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RNAa
Expand Disease Spaces By Targeting
Nearly Any Beneficial Gene

‘Turn up’ Transcription

RNAa uses short duplex RNA known as saRNA to target and ‘turn up’ transcription of an endogenous gene leading to
restoration of endogenous protein function.

RNAa Offers Unprecedented Advantages:

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EXPANSION & RESTORATION

Expanded Disease Spaces

Expanded disease spaces by targeting nearly any beneficial gene, instead of disease-causing genes, offering new hope for many diseases undruggable by conventional approaches such as those caused by epigenetic silencing or down regulation of target gene expression.

Faithful Restoration of
Natural Gene Function

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EXPRESSION & ACCELERATION

Persistent Expression
of Target Gene

Acceleration of In-house
Drug Development

Pre-established medicinal chemistries and drug delivery platforms developed for gene silencing techniques are readily implemented accelerating in-house drug development.

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COST REDUCTION & MAXIMIZATION

Our In-house Expedited and
Cost-saving Screening Process

Our in-house expedited and cost-saving screening process rapidly identifies numerous saRNAs for targeted genes of choice. Each represents a candidate API.

Maximize Transcriptional Output

Additional tweaks improve medicinal properties and maximize transcriptional output for selected lead candidates.

Mechanism of RNAa

1: INPUT

saRNA Targets Non-coding Regulatory Sequence

saRNA is loaded into the AGO2 protein and processed into a single-strand.

2: SYSTEM

Cells with Target Endogenous Gene

AGO-saRNA complex enters the nucleus and recognizes a non-coding regulatory
sequence embedded in chromatin proximal to targeted gene and recruits
additional proteins to form to form the RITA complex.

3: OUTPUT

Elevated Target Gene Protein Levels

Formation of the RITA complex augments steps of transcription
initiation/elongation via RNAPII enrichment/ phosphorylation
and epigenetic modification indicative of relaxed chromatin.

Enhanced target gene transcription boosts mRNA output
leading to an increase in protein production.

Scientific illustration showing Mechanism of RNAa in 3 stages: Input, systems, and ouput