Gene EditingTX
Gene EditingTX
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  • Our Technologies
  • Therapeutics
  • Team
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    • About Us
    • Our Technologies
    • Therapeutics
    • Team
    • Partnership & Investment
    • Newsroom
  • About Us
  • Our Technologies
  • Therapeutics
  • Team
  • Partnership & Investment
  • Newsroom

Our Technologies

At GETx, we develop state-of-the-art gene editing platforms that advance and drive innovation in gene editing therapeutic landscape. Our proprietary technologies drive development of next-generation genetic medicines, ensuring the most effective and safest gene editing therapies for patients with genetic disorders.

REMAXPCOC

REMAX: High-Efficiency Genetic Reframing

What is REMAX?

REMAX is our proprietary gene editing platform, specifically designed for highly efficient genetic reframing in diseases where restoring the reading frame is a viable therapeutic strategy, such as Duchenne Muscular Dystrophy (DMD).

Key Features of REMAX

  

  • Dual-Cut Nuclease Strategy: REMAX utilises a dual-cut mechanism, targeting critical genomic regions such as exons and splice sites to enhance therapeutic efficiency.
  • Optimised Repair Pathways: The dual-cut approach leads to two primary repair outcomes; intervening deletions or local indels. With REMAX, both outcomes restore the reading frame and significantly increase therapeutic efficacy.
  • Superior to Conventional CRISPR Approaches: REMAX dramatically improves functional dystrophin restoration in DMD-affected cells, outperforming conventional CRISPR strategies.
  • Single AAV Delivery: By leveraging small-size nucleases, REMAX enables highly efficient in vivo gene editing and is fully packaged within a single AAV vector, ensuring scalability and clinical feasibility.

With its exceptional reframing efficiency, REMAX represents a groundbreaking advancement in gene editing therapy for diseases amenable to reframing, such as Duchenne Muscular Dystrophy (DMD)—a devastating disorder affecting over 300,000 individuals worldwide. By enabling efficient genetic correction, REMAX offers a transformative, one-time treatment with the potential to effectively restore functional dystrophin expression and significantly improve patient outcomes.

PCOC: Prime-Chop-or-Change

PCOC: Prime Chop-or-Change for Dominant Mutations

Prime Chop-or-Change (PCOC) is our proprietary technology engineered to treat diseases caused by dominant-negative or toxic-gain-of function (DN-TGOF) mutations, such as autosomal dominant Retinitis Pigmentosa (adRP).

PCOC - Key Features

  • Dual-Action Approach: PCOC integrates prime editing with Cas9-induced double-strand breaks (DSBs) to correct mutant alleles while eliminating unedited toxic alleles.
  • Maximizing Functional Alleles: This approach significantly increases the proportion of functional alleles, making PCOC the most effective potential therapy for adRP and similar conditions.
  • Breakthrough in adRP Treatment: Since adRP desirably requires both mutant allele elimination and functional allele enhancement, PCOC offers a novel, comprehensive solution.

Undisclosed Innovations

We are continuously developing next-generation gene editing technologies to further enhance efficiency and safety. By staying ahead of the curve, we aim to create best-in-class therapies for a wide range of genetic diseases.

Gene Editing Therapeutics

The University of Adelaide, Adelaide SA 5000, Australia

Email: contact@geneeditingtx.com

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