A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/CAS9-Based Coagulation Factor IX Gene Insertion Therapy in Participants with Hemophilia B

Hemophilia B

Part 1: Incidence and severity of treatment-emergent adverse events (TEAEs) up to 104 weeks., Part 1: Coagulation Factor IX (FIX) functional activity at day 29 measured using the chromogenic substrate assay., Part 2A: Change from baseline in FIX functional activity in plasma at week 26 after REGN131-LNP1265 dosing at the recommended dose for expansion (RDE) measured using the chromogenic substrate assay., Part 2A: Annualized bleeding rate (ABR) over 52 weeks following sustained FIX functional activity (weeks 26-78 post-REGV131-LNP1265 dosing) among participants receiving RDE.

Part 1: Change from baseline in FIX functional activity in plasma at 26 weeks after REGV131-LNP1265 dosing at the RDE, measured using the chromogenic substrate assay., Part 1: ABR over 52 weeks following sustained FIX functional activity (weeks 26-78 post-REGV131-LNP1265 dosing) among participants receiving the RDE., Part 1 and Part 2A: FIX functional activity in plasma over time during the study period using the chromogenic substrate assay up to 104 weeks., Part 1 and Part 2A: Annualized treated bleeding rate (tABR) over 52 weeks following sustained FIX functional activity (weeks 26-78 post-REGV131-LNP1265 dosing) among participants receiving the RDE., Part 1 and Part 2A: Annualized utilization (IU/kg/year) of FIX replacement therapy over 52 weeks following sustained FIX functional activity (weeks 26-78 post-REGV131-LNP1265 dosing) among participants receiving the RDE., Part 1 and Part 2A: Remaining free of FIX replacement therapy among those receiving the RDE over 52 weeks following sustained FIX expression (weeks 26-78 post-REGV131-LNP1265 dosing)., Part 1 and Part 2A: Remaining zero spontaneous bleeding events among those receiving the RDE over the 52 weeks of sustained FIX functional activity period (weeks 26-78 post-REGV131-LNP1265 dosing)., Part 1 and Part 2A: Concentrations of REGV131 components up to 29 days., Part 1 and Part 2A: Concentrations of LNP1265 components up to 29 days., Part 1 and Part 2A: Detection of antibodies to the F9 transgene product FIX protein up to 104 weeks., Part 1 and Part 2A: Detection of total binding antibodies (TAbs) to the adeno-associated virus 8 (AAV8) capsid proteins up to 104 weeks., Part 1 and Part 2A: Detection of neutralizing antibodies/transduction inhibitors (NAb/TI) to the adeno-associated virus 8 (AAV8) capsid proteins up to 104 weeks., Part 1 and Part 2A: Detection of antibodies to LNP1265 up to 104 weeks., Part 1 and Part 2A: Detection of antibodies to CRISPR-associated protein 9 (Cas9) protein up to 104 weeks., Part 1: Detection of vector DNA in blood, saliva, nasal secretions, semen, urine, and feces over time (Part 1 dose confirmation cohort only), Part 2A: Incidence and severity of TEAEs up to 104 weeks., Part 2A: Detection of vector DNA in relevant matrices based on data analysis of Part 1 Dose Confirmation Cohort up to 104 weeks.

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