Ultragenyx Rare Entrepeneur Bootcamp!
At the end of April this year, I had the amazing opportunity to attend the single best gathering to support parent advocates developing drugs for their children — the Ultragenyx Rare Entrepenuer Bootcamp.
This free 3-day comprehensive bootcamp is generously hosted by Ultragenyx, a biotech focused on Rare Disease and sponsored by Global Genes, BridgeBio Pharma, drug development companies and many others.
The gathering first launched in 2022 by Ultragenyx CEO Dr. Emil Kakkis. Dr. Kakkis and his team have for many years advised families on an ad hoc basis about how to successfully develop rare disease treatments. He wanted to make essential information, practical know-how and education available to patients, caregivers and advocates interested in developing their own rare disease treatments.
Here’s what he said in an Ultragenyx Press Release about why they did it:
“Our policy is if someone asks us for our help, we help them. Because we believe sharing our knowledge helps advance the entire field of rare disease medicine.” Conducting drug development without any previous biotech industry experience, as in the case of most parents of an affected child, can be a near impossible barrier to overcome in the rare disease space. Amen.
“We don’t want to hold our knowledge as proprietary,” said Dr. Kakkis, adding, “We alone cannot possibly develop all the rare disease treatments – we can however gain great satisfaction in helping others treat as many rare diseases as possible.”
The event was a true comprehensive bootcamp that covered the drug development process from beginning to end. Day 1 started with each of us giving a pitch on stage, during which a public speaking expert was on hand to give us candid feedback on everything from our message to our body language.
Even though I prepared my speech ahead of time, I broke down in tears as I spoke about Olive. I always do. I explained that there are enough rare disease patients in the US to fill every single NFL stadium at the same time. Yet 95% of these patients (the majority of which are children) have no treatment. I practiced making a fundraising ask towards our $400K gene therapy mouse model study to save my daughter and children like her from this devastating disease. I got great feedback from the group, and was encouraged to put Olive’s joyful face front and center in my asks.
The event was held at the Ultragenyx headquarters in Novato, California, and attended by about 40 patient advocates like me. These are parents who are dedicating their lives to save their children, alongside full time jobs, and caregiving for their sick kids. I got to meet so many people like me who get it, and some my favorite moments were when we got to all sit at a table and eat dinner together.
None of us ever thought we would be doing this, but here we are. Learning how to be scientists, drug developers, community organizers, and fundraisers.
We got to listen to sessions about everything from basic science, animal models, different therapeautic avenues like ASOs, gene therapies, CRISPR gene editing, and drug repurposing. We learned about what to look out for in contracting with academia and commercial partners, and how to make your self attractive to biotechs and VCs. We learned about natural history studies and how to gather patient data. Developing biomarkers and engaging the FDA. The amount of work that this effort takes is a monumental undertaking. And these are parents who are doing it.
We also heard stories from those who are ahead of us on this journey. My favorite speech was from Allison Berent of FAST, a veterinarian and mother to a daughter with Angelman syndrome, who has moved mountains and raised millions of dollars to push forward a series of research goals towards a cure. Her words were so encouraging. She told me that I would do this… and that she would help me in any way that she can.
Not only did we get to learn about all of these topics from experts, we also got to schedule office hours, and received the contact information from all of them, with generous offers to help us in any way they can after bootcamp. I finally got to meet several rare disease parents in person who I have met online through this journey, and although I drank from the firehouse and this is an incredibly difficult journey, I am so encouraged.
I truly can’t believe this event exists, and am so grateful for Ultragenyx for putting it together.
Onwards!