相关临床试验
125
29 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）
3
2.4%
已完成
23
18.4%
Enrolling By Invitation
3
2.4%
尚未招募
23
18.4%
招募中
50
40.0%
Unknown
22
17.6%
撤回
1
0.8%
暂无批准数据
- GenEditBio has dosed the first patient in a groundbreaking investigator-initiated trial of GEB-101, marking the world's first clinical study of an in vivo CRISPR-Cas ribonucleoprotein-based genome editing therapy for TGFBI corneal dystrophy. - The open-label, dose-escalation study is investigating GEB-101's tolerability when combined with standard phototherapeutic keratectomy in adults with corneal dystrophy, with the first patient discharged without observable adverse events. - GEB-101 represents a potential once-and-done treatment using CRISPR-Cas technology delivered via GenEditBio's proprietary protein delivery vehicle, targeting mutations in the TGFBI gene that cause abnormal protein buildup in the corneal stroma. - The therapy demonstrated high safety profiles with virtually undetectable off-target effects in non-human primate studies, earning recognition with an Excellence in Research Award at the American Society of Gene and Cell Therapy meeting.
- Five children with DFNB9 inherited deafness gained hearing in both ears after receiving bilateral gene therapy targeting OTOF gene mutations in a world-first clinical trial. - The children demonstrated dramatic improvements in speech perception, sound localization, and ability to appreciate music within weeks of treatment. - This bilateral approach represents a significant advancement over previous single-ear treatments, enabling three-dimensional hearing crucial for communication and daily activities. - The therapy uses adeno-associated virus to deliver functional OTOF gene copies to restore otoferlin protein production in the inner ear.
