SAN DIEGO, Sept. 23, 2025 (GLOBE NEWSWIRE) -- NeuraClick, Inc., an innovative, preclinical-stage biopharmaceutical company reimagining patient health through the development of novel, long-acting neurotherapeutics, today announced the appointment of industry veteran and biotech executive Daniel M. Bradbury as Strategic and Metabolic Programs Advisor. “We are thrilled to welcome Dan to NeuraClick,” said Dr. Subhi Marwari, Chief Executive Officer. “For an early-stage venture, experienced guidance is essential ...

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核心观点 - 美国FDA授予赛诺菲基因疗法SAR446268快速通道资格 用于治疗非先天性1型强直性肌营养不良症(DM1) [1] - 该疗法采用RNA干扰技术靶向沉默DMPK基因表达 是目前唯一进入临床研究阶段的DM1候选疗法 [2][7] - 全球DM1患者发病率约为1/2300 目前尚无任何获批治疗方案 [4][7] 产品特性 - 采用腺相关病毒载体(AAV)递送RNA干扰(RNAi)技术 通过单次给药实现DMPK基因沉默 [2] - 作用机制为减少DMPK转录本 消除肌肉组织中有毒RNA foci 恢复正常剪接功能并改善肌肉功能 [2] - 针对疾病核心症状包括进行性肌无力、肌强直以及心脏/肺部/内分泌系统等多系统功能障碍 [2][4] 临床进展 - 目前处于首次人体I/II期临床研究阶段(NCT06844214) 重点评估安全性、耐受性和有效性 [3] - 首例患者计划于2025年末入组 [3] - 已获得美国(2024年7月)和欧盟(2024年10月)孤儿药资格认定 [3] 疾病背景 - 1型强直性肌营养不良症(Steinert病)为遗传性进行性罕见疾病 由DMPK基因突变引起 [4] - 临床表现包括从轻度成人病例到严重先天性形式的不同程度症状 严重影响患者日常生活能力和独立功能 [4] - 疾病可发生于任何年龄段 对维持生命重要功能产生深远影响 [4] 公司战略 - 赛诺菲定位为研发驱动型人工智能生物制药企业 专注于免疫系统相关药物和疫苗开发 [5] - 公司致力于通过解决最紧迫的医疗健康挑战来推动进步并产生积极影响 [5] - 在泛欧交易所(EURONEXT: SAN)和纳斯达克(NASDAQ: SNY)双重上市 [6]

Ad Hoc Announcement Pursuant to Art. 53 LR Geneva, Switzerland, September 23, 2025 - Addex Therapeutics (SIX and Nasdaq: ADXN), a clinical-stage biopharmaceutical company focused on developing a portfolio of novel small molecule allosteric modulators for neurological disorders, today announced the appointment the Bank of New-York Mellon (BNY) as its new ADS depositary agent. This change is expected to become effective on October 6, 2025.  The appointment of BNY has no impact on the fees paid by ADS holders. ...

Sanofi’s SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1 Designation earned for one-time AAV gene therapy SAR446268, designed to silence DMPK expressionMyotonic dystrophy type 1 (DM1) is a rare, genetic disorder that causes progressive muscle weakness and wasting, with no currently approved medicines Paris, September 23, 2025. The US Food and Drug Administration (FDA) has granted fast track designation to SAR446268, Sanofi's one-time AAV gene therapy f ...

合作与产品授权 - 公司通过子公司与重庆智翔金泰生物制药股份有限公司就Vecantoxatug注射液(GR2001)和Silevimig注射液(GR1801)签订两项独家合作协议 [1] - 获得两款产品在中国大陆的独家商业化权及亚太地区、中东和北非的独家许可权 [1] - 合作期限为产品在中国大陆获批上市后10年 期满后可自动续约10年 [1] Vecantoxatug产品特性与进展 - Vecantoxatug为被动免疫破伤风药物 安全性优于人破伤风免疫球蛋白(HTIG) 能提供快速持久免疫保护 [2] - 三期临床试验达到主要疗效终点 2024年5月获国家药监局药品审评中心突破性治疗药物认定 [2] - 2025年5月22日新药上市申请获正式受理 [2] - 采用重组人源化单克隆抗体技术 靶向破伤风毒素重链片段C结构域 有效阻断毒素进入神经元 [5] - 中国专利已获授权 [5] 破伤风市场现状与需求 - 破伤风是由破伤风梭菌引起的急性感染性疾病 全球年发病数约50-100万例 死亡率高达30-50% [6] - 现有被动免疫产品存在安全性问题(过敏反应风险、潜在病原体传播)和可及性限制 [7] - 破伤风梭菌广泛存在于土壤和哺乳动物肠道中 预防是最有效策略 [7] Silevimig产品特性与进展 - 全球首款重组全人源双特异性抗体 靶向狂犬病毒糖蛋白表位I和/或III 符合WHO推荐的"鸡尾酒"疗法方案 [3] - 具有广谱中和性、低免疫原性、对疫苗诱导主动免疫干扰小、生产成本可控等优势 [3] - 成人用上市申请于2025年1月14日获受理 儿童青少年用三期临床试验于2025年7月获批 [3] - 三期临床试验显示其保护效果不劣于人狂犬病免疫球蛋白(HRIG) [10] - 中国专利已获授权 [9] 狂犬病市场现状与需求 - 狂犬病死亡率接近100% 中国每年暴露人群超4000万人 其中三级暴露者约1600万人(40%) [11] - 目前仅约15%的三级暴露者接受被动免疫 受限因素包括认知不足、费用高昂、可及性差等 [11] - 现有被动免疫产品HRIG存在血源感染风险(如HIV、乙肝、丙肝) ERA存在血清病和过敏性休克风险 [12] - 中国目前仅批准两种狂犬病抗体产品 [12] 公司战略定位 - 公司专注于具有临床价值和差异化优势的创新产品布局 [4] - 重点布局全球首创(FIC)和最佳同类(BIC)创新产品 [15] - 在心脑血管/消化/眼科/皮肤健康领域建立竞争优势 其中皮肤健康业务已成为行业领先企业 [16] - 持续深化东南亚和中东地区业务发展 [16] 合作伙伴背景 - 智翔金泰(股票代码688443)成立于2015年 专注于自身免疫疾病、感染性疾病和肿瘤领域的抗体药物研发 [13] - 具备抗体分子发现、工艺开发、质量研究、临床试验和规模化商业化的全产业链能力 [13]

AbbVie Inc. (NYSE:ABBV) is one of the 15 Best Stocks to Invest in for Financial Stability. Piper Sandler Reiterates Its ‘Overweight’ Rating on AbbVie Inc. (ABBV) with a $231 PT On September 12, 2025, Piper Sandler reiterated its ‘Overweight’ rating on AbbVie Inc. (NYSE:ABBV) with a $231 price target. This comes after the company settled with abbreviated New Drug Application (ANDA) filers over Rinvoq. The agreement delays generic entry until April 2037, which is four years beyond the expiration of key co ...

CARMEL, Ind., Sept. 22, 2025 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc. (Nasdaq: MBX), a clinical-stage biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders, today announced that it has filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission (SEC) relating to a proposed underwritten public offering of 10,000,000 shares of its common stock. Furthermore, MBX Biosciences ...

Cash and cash equivalents totaled €107.5 million as of June 30, 2025, excluding the €26.5 million milestone invoiced in May 2025 (received in July 2025) upon pricing and reimbursement approval of Iqirvo® (elafibranor) in three major European countries€33.5 million in revenues, including the €26.5 million milestone invoiced in May 2025Strong 1H25 sales trajectory reported by Ipsen for Iqirvo® in PBC in July, followed by U.S. market exit of key competitor in SeptemberFollowing discontinuation of the VS-01 pro ...

Interim Clinical Study Results for Cohort 1 of the BB-301 Phase 1b/2a Treatment Study Anticipated in Q4 2025 Enrollment of the First Subject into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study Expected in Q4 2025 HAYWARD, Calif., Sept. 22, 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 (“ddR ...