Investor Presentaiton
Appendix (Research and Development)
New Chemical Entity: SP-101
✓ Target indication: Cystic Fibrosis
✓ Origin: In-house (Spirovant Sciences, Inc.)
✓ Mechanism of action: CFTR gene transfer to lung
✓ Stage: Phase 1/2 in the U.S.
✓ Expected profile:
SP-101 is a novel adeno-associated viral (AAV) vector engineered to efficiently transduce
human airway epithelia from the apical (lumen) surface. It is designed to deliver a
shortened but fully functional cystic fibrosis transmembrane conductance regulator
(CFTR) gene to the airways of people living with Cystic Fibrosis (CF)
Based on preclinical data, the addition of doxorubicin substantially improves SP-101
transduction and subsequent expression of the CFTR gene. SP-101 followed by
doxorubicin administered via a nebulizer is being developed as a combination product for
the treatment of CF. SP-101 is expected to restore CFTR function and halting disease
progression in the lungs of people living with CF
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