Drug Profile


Alternative Names: AAV2-CB-RS1; rAAV hRS1; rAAV2tYF CB hRS1; Recombinant adeno associated virus vector expressing retinoshcisin; X-linked retinoschisis gene therapy-AGTC; XLRS gene therapy

Latest Information Update: 30 May 2017

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At a glance

  • Originator Applied Genetic Technologies Corporation
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; RS1 gene expression stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Retinoschisis
  • New Molecular Entity No

Highest Development Phases

  • Phase II Retinoschisis

Most Recent Events

  • 10 May 2017 Safety data from a phase I/II trial in Retinoschisis released by Applied Genetics Technology Corporation
  • 10 Jun 2016 Phase-II clinical trials in Retinoschisis in USA (Intravitreous)
  • 30 Sep 2015 Interim pharmacodynamics and adverse events data from preclinical studies in X-linked retinoschisis released by AGTC
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