Healthcare venture research · snapshot
Where unmet need remains in cystic fibrosis after highly effective modulators.
This is a public-data research memo with a website on top. It is not a company pitch, not medical advice, and not a recommendation to buy or sell anything. Data as of the Cystic Fibrosis Foundation 2024 Patient Registry and a ClinicalTrials.gov download from 9 Sep 2026.
Cystic fibrosis is a genetic disease. For most people with the right gene changes, a class of pills called CFTR modulators (Trikafta, and later Alyftrek) changed survival. This project asks what is still unsolved, where companies are already working, and which leftovers are worth a closer look.
The first three numbers are published facts. The 77 is a score this project computed. Registry source: CFF Patient Registry 2024 Annual Data Report.
The question
Where do meaningful unmet needs remain, how well is the current therapeutic and clinical-development ecosystem addressing them, and which areas appear most promising for future innovation or company formation?
The pages follow that order: Landscape shows leftover disease, Map shows which leftovers already have companies, and Opportunities ranks the gaps and attaches a thesis you can kill.
FACT
A published number with a source and a date. You can check it.
ANALYSIS
A filter or count this project computed, such as the reviewed CF map.
HYPOTHESIS
A score or thesis. Always shown with evidence and a way to kill it.
Words used on this site
- Modulator / HEMT
- Pills that help some broken CFTR proteins work. Highly effective modulator therapy usually means Trikafta and now Alyftrek.
- CFF Registry
- Yearly US report from accredited cystic fibrosis care centers. It covers people who consented, not every person with CF on earth.
- TAM
- Total addressable market — a commercial sizing number. 33,989 is a headcount, not a TAM.
- Crowding / whitespace
- Crowding means companies are already working on this problem. Whitespace means few or none. Empty is interesting. Empty is not proof a new company should exist.
- Need (N1–N8)
- Eight leftover problem buckets this project scored. N5 is CF-related diabetes. N1 is mutation-agnostic CFTR restoration.
- Thesis (H1–H7)
- A specific hypothesis hanging off a need. Each one has a bear case and kill criteria. The score is inherited from the parent need.
- Kill criteria
- A pre-committed rule: if this happens, drop the idea. Without it, a score is just a cheerleading number.
- NCFB
- Non-CF bronchiectasis — a different lung disease that shows up in a raw “cystic fibrosis” trial search. Those programs stay off the map.
Start here
You can jump, or use the Next button at the bottom of every page to read in order.