Intellia Therapeutics Presents Data On Redosing With In Vivo CRISPR Therapy; 55 Mg NTLA-2001 Dose Shows 90% Median Reduction In Serum TTR At Day 28; Well Tolerated Across All Patients; Potential Redosing Advantage For Future Therapies
Intellia Therapeutics Presents Data On Redosing With In Vivo CRISPR Therapy; 55 Mg NTLA-2001 Dose Shows 90% Median Reduction In Serum TTR At Day 28; Well Tolerated Across All Patients; Potential Redosing Advantage For Future Therapies
intellia therapeutics展示了关于体内CRISPR治疗再剂量的数据；55毫克NTLA-2001剂量显示在第28天时血清TTR中位数减少了90％；所有患者都能良好耐受；为未来治疗提供潜在再剂量优势。
- First-ever clinical data demonstrating redosing with an investigational in vivo CRISPR-based therapy
- Follow-on dosing with a 55 mg dose of NTLA-2001 led to a 90% median reduction in serum TTR at day 28 in the three patients who previously received the lowest dose in the Phase 1 dose-escalation study
- 55 mg follow-on dose was well tolerated across all patients
- While redosing is not planned for the NTLA-2001 program in transthyretin (ATTR) amyloidosis, a redosing option could be an important advantage of Intellia's non-viral, lipid nanoparticle (LNP)-based delivery platform for future investigational therapies where a target additive effect is desired
- 首次临床数据证明，在体内CRISPR基因编辑疗法的治疗方案中再次施用的有效性
- 在第一阶段剂量递增研究中，使用55毫克的Ntla-2001剂量进行随访治疗，三名之前接受最低剂量的患者在第28天时血清TTR减少了90％
- 所有患者均能很好地耐受接受55毫克的随访剂量。
- 虽然在转甲状腺素运输蛋白（ATTR）淀粉样变（amyloidosis）中，NTLA-2001计划中并不打算再次给药，但再次施用选项可能是Intellia非病毒、脂质纳米颗粒（LNP）为基础的递送平台在未来研究中寻求目标效应时的重要优势。