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Clinical Trials

Date: 2017-08-24

Type of information: update on patient enrollment

phase: 1-2

Announcement: update on patient enrollment

Company: MeiraGTx (USA - NY)

Product: AAV2/8-hCARp.hCNGB3 - adenovirus associated viral vector serotype 8 containing the human CNGB3 gene

Action mechanism:

  • gene therapy.

Disease: achromatopsia due to mutations in the CNGB3 gene

Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases

Country: UK

Trial details: (NCT03001310)

Latest news:

  • • On August 24, 2017, MeiraGTx announced that the first patient in the 2nd cohort in the clinical study of AAV-mediated gene therapy AAV2/8-hCARp.hCNGB3 for achromatopsia patients with mutations in the CNGB3 gene has been treated at Moorfields Eye Hospital. This follows the safe completion of treatment of the first three patients in the low dose cohort in the CNGB3 clinical trial. MeiraGTx has also treated 7 patients in the PhaseI/II clinical study of AAV2/5-OPTIRPE65 in LCA2 patients with RPE65 deficiency, with the final two patients in the high dose cohort to be treated during Q3 2017.
  • • On February 8, 2017,  MeiraGTx announced  the first patient in its clinical study for achromatopsia due to mutations in the CNGB3 gene was treated at the Moorfields Eye Hospital in London. The AAV-mediated gene therapy study design for CNGB3 deficiency is an open label, multi-center, Phase I/II dose escalation trial.
 

Is general: Yes