Reviewed competitive map

Every row here has been reviewed as CF.

This is the cleaned-up picture of companies and programs. Unreviewed trials never enter this page. Programs for a different lung disease (non-CF bronchiectasis, including ensifentrine and brensocatib) stay off.

Approach	Preclinical	Phase 1	Phase 2	Phase 3	Approved	Standard of care
Anion bypass		1
Anti-inflammatory			2
Antimicrobial		1			1
ASO / splicing			1
CFTR modulator		3	1		2
Gene editing	1
Gene therapy	1	3	1
Genetic therapy	1
GI / nutrition			2		1
CFRD / metabolic				1
mRNA	1	2	2
Mucociliary					1	1
Nonsense readthrough	1
Other			1
Phage		1	1

ApproachLeftover problem

54 programs in this list

Try leftover problem N5 (diabetes) then N1 (restoration). Reset to All before you leave. “Counts as competition” means the program is investigational, not already-approved standard care.

Program	Company	Approach	Leftover problem	Stage	Counts as competition
ABCI (amphotericin B cystetic for inhalation)	Cystetic Medicines	Anion bypass	N1 · Mutation-agnostic CFTR restoration	Phase 1	Yes
Anakinra	Heidelberg University	Anti-inflammatory	N3 · Residual lung disease	Phase 2	Yes
Losartan as adjunct to modulators	University of Kansas Medical Center	Anti-inflammatory	N3 · Residual lung disease	Phase 2	Yes
High-dose ibuprofen	generic	Anti-inflammatory	N3 · Residual lung disease	Standard of care	No
Cayston (aztreonam)	Gilead	Antimicrobial	N4 · Chronic infection	Approved	No
TOBI Podhaler	Novartis / later holders	Antimicrobial	N4 · Chronic infection	Approved	No
Inhaled tobramycin	multiple	Antimicrobial	N4 · Chronic infection	Approved	No
Intravenous gallium	—	Antimicrobial	N4 · Chronic infection	Phase 1	Yes
Intensive-phase NTM combination including bedaquiline	The University of Queensland	Antimicrobial	N4 · Chronic infection	Phase 2	No
Azithromycin	generic	Antimicrobial	N4 · Chronic infection	Standard of care	No
SPL84	SpliSense	ASO / splicing	N1 · Mutation-agnostic CFTR restoration	Phase 2	Yes
Alyftrek (vanzacaftor/tezacaftor/deutivacaftor)	Vertex Pharmaceuticals	CFTR modulator	N2 · Eligible but not prescribed	Approved	No
Kalydeco (ivacaftor)	Vertex Pharmaceuticals	CFTR modulator	N2 · Eligible but not prescribed	Approved	No
Orkambi (lumacaftor/ivacaftor)	Vertex Pharmaceuticals	CFTR modulator	N2 · Eligible but not prescribed	Approved	No
Symdeko (tezacaftor/ivacaftor)	Vertex Pharmaceuticals	CFTR modulator	N2 · Eligible but not prescribed	Approved	No
Trikafta (elexacaftor/tezacaftor/ivacaftor)	Vertex Pharmaceuticals	CFTR modulator	N2 · Eligible but not prescribed	Approved	No
SION-451	Sionna Therapeutics	CFTR modulator	N2 · Eligible but not prescribed	Phase 1	Yes
VX-581	Vertex Pharmaceuticals	CFTR modulator	N2 · Eligible but not prescribed	Phase 1	Yes
VX-272	Vertex Pharmaceuticals	CFTR modulator	N2 · Eligible but not prescribed	Phase 1	Yes
SION-719	Sionna Therapeutics	CFTR modulator	N2 · Eligible but not prescribed	Phase 2	Yes
Prime Medicine	Prime Medicine	Gene editing	N1 · Mutation-agnostic CFTR restoration	Preclinical	Yes
KB407	Krystal Biotech	Gene therapy	N1 · Mutation-agnostic CFTR restoration	Phase 1	Yes
SP-101	Spirovant Sciences	Gene therapy	N1 · Mutation-agnostic CFTR restoration	Phase 1	Yes
BI 3720931	Boehringer Ingelheim	Gene therapy	N1 · Mutation-agnostic CFTR restoration	Phase 1	Yes
4D-710	4D Molecular Therapeutics	Gene therapy	N1 · Mutation-agnostic CFTR restoration	Phase 2	Yes
Gensaic	Gensaic	Gene therapy	N1 · Mutation-agnostic CFTR restoration	Preclinical	Yes
ViaNautis Bio	ViaNautis Bio	Genetic therapy	N1 · Mutation-agnostic CFTR restoration	Preclinical	Yes
Pancrelipase enzyme products	multiple	GI / nutrition	N6 · GI and CF liver disease	Approved	No
RELiZORB	Alcresta	GI / nutrition	N6 · GI and CF liver disease	Approved	No
Linaclotide for CF GI disease	University of Nottingham	GI / nutrition	N6 · GI and CF liver disease	Phase 1	Yes
ANG003	—	GI / nutrition	N6 · GI and CF liver disease	Phase 2	Yes
Maralixibat for CF constipation	Children's Hospital Los Angeles	GI / nutrition	N6 · GI and CF liver disease	Phase 2	Yes
NHS7108	Aimmune Nestlé Health Science US R&D, LLC	GI / nutrition	N6 · GI and CF liver disease	Phase 2	Yes
Tenapanor for CF-related constipation	Massachusetts General Hospital	GI / nutrition	N6 · GI and CF liver disease	Phase 3	Yes
AquADEKs	—	GI / nutrition	N6 · GI and CF liver disease	Standard of care	No
Dorzagliatin in pancreatic-insufficient CF	Michael R. Rickels, MD, MS	CFRD / metabolic	N5 · CFRD / diabetes and metabolism	Phase 1	Yes
Metformin in CFRD	University of Kansas Medical Center	CFRD / metabolic	N5 · CFRD / diabetes and metabolism	Phase 2	Yes
Dulaglutide in CF-related glucose intolerance	University of Pennsylvania	CFRD / metabolic	N5 · CFRD / diabetes and metabolism	Phase 2	Yes
Empagliflozin in CFRD	University of Minnesota	CFRD / metabolic	N5 · CFRD / diabetes and metabolism	Phase 2	Yes
Verapamil in CF-related dysglycemia	Rhode Island Hospital	CFRD / metabolic	N5 · CFRD / diabetes and metabolism	Phase 2	Yes
Bionic Pancreas	—	CFRD / metabolic	N5 · CFRD / diabetes and metabolism	Phase 3	Yes
VX-522 mRNA	Vertex Pharmaceuticals	mRNA	N1 · Mutation-agnostic CFTR restoration	Phase 1	Yes
BMD003	Peking Union Medical College Hospital	mRNA	N1 · Mutation-agnostic CFTR restoration	Phase 1	Yes
ARCT-032 (LUNAR-CF)	Arcturus Therapeutics	mRNA	N1 · Mutation-agnostic CFTR restoration	Phase 2	Yes
RCT2100	ReCode Therapeutics	mRNA	N1 · Mutation-agnostic CFTR restoration	Phase 2	Yes
Nanite	Nanite	mRNA	N1 · Mutation-agnostic CFTR restoration	Preclinical	Yes
Bronchitol (inhaled mannitol)	Pharmaxis / Chiesi	Mucociliary	N3 · Residual lung disease	Approved	No
Pulmozyme (dornase alfa)	Genentech / Roche	Mucociliary	N3 · Residual lung disease	Approved	No
Hypertonic saline	multiple	Mucociliary	N3 · Residual lung disease	Standard of care	No
Southern Research nonsense program	Southern Research	Nonsense readthrough	N1 · Mutation-agnostic CFTR restoration	Preclinical	Yes
Tamoxifen in modulator-ineligible CF	Azienda Ospedaliera Universitaria Integrata Verona	Other	N1 · Mutation-agnostic CFTR restoration	Phase 2	Yes
AchromoPhage	Ghady Haidar	Phage	N4 · Chronic infection	Phase 1	Yes
IV bacteriophage cocktail plus IV antibiotics	University of California, San Diego	Phage	N4 · Chronic infection	Phase 1	Yes
BX004-A	BiomX	Phage	N4 · Chronic infection	Phase 2	Yes