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His deep immersion in the literature and wide network provided many leads for investigation, and at that point the stories often took a twist, as I recount below…. Gaucher disease was our second program.
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Chris found our candidate in a Swedish research program that was winding down. We had to move quickly, jumping on a plane and wiring them money to thaw mouse embryos and start breeding Gaucher mice again.
We wanted to jump-start the experiments while we were still negotiating contracts! Then disaster struck — the postdoc who had done the proof of concept PoC work left the project for another role.
Suddenly, we were recruiters, headhunting the missing researcher and convincing her to come back to her own research project. After some negotiation, and some complex international contracting, she agreed to return and brought the project back to life. We breathed a sigh of relief. But the relief was short-lived. As it turns out, professors rather than their institution in Sweden own their IP, which makes negotiating very complicated, so again our young program seemed in peril.
We needed to get creative and fast with both American and Swedish lawyers to negotiate a technology license that worked for both parties. We wore many hats in a short time. In fact, that whole program was a wonderful example of how the pursuit of new medicines requires the ability to rapidly become a temporary expert on many topics.
https://cuhealhuovern.ga We continued to expand our lysosomal storage disease focus, looking next at Pompe disease. Treating this disease is a hard nut to crack as the enzyme is needed in difficult-to-reach target organs, including brain and muscle. Chris figured out a possible solution, namely, that using a GILT tag could help target our gene therapy to these cells and organs in a way that might deliver enhanced outcomes over competing AAV approaches.
We were onto something special. So I made the call, and a few months later, even though our initial internal champion at BioMarin had left, the deal got done.
After that adventure, we embarked on a global effort to find our next candidate, for cystinosis. We sent a scout to scientific conferences in Europe and the U.
We certainly found them, though some were wrapped up and stored in research lab attics and basements. We were really excited to find a cystinosis gene therapy program close to home, led by Dr. Stephanie Cherqui, an associate professor at University of California, San Diego and a champion for patients with cystinosis. Initially, Dr.
A couple of months later, we tried again with a different team. Again, she politely took the meeting… and this time, we focused on discussing our common goal: getting this potentially life-saving treatment to patients as quickly as possible. We brought a Gantt chart demonstrating the value of partnering from an early stage: we proposed that Dr.
Our shared commitment to getting this therapy to patients was the starting point needed to cement a a strong partnership with Dr.