Be Biopharma Initiates First-in-Human Trial of CRISPR-Based Gene Therapy for Haemophilia B
核心洞察
Be Biopharma (搜索) has dosed the first patient in the BeCoMe-9 Phase I/II trial of BE-101, marking the first clinical application of B Cell Medicines technology for haemophilia B (搜索) treatment.
BE-101 uses CRISPR/Cas9 gene editing to insert the human Factor IX gene into patients' own B cells, designed to continuously secrete Factor IX without requiring preconditioning or immunosuppression.
The two-part trial will enroll up to 24 subjects with moderately severe to severe haemophilia B (搜索), with participants monitored for 52 weeks to evaluate safety and clinical activity.
Be Biopharma (搜索) has achieved a significant milestone in gene therapy development by dosing the first patient in its BeCoMe-9 Phase I/II trial of BE-101, a novel CRISPR-based treatment for haemophilia B (搜索). This marks the first clinical application of B Cell Medicines (BCMs) technology, representing a potentially transformative approach to treating this rare bleeding disorder.
Novel CRISPR-Based Approach
BE-101 represents a groundbreaking therapeutic strategy that uses CRISPR/Cas9 gene editing to precisely insert the human Factor IX (FIX (搜索)) gene into patients' own B cells. Once infused, the engineered cells are designed to engraft and continuously secrete Factor IX into circulation to restore clinically meaningful levels of active FIX. Notably, the therapy is administered without the need for preconditioning or immunosuppression, distinguishing it from other gene therapy approaches.
The autologous nature of BE-101 means it utilizes each patient's own cells, which undergo leukapheresis collection to support manufacturing before administration. According to Be Biopharma (搜索), the therapy is designed to be re-dosable and titratable, offering flexibility in treatment management.
Trial Design and Objectives
The BeCoMe-9 study is structured as a two-part, first-in-human, multi-center, open-label, dose-escalation trial targeting adults with moderately severe or severe haemophilia B (搜索). Part I employs an ascending-dose design to evaluate increasing doses in a stepwise manner, with the primary objective of identifying the dose required to achieve desired FIX (搜索) activity levels 28 days post-infusion.
Part II will expand on these findings through an expansion phase that will include up to six adult participants to further characterize the safety and activity of BE-101 at the selected dose. The trial plans to enroll up to 24 subjects total, with 18 participants in Part I and six in Part II. Additional cohorts for adolescents and redosing opportunities for Part I participants will be considered following data availability.
Clinical Monitoring and Regulatory Status
Participants will undergo comprehensive monitoring for clinical activity and safety over approximately 52 weeks following BE-101 administration. This extended follow-up period will provide crucial data on the therapy's durability and long-term safety profile.
BE-101 has secured both fast-track and orphan drug designations from the US Food and Drug Administration (搜索), reflecting the significant unmet medical need in haemophilia B (搜索) treatment and the therapy's potential to address this gap.
Leadership Perspective
"Dosing the first participant in our first-in-human study of BE-101 marks a significant milestone for Be Bio and for people with haemophilia B (搜索)," said Joanne Smith-Farrell, CEO and president of Be Biopharma (搜索). "BE-101 is our first BCM to enter the clinic, and it holds the promise of transforming the treatment landscape for haemophilia B while demonstrating the power of BCMs to deliver groundbreaking in vivo biologics."
The initiation of this trial represents a critical step forward in applying CRISPR technology to rare disease treatment, potentially offering a new therapeutic paradigm for patients with haemophilia B (搜索) who currently rely on regular Factor IX replacement therapy or other treatment modalities.