Trial Profile

A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 24 Mar 2025

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

17 Mar 2025 Status changed from active, no longer recruiting to completed.
20 Jun 2024 According to a Tectonic Therapeutic media release, AVROBIO has been merged with Tectonic Therapeutic to form Tectonic Therapeutic.
18 May 2023 Results presented in the AVROBIO Media Release.

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 (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com