Oral Presentation on Saturday to Highlight Interim Safety and Efficacy Results from the MyPEAK™-1 Phase 1b/2a Clinical Trial in Adults with MYBPC3-Associated Hypertrophic Cardiomyopathy Second Late-Breaking Presentation on Sunday to Showcase Results of Cellular Reprogramming Gene Therapy Treatment in Pig Model of Ischemic Heart Failure Tenaya Management to Host Webcast Conference Call on Monday, November 10, 2025, to Review MyPEAK-1 Data SOUTH SAN FRANCISCO, Calif., Nov. 04, 2025 (GLOBE NEWSWIRE) -- Tenaya ...

Key Takeaways Vertex's Q3 EPS of $4.80 beat estimates, rising 10% year over year on $3.08B in revenues.Trikafta and Alyftrek fueled growth, while Casgevy sales fell 44% sequentially to $16.9M.VRTX tightened its total revenue guidance for 2025 from $11.85-$12 billion to $11.9 to $12.0 billion.Vertex Pharmaceuticals (VRTX) reported adjusted earnings of $4.80 per share for the third quarter of 2025, surpassing the Zacks Consensus Estimate of $4.55. Earnings rose around 10% year over year.Total revenues of $3.0 ...

TESLA WATCH: Elon Musk Pay Deal In FocusExact Sciences (EXAS) stock surged Tuesday on a broad third-quarter beat that "should allay fears of a market slowdown," an analyst said.The company is well known for Cologuard, a stool-based test that detects colon cancer. Screening revenue, which includes Cologuard tests, grew 22% to $666 million, easily topping forecasts for $637 million. Precision oncology revenue, which includes a breast cancer test, jumped 13% to $184 million. That beat expectations for $173 mil ...

Taysha Gene Therapies (NasdaqGS:TSHA) Q3 2025 Earnings Call November 04, 2025 08:30 AM ET Speaker0Good day, everyone, and welcome to the Tacea Gene Therapies Third Quarter twenty twenty five Earnings Call. At this time, all participants are in a listen only mode. Later, you will have the opportunity to ask questions during the question and answer session. Please note this call may be recorded and I will be standing by if you should need any assistance. Is now my pleasure to turn the conference over to Haley ...

核心观点 - Solid Biosciences公司公布了其杜氏肌营养不良症基因疗法SGT-003在1/2期临床试验中的积极中期数据，显示其在微肌营养不良蛋白表达、肌肉保护及心脏功能方面具有良好潜力，并计划于2026年上半年与FDA讨论注册路径[1][5][25][26] - 公司其他管线项目，包括针对弗里德赖希共济失调的SGT-212和针对儿茶酚胺敏感性多形性室速的SGT-501，也取得临床进展[29][31] - 公司财务状况稳健，截至2025年第三季度末拥有2.361亿美元现金及等价物，预计现金可支撑运营至2027年上半年[35] 主要候选产品SGT-003（杜氏肌营养不良症）临床进展 - 截至2025年10月31日，INSPIRE DUCHENNE试验中已有23名参与者接受给药，公司预计到2026年初将完成总共30名参与者的给药[1][6] - 第90天活检数据显示，10名接受治疗的参与者（年龄5-10岁）全部产生治疗反应，平均微肌营养不良蛋白表达水平为58%（蛋白质印迹法和质谱法），平均微肌营养不良蛋白阳性纤维为51%（免疫荧光法）[3][11] - 观察到微肌营养不良蛋白表达水平与肌营养不良蛋白相关蛋白复合物关键组分（如β-肌聚糖，r=0.95）之间存在强统计相关性，表明治疗介导的蛋白复合物恢复[4][7][9] - 肌肉损伤生物标志物呈现有利下降，第90天平均肌酸激酶降低34%，第360天降低42%[14] - 心脏安全监测显示心脏损伤减少和心脏功能正常化的早期信号，第90天平均血清心肌肌钙蛋白I降低31%，第360天降低70%[17][18] 安全性数据 - 截至2025年10月31日，SGT-003在23名给药参与者中普遍耐受性良好，仅使用类固醇作为预防性免疫调节方案[21] - 试验中报告了1例治疗相关的严重不良事件，为3级免疫介导的肌炎，患者对类固醇治疗反应迅速，数据安全监测委员会建议继续给药[22] - 未观察到药物性肝损伤病例，肝损伤特异性标志物γ-谷氨酰转移酶水平保持稳定[23][24] 监管与后续开发计划 - 公司计划在2026年上半年与FDA会面，讨论SGT-003的潜在注册路径，包括加速批准途径[1][26] - 公司已与FDA就SGT-003的效价测定策略达成一致，预计将在2026年完成工艺性能确认生产批次[27] - 针对美国以外地区的3期随机、双盲、安慰剂对照临床试验IMPACT DUCHENNE已于2025年10月启动首个临床试验中心并开始筛选参与者[28] 其他研发管线进展 - 针对弗里德赖希共济失调的SGT-212：其首次人体1b期临床试验FALCON已于2025年10月启动首个试验中心并开始筛选参与者，这是首个采用双给药途径的FA研究性基因疗法[29][30] - 针对儿茶酚胺敏感性多形性室速的SGT-501：预计在2025年第四季度启动其首次人体1b期临床试验ARTEMIS的首个试验中心[31][32] 平台技术与财务状况 - 公司专有的下一代衣壳AAV-SLB101已在超过30份与公司、机构和学术实验室的协议中获得授权使用[33][34] - 截至2025年9月30日，公司拥有现金、现金等价物和可供出售证券总计2.361亿美元，预计资金可支撑运营至2027年上半年[35] - 2025年第三季度研发费用为3890万美元，同比增长1150万美元，主要由于SGT-003相关制造、监管和临床成本增加[35]

uniQure N.V. (NASDAQ:QURE) is one of the 12 Best Performing Stocks in the Last 3 Months. On October 8, RBC Capital reiterated a Buy rating on uniQure N.V. (NASDAQ:QURE) and increased its price target from $55 to $65. Moreover, on October 6, H.C. Wainwright also raised its price target on uniQure N.V. (NASDAQ:QURE) from $70 to $110 and kept a Buy rating. uniQure (QURE) Price Targets Raised by Analysts After Positive Trial Results These updates came after the company reported positive topline data from it ...

RESEARCH TRIANGLE PARK, N.C., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Opus Genetics (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs), announced today that its pioneering work was featured on Good Morning America (ABC News) in honor of World Blindness Awareness Month. The national feature highlighted the story of Lindsey Rambo, the second participant in Opus Genetics’ ongoing Phase 1/2 trial of gene therapy OPGx-LCA5, aimed a ...

Lexeo Therapeutics (NASDAQ:LXEO) is one of billionaire Daniel Sundheim’s stock picks with huge upside potential. On October 21, Leerink’s Mani Foroohar lowered the price target on Lexeo Therapeutics (NASDAQ:LXEO) from $20 to $18 but maintained an Outperform rating. Is Lexeo Therapeutics (LXEO) One of Billionaire Daniel Sundheim's Stock Picks with Huge Upside Potential Likoper/Shutterstock.com The revision followed Lexeo’s $135 million capital raise through a secondary offering and private placement, alon ...

Third-Quarter 2025 Earnings Presentation October 30, 2025 1 Forward Looking Statements The forward-looking statements in this presentation are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timings discussed, projected, anticipated or indicated in any fo ...

Accessibility StatementSkip Navigation ROCKVILLE, Md., Oct. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the completion of enrollment in the AFFINITY DUCHENNE pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, as well as the successful production of the first batches intended for commercial supply. "The Duchenne community urgently needs new treatment options that provide durable, safe outcomes and can meaningfully change the ...