Who Owns the Cure? The Case for Patient Control in Rare Disease Drug Development
Key Takeaways:
Families funding their own rare disease therapies can lose access to the IP, manufacturing know-how, and clinical data they paid for when partners go bankrupt, get acquired, or deprioritize the program.
Four contractual moves — holding IP in the patient entity, signing MTAs early, contracting vendors fee-for-service with right of reference, and building reacquisition rights into every commercial license — keep families in control of the programs they fund.
Nome is built this way from the ground up: every program is structured so the family or foundation holds the IP from day zero, executed across a fee-for-service vendor network of more than 30 partners.
Families who fund their own therapies should hold the IP from day one. The path to do that all the way to market now exists.
A family raises money for their child's therapy. They sign a license with a biotech or fund an academic group to do the work. The biotech goes bankrupt, gets acquired, or deprioritizes the program. The academic group moves on to the next grant. Priorities change. The IP, the manufacturing know-how, the clinical data: all sit somewhere the family cannot reach.
Partnership is necessary in drug development. Done right, partnerships between academia, biotech, CROs, and patient organizations can work well, and that approach has already produced meaningful therapies. The common thread: patient organization control.
I have personally been on the family side of these contracts. I founded a 501(c)(3) to develop a gene therapy for my own disease. What follows is a practical guide, drawn from that experience, to the key moves families should make early to ensure the therapeutics they fund make it to the finish line.
The Practical Playbook
Four contractual moves keep families in control of the work they fund. Each one is simple. Each one is routinely skipped. Each one is the difference between a program you can move forward and a program stuck on someone else's shelf.
Move 1: Hold the IP in the patient entity's name
The foundation or patient entity owns the composition of matter, the method of use, the manufacturing know-how, and the clinical data. New IP generated during the program is assigned to the patient entity, not the vendor. The asset stays with the people who funded it, regardless of what happens to any single operator.
Red flag: a partner who insists on owning new IP generated during work you are paying for, without any access carve-outs for your use. This blocks your ability to receive the therapy you helped to fund.
Move 2: Sign Material Transfer Agreements as early as possible
A Material Transfer Agreement signed before any biological material moves governs who owns derivatives and inventions. Without one, the receiving lab's default IP policy often claims ownership. Further, if you need to transfer materials to a new vendor later in development, even when the existing partner is aligned on sharing, MTAs represent programs moving out of their lab — making the paperwork a low priority that can add months to a project timeline. Mitigate this risk when you have the most leverage, as you are starting the relationship with the partner.
Red flag: a partner who places restrictions on materials transfer.
Move 3: Contract vendors fee-for-service, with right of reference and escrow
Contract manufacturers, CROs, analytics providers, and regulatory consultants are paid for work done. The family owns the work product. Every vendor grants irrevocable right of reference to their master files, so the sponsor can switch operators without a regulatory restart. Master cell banks, clinical data, and FDA correspondence are dual-stored under the sponsor's name, with contractual return within thirty days of any termination.
Red flag: a vendor demanding royalties for routine work, or a manufacturer who will not provide right of reference.
Move 4: Build reacquisition rights into every commercial license
As programs progress, one way to continue funding them is to license the preclinical work to a biopharma company that will take the program forward. This can be a successful strategy, but it requires attention to detail. Any license to a commercial sponsor must include step-in rights, diligence obligations tied to specific development milestones, and automatic IP reversion if the sponsor deprioritizes or misses milestones. If the partner's priorities change, the program comes home.
Without these clauses, the foundation can watch its assets sit idle on a shelf indefinitely while the patients who funded it continue to wait.
Nome executes these moves — and more — to protect patient access to rare disease medicines.
Nome is the only company built this way from the ground up. We structure every program with the family or foundation holding the IP from day zero. We run fee-for-service contracts across a vendor network of more than 30 partners, negotiate MTAs as early as possible, and ensure patients keep access to the work they fund.
If you are a family, a foundation, or a clinician navigating a rare disease program and want to discuss how to structure it for long-term patient control, submit your case at nome.bio.
About Nome
Nome Therapeutics is the Operating System for Personalized Therapeutics, turning every rare disease patient's genome into an actionable treatment plan and helping execute on it at the lowest cost and highest speed in the industry.
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