Drug Profile
RP A501
Alternative Names: AAV9.LAMP2B; RP-A501Latest Information Update: 08 Nov 2023
Price :
$50
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At a glance
- Originator Rocket Pharmaceuticals
- Class Gene therapies
- Mechanism of Action Gene transference; Lysosomal-associated membrane protein 2 replacements
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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 II Glycogen storage disease type II
Most Recent Events
- 06 Nov 2023 Rocket Pharmaceuticals reached an alignment with the US FDA on the global phase II pivotal trial of RP A501 for Glycogen storage disease
- 06 Nov 2023 Rocket Pharmaceuticals submits Clinical Trial Application (CTA)/Investigational Medicinal Product Dossier (IMPD) to EMA and MHRA for Glycogen storage disease
- 06 Nov 2023 Rocket Pharmaceuticals plans a phase II trial in Glycogen storage disease in Europe and United Kingdom