TSHA-102 granted Breakthrough Therapy designation by FDA Finalized FDA alignment on REVEAL pivotal trial protocol and SAP, including 6-month interim analysis that may expedite BLA submission, which was enabled by the rigorous developmental milestone evaluation in Part A REVEAL Phase 1/2 trials showing an unprecedented response rate Dosing of first patient in REVEAL pivotal trial scheduled for Q4 2025, with enrollment of additional patients expected to continue at multiple sites this quarter Presented new su ...

（来源：一度医药） 转自：一度医药 积极的数据表现曾推动uniQure股价暴涨300%，也让该疗法成为陷入困境的基因治疗领域的重要提振。 AMT-130的审批受阻并非个例，近期FDA对细胞和基因疗法的监管态度正呈现收紧态势。今年7月， Capricor Therapeutics治疗杜氏肌营养不良相关心脏疾病的细胞疗法因"有效性证据不足"遭FDA拒绝； Replimune的黑色素瘤溶瘤病毒疗法也因类似原因被驳回加速审批申请，尽管该公司后续重新提交申请 已获受理。 去年年底，uniQure还与FDA达成协议，获准基于这一1/2期临床试验数据，通过加速审批通道提交BLA 申请。 此次FDA的立场转变完全超出预期。uniQure首席执行官Matt Kapusta表示，FDA的最新反馈与此前提供 的指导意见"存在巨大差异"，公司对此感到意外。FDA认为，仅依靠外部对照组的1/2期数据不足以支 撑上市申请，这与双方此前达成的共识相悖。 受此消息影响，uniQure股价在11月3日盘前交易中暴跌66%。 曾被业内寄予厚望的亨廷顿舞蹈症基因疗法AMT-130，近日遭遇美国FDA的审批急转弯。研发企业 uniQure于11月3 ...

Whales with a lot of money to spend have taken a noticeably bullish stance on uniQure.Looking at options history for uniQure (NASDAQ:QURE) we detected 53 trades.If we consider the specifics of each trade, it is accurate to state that 60% of the investors opened trades with bullish expectations and 35% with bearish.From the overall spotted trades, 16 are puts, for a total amount of $1,759,721 and 37, calls, for a total amount of $3,331,788.Projected Price TargetsBased on the trading activity, it appears that ...

U.S. stocks traded higher this morning, with the Nasdaq Composite gaining 1% on Monday.Following the market opening Monday, the Dow traded up 0.04% to 47,583.87 while the NASDAQ rose 1.01% to 23,964.61. The S&P 500 also rose, gaining, 0.48% to 6,872.88.Check This Out: How To Earn $500 A Month From Pfizer Stock Ahead Of Q3 EarningsLeading and Lagging SectorsConsumer discretionary shares jumped by 1.8% on Monday.In trading on Monday, utilities stocks fell by 0.8%.Top HeadlineON Semiconductor Corporation (NASD ...

-Dr. Mates served as the Chairman, Chief Executive Officer, and Co-founder of Intra-Cellular Therapies, a mental health company, from June 2002 until it was acquired by Johnson & Johnson in 2025-HAYWARD, Calif., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today a ...

PARIS--(BUSINESS WIRE)--Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the Company's gene therapy GS010/LUMEVOQ® has been granted regulatory authorizations for an Individual Patient Expanded Access in the United States. The authorizations mark the first set of regula. ...

公司财务与活动安排 - 公司将于2025年11月4日美国东部时间上午8:30公布2025年第三季度财务业绩并举行企业最新情况电话会议及网络直播 [1] 公司业务与战略 - 公司是一家临床阶段生物技术公司，专注于开发基于腺相关病毒（AAV）的基因疗法，用于治疗中枢神经系统的严重单基因疾病 [2] - 公司的主要临床项目TSHA-102正在开发中，用于治疗Rett综合征，这是一种尚无获批针对疾病遗传根源的改良疗法的罕见神经发育障碍 [2] - 公司致力于开发变革性药物，以解决严重的未满足医疗需求，并显著改善患者及其护理人员的生活 [2] - 公司管理团队在基因疗法开发和商业化方面拥有成熟经验，并利用其生产工艺及临床和商业上得到验证的AA9衣壳，以加速将治疗从实验室推向临床 [2]

Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is one of the best NASDAQ stocks under $5 to buy now. On October 14, Rocket Pharmaceuticals announced that the US FDA accepted the resubmission of the Biologics License Application/BLA for KRESLADI (marnetegragene autotemcel; marne-cel). KRESLADI is a lentiviral vector/LV-based investigational gene therapy intended for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I), which is a rare genetic immune disorder. The BLA resubmission is supported by posi ...

公司概况与市场定位 - 公司为临床阶段生物技术公司 专注于开发基于腺相关病毒（AAV）的基因疗法 用于治疗影响中枢神经系统的罕见单基因遗传病 [4] - 公司市值为12.8亿美元 拥有现金充裕的资产负债表 [2] - 公司是亿万富翁Steve Cohen看好的具有巨大上涨潜力的小盘股之一 [1] 分析师观点与评级 - Raymond James于10月20日首次覆盖公司 给予“强力买入”评级 目标价为13美元 与更广泛的分析师共识一致 [1] 核心产品研发进展 - 针对Rett综合征的1/2期REVEAL试验结果积极 并获得FDA正面反馈 是主要驱动因素 [2] - 近期里程碑包括：获得突破性疗法认定 与FDA就方案达成一致 并获准进行6个月中期分析 这可能加快监管申报速度 [3] - REVEAL试验B部分患者招募正在进行中 最终给药预计在2026年中期完成 潜在商业化时间点为2027年 [3] - 公司专注于基因定义明确的Rett综合征患者群体 提供了清晰的治疗机会 [3]

公司股价表现 - Wellgistics Health Inc (NASDAQ:WGRX) 股价飙升至108美元，涨幅达168.16% [1][5] - MaxsMaking Inc (NASDAQ:MAMK) 股价上涨至743美元，涨幅为148.70% [1][5] - Inhibrx Biosciences Inc (NASDAQ:INBX) 股价升至5513美元，涨幅为94.43% [2][5] - Genenta Science SpA (NASDAQ:GNTA) 股价上涨至622美元，涨幅达92.72% [3] 公司业务与驱动因素 - Wellgistics Health 为药品批发分销商，专注于仿制药和品牌药分销以及第三方物流服务，其股价上涨与宣布同Datavault AI Inc (NASDAQ:DVLT) 就整合区块链智能合约至处方药追踪系统的无约束力意向书有关 [1] - MaxsMaking 为中国定制消费纺织品制造商，采用专有系统实现高效生产，其股价上涨与成功完成首次公开募股筹集650万美元并于2025年7月7日在纳斯达克资本市场开始交易相关 [1][5] - Inhibrx Biosciences 为临床阶段生物制药公司，专注于开发针对危及生命疾病的生物疗法，其股价上涨与其中期试验显示实验性药物有效减缓一种罕见侵袭性骨癌的进展相关 [2][5] - Genenta Science 致力于开发造血干细胞基因疗法，其股价上涨与同ANEMOCYTE建立战略合作伙伴关系以推进现成慢病毒载体质粒DNA生产相关 [3] 交易量与行业背景 - Wellgistics Health 交易量超过6.14亿股，远高于其平均水平 [1] - MaxsMaking 交易量超过140万股 [1] - Inhibrx Biosciences 交易量超过500万股 [2] - Genenta Science 交易量超过2000万股 [3] - 股价变动反映了从医疗保健、生物技术到消费品和科技等多个行业的动态，临床试验结果、战略合作、创新产品供应和市场趋势是主要驱动因素 [4]