A Two-Part Open-Label Study of REGV131-LNP1265, a CRISPR/Cas9-Based Coagulation Factor IX Gene Insertion Therapy in Participants with Hemophilia B (BEYOND-9)

Research summary

This is a phase 1/2 open-label study of REGV131-LNP1265 in participants with severe and moderately severe congenital haemophilia B. The study consists of a pre-screening period, a screening period, a lead-in period, and a treatment and follow-up period. This study has been designed to identify a recommended dose for expansion (RDE) of a single IV administration of REGV131-LNP1265, with the aim to demonstrate safety and tolerability and achievement of sustained normal or near normal levels of plasma FIX functional activity. The study will be conducted in 2 parts: 1. Part 1: Dose Escalation and Dose Confirmation in participants ≥ 18 years of age. • Dose escalation cohorts to determine the RDE of REGV131-LNP1265. • Dose confirmation cohort to gain further confidence in safety, tolerability, and FIX functional activity data at the RDE. 2. Part 2A: Dose Expansion at the RDE in participants ≥ 18 years of age.

Principal Investigator

Dalia Khan

Contact us

Email: christopher.deane@ouh.nhs.uk

IRAS number

1009750