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Delivery Platforms
Beyond Boundaries: Advanced Oligonucleotide Delivery Solutions  

Our Proprietary Delivery Platforms

Ractigen Therapeutics has made significant strides in oligonucleotide delivery technologies, developing proprietary platforms that facilitate the clinical development of these therapeutics. Their innovative approaches have enabled access to tissues and organs previously unreachable by oligonucleotide treatments.

Simplified Manufacturing

 The manufacturing process for our delivery technologies is streamlined, using classic oligonucleotide synthesis. This approach eliminates complex secondary manufacturing steps, making it more efficient than many traditional methods.

SCAD™

Smart chemistry-aided delivery (SCAD™): Pioneering RNA therapeutics for the CNS

Ractigen Therapeutics’ Smart chemistry-aided delivery (SCAD™) platform is a groundbreaking system specifically designed for the efficient delivery of duplex RNA (siRNA or saRNA) directly to the CNS via intrathecal injection. By conjugating the duplex RNA to a proprietary accessory oligonucleotide (ACO), SCAD™ effectively tackles a key hurdle in treating neurological disorders, holding immense potential for advancing novel therapies.

Key features & benefits:

  • Addresses neurological disorders with targeted intrathecal delivery directly to the CNS (brain and spinal cord).
  • Achieves broad and durable distribution throughout the CNS, leading to significant and sustained target modulation.
  • Validated safety profile: Supported by GLP toxicology studies and clinical data, building on established ASO chemistry used in approved CNS therapies.
  • Flexible modular design enables independent optimization of RNA payload and delivery properties.
  • Streamlined CMC process supports efficient scale-up, offering significant time and cost savings.
  • Platform versatility: Compatible with diverse duplex RNA payloads (e.g., siRNA, saRNA).

Partner with Ractigen to leverage the validated power of SCAD™ and develop transformative therapies for neurological diseases.

LiCO™

Lipid-conjugated oligonucleotide (LiCO™): Unlocking RNA therapeutics beyond the liver

Ractigen’s proprietary Lipid-conjugated oligonucleotide (LiCO™) platform overcomes the extrahepatic delivery challenge for RNA therapeutics. LiCO™ technology involves the conjugation of a carefully selected lipid moiety to a duplex RNA (siRNA or saRNA) via our proprietary Smart Diverse Linker (SDL™). This versatile system effectively delivers the RNA payload to a wide range of tissues and organs beyond the liver.

Key features & benefits:

  • Proven extrahepatic delivery: Successfully targets challenging tissues beyond the liver.
    • Systemic: Effectively reaches muscle, heart, and adipose tissue via SC or IV injection.
    • Local: Demonstrates potent activity in eye (intravitreal) and bladder (intravesical) applications.
  • Exceptional durability: Achieves significant therapeutic effects lasting at least 6 months with optimized dosing regimens. (Note: Systemic delivery validated for at least 3 months in NHPs).
  • Flexible administration: Validated efficacy via multiple routes: Subcutaneous (SC), Intravenous (IV), Intravitreal, and Intravesical.
  • Extensively de-risked & validated: Proven across multiple species (incl. NHPs), advancing into human trials, with extensive toxicology studies demonstrating a favorable safety profile.

Proven versatility – powering Ractigen’s pipeline:

  • RAG-01 (NMIBC): Phase I saRNA via Intravesical instillation (Local).
  • RAG-1C (PVR): Phase I-ready saRNA via Intravitreal injection (Local).
  • RAG-18 (DMD): IND-enabling saRNA via IV injection (Systemic).
  • RAG-24 (Obesity): PoC stage saRNA via SC dosing (Systemic).

Partner with Ractigen to leverage the power of LiCO™ and reach challenging therapeutic targets.

GLORY™

GaINAc-Oligonucleotide Liver Delivery (GLORY™)

By leveraging our SDL™ technology, Ractigen has created the GLORY™ platform that compares favorably with the best of industry benchmarks for liver delivery of duplex RNA.

 

 

 

 

 

 

 

 

 

How Can Our Delivery Platforms Help You?

We seek to explore the broad potential of our delivery platforms through research collaborations and licensing agreements in multiple therapeutic areas and applications.

For more information, please contact us.

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