NGF Blog


AVROBIO to End Gaucher Disease Trials, Explore Sale

AVROBIO, a promising developer of clinical-stage gene therapy solutions, has announced that they are stopping development of their programs, including their trials for patients with Gaucher disease.

In a press release, AVROBIO says they will “conduct a comprehensive exploration of strategic alternatives focused on maximizing shareholder value” while they halt all of the programming. While they consider an acquisition, merger, or some other transaction, they did not share a timetable for the evaluation process or the likelihood that any transaction even occurs.

The decision was made after a review of their business, including their resources, programs, and capabilities, according to the press release.

Dani Thomas of AVROBIO provided the following information to NGF, confirming the end of the GUARD 1 trials for Type 1 patients and the development plans for GUARD 3 trials for Type 3 patients.

Based on review of the business, including the status of its programs, resources, and capabilities, AVROBIO has made the difficult determination to halt further development of its programs and to conduct a comprehensive exploration of strategic alternatives for the company.  This means that the GUARD 1 trial will not be recruiting any new subjects and that the planned development plans in GD3 and the GUARD 3 trial will be halted at this time.

This has been a difficult decision for AVROBIO, and we know that it has a big impact on the patients and families whom we have had the privilege to get to know over the years. However, we believe that this is the most prudent decision that will allow us as an organization the opportunity to consider and potentially pursue alternative options for the programs.

This news is disappointing, however, there will be other gene therapy studies that will take place in the future. We will continue to follow the various therapeutic developments and will keep the Gaucher community posted.

– Robin Ely MD, Founder, Clinical Director, National Gaucher Foundation

Living a Better Today

Skip to content