Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency

Next Previous
X
Search
Trial Profile

A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 10 Jun 2024

Price :

$35 *

Note:
  • Adis is an information provider.
  • 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 trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

  • Drugs GS 100 (Primary)
  • Indications Inborn genetic disorders
  • Focus Adverse reactions; First in man
  • Sponsors Grace Science

    • Most Recent Events

    • 03 Jun 2024 According to a Grace Science media release, company that the 2nd NGLY1 Deficiency patient was successfully treated with GS-100 in May. A dose escalation is planned for the 3rd patient (second cohort) later this summer provided all safety signals remain positive in the first cohort.
    • 03 Jun 2024 According to a Grace Science media release, company announced that the IND for GS-100 for NGLY1 Deficiency, was accepted into the FDA's Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.
    • 25 Mar 2024 event According to a Grace Science media release, the company is planning to dose the second patient in May 2024, for NGLY1 Deficiency treatment.

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
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top