Drug Profile

RP A501

Alternative Names: AAV9.LAMP2B; RP-A501

Latest Information Update: 29 Aug 2025

Price :

$50 ^*

Note:

Adis is an information provider. We do not sell or distribute actual drugs.
Final gross price and currency may vary according to local VAT and billing address.
Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

Originator Rocket Pharmaceuticals
Class Gene therapies
Mechanism of Action Gene transference; Lysosomal-associated membrane protein 2 replacements

Orphan Drug Status

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

Yes - Glycogen storage disease type II
New Molecular Entity No

Highest Development Phases

Phase II Glycogen storage disease type II

Most Recent Events

20 Aug 2025 US FDA lifts a clinical hold on phase II trial for Glycogen storage disease type II (NCT06092034)
23 May 2025 US FDA placed a clinical hold on phase II trial for Glycogen storage disease type II
18 Nov 2024 Efficacy and adverse events data from a phase-I clinical trials in Glycogen storage disease type II released by Rocket Pharmaceuticals

You need to be a logged in or subscribed to view this content

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com