FDA approval extends the indication of Evkeeza to treat younger patients with homozygous hypercholesterolemia (HoFH) HoFH is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) Initial Evkeeza approval based on placebo-controlled trial showing Evkeeza, when added to standard lipid-lowering therapies, could lower LDL-C by about 50% compared to placebo in this high unmet need population TARRYTOWN, N.Y., Sept. 26, 2025 (GLOBE NEWSWIRE) -- Regeneron Phar ...

About Soligenix Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. The Company's Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryteâ"¢ (SGX301 or synthetic hypericin sodium) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T- cell lymphoma (CTCL). With successful completion of the second Ph ...

Accessibility StatementSkip Navigation MINNEAPOLIS, Sept. 26, 2025 /PRNewswire/ -- Bio-Techne Corporation (NASDAQ: TECH), a global provider of life science tools, reagents and diagnostic products, today announced new advancements in its spatial biology portfolio for the RNAscopeâ"¢ ISH technology and the Lunaphore COMETâ"¢. These solutions help researchers and pathologists bridge the gap between translational research and clinical applications by enabling the detection of RNA and protein biomarkers on the s ...

智通财经APP获悉，高盛最新深度分析报告聚焦uniQure NV(QURE.US)基因疗法AMT-130在亨廷顿病治 疗中的突破性进展及商业前景。据披露，该疗法已在两项剂量组临床试验中展现显著疗效——高剂量组 17例患者完成36个月随访后，复合亨廷顿病评定量表显示疾病进展较自然病史减缓75%，总功能能力评 分变化减缓60%，脑脊液神经丝轻链蛋白水平平均下降8.2%;低剂量组12例患者虽未直接给出对比数 据，但整体趋势与高剂量组一致。生物标志物及多项次要终点指标均支持AMT-130的神经保护作用。近 日，该股已因为该疗法消息出现大涨。 在FDA加速批准路径下，uniQure计划于2026年第一季度提交生物制品许可申请，基于高剂量组三年期 的复合亨廷顿病评定量表(CUHDRS)变化数据，并采用ENROLL-HD自然病史数据集作为外部对照进行 倾向评分匹配分析。FDA已认可其制造工艺验证路径，要求补充全规模良好生产规范批次及单个工艺性 能确认批次以支持生物制品许可申请(BLA)提交。 安全性方面，自2022年12月起未新增严重不良事件，给药相关不良反应均已解决;第三队列联合免疫抑 制方案试验中，三例严重不良事件通过 ...

September 26, 2025News Release BioPorto Sponsors Conferences on Pediatric Critical Care with New NGAL Scientific Data Presented COPENHAGEN, DENMARK and BOSTON, MA, USA, September 26 – BioPorto A/S (“BioPorto” or the “Company”) (CPH:BIOPOR), is pleased to support conferences focused on pediatric solid organ transplants and critical care nephrology. Clinical researchers present posters, abstracts and new publications using NGAL in their patients, leading to better identification for risk of Acute Kidney Injur ...

ST. GALLEN, Switzerland, and SAN DIEGO, Sept. 26, 2025 /PRNewswire/ -- CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) support the recent publication of the updated clinical practice guidelines for the treatment of IgA Nephropathy (IgAN), Kidney Disease: Improving Global Outcomes (KDIGO) 2025 clinical practice guideline for the management of IgA Nephropathy and Immunoglobulin A vasculitis. The updated guidelines define diagnostic criteria, treatment goals and treatment approaches, aiming for progre ...

NEW YORK, Sept. 26, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today reiterated that its allogeneic cell therapy products are manufactured from U.S. donors in the U.S. and designated as U.S. origin products not subject to tariffs on imported branded or patented pharmaceutical products. Ryoncil® (remestemcel-L) is the only allogeneic mesenchymal stromal cell therapy approved by U.S. Food and Drug Administration ...

NEW YORK, Sept. 26, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today reiterated that its allogeneic cell therapy products are manufactured from U.S. donors in the U.S. and designated as U.S. origin products not subject to tariffs on imported branded or patented pharmaceutical products. Ryoncil® (remestemcel-L) is the only allogeneic mesenchymal stromal cell therapy approved by U.S. Food and Drug Administration ...

OSE Immunotherapeutics, Inserm Transfert and Nantes University Announce Strategic Partnership to Accelerate Innovation in Immunotherapy NANTES, France – September 26, 2025, 7:00 a.m. – OSE Immunotherapeutics SA (ISIN: FR0012127173; Mnemo: OSE), Inserm Transfert (on behalf of Inserm, a French public organization dedicated to human health), and Nantes University today announce the signing of a strategic partnership agreement aimed at accelerating the development of innovative projects in the fields of immuno ...

Several small-cap names posted sharp gains in Thursday's after-hours session, driven by fresh corporate updates, clinical developments, and strategic pivots. Below are five stocks that stood out for their notable post-close moves and underlying catalysts.Shares of MEDIROM Healthcare Technologies Inc. (MRM) surged dramatically in after-hours trading on Thursday, climbing 282.8% to $5.55 as of 11:41 PM EDT. The sharp spike followed news that the company will double the installation of its biometric authentic ...