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相关临床试验
12
3 进行中
药物批准
0
批准总数
监管机构
监管机构数
成立时间
N/A
进行中(未招募)
2
16.7%
已完成
5
41.7%
尚未招募
1
8.3%
招募中
4
33.3%
暂无批准数据
- Opus Genetics' investigational gene therapy OPGx-LCA5 is helping individuals born blind regain partial sight in an ongoing Phase 1/2 clinical trial. - The therapy targets mutations in the LCA5 gene by delivering a functional copy directly to retinal cells to restore photoreceptor function. - Patient Lindsey Rambo, the second participant in the trial, shared her experience on Good Morning America, highlighting the life-changing potential of the treatment. - The company is advancing multiple gene therapy programs for inherited retinal diseases, including trials for both LCA5 and BEST1 mutations.