Cystic fibrosis digest - 2021

Cystic fibrosis digest - 2021

Preclinical data was presented at the 44th European Cystic Fibrosis Conference (ECFS)

RCT223 (tRNA) & RTX0001 (mRNA) – ReCode Therapeutics

  • RNA-based molecular therapy program for cystic fibrosis (CF) was delivered using LNP platform, including three-component Zwitterionic Amino Lipids (ZALs) and five-component selective organ targeting lipid nanoparticles (SORT-LNPTM)
  • RCT223 (tRNA) restored CFTR function in patient-derived hBE cells with both single and repeat dosing
  • RTX0001 (mRNA) was well-tolerated after single and repeat doses delivered to primary hBE cells and to the lungs of mice; demonstrated restoration of CFTR function in patient-derived hBE cells (For full story click here)

SPL23-ASO – SpliSense

  • SpliSense preclinical data for SPL23-ASO, an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of the CF, showed that the ASO enables the production of a functional CFTR protein (For full story click here)

Novel RNA-based approaches from early-stage companies showed promise in animal studies

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CI Scientists Commentary:

  • ReCode Therapeutics is following two approaches in developing CF therapies
    • Target nonsense mutations – RCT223
    • Mutation-independent – RTX0001
  • Their proprietary lipid nanoparticle (LNP) supports the delivery of all programs
  • Next steps: RCT223 – Rodent studies; RTX0001 – Non-human primate studies in 2021
  • SpliSense is developing a broad portfolio for CF

Approach

Program

Stage of development

Correction of splicing mutation

SPL84-23-1

IND enabling studies

Undisclosed

Discovery

Exon skipping

EXON 23

Lead selection

EXON 24

Discovery

Splicing variants

Undisclosed

Discovery

– Dr. Kowndinya, CI Scientists

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