Technology

Delivery Platform

A proprietary drug delivery technology that takes advantage of tissue targeting via a modified / optimized antibody (3E10) that has intracellular-delivery capabilities dependent on membrane expression of the nucleoside transporter (ENT2). Because the ENT2 transporter is naturally enriched in muscle and neurons (and elevated in certain cancers), Valerion is able to generate proprietary product candidates utilizing substrates (proteins, enzymes, oligonucleotides, etc.) not previously deliverable to target tissues (ex. heart & skeletal muscle fibers); thereby, providing a novel way to treat a number of diseases with limited or no current therapeutic options.

 

Key References

  • 3E10Fv Review Paper:  Antibody mediated transduction of therapeutic proteins into living cells.
  • Hansen et al. The Scientific World Journal (2005) vol: 5, pp.782-788.

  • ENT2 Transport Identification:  Intranuclear Protein Transduction through a Nucleoside Salvage Pathway.
  • Hansen et al. JBC (2007) vol. 282, no. 29, pp. 20790–20793.

  • Myotonic Dystrophy Rationale (Viral-mediated replacement previously demonstrated): Reversal of RNA missplicing and myotonia after muscleblind over-expression in a mouse poly(CUG) model for myotonic dystrophy.
  • Kanadia et al. PNAS (2006) vol. 103, no. 31.

  • Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy.
  • Lawlor MW, Armstrong D, Viola MG, Widrick JJ, Meng H, Grange RW, Childers MK, Hsu CP, O’Callaghan M, Pierson CR, Buj-Bello A, Beggs AH.

    Hum Mol Genet. 2013 Apr 15;22(8):1525-38.