相关临床试验
173
16 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）
6
3.5%
已完成
111
64.2%
尚未招募
10
5.8%
招募中
20
11.6%
暂停
1
0.6%
Unknown
25
14.4%
暂无批准数据
- A 38-year-old Italian man with Usher syndrome type 1b became the first patient worldwide to regain sight through an experimental dual-vector gene therapy targeting the MYO7A gene. - The innovative treatment splits the large MYO7A gene across two viral vectors, allowing retinal cells to reassemble and produce the missing protein essential for vision. - Eight patients have undergone the procedure with no serious adverse events, showing rapid improvement within two weeks and sustained vision restoration after one year. - The therapy addresses a previously untreatable genetic eye disorder and offers potential hope for other inherited retinal diseases currently lacking treatment options.
- The European Medicines Agency has provided positive scientific advice on Variant's VAR002, an adeno-associated viral vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations. - VAR002's non-clinical development plan was deemed suitable for advancing to first-in-human clinical trials, with the EMA confirming that planned pivotal toxicology studies align with requirements for future EU marketing authorization. - The gene replacement therapy shows potential beyond CRX-related disorders, with preclinical studies demonstrating efficacy in diseases associated with phototransduction pathway defects and ciliopathies, including CEP290-associated conditions.
