RNA Therapeutics Platform

One RNA.
Multiple functions.
Real medicine.

A proprietary platform that integrates multiple gene editing and regulatory components into a single, deliverable RNA molecule — paired with large-fragment delivery systems engineered to overcome the limits of conventional genetic medicine.

Platform

A new architecture for genetic medicine

Traditional approaches fragment complexity across multiple delivery vectors and editing systems. Our platform unifies them into a single molecule.

RNA All-in-One

A single IVT RNA encoding all required functional components for coordinated therapeutic action.

Multi-component integration in one transcript
Synchronized intracellular release
Reduced variability

Large-Payload LNP Engine

A delivery system purpose-built for next-generation RNA therapeutics.

Large RNA payloads (9kb+)
Hepatic targeting & systemic delivery
Scalable for clinical translation

Process

From design to execution

1

Encode

Multiple functional elements are designed within a single RNA construct, enabling coordinated multi-component activity from one molecule.

2

Deliver

Proprietary LNP systems transport large RNA payloads into target tissues with high efficiency and cell-type specificity.

3

Execute

Intracellular processing releases independent modules, enabling coordinated biological function from a single delivery event.

Capabilities

A versatile engine for genetic modulation

Three core modalities, unified within a single platform architecture.

Epigenetic Editing

CRISPRi / Modulation

Programmable, non-permanent control of gene expression without altering the underlying DNA sequence.

  • Multi-target silencing in one construct
  • Tumor & viral transcriptional reprogramming

Genome Editing

Targeted Gene Integration

Approaches toward sequence integration beyond single-base editing, with a focus on defined genomic loci relevant to cell engineering.

  • ~2 kb sequence integration
  • Targeted loci (e.g., TRAC locus)
  • In vivo cell engineering (e.g. CAR-T)

Protein Replacement

In Vivo RNA Expression

Direct expression of functional proteins via high-expression IVT RNA constructs, enabling therapeutic protein delivery beyond the limits of AAV.

Validation

Engineered for real-world performance

Our platform is built not only for innovation — but for translatability. Every component is designed with clinical development in mind.

>9 kb

Successfully delivered large RNA constructs exceeding conventional payload limits

Multi

Demonstrated coordinated multi-component expression from a single transcript

LNP+

Enhanced delivery performance compared to conventional transfection systems

Applications

Targeting diseases where simpler tools fail

Designed for therapeutic challenges that cannot be solved with single-edit approaches.

Liver-Directed RNA Medicine

Precision hepatic gene modulation for chronic conditions with high unmet need.

Chronic viral diseases (HBV)
High-burden protein deficiencies
Precision gene modulation

Genetic Disorders

Multi-component strategies for diseases beyond the reach of single-edit tools.

Targeted sequence modification
Defined mutation sites
Delivery-enabled contexts

Precision Oncology

Epigenetic reprogramming and multi-target intervention in a single therapy.

Tumor pathway reprogramming
Microenvironment modulation
Multi-target single therapy

Let's build the next generation of RNA medicine

We collaborate with leading biopharma and research organizations to unlock new therapeutic possibilities — from multi-component genetic engineering to large payload delivery and strategies beyond conventional CRISPR.

Company

Driven by science.
Built for impact.

Astral Genetics is advancing a new class of RNA therapeutics designed to address the most challenging diseases — where precision, scale, and coordination are essential. We combine innovative molecular design with next-generation delivery systems to transform genetic medicine from fragmented solutions into unified platforms.