THERAPY DOSSIER · th-c58ac57a012f

Hepatic delivery of functional human ABCC6 by hydrodynamic tail vein injection

Other

Evidence & regulatory context

Mechanism: Not recorded
Target: Not recorded
Injury models: Other
Reported indication: Not recorded
First approval: Not recorded
Development stage: Not recorded
ChEMBL record: Not recorded
ChEMBL max phase: Not recorded
Synonyms: Not recorded
Note: Not recorded

Clinical trials 1

0 linked trials carry an injury-indication flag. These flags support record-level exploration and sit alongside the stringent aggregate counts described in Methods.

NCT06462547 ↗PHASE2recruiting

ADAPT: open-label long-term safety of INZ-701 in ENPP1 deficiency and ABCC6 deficiency

Pseudoxanthoma Elasticum · ABCC6 deficiency · Arterial Calcification

Enrolment: 200 · Results not recorded

Preclinical evidence 1

1 studies reported at therapy level; 1 evidence entries itemised.

PMID 24479134 ↗2014Reported benefit

The level of hepatic ABCC6 expression determines the severity of calcification after cardiac injury.

MouseOtherC57BL/6 background, Abcc6-/- and Abcc6+/- miceHydrodynamic tail vein injection

Model: Cardiac infarction by non-ischaemic cryoinjury and by coronary artery ligation

Delivery of human ABCC6 to approximately 13% of mouse hepatocytes significantly reduced the calcification response to cardiac cryoinjury; Abcc6+/- mice with approximately 38% of wild-type ABCC6 expression showed no calcium deposits after injury.

Extraction: extract · Supplied extraction confidence: 0.65. Machine-extracted fields require source verification.

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