相关临床试验
30
21 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
2012
进行中（未招募）
20
66.7%
已完成
6
20.0%
No Longer Available
1
3.3%
尚未招募
1
3.3%
招募中
2
6.7%
暂无批准数据
- GenSight Biologics confirmed the viral genome titer of the GS010/LUMEVOQ drug substance was within target specifications, indicating a repeatable manufacturing process transfer to Catalent. - All remaining patients for the dose-ranging REVISE study in France have been identified, with the last treatment scheduled for December 2026. - Named patient early access treatments have resumed in France, with the first early access treatment in Israel performed in July 2026. - GenSight Biologics will publish its 2026 half-year financial statement after market close on September 29, 2026.
- Nitza Thomasson joined Servier in March 2025 as Executive Director and Global Head of R&D Neurology to build the company's rare neurology therapeutic area from scratch. - Servier currently has three neurology drugs in its pipeline, targeting rare refractory epilepsies, movement disorders, neuromuscular disorders, and other conditions. - The company recently strengthened its portfolio through the acquisition of Edgewise Therapeutics' neuromuscular dystrophy pipeline. - Thomasson emphasizes that success in rare neurology requires embracing scientific uncertainty, adapting approaches to each disease, and building a team culture grounded in trust and high expectations.
- GenSight Biologics completed a €2.9 million fundraising round subscribed by Heights Capital, Invus, and Alumni Capital, following €10.2 million raised earlier in 2025. - The funds will primarily support continued development of GS010/LUMEVOQ®, the company's lead gene therapy candidate for Leber Hereditary Optic Neuropathy currently in Phase III trials. - The company secured compassionate use approvals in the United States, France, and Israel in 2025, representing significant regulatory milestones for the retinal gene therapy program. - GenSight expects revenues from compassionate access programs to ensure operational continuity through 2026, while continuing to pursue additional funding for the RECOVER Phase III trial.
- GenSight Biologics received French regulatory approval for the REVISE dose-ranging study investigating GS010/LUMEVOQ gene therapy for ND4-LHON, a rare mitochondrial disease causing irreversible vision loss. - The French medicines agency ANSM granted compassionate use authorization (AAC) for GS010/LUMEVOQ, enabling named patient early access for eligible patients with serious unmet medical needs. - The Phase II REVISE study will enroll 14 patients and investigate two clinical doses, with the primary endpoint measuring visual acuity changes 1.5 years post-treatment versus baseline.
- GenSight Biologics received FDA authorization and IRB approval for expanded access treatment of one patient with its gene therapy GS010/LUMEVOQ® at the University of Pittsburgh School of Medicine in November 2025. - The authorization represents the first regulatory approval for the candidate since the company withdrew its European marketing authorization application in 2023. - GS010/LUMEVOQ® targets Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial genetic disease that causes irreversible vision loss and legal blindness in teens and young adults. - The company is preparing for a pivotal Phase III study RECOVER in H2 2026 while completing technology transfer to manufacturing partner Catalent to produce new batches for clinical and early access needs.
- The gene therapy market for rare diseases has reached $11.4 billion in 2025 and is projected to grow at 20% CAGR to $58.87 billion by 2034, driven by strategic partnerships between biotechs and CDMOs. - Contract Development and Manufacturing Organizations now capture over 87.8% of the cell and gene therapy manufacturing market share, with companies like Viralgen and Catalent enabling scalable production for ultra-rare disease treatments. - Patient-driven initiatives and caregiver-led collaborations are accelerating development timelines from years to six months through platform-based approaches and shared resources. - Small biotechnology companies leverage their agility to advance rare disease treatments, with 80% of rare diseases being genetic in nature, benefiting from recent advances in genome sequencing.
- NICE has recommended idebenone (Raxone) as the first NHS treatment for Leber's hereditary optic neuropathy (LHON) in England, benefiting an estimated 250 eligible patients aged 12 and over. - The approval ends a disparity in UK access, as the drug has been available in Scotland, Wales, and Northern Ireland for several years, with NHS England set to provide treatment within three months. - Clinical evidence from RHODOS, LEROS, and PAROS trials demonstrates that idebenone can improve vision and reduce eyesight deterioration by restoring cellular energy production in affected eye cells. - LHON primarily affects young men and boys, causing rapid progression from painless central vision blurring to potential blindness in both eyes within months, with devastating impacts on independence and employment prospects.
- A new exploratory analysis published in Investigational Ophthalmology and Visual Science identifies key predictive factors for improved visual outcomes in LHON patients treated with GenSight's gene therapy LUMEVOQ®. - Better baseline visual acuity and thicker retinal nerve fiber layer and ganglion cell layer measurements predict superior visual outcomes 1.5 years after treatment. - Patients treated during the dynamic phase of disease achieve better final visual outcomes than those treated during the subacute phase, challenging conventional treatment timing wisdom. - The study analyzed 113 eyes from three Phase III trials (RESCUE, REVERSE, and REFLECT) and supports bilateral treatment recommendations for LHON patients.
- GenSight Biologics has successfully transferred the upstream manufacturing process for LUMEVOQ, its gene therapy for Leber Hereditary Optic Neuropathy (LHON), to manufacturing partner Catalent Inc. - The partnership is expected to improve yield and upgrade analytical methods ahead of clinical use and regulatory submissions for the rare mitochondrial disease treatment. - Catalent manufactured a drug product batch released as safe for human use in November 2024, which will supply France's named patient early access program and dose-ranging study. - The collaboration positions GenSight for its planned global Phase III RECOVER trial beginning in 2026 and upcoming regulatory submissions to FDA and EMA.
- GenSight Biologics reached agreement with French regulatory agency ANSM to expedite opening of early access program for LUMEVOQ gene therapy following approval of a dose-ranging study. - The company targets Q3 2025 for clinical trial application submission and Q4 2025 for program launch, providing treatment access for LHON patients in France. - GenSight plans global Phase III trial initiation in early H2 2026 while implementing financing strategy to bridge operations through regulatory milestones. - The agreement represents a critical pathway for patients with Leber Hereditary Optic Neuropathy, a rare mitochondrial disease causing irreversible blindness in young adults.
