相关临床试验
5
1 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
2008
进行中（未招募）
1
20.0%
已完成
3
60.0%
招募中
1
20.0%
暂无批准数据
- Evox Therapeutics has initiated a collaboration with the Rett Syndrome Research Trust to evaluate its ExoEdit exosome-based delivery platform for developing gene editing treatments for Rett syndrome. - The partnership aims to address the critical challenge of safe and efficient gene editing delivery to the central nervous system for treating this severe neurodevelopmental disorder. - Over 90% of Rett syndrome cases are caused by mutations in the MECP2 gene, making it a prime target for gene editing approaches that could potentially reverse symptoms even after disease onset. - If successful, the collaboration could establish the foundation for an expanded preclinical program targeting the majority of Rett syndrome patients whose disease stems from MECP2 gene mutations.
- ProQR Therapeutics announced encouraging initial safety and pharmacokinetic data from the first cohort of healthy volunteers in its Phase 1 trial of AX-0810, showing no serious adverse events after 4 weeks of dosing. - The company selected development candidates for two pipeline programs: AX-2402 targeting MECP2 for Rett syndrome and AX-2911 targeting PNPLA3 for metabolic-associated steatohepatitis (MASH). - AX-2402 demonstrated statistically significant functional improvements in a mouse model of Rett syndrome, while AX-2911 showed over 80% reduction in hepatic fat content in preclinical studies. - ProQR's strategic collaboration with Eli Lilly achieved $4.5 million in milestones during 2025, contributing to the company's financial runway extending into mid-2027.
- Profluent Bio has announced a research collaboration with the Rett Syndrome Research Trust to design novel base editors using AI technology to permanently correct mutations causing Rett syndrome. - The partnership will leverage Profluent's frontier AI models to engineer compact base editors targeting recurrent "hot-spot" mutations in the MeCP2 gene, initially focusing on the T158M mutation. - The collaboration aims to create personalized genomic medicines that can fit inside a single AAV vector for efficient delivery to the central nervous system. - RSRT has raised almost $110 million for Rett research and launched a $40 million initiative in 2024 to bring three genetic medicines to clinical trials by 2028.
- Apertura Gene Therapy and Rett Syndrome Research Trust announced a collaboration to license Apertura's TfR1-targeted AAV capsid technology for developing genetic medicines to treat Rett syndrome. - The TfR1 CapX platform enables intravenous delivery of genetic medicines across the blood-brain barrier, achieving over 50% neuronal and 90% astrocyte transduction in preclinical studies. - The partnership will integrate the capsid technology into RSRT's MECP2 Editing Consortium, a $40 million initiative aimed at bringing three genetic medicines to clinical trials by 2028. - Rett syndrome affects primarily girls due to MECP2 gene mutations, causing severe developmental regression and requiring round-the-clock care throughout life.
- ProQR Therapeutics is advancing its Axiomer™ RNA editing platform across liver and CNS programs, with its lead program AX-0810 for cholestatic diseases on track for CTA filing in Q2 2025. - The company expects up to four clinical data readouts in 2025-2026, including first clinical data for AX-0810 in Q4 2025, supported by a strong financial position of €149.4 million providing runway into mid-2027. - ProQR's strategic partnerships include a $3.9 billion collaboration with Eli Lilly and a partnership with the Rett Syndrome Research Trust, reinforcing its leadership in the emerging field of RNA editing therapeutics.
