THERAPY DOSSIER · th-6d506ad00f2f

Deoxyribozyme against xylosyltransferase-1 mRNA (DNAXT-1as)

Other · CLASS-LEVEL / MODALITY match only, recorded knowingly. No human trial targets xylosyltransferase-1 mRNA. The recorded trials are DNAzymes against unrelated targets (GATA-3 in asthma, EBV-LMP1 in nasopharyngeal carcinoma) and establish that the deoxyribozyme modality has reached Phase 2 in humans, not that this agent has.

Non-drug Suggest a correction

Evidence & regulatory context

Mechanism: CLASS-LEVEL / MODALITY match only, recorded knowingly. No human trial targets xylosyltransferase-1 mRNA. The recorded trials are DNAzymes against unrelated targets (GATA-3 in asthma, EBV-LMP1 in nasopharyngeal carcinoma) and establish that the deoxyribozyme modality has reached Phase 2 in humans, not that this agent has.
Target: Not recorded
Injury models: Spinal cord injury
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 5

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

NCT01743768 ↗PHASE2completed

Human GATA-3-specific DNAzyme SB010 in patients with mild allergic asthma

Asthma

Enrolment: 38 · Results not recorded

NCT01449942 ↗PHASE1/PHASE2completed

EBV-LMP1 targeted DNAzyme (DZ1) in nasopharyngeal carcinoma

Nasopharyngeal Carcinoma

Enrolment: 40 · Results not recorded

NCT01470911 ↗PHASE1completed

Orally inhaled single doses of SB010, a human GATA-3-specific DNAzyme, in healthy male subjects

Asthma

Enrolment: 46 · Results not recorded

NCT01577953 ↗PHASE1completed

Orally inhaled single doses of SB010 in male patients with stable allergic asthma

Asthma

Enrolment: 24 · Results not recorded

NCT01554319 ↗PHASE1completed

Orally inhaled multiple doses of SB010 in healthy male subjects

Asthma

Enrolment: 36 · Results not recorded

Preclinical evidence 1

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

PMID 26428904 ↗2015Reported benefit

Long term study of deoxyribozyme administration to XT-1 mRNA promotes corticospinal tract regeneration and improves behavioral outcome after spinal cord injury.

RatSpinal cord injuryHemisection / partialWistarFemaleOsmotic minipump deliveryAdministration started acutely after the hemisection

Model: Dorsal hemisection spinal cord injury

DNAXT-1as digested XT-1 mRNA, gave overall behavioural improvement at 8, 10 and 14 weeks after injury compared with controls, promoted corticospinal tract growth over a 19 week survival period, and reduced lesion size, indicating tissue-protective effects.

Extraction: extract · Subtype source: rule · Supplied extraction confidence: 0.6. Machine-extracted fields require source verification.

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Deoxyribozyme against xylosyltransferase-1 mRNA (DNAXT-1as)