相关临床试验
11
5 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
N/A
进行中（未招募）
5
45.5%
已完成
5
45.5%
招募中
1
9.1%
暂无批准数据
- eXmoor Pharma has established two strategic partnerships to create integrated pathways for cell and gene therapy development and manufacturing in the UK. - The collaboration with Royal Free London NHS Foundation Trust connects early-phase GMP capabilities with later-stage clinical manufacturing expertise to accelerate therapy development timelines. - A separate partnership with Anthony Nolan Cell Therapy & Laboratory Services provides end-to-end services from donor sourcing through clinical supply, leveraging a donor register of over 900,000 HLA-typed individuals. - These partnerships aim to reduce development complexity and strengthen the UK's position as a global leader in advanced therapeutics manufacturing.
- Updated Phase 1/2 STAAR study data demonstrates sustained α-Gal A enzyme activity for up to 47 months, with all 18 ERT-dependent patients successfully discontinuing enzyme replacement therapy. - Patients showed significant kidney function improvements with a positive mean annualized eGFR slope of 3.061 mL/min/1.73m2/year, indicating potential disease-modifying effects. - Sangamo plans BLA submission in second half of 2025 following FDA's agreement to consider Accelerated Approval pathway based on 52-week eGFR data.
