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?

Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency

X
Trial Profile

Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 08 Aug 2022

Price : $35 *
  • 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 Simoladagene autotemcel (Primary) ; Busulfan; Elapegademase
  • Indications Adenosine deaminase deficiency
  • Focus Therapeutic Use
  • Acronyms UCLA Cryo Study
  • Sponsors Orchard Therapeutics
  • Most Recent Events

    • 11 May 2021 Results (n=50) from NCT01852071, NCT02999984, and NCT01380990 assessing overall and event-free survival in autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency, published in the New England Journal of Medicine
    • 11 May 2021 Data from three Phase 1/2 clinical studies (n=40), and a compassionate use program (n=10) presented in an Orchard Media Release.
    • 11 May 2021 According to an Orchard Therapeutics media release, data from three Phase 1/2 clinical studies (n=40), and a compassionate use program (n=10) published in the New England Journal of Medicine (NEJM) .
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