Drug Profile

SPK 10001

Alternative Names: SPK-10001

Latest Information Update: 28 Mar 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 Spark Therapeutics
Class Gene therapies
Mechanism of Action Gene transference; Huntingtin protein expression inhibitors

Orphan Drug Status

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

No
New Molecular Entity No

Highest Development Phases

Phase I/II Huntington's disease

Most Recent Events

21 Feb 2025 Phase-I/II clinical trials in Huntington's disease in USA (Intracerebral) (NCT06826612)
14 Feb 2025 Preclinical trials in Huntington's disease in USA (Intracerebral), prior to February 2025
14 Feb 2025 Pharmacodynamics and adverse event data from a preclinical trial in Huntington's disease released by Spark therapeutics

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

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

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