Drug Profile

Research programme: gene therapies - Spark Therapeutics

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

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

No development reported Haematological disorders; Neurodegenerative disorders

28 Mar 2025 No recent reports of development identified for preclinical development in Neurodegenerative-disorders in USA (Parenteral)
27 Mar 2023 Preclinical development in Neurodegenerative disorders is ongoing in USA (Parenteral) (Spark Therapeutics pipeline, March 2023)
28 Feb 2021 No recent reports of development identified for preclinical development in Haematological-disorders in USA (Parenteral)

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