Below, in their own words, is how we assisted rare blood disorder gene therapy uniQure Biopharma, launch and accelerate 4-6 months off of their study start-up. For more examples of our clients, see our collaborators page
When I started, we were planning to move into Phase 3, so we definitely needed a foothold in the USA. When I started, we knew nothing about genetic therapy. And the team (again, all in Europe at the time) knew nothing about USA regulations. We were basically stuck. I did my research and found a few people who offered support for IBC services.
We initially went towards a large firm as a way to centralize services, but we quickly realized that the firm wasn’t as centralized as we had thought, and more importantly, the firm did not support sites who had their own IBC.
CBS was different. Chris spent time with me, he came out and educated my newly formed USA team (and my Amsterdam based team by phone) around the IBC regulations. He helped us write an IBC executive summary, which has sped us through every IBC we have submitted to (1st pass approval every time so far). He also helped us write the Appendix M.
All things we didn’t really know how to do.
But what really set CBS apart has been their “white glove” service for sites. Many of our sites have their own IBCs, but not all of those IBCs are as organized as we would like – coupled with the fact that many of our coordinators have not worked on gene therapy before, so didn’t know how to fill out the form. CBS has supported each of those sites as much as they required. In some cases, that was fully completing the form. In other cases, it mean just proofreading it, and in some cases, it was just inserting a couple of lines in some sections.
It was this boutique style that sets them apart. It has had a direct effect in making our study successfully navigate difficult timelines, and it has done wonders for building our reputation as a sponsor who wants to help.
Honestly, don’t look back. Grab up CBS. You won’t regret it.
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Raul P. Lima, MA, MBA
VP, Global Head of Clinical Operations
uniQure
