This week, our Chief Executive Bob Stevens and RDRP colleague Bennie Marshall-Andrews have been on a vital whirlwind tour of some of our pharma partners in the USA. Bob shares some important updates from the trip.
Denali
We recently visited our great partners Denali who have done the most amazing science and have developed blood-brain crossing therapies.
If trials are successful, and I believe they will be, it will represent a big leap forward in treating rare genetic conditions that not only affect the body but also the brain.
What is equally impressive, they have built their business around advocacy and really listen to the patient’s needs. Ryan and his team are doing something magical in my eyes and Kim Ramsey is just the most fabulous Senior Director of Patient Advocacy who works tirelessly with her team to do the very best for the global patient community. I am honoured to be considered a honoury Denalian.
Biomarin
Under Alexander's leadership, Biomarin has a renewed focus that is impressive to say the least.
Together with his whole staff team they are laser focused on bringing new treatments to our communities.
It was inspiring for me to see Biomarin back on its way to its best and I have to give a big shout out to the lab staff who are so enthusiastic about the challenges and opportunities ahead.
I would gladly spend a whole week quizzing and learning from their unique perspectives. Part of the MPS Society's success was built with Biomarin as collaborative partners and today that partnership has been renewed. Thank you Alexander and Team Biomarin, you are indeed back!
Alexander Hardy, President and Chief Executive Officer of Biomarin, reflects on the visit:
It was really meaningful to speak with Bob about the challenges and opportunities facing people living with these rare genetic conditions.
We bonded over our beliefs that in our professional lives, patients should always come first, but that our most important roles in life are as fathers – in Bob’s case, as a rare dad.
Regenxbio
Bennie and I had the opportunity at last to meet their CEO Curran Simpson face to face rather than via a computer screen and we had a great conversation about the regulatory environment for clinical trials. We then had a lab tour, which is always impressive.
It felt like being in a science fiction film with all the amazing high-tech they have.
Finally, we got to spend time with the wider team and the photo is with two of my favourite people in the world. Thanks, Vivian and Jen, for arranging all of this, I will be back in the near future.
