相关临床试验
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8 进行中
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进行中（未招募）
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50.0%
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37.5%
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- ProQR Therapeutics announced that the first participant has been dosed in a Phase 1 trial of AX-0811 in healthy volunteers. - AX-0811 is an AI-discovered RNA editing oligonucleotide designed to modulate NTCP for cholestatic liver diseases including biliary atresia. - The Phase 1 study assesses safety, tolerability, pharmacokinetics and pharmacodynamic biomarkers, with target engagement data from the first two cohorts expected in early January 2027. - ProQR will host a virtual investor and analyst event on September 30, 2026, reviewing AX-0810 Phase 1 target engagement data and its NTCP strategy in biliary atresia.
- ProQR Therapeutics announced a strategic partnership with Ginkgo Bioworks to leverage autonomous laboratory technology for high-throughput data generation supporting AI-enabled drug discovery for its Axiomer RNA editing platform. - The company established an AI Advisory Board comprising industry leaders from organizations including NVIDIA, Owkin, and Hugging Face to guide the development of AI strategies for optimizing Axiomer editing oligonucleotides. - ProQR expects to advance its first AI-discovered development candidate into clinical trials with a CTA filing anticipated in mid-2026 and initial clinical data expected by year-end 2026. - The partnership provides access to Ginkgo's 50+ instrument autonomous lab called Nebula, which is designed to remove bottlenecks in AI-enabled drug discovery by increasing experimental data generation speed and scale.
- 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.
- Faron Pharmaceuticals has appointed Jurriaan Dekkers as Chief Financial Officer, bringing over 20 years of biopharma experience including roles at AstraZeneca Netherlands and ProQR Therapeutics. - The appointment comes as Faron's lead immunotherapy asset bexmarilimab prepares to enter registrational studies, with CEO Juho Jalkanen highlighting Dekkers' fundraising and strategic growth expertise. - Current CFO Yrjö Wichmann will retire after playing a key role in the company's strategic transition and growth, remaining through Q1 2026 for continuity.
- ProQR Therapeutics received authorization from the Central Committee on Research Involving Human Subjects (CCMO) following EMA centralized review to initiate a Phase 1 study of AX-0810 in healthy volunteers. - AX-0810 is a first-in-class investigational RNA editing oligonucleotide targeting NTCP, developed for treating cholestatic diseases like primary sclerosing cholangitis and biliary atresia. - The Phase 1 study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics via biomarkers to establish proof of target engagement in the Netherlands. - AX-0810 represents the first program from ProQR's Axiomer RNA editing pipeline to enter clinical development, with initial clinical data expected in Q4 2025.
- ProQR Therapeutics has submitted a Clinical Trial Application to the European Medicines Agency for AX-0810, marking the first clinical advancement of its Axiomer RNA editing platform. - The Phase 1 study will evaluate safety, tolerability, and target engagement of AX-0810 in healthy volunteers, with initial data expected in Q4 2025. - AX-0810 targets NTCP to reduce toxic bile acid accumulation in cholestatic liver diseases, representing a novel therapeutic approach for conditions with high unmet medical need. - The investigational drug uses ADAR-mediated RNA editing to selectively modulate NTCP function, supported by human genetics data showing naturally occurring NTCP variants safely reduce bile acid reuptake.
• ProQR Therapeutics has appointed Dennis Hom as CFO and Dr. Cristina Lopez Lopez as CMO to support the advancement of its Axiomer™ RNA editing technology platform into clinical development. • Dennis Hom brings over 25 years of financial leadership experience, having raised more than $4.5 billion in capital and executed transactions exceeding $57 billion in value throughout his career. • Dr. Cristina Lopez Lopez contributes 20+ years of global R&D leadership with expertise in translational science, previously serving as Global Head of Neurodegeneration at Johnson & Johnson.
- The global RNA therapy clinical trials market is projected to grow from $2.85 billion in 2024 to $4.16 billion by 2034, with a CAGR of 3.85%, driven by advancements in mRNA, siRNA, and antisense oligonucleotide-based therapies. - Over 80 companies are currently evaluating more than 100 RNA therapies across various development stages, with significant activity in rare diseases, oncology, and genetic disorders. - Recent breakthroughs include FDA clearance for the first CRISPR/Cas13 RNA-editing therapy for neovascular age-related macular degeneration and promising results for RNA therapies targeting rare muscular dystrophies.
- ProQR Therapeutics secures $8.1 million in additional funding from the Rett Syndrome Research Trust to advance AX-2402 into clinical trials. - AX-2402, based on ProQR's Axiomer platform, targets the R270X mutation in the MECP2 gene, which causes Rett syndrome. - The funding will support optimizing therapeutic candidates targeting Methyl CpG binding protein 2 (MECP2) and progressing them towards clinical development. - ProQR's Axiomer technology uses RNA editing to correct disease-causing mutations at the mRNA level, potentially restoring normal cell function.
• Beam Therapeutics reported a patient death in their BEAM-101 sickle cell disease trial, likely due to the conditioning regimen. • AstraZeneca scientists engineered PsCas9 for therapeutic genome editing in mouse liver, showing promise for hypercholesterolemia treatment. • YolTech Therapeutics' novel LNP system delivers base editor mRNA to bone marrow cells, activating foetal haemoglobin production for blood disorder treatment.
