A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)

NCTID	NCT06526923 (View at clinicaltrials.gov)
Description	This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to CFTR modulator therapy. (Show More)
Development Status	Active
Indication	Cystic Fibrosis
Disease Ontology Term	DOID:1485
Compound Name	SP-101
Compound Description	AAV2.5T-SP183-hCFTRΔR
Sponsor	Spirovant Sciences, Inc.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	15
Results Posted	Not Available

Target Gene/Variant	CFTRΔR
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Inhalational
Drug Product Type	Viral vector
Target Tissue/Cell	Human airway epithelia
Delivery System	Viral transduction
Vector Type	AAV2.5T
Editor Type	none
Dose 1	Undisclosed dose
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	First patient dosed November 2024