IDefine attends Ultragenyx Rare Entrepreneur Bootcamp

Ultragenyx, a biopharmaceutical company involved in the development of novel products for treatment of rare genetic diseases, has been advising rare disease non-profits on an ad hoc basis for years.

However, in 2017, Ultragenyx decided to develop a more formal curriculum on the rare drug development process, and the “Rare Bootcamp” was born. Since then, more than 65 patient and family organizations have participated in the Bootcamp event series.

To ensure that the bootcamps are personal and impactful, only a small number of organizations that have typically already started their drug development journey are invited to attend.

IDefine was invited and represented at the two-and-a-half-day event in Novato, Calif., by Geoff Rhyne, CEO, and Eric Scheeff, chief scientific officer (CSO).

“It’s an honor to be invited because it means that IDefine is viewed as ready enough as an organization to really hear this message,” said Scheeff. “It was exciting to get the invitation and a golden opportunity that we couldn’t miss. My expectations were high, and they were massively exceeded.”

The curriculum

Expert speakers from Ultragenyx and other companies covered topics such as clinical trial design, collaboration with academia, treatment approaches, manufacturing, intellectual property considerations, licensing, and presentation skills training.

“A really great part of the conference was honing your elevator pitch, really getting good at telling your story in two minutes or less, and then being able to tailor that story, depending on the particular person you’re talking to,” said Scheeff. “As Geoff and I refined ours what we kept finding was that as we took out material, it got better. They also did coaching on the actual presentation itself, that is standing in front of a group of people and using the right body language to better deliver your message.”

IDefine’s elevator pitch focuses on three things:

• We know the gene that causes Kleefstra Syndrome.
• We know what is wrong with that gene in KS.
• And there are new technologies coming out that allow you to modulate genes in ways that weren’t possible in the past, and that gives us a path to a potential treatment.

The impact

Scheeff says the entire Bootcamp was great, but a couple of presentations were particularly impactful.

Emil Kakkis, CEO of Ultragenyx, opened the event by sharing his personal journey from academic researcher, to developing the first ever treatment for the rare disease MPS I, to CEO of Ultragenyx and developing drugs for additional rare diseases.

Allyson Berent, CSO for the Foundation for Angelman Syndrome Therapeutics (FAST), also shared her story.

“Her presentation was very impactful because she went all the way from, ‘I got my child’s diagnosis, to I joined up with FAST, to we are now in a clinical trial.’” “She has walked the entire path that we are now on, and she shared lessons learned at each step.”

Call to action 

Kakkis and several other speakers stressed that patient organizations like IDefine must lead the push to a treatment, because drug companies and other outside entities have a lot of competing pressures. And while patient organizations need to take the lead for a treatment, individual members of those communities also need to contribute by signing up for things like AllStripes, Rare-X and the Kleefstra Syndrome World Map.

“When we go talk to drug developers or researchers, they ask how many patients are there? Do you know where they are? How old are they? They have to know that the patients are available and this is viable for them.”

Rhyne said the Bootcamp was a gamechanger for IDefine.

“Eric and I both have been saying repeatedly there was before the bootcamp and after the bootcamp. We have a different understanding now of what we need to do as an organization. It was truly transformative to attend. We are so thankful to Yael Weiss, the CEO of Mahzi Therapeutics and former vice president of business development at Ultragenyx, who has given us a lot of really helpful advice and invited us to the Rare Bootcamp.”