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

Next Previous
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 *
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 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) .

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