Science & Technology
Our Approach
Our <u>Approach</u>

Integrative Technology for Complex Neurological Disorders

At Alcyone, we have developed a three-pronged approach to treating neurological disorders. We have engineered four platform technologies, across which there are several gene-based therapeutics. Each of our therapeutics is manufactured through our quality CMC process. Our novel precision delivery system Falcon™ optimizes administration to the patient.

Learn more about our gene-based platform technologies, CMC process, and Falcon™ below.

Gene Replacement

Gene Replacement therapy introduces a functional copy of a mutant gene to restore functional gene expression to the patient.

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X-Reactivation technology reactivates the silenced X chromosomes in X-linked genetic disorders, facilitating expression of the patient's own functional gene.

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Exon Skipping

Exon Skipping technology shifts translation of misaligned genes into a position that results in expression of a functional protein.

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Promoter Modulation

Promoter Modulation increases or decreases the expression of genes in a therapeutic direction.

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Chemistry, Manufacturing, and Controls

Alcyone has developed a customized approach to Chemistry, Manufacturing, and Controls, an integral part of pharmaceutical production that is particularly challenging for gene-based therapeutics.

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Delivering therapeutics to the brain is a critical but challenging aspect of treating neurological conditions. The Falcon™ precision delivery technology utilizes computational algorithms to tailor drug delivery for each patient.

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CMC Exon Skipping Falcon Gene Replacement Promoter Modulation X-reactivation How it works