Drug Profile

GCB 001

Alternative Names: GCB-001

Latest Information Update: 28 Feb 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 Genecombio
Class Spinal muscular atrophy gene therapies
Mechanism of Action Gene transference; Survival of motor neuron 1 protein replacements

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

13 Jan 2025 Preclinical trials in Spinal muscular atrophy in China (Intrathecal)
13 Jan 2025 Genecombio plans a phase I GITOPWTSMA trial for Spinal muscular atrophy (In children) in China (Intrathecal) in February 2025 (NCT06772402) (ChiCTR2500095667)

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