Ractigen Secures FDA Rare Pediatric Disease Designation for RAG-18

Ractigen Secures FDA Rare Pediatric Disease Designation for RAG-18

By, Admin 27 July 2024

Ractigen Secures FDA Rare Pediatric Disease Designation for RAG-18 in Duchenne Muscular Dystrophy Treatment

Overview

Ractigen Therapeutics, a leader in small activating RNA (saRNA) therapies, has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for its saRNA candidate RAG-18, designed to treat Duchenne Muscular Dystrophy (DMD). This designation highlights RAG-18's potential as an innovative treatment for both DMD and Becker Muscular Dystrophy (BMD), irrespective of specific mutations in the DMD gene.

RPDD Grant

  • The RPDD is granted for treatments addressing serious or life-threatening diseases affecting fewer than 200,000 children in the U.S. 
  • If RAG-18’s New Drug Application (NDA) is approved, Ractigen could obtain a Priority Review Voucher (PRV). 
  • This voucher enables expedited review of another product's marketing application and can be sold, with past PRVs selling for between $100 million and $350 million, offering substantial financial potential for Ractigen.

From the CEO of Ractigen Therapeutics

Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, stated, ""Securing the RPDD for RAG-18 is a major achievement, underscoring our dedication to developing groundbreaking therapies for rare diseases. This designation accelerates RAG-18’s development and opens doors for further advancements in RNAa therapies, aiming to enhance the lives of patients with DMD and other rare diseases.""

About RAG-18

  • RAG-18 is an innovative saRNA candidate intended to activate UTRN gene expression in muscle cells using the RNAa mechanism. 
  • The utrophin protein, produced by the UTRN gene, is similar to dystrophin in structure and function. 
  • Increasing utrophin levels could compensate for the lack of dystrophin in DMD muscle cells, making this treatment applicable to all DMD patients regardless of their specific genetic mutation.
  • Preclinical research indicates that RAG-18, delivered via subcutaneous injection with Ractigen’s LiCOTM (lipid-conjugated oligonucleotide) technology, effectively reduces muscle damage, showing promise for DMD treatment.

Duchenne Muscular Dystrophy

  • Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) are genetic disorders caused by mutations in the dystrophin gene, resulting in insufficient functional dystrophin protein. 
  • This protein is vital for muscle fiber stability, and its absence leads to muscle cell damage and progressive weakness and degeneration. 
  • The dystrophin gene, the largest in the human genome with 79 exons, is the target of current treatments like antisense oligonucleotides (ASO) mediated exon skipping, gene therapy, and gene editing. 
  • These treatments, however, have limitations, highlighting the urgent need for innovative therapies that address DMD's root cause for more effective and lasting benefits.

RNAa

  • RNAa is a clinically validated technology platform developed by Dr. Long-Cheng Li and his team. 
  • It uses saRNAs to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. 
  • This technology holds great promise for developing treatments for various diseases, particularly genetic disorders, where traditional methods are inadequate.