the Company's proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone's lead programs utilize X-chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMPB-2 related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). For more information, visit http://www.alcyonetx.com.

About Alcyone's Strategic Collaboration with the Abigail Wexner Research Institute at Nationwide Children's HospitalAlcyone works closely with scientists from the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI) in Columbus, Ohio, where four discrete gene therapy platform technologies, each with novel and differentiated mechanisms of action, including X-reactivation, conventional transgene replacement, vectorized exon skipping, and promoter modulation were designed, developed, and are being advanced towards the clinic. Alcyone has optioned the four programs and is funding research to explore the potential for the clinical application of these therapeutics using FalconTM, its proprietary CNS precision drug delivery and dosing technology platform, to improve the lives of people impacted by severe neurological conditions. The research is led by Kathrin Meyer, Ph.D., and Nicolas Wein, Ph.D., Principal Investigators in the Center for Gene Therapy at AWRI. Both Dr. Meyer and Dr. Wein sit on Alcyone's Scientific Advisory Board (SAB), with Dr. Meyer serving as Chief Scientific Advisor and Chair of Alcyone's SAB.

SOURCE Alcyone Therapeutics

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Alcyone Announces Two Oral Presentations on its Gene Therapy Platforms at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting -...

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