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?

ATA 100

Next Previous
Drug Profile

ATA 100

Alternative Names: ATA 100; GNT-0006; LGMD2I/R9 gene therapy - Atamyo Therapeutics; rAAV9-hFKRPco_miR-208a

Latest Information Update: 28 Nov 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 Atamyo Therapeutics
  • Class Gene therapies
  • Mechanism of Action FKRP protein replacements; Gene transference; Glycosyltransferases replacements
  • Orphan Drug Status

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

    Yes - Limb girdle muscular dystrophies
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Limb girdle muscular dystrophies

Most Recent Events

  • 28 Nov 2025 No recent reports of development identified for preclinical development in Limb-girdle-muscular-dystrophies in USA (Parenteral)
  • 03 Apr 2025 ATA 100 receives Fast Track designation for Limb girdle muscular dystrophies [Parenteral] in USA
  • 03 Apr 2025 ATA 100 receives Rare Pediatric Disease Designation forLimb girdle muscular dystrophies in the USA

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

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: 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