From Nonprofit to Neurotech Powerhouse
How Rafa’s Moonshot Is Rewiring Rare Disease Development.
Life Sciences Today This Week
My guest this week was Sagi Gidali from Rafa’s Moonshot.
Danny and Sagi discuss how Rafa’s Moonshot is turning a rare genetic disorder (STXBP1) into an investable, de‑risked therapeutic opportunity.
Sagi explains how his tech/entrepreneur background shapes a for‑profit mindset around fundraising, biopharma partnerships, and making rare disease indications attractive to industry. They cover building a global network, leveraging grants and consortia, working with academia/CROs, and planning the flip from nonprofit to for‑profit. They also dig into industry “anti‑patterns,” including copy‑paste development strategies, and explore a start from the drug label and work backwards approach.
Rafa’s Moonshot is building a full-stack engine to take an ultra‑rare genetic disorder, STXBP1, all the way from scattered case reports to real, clinic-ready therapies. They start by doing what traditional drug developers often ignore: systematically finding and organizing the global patient population (about 1,400 diagnosed so far), running natural history studies, and deeply understanding disease mechanisms.
On top of that data and insight, they assemble consortia of academic labs, CROs, and biotechs, then use philanthropic capital to unlock 3–4x leverage in non‑dilutive grants for their research partners. The long‑term play is to flip from a nonprofit hub into a for‑profit therapeutic company once there’s enough de‑risked biology, IP, and clinical direction.
Who needs what they’re building?
Biopharma and biotech looking for de‑risked, ready‑to‑develop rare disease programs.
Academic groups needing funding, infrastructure, and industry‑grade coordination.
Investors hunting for high‑impact, capital‑efficient CNS/neurogenetic plays.
Most importantly, families living with STXBP1 and similar CNS disorders, who need a path to actual treatments rather than yet another descriptive paper.
Sagi closes with his vision for a complete STXBP1 solution, a new CNS neurogenetic research center in Israel, and a stronger Israeli life sciences ecosystem.
You can see the episode here
In 2026, I’m helping 100 founders succeed more
I’m giving 100 founders access to my pattern library and a free strategic audit.
I’ll review your 2026 plans and tell you what breaks first. After our 60’ phone call, I’ll send you a written assessment and circle back with 30 and 90 day follow-up calls.
Get it here
About me
I’m an entrepreneur, writer, host of Life Sciences Today podcast, ex-pharmatech founder, father of 4.
I’ve been building in tech, cyber, privacy, and clinical data for 25+ years across Israeli medical device startups, Verily, Amgen, and the Fortune 1 company.
I work at the intersection of business, engineering, AI and clinical data.
If you love my writing — share it