Drug Profile
Leber congenital amaurosis gene therapy - Beacon Therapeutics
Alternative Names: AAV2-RPE65 Vector; rAAV2-CB-hRPE65Latest Information Update: 29 Jun 2023
Price :
$50
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At a glance
- Originator Applied Genetic Technologies Corporation
- Developer Beacon Therapeutics; University of Massachusetts Medical School
- Class Gene therapies
- Mechanism of Action Gene transference
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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 Leber congenital amaurosis
Most Recent Events
- 22 Sep 2017 Applied Genetic Technologies Corp completes a phase I/II trial in Leber congenital amaurosis in USA (Intraocular) (NCT00749957)
- 22 Apr 2016 Interim efficacy and adverse events data from a phase I/II trial in Leber congenital amaurosis released by Applied Genetic Technologies
- 25 Jun 2015 Phase I/II development is ongoing in USA