相关临床试验
7
2 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
2016
进行中（未招募）
2
28.6%
招募中
4
57.1%
Unknown
1
14.3%
暂无批准数据
- SparingVision successfully completed dosing in its Phase I/II PRODYGY trial with 33 patients enrolled, including 27 patients receiving SPVN06 and six controls, with initial data readout expected in 2027. - SPVN06 represents a novel gene-agnostic approach using AAV-based gene therapy to protect cone photoreceptors responsible for visual acuity and color vision in retinitis pigmentosa patients. - All patients dosed with SPVN06 continue to demonstrate a favorable safety profile, reinforcing confidence in this neuroprotective therapy that could benefit patients regardless of genetic mutation. - The company plans to share data with regulators in 2026 and aims to initiate a pivotal trial in 2027, leveraging AI-powered machine learning tools to analyze efficacy signals.
- SparingVision has dosed the first patient in the NYRVANA clinical trial, marking the first-in-human study of SPVN20 for advanced retinitis pigmentosa. - SPVN20 is a gene-agnostic AAV-based gene therapy that delivers a GIRK channel gene to reactivate dormant cone photoreceptors through intravitreal injection. - The open-label, dose-escalation study will evaluate safety, tolerability, and preliminary efficacy over 6 months with 5-year follow-up across Belgium, France, and Ireland. - The company plans to explore combining SPVN20 with its other lead product SPVN06 as SPVN30 to address a broader population of retinitis pigmentosa patients.
- Abeona Therapeutics has appointed James A. Gow, MD, MBA, MS, MHCM, as Senior Vice President, Head of Clinical Development & Medical Affairs, effective immediately. - Dr. Gow brings over 20 years of industry experience in clinical development and medical affairs, with recognized expertise in gene therapy, particularly in ophthalmology. - His clinical development track record includes leading programs from Phase 1 through post-marketing studies that resulted in FDA approvals of multiple drugs including Xibrom, Bromday, Prolensa, and Bepreve. - The appointment strengthens Abeona's leadership as the company advances its pipeline of cell and gene therapies, including ZEVASKYN for recessive dystrophic epidermolysis bullosa and AAV-based gene therapies for ophthalmic diseases.
- Beacon Therapeutics has appointed Dr. Daniel Chung as Chief Medical Officer, bringing over three decades of experience in clinical ophthalmology and gene therapy development. - Dr. Chung previously played an instrumental role in developing Luxturna, the first FDA and EMA-approved gene therapy for a blinding genetic disease. - The appointment comes as Beacon approaches completion of enrollment for its registrational Phase 2/3 VISTA trial of laru-zova for X-linked retinitis pigmentosa. - Beacon recently announced positive six-month interim safety and efficacy results from the Phase 2 DAWN trial of laru-zova in patients with X-linked retinitis pigmentosa.
