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?

AGTC 0106

Drug Profile

AGTC 0106

Alternative Names: AGTC-0106; Alpha-1 antitrypsin deficiency gene therapy - Beacon Therapeutics; rAAV1-CB-hAAT; Recombinant AAV-AAT vector gene therapy - Beacon Therapeutics

Latest Information Update: 29 Jun 2023

Price : $50 *
  • 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 Applied Genetic Technologies Corporation
  • Developer Beacon Therapeutics; National Heart, Lung and Blood Institute
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Alpha 1-antitrypsin deficiency
  • New Molecular Entity No

Highest Development Phases

  • Phase II Alpha 1-antitrypsin deficiency

Most Recent Events

  • 07 Feb 2019 Phase II development is ongoing for Alpha 1-antitrypsin deficiency in Ireland and USA
  • 05 May 2016 Pharmacodynamics data from a phase I trial in Alpha-1-antitrypsin deficiency released by Applied Genetic Technologies Corporation
  • 20 Jan 2016 Pharmacodynamics data from a phase II trial in Alpha-1-antitrypsin deficiency released by Applied Genetic Technologies Corporation
Subscriber content

You need to be a logged in subscriber to view this content.

If your organization has a subscription then there are several options available to help you access AdisInsight, even while working remotely.

  • IP authentication when working within your organization’s network.
  • with username/password or try via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days.
  • Contact us at AsktheExpert.AdisInsight@springer.com for assistance.

If your organization does not have a subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Buy a PDF version of the profile.
  • Request a free trial
Back to top