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Cell:先导编辑里程碑!刘如谦团队利用体内先导编辑成功治疗儿童脑病
生物世界· 2025-07-22 03:27
撰文丨王聪 编辑丨王多鱼 排版丨水成文 儿童交替性偏瘫 ( Alternating Hemiplegia of Childhood, AHC) 是一种神经发育障碍,通常在出生后的前 18 个月内出现,表现为反复发作的阵发性症状, 包括偏瘫 (身体一侧瘫痪) 、肌张力障碍 (疼痛性不自主肌肉收缩) 、异常眼球运动和癫痫发作,患者还会表现出非阵发性肌张力低下、发育迟缓以及智力障 碍。AHC 极为罕见,据估计每 100 万人中仅有 1 例,目前尚无能够改变疾病进程的治疗方法。 大约 70% 的 AHC 病例与 ATP1A3 基因的致病突变有关 ,该基因编码 钠钾泵 (Na + /K + -ATP酶) 的 α3 亚基, 其对神经元功能至关重要, 功能异常会导 致神经元过度兴奋或能量代谢失衡 ,引发发作性症状与神经发育缺陷。尽管已报道了 50 多种 AHC 相关 ATP1A3 致病突变,但 ATP1A3 的 D801N、E815K 和 G947R 这三种突变占 65% 以上,发生率分别为 36%、22%和 9%。 之前的研究揭示了 AHC 相关 ATP1A3 突变的 显性负效应致病机制 (Dominant-Negati ...
连连数字(02598.HK):受益于WEB3的领先跨境支付服务商
Ge Long Hui· 2025-06-29 10:38
Core Viewpoint - The company is well-positioned to benefit from the growing interest in stablecoins and has a strong foundation for growth in its payment business, supported by its licenses, compliance, technology, partnerships, and customer resources [1][2]. Group 1: Business Model and Market Position - The company focuses on providing payment services to B2B merchants, differentiating itself from consumer-focused e-wallets and third-party payment processors [1]. - In 2024, the company's digital payment revenue is expected to contribute 88% from TPV fees, with cross-border payments accounting for 61% and domestic payments for 26% [1]. - The company has a unique competitive advantage with its self-built licensing system and an open cooperation ecosystem, supported by 65 global payment licenses [1]. Group 2: Growth Expectations and Future Drivers - The company is expected to maintain a stable growth trajectory with a projected payment revenue growth rate of over 20% in the coming years, driven by a low dependency on any single market [1]. - The company plans to enhance its global licensing and local service capabilities while expanding its value-added services to support comprehensive business operations [1]. - The company has begun exploring opportunities in the Web3 space, which aligns with the application of stablecoins in cross-border payments, potentially improving global payment efficiency [2]. Group 3: Profitability and Valuation - The company maintains its profit forecast, currently trading at 7x/5x P/S for 2025/2026, with a target price increase of 6% to HKD 15.3, reflecting a 45% upside potential [3].
Esperion Reaches Settlement Agreement with Second ANDA Filer Not to Market Generic Version of NEXLETOL® (bempedoic acid) Prior to April 19, 2040
Globenewswire· 2025-06-02 12:00
Core Insights - Esperion has entered into a settlement agreement with Hetero USA, resolving patent litigation related to the generic version of NEXLETOL, preventing Hetero USA from marketing the generic in the U.S. before April 19, 2040, unless specific circumstances arise [1] - Ongoing patent litigation against other defendants regarding NEXLETOL and NEXLIZET continues, with no assurance on the outcome or potential market entry of generics before the 2040 date [2] Company Overview - Esperion Therapeutics is a commercial stage biopharmaceutical company focused on developing and marketing new medicines for patients at risk for cardiovascular disease, specifically offering FDA-approved oral, once-daily, non-statin medications for elevated LDL-C [3] - The company is advancing its next-generation program aimed at developing ATP citrate lyase inhibitors (ACLYi), leveraging new insights for rational drug design and the creation of potent inhibitors [3][4] - Esperion is evolving into a leading global biopharmaceutical company through commercial execution, international partnerships, collaborations, and the advancement of its pre-clinical pipeline [4]
陶哲轩转发!DeepMind开源「AI数学证明标准习题集」
量子位· 2025-05-31 03:34
Core Viewpoint - DeepMind has launched an open-source formal mathematical conjecture library, which includes a collection of formally stated mathematical conjectures, addressing the scarcity of resources for open conjectures and aiding AI models in enhancing mathematical reasoning and proof capabilities [1][6][8]. Group 1 - The conjecture library contains a diverse set of mathematical conjectures formalized using Lean, sourced from various avenues [9]. - The library serves as a formal "exercise set" for computers, allowing traditional automated theorem proving (ATP) systems to conduct proof searches based on the conjectures within [11][12]. - Users can contribute by formalizing new conjectures, suggesting desired formal problems, improving citations, and correcting inaccuracies in existing formalizations [16][17][18]. Group 2 - The library is expected to become a benchmark for testing automated theorem proving or formal tools, thereby assisting AI models in improving their mathematical reasoning and proof capabilities [7][8]. - The collaboration between DeepMind and mathematician Terence Tao has been significant, with Tao endorsing the potential of AI in mathematical discovery [28][29]. - The AlphaEvolve project, developed by DeepMind, has made strides in solving long-standing geometric challenges, demonstrating the potential of AI in mathematics [35][41].
Esperion(ESPR) - 2025 FY - Earnings Call Transcript
2025-05-20 15:00
Esperion Therapeutics (ESPR) FY 2025 Conference May 20, 2025 10:00 AM ET Speaker0 Okay. Welcome back, everybody. My name's Joe Panginas, managing director here at, HC Wainwright. Very happy to have with us Esperion Therapeutics. And, also nice to have the second commercial stage, company that I'm, doing a fireside chat with today. So it's very refreshing in the small cap biotech space, which, you don't necessarily apply to. So presenting for the company is Ben Halliday, chief financial officer. Ben, thank y ...
Esperion Reaches Settlement Agreement with ANDA Filer Not to Market Generic Version of NEXLETOL® (bempedoic acid) Prior to April 19, 2040
Globenewswire· 2025-05-12 12:00
ANN ARBOR, Mich., May 12, 2025 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that it has entered into a settlement agreement with Micro Labs USA, Inc. and its affiliate Micro Labs Limited (together, Micro Labs). This agreement resolves the patent litigation brought by Esperion against Micro Labs in response to Micro Labs’ Abbreviated New Drug Application (ANDA) seeking approval to market a generic version of NEXLETOL prior to the expiration of the applicable patents. Pursuant to the agreement, ...
Esperion to Participate in H.C. Wainwright's 3rd Annual BioConnect Investor Conference
GlobeNewswire News Room· 2025-05-09 12:00
ANN ARBOR, Mich., May 09, 2025 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that the company will be participating in the webcasted H.C. Wainwright 3rd Annual BioConnect Investor Conference on May 20, 2025, at 10 a.m. ET. The live webcast can be accessed on the investor and media section of the Esperion website. Access to the webcast replay will be available approximately two hours after the completion of the call and will be archived on the Company’s website for approximately 90 days. Esperi ...