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Enum: OligonucleotideDeliveryPlatformEnum

How a therapeutic oligonucleotide is carried to its target tissue. This is the delivery strategy, not the targeting ligand (see conjugation) and not the route of administration: it is what solves the stability, cellular-uptake, and endosomal-escape problem for a given drug. The distinction is clinically load-bearing - patisiran and vutrisiran silence the same transcript, but the lipid-nanoparticle formulation is dosed intravenously every three weeks with premedication, while the GalNAc conjugate is dosed subcutaneously every three months without it.

URI: dismech:enum/OligonucleotideDeliveryPlatformEnum

Permissible Values

Value Meaning Description Additional Info
UNFORMULATED None Chemically stabilized oligonucleotide administered without a carrier or targe... Title: Unformulated / free uptake
CONJUGATE None Covalently conjugated to a targeting ligand that drives receptor-mediated upt... Title: Ligand conjugate
LIPID_NANOPARTICLE None Encapsulated in an ionizable-lipid nanoparticle that protects the payload and... Title: Lipid nanoparticle (LNP)
POLYMER_NANOPARTICLE None Encapsulated in a polymeric or dendrimer nanoparticle
VIRAL_VECTOR None Delivered by an engineered viral vector (e
EXOSOME None Delivered in an exosome or other extracellular vesicle Title: Exosome / extracellular vesicle
OTHER None Delivery platform not covered by the above categories

Slots

Name Description
delivery_platform How the oligonucleotide is carried to its target tissue - unformulated, ligan...

Identifier and Mapping Information

Schema Source

  • from schema: https://w3id.org/monarch-initiative/dismech

LinkML Source

name: OligonucleotideDeliveryPlatformEnum
description: 'How a therapeutic oligonucleotide is carried to its target tissue. This
  is the delivery strategy, not the targeting ligand (see conjugation) and not the
  route of administration: it is what solves the stability, cellular-uptake, and endosomal-escape
  problem for a given drug. The distinction is clinically load-bearing - patisiran
  and vutrisiran silence the same transcript, but the lipid-nanoparticle formulation
  is dosed intravenously every three weeks with premedication, while the GalNAc conjugate
  is dosed subcutaneously every three months without it.'
from_schema: https://w3id.org/monarch-initiative/dismech
rank: 1000
permissible_values:
  UNFORMULATED:
    text: UNFORMULATED
    description: Chemically stabilized oligonucleotide administered without a carrier
      or targeting ligand, relying on free tissue uptake (e.g., intrathecal nusinersen,
      subcutaneous inotersen)
    title: Unformulated / free uptake
  CONJUGATE:
    text: CONJUGATE
    description: Covalently conjugated to a targeting ligand that drives receptor-mediated
      uptake; the specific ligand is recorded in conjugation (e.g., GalNAc for hepatocyte
      ASGR1 uptake)
    title: Ligand conjugate
  LIPID_NANOPARTICLE:
    text: LIPID_NANOPARTICLE
    description: Encapsulated in an ionizable-lipid nanoparticle that protects the
      payload and destabilizes the endosomal membrane on acidification (e.g., patisiran)
    title: Lipid nanoparticle (LNP)
  POLYMER_NANOPARTICLE:
    text: POLYMER_NANOPARTICLE
    description: Encapsulated in a polymeric or dendrimer nanoparticle
  VIRAL_VECTOR:
    text: VIRAL_VECTOR
    description: Delivered by an engineered viral vector (e.g., AAV-expressed short
      hairpin RNA)
  EXOSOME:
    text: EXOSOME
    description: Delivered in an exosome or other extracellular vesicle
    title: Exosome / extracellular vesicle
  OTHER:
    text: OTHER
    description: Delivery platform not covered by the above categories