本文来自微信公众号：返朴 （ID：fanpu2019），受访者：付向东，访谈人：叶水送，原文标题：《专访著名生物学家付向东：治疗老年痴呆、逆转衰 老，RNA疗法为人类带来新的希望》，题图来自：AI生成 RNA，作为细胞信号传导的重要使者，在许多疾病中扮演着重要的角色，无论是神经退行性疾病还是衰老，都有研究者在开发相应的RNA疗法。 西湖大学讲席教授付向东自1992年建实验室时起，就一直在做RNA方面的基础研究和转化应用。他于2010年入选美国科学促进会成员，2016年获Ray Wu Society终身成就奖，是国际知名的RNA研究专家。在近期我们对付向东教授的一次专访中，他表示："RNA既是一个调节因子，也是一个药物靶标，同时 也可以自身作为药物。" 近年，付向东教授将工作重点转移至开发各种RNA疗法上，他认为对于复杂疾病的治疗近年取得的真正进展还是比较小，但随着RNA领域一些基础理论 取得突破，可以带来全新的希望。 问：首先请您介绍下什么是RNA？它跟DNA、蛋白质之间的关系是怎样的？ 付向东：一提到这个话题，我们大概会想到中心法则：从DNA到RNA再到蛋白质，RNA发挥了一种承上启下的功能，只要有细胞都符 ...

背景介绍 ： 近年来， t au 蛋白 因其在神经退行性疾病机制中的 核心 作用 ，以及作为治疗靶点的巨大潜力， 已然成为研究热点 。 作为中枢神经系统中关键的微管相关蛋白 ， tau 蛋白对维持神经元结构稳定与轴突运输功能至关重要。然而，当 tau 蛋白发生异常翻译后修饰 （例如过度磷酸化） 时，会脱离微管并聚集形成 神经原纤维缠结 （NFT） ——而这正 是 阿尔茨海默病 （AD） 等多种神经退行性疾病的标志性病理特征 之一 。 深入 解析 t au 蛋白的致病机制、探索 t au 蛋白相关 生物标志物以及靶向 tau 蛋白的 治疗策略，不仅意义重大，更拥 有广阔前景，为基础研究与临床试验提供了 新思路 。 tau 的结构·修饰·致病：从基础功能到神经退行性疾病的诊断与治疗突破 本次义翘讲堂， 义翘神州 联合 生物世界 ，推出线上讲座—— tau 的结构·修饰·致病：从基础功能到神经退行性疾病 的诊断与治疗突破 ，该讲座将全方位聚焦 t au 蛋白，串联 并解构 其 "双面角色"—— 从神经元的"守护者"到神经退 行性疾病的"关键推手"。 本次讲座特邀 Sino Biological US Inc. （义 ...

Core Viewpoint - Alzheimer's disease (AD) is a prevalent neurodegenerative disorder characterized by memory decline and cognitive impairment, with significant challenges in drug development despite substantial investments from major pharmaceutical companies [3][4]. Group 1: Alzheimer's Disease Overview - Alzheimer's disease is linked to the abnormal accumulation of tau protein and beta-amyloid (Aβ) in the brain, with tau protein aggregation being more closely associated with cognitive symptoms and severity [3]. - Major pharmaceutical companies, including Pfizer, Johnson & Johnson, and Roche, have invested over $10 billion in research for Alzheimer's treatments, but success has been limited [3]. Group 2: Recent Research Findings - A recent study published in Nature by Dr. Ke Hou from UCLA presents a potential strategy to reduce tau protein neurofibrillary tangles (NFTs) in Alzheimer's patients, which may halt disease progression [4]. - The study reveals that D-type peptides can disassemble tau fibrils by assembling into amyloid-like fibers that induce stress release, leading to fiber breakage without the need for enzymatic activity or external energy sources [4][10]. Group 3: Mechanism of Action - The research team explored the mechanism of D-type peptides in disassembling tau protein fibers, identifying D-TLKIVWI as the most effective variant in vitro [6][7]. - D-type peptides assemble into amyloid-like fibers that create torsional stress on tau fibers, resulting in the disruption of local hydrogen bonds and subsequent fiber breakage [8][10]. Group 4: Implications for Treatment - The study highlights the stability, protease resistance, and good biocompatibility of D-type peptides, which can cross the blood-brain barrier without eliciting harmful immune responses [10]. - This research provides a promising new avenue for treating Alzheimer's and other amyloid-related diseases, such as Parkinson's and Huntington's disease, potentially transforming the treatment landscape for neurodegenerative disorders [4][10].

Core Viewpoint - Athira Pharma reported zero revenue for Q2 2025, focusing on cost containment and strategic pipeline refocus, particularly on its lead candidate ATH-1105 for ALS trials [1][5][10] Financial Performance - EPS (GAAP) for Q2 2025 was $(0.18), an improvement of 74.3% from $(0.70) in Q2 2024 [2] - Research and Development Expenses decreased to $3.7 million, down 83.4% from $22.2 million in Q2 2024 [2][5] - General and Administrative Expenses were reduced to $3.6 million, a 38.8% decrease from $5.9 million in Q2 2024 [2][5] - Net Loss for the quarter was $7.0 million, significantly lower than the $26.9 million loss in Q2 2024, marking a 74.0% reduction [2][5] - Cash reserves stood at $29.8 million as of June 30, 2025, down from $51.3 million at the end of 2024 [6] Strategic Focus - Athira Pharma is concentrating its efforts on ATH-1105, a candidate targeting the HGF pathway to address ALS, following the halt of its previous Alzheimer's candidate, fosgonimeton [3][4][9] - The company completed a Phase 1 clinical trial for ATH-1105, demonstrating a favorable safety profile and the ability to cross the blood-brain barrier [7][10] - Management is exploring strategic alternatives, including potential licensing deals or partnerships, to maximize shareholder value [8][10] Operational Outlook - The company has not provided forward financial or trial guidance for the remainder of 2025 or 2026, creating uncertainty for shareholders [11] - The focus on a single program (ATH-1105) necessitates successful clinical development and securing external partnerships to ensure future funding and operational viability [10]

Group 1 - Coya Therapeutics, Inc. (COYA) shares increased by 10.7% to close at $6.72, driven by notable trading volume [1] - The rise in stock price is linked to positive investor expectations regarding Coya Therapeutics' pipeline for neurodegenerative diseases, particularly the upcoming phase II study of COYA-302 for amyotrophic lateral sclerosis [2] - The company is projected to report a quarterly loss of $0.22 per share, a year-over-year decline of 15.8%, while revenues are expected to reach $4.2 million, reflecting a 22.8% increase from the previous year [3] Group 2 - The consensus EPS estimate for Coya Therapeutics has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without earnings estimate revisions [4] - Coya Therapeutics is classified under the Zacks Medical - Biomedical and Genetics industry, where another company, Absci Corporation (ABSI), experienced a 0.3% decline in its stock price [4] - Absci Corporation's consensus EPS estimate has decreased by 10% over the past month, with a year-over-year change of +9.1%, and it currently holds a Zacks Rank of 4 (Sell) [5]

SANTA ANA, Calif., July 21, 2025 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (OTC: NKGN) ("NKGen" or the "Company"), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer ("NK") cell therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Expanded Access Program (EAP) authorization for its IND for an open-label, non-randomized, multi-center intermediate size expanded access protocol for us ...

Summary of BioV Virtual KOL Event Company and Industry - **Company**: BioV - **Industry**: Neurodegenerative diseases, specifically focusing on Parkinson's disease and Alzheimer's disease Core Points and Arguments 1. **Introduction of Bezesterone**: Bezesterone is a novel orally bioavailable anti-inflammatory agent that targets inflammation and oxidative stress in neurodegenerative diseases, particularly Parkinson's disease [27][28][29] 2. **Mechanism of Action**: Bezesterone acts on the ERK signaling pathway, reducing tumor necrosis factor expression and activity, and is designed to improve insulin signaling without interfering with homeostasis [30][31][32] 3. **Neuroinflammation's Role**: Neuroinflammation is a significant factor in Parkinson's disease, driving both symptoms and disease progression. Bezesterone aims to reduce this inflammation, potentially improving both motor and non-motor symptoms [35][38] 4. **Clinical Study - Sunrise PD**: The Sunrise PD study is a phase two double-blind randomized controlled trial designed to evaluate the efficacy of bezesterone in early-stage Parkinson's patients who have not been exposed to dopaminergic therapy [52][81] 5. **Endpoints and Measurements**: The primary endpoint is the change in the modified MDS UPDRS rating scale, focusing on motor symptoms, while secondary endpoints include various non-motor symptoms and exploratory biomarkers [53][54] 6. **Comparison with Exenatide**: The panel discussed the limitations of exenatide in Parkinson's treatment and expressed confidence that bezesterone's dual action on inflammation and insulin signaling could yield better results [68][72] 7. **Home-Based Trial Design**: The trial allows for home assessments, where trained nurses conduct evaluations, ensuring patient comfort and potentially increasing participation rates [87][88] 8. **Potential for Disease Modification**: Preclinical data suggest that bezesterone has neuroprotective effects and may modify disease progression, with evidence from various CNS models showing reduced neuronal death [85][86] 9. **Future Plans for Alzheimer's Program**: BioV is also interested in pursuing Alzheimer's research, pending funding availability, with a focus on the adverse event profile observed in previous studies [92] Other Important Content 1. **Expert Presentations**: The event featured presentations from leading experts in neurology, discussing the pathophysiology of Parkinson's disease and the importance of addressing neuroinflammation and oxidative stress [4][5][7] 2. **Patient-Centric Approach**: The trial design emphasizes patient convenience and aims to standardize assessments across remote and traditional sites, potentially improving the quality of data collected [49][62] 3. **Discussion on Non-Motor Symptoms**: The panel acknowledged the significance of non-motor symptoms in Parkinson's disease and their role in diagnosis and treatment [82] 4. **Adverse Event Profile**: Bezesterone has shown a favorable safety profile in previous studies, with adverse effects similar to placebo, which is crucial for patient acceptance [38][60] This summary encapsulates the key points discussed during the BioV virtual KOL event, highlighting the company's focus on innovative treatments for neurodegenerative diseases and the ongoing clinical research efforts.

Group 1 - Sanofi announced the acquisition of Vigil Neuroscience for $470 million in cash, with the total price potentially rising to $600 million upon achieving certain development milestones [1] - The acquisition price of $8 per share represents a 236% premium over Vigil's closing price prior to the announcement, and shareholders may receive an additional $2 per share if Vigil's Alzheimer's treatment drug VG-3927 achieves commercial sales within a specified timeframe [1] - This acquisition focuses on the neurology field, which is one of Sanofi's four core strategic disease areas, and aims to strengthen its early-stage research pipeline in neurodegenerative diseases [1] Group 2 - The global aging trend has created significant unmet medical needs in the treatment of neurodegenerative diseases like Alzheimer's, prompting Sanofi to adopt a dual strategy of "in-house development + targeted acquisitions" to build a differentiated central nervous system drug pipeline [2]