Drug Profile
SPK 7001
Alternative Names: AAV2 hCHM; RG 6367; SPK-7001; SPK-CHMLatest Information Update: 11 Nov 2022
Price :
$50
*
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.
- New Molecular Entity No
Highest Development Phases
- Phase I/II Retinal dystrophies
Most Recent Events
- 12 Oct 2022 Spark Therapeutics completes a phase I/II clinical trials in Eye disorders in USA (Intraocular) (NCT02341807)
- 17 Dec 2019 Spark Therapeutics has been acquired by Roche
- 27 Mar 2017 SPK 7001 is still at phase I/II development stage for Retinal dystrophies (Choroideremia) in USA (Intraocular)