Annette Bakker is no stranger to upending the status quo in rare disease drug development. The CEO of the nonprofit Children’s Tumor Foundation, Bakker once turned the tables for a drug that had been shelved by Pfizer for neurofibromatosis, a group of genetic conditions that cause tumors on nerves throughout the body. After seeing its potential, Bakker convinced Pfizer to give it another look, and the large pharma eventually spun out a company to develop it and other assets. The drug won approval in 2025.
Now, she’s leading the charge on a new strategy for dealmaking in rare disease R&D that involves an agreement giving CTF an equity interest in a preclinical drug program at GondolaBio, a clinical-stage biotech.
Instead of investing cash in GondolaBio’s preclinical neurofibromatosis type 1 program, CTF will provide what Bakker calls “sweat equity.” It will guide preclinical and clinical development using its deep knowledge of NF1, scientific and clinical expertise, and relationships across the NF community. In exchange, “CTF will receive an equity stake in the GondolaBio NF1 program,” Morgan Paull, COO of GondolaBio, told PharmaVoice via email.
Both organizations believe the deal is the first of its kind. They are not disclosing specific terms.
“CTF has already been a fantastic partner to GondolaBio. The infrastructure for NF drug development that they have built facilitates better and faster drug development in NF,” Paull said. “GondolaBio will continue to benefit from these efforts, but ultimately we are most excited to recognize and support the work CTF is doing on behalf of the patients we both seek to serve.”
Rethinking ‘venture philanthropy’
It’s not uncommon for patient advocacy groups to gain a financial stake in drug development.
In 2000, the Cystic Fibrosis Foundation pioneered “venture philanthropy,” investing $40 million in Aurora Biosciences, which Vertex Pharmaceuticals later acquired. Rather than acting as a passive donor, the foundation supported research that ultimately produced approved cystic fibrosis therapies. In 2014, it sold related royalty rights for $3.3 billion.
Since then, more patient advocacy groups have followed in the Cystic Fibrosis Foundation’s footsteps. Among them are the Rett Syndrome Research Trust, which backed biotechs like ProQR Therapeutics and Reverta, and the Foundation for Prader-Willi Research, which recently added ConSynance Therapeutics to its venture philanthropy portfolio.
But not every patient advocacy group is in a financial position to make a $40 million gamble, and even if they were, the odds of later scoring a windfall payout are incredibly small, Bakker said.
“Instead of us being an investor, which I don't think we're big enough to be, let us be your tool shop.”
Annette Bakker
CEO, Children’s Tumor Foundation
“In 90% of the cases, it's not going to hit, and the investment that you have to make … is really massive. A foundation of our size is not able to do that,” she said. “Instead of us being an investor, which I don't think we're big enough to be, let us be your tool shop.”
Rather than building patient relationships and a drug development infrastructure from scratch, drug developers can tap existing resources within patient advocacy groups. That’s especially important in rare disease research, where patient recruitment is a persistent challenge. A 2019 analysis of 659 rare-disease trials found that 30.2% were discontinued, with inadequate recruitment accounting for about a third of those discontinuations.
“You have to know the community,” she said.
What patient groups bring to the table
Patient advocacy groups already invest much more than money to get drugs onto the market.
For instance, Bakker said CTF can quickly and efficiently find and test preclinical models, pre-negotiate agreements with academic institutions and CROs, recruit patients and develop platform basket trials, shaving time and money off of the drug development process.
“The clinical and scientific infrastructure that CTF has built over the past 45 years has already made it possible to advance better and faster potential treatments, and it will continue to do so,” Paull said.
But philanthropy is “incredibly unpredictable” and “R&D cannot run on unpredictable income,” Bakker said.
“If we want to be able to keep doing that there has to be some share in the financials,” she said.
While Bakker admits that gaining an equity stake without a financial investment is a “hard sell” for most companies, she said the GondolaBio deal represents a biotech “finally” recognizing the value smaller patient advocacy groups like CTF add to the R&D ecosystem.
Bakker believes this kind of equity agreement is a “big shift” in the way companies and patient advocacy organizations can work together.
“I think that is what makes me so happy about this. [It] was always my dream that one day, companies would start valuing us. Valuing in the sense of not just paying [for] a service but actually letting us in, at their table, with the other investors, as a sweat equity investor,” she said.
