SPK 7001

Drug Profile

SPK 7001

Alternative Names: AAV2 hCHM; SPK-7001; SPK-CHM

Latest Information Update: 27 Mar 2017

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At a glance

  • Originator University of Pennsylvania
  • Developer Spark Therapeutics; The Childrens Hospital of Philadelphia; University of Pennsylvania
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action CHM protein expression stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Retinal dystrophies
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Retinal dystrophies

Most Recent Events

  • 27 Mar 2017 SPK 7001 is still at phase I/II development stage for Retinal dystrophies (Choroideremia) in USA (Intraocular)
  • 28 Apr 2015 Spark Therapeutics enters into an option agreement with Clearside Biomedical for exclusive licensing rights for the micoinjector technology for delivering gene therapies to the back of the eye
  • 21 Jan 2015 Phase-I/II clinical trials in Eye disorders in USA (Intraocular) (NCT02341807)
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