"宁可与渐冻症战死，不会面对绝症屈辱等死。" 近日，国内渐冻症(肌萎缩侧索硬化症，ALS)"斗士"蔡磊已丧失语言能力的消息受到广泛关注。蔡磊在 最新回应中坦言"现在五体瘫软无法言语，压痛、口水、呛咳、吞咽、憋气每天都在折磨"，其同时还表 示，每天依然通过眼控在高效处理工作，"近期，我刚宣布了过去20个月对外科研资助超过5000万元， 这背后是上百个科研项目的合作"。 据《每日经济新闻》记者了解，今年以来，国内渐冻症领域研发进展消息频传，但所涉公司多为初创企 业，产品管线多处于IIT(研究者发起的临床研究)或临床早期阶段，融资仍然是一道关卡。 "蔡磊一直在为这些出了有效成果的药企呼吁、联系融资，但是今年依然很难。"9月28日，蔡磊团队方 面接受《每日经济新闻》记者采访时表示，隐藏在资金难题背后的，是ALS药物研发本身所存在的巨大 难度。 首个对因治疗药物进入中国，多款核酸药物处于早期临床阶段 中国药企成为ALS药物研发主力军，IIT研究破解研发困局 作为一种运动神经元病，渐冻症的病因尚未完全明确。近200年来全球有1000多万人因此死亡，治愈率 为0，绝大多数患者在患病后2至5年内死亡。 过去几年，在蔡磊不断努 ...

"蔡磊总一直在为这些有有效成果的药企呼吁、联系融资，但是今年依然很难。"9月28日，蔡磊方面接受《每日经济新闻》记者采访时表示，隐藏在资金难 题背后的，是ALS药物研发本身所存在的巨大难度。 首个对因治疗药物进入中国，多款核酸药物处于早期临床阶段 作为一种运动神经元病，渐冻症的病因尚未完全明确。近200年来全球有1000多万人因此死亡，治愈率为0，绝大多数患者在患病后2至5年内死亡。 过去几年，在蔡磊不断努力下，渐冻症已成为大众所知晓的疾病，药物研发进展也不断加快。而谈到今年以来，国内渐冻症领域最值得关注的进展，有业内 人士认为是渤健（Biogen）的反义寡核苷酸（ASO）药物——Tofersen（托夫生注射液）进入中国。 一方面，托夫生注射液是全球获批的3款ALS治疗药物之一，可用于携带SOD1（超氧化物歧化酶1）基因突变的ALS成人患者，其在中国获批，既填补了国 内没有针对SOD1-ALS患者的药物空白，也意味着国内ALS患者的治疗选择已经与国际同步。 另一方面，托夫生注射液是全球首个且唯一"对因治疗"的渐冻症药物，与利鲁唑和依达拉奉不同，该药物可以从源头上减轻运动神经元损伤，在2023年4月 获得FDA ...

Core Viewpoint - The article highlights the significant contributions of Dr. Fan Dongsheng in the research and treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, emphasizing the hope brought by new drug trials and the importance of patient involvement in clinical research [1][2][4]. Group 1: Dr. Fan Dongsheng's Contributions - Dr. Fan Dongsheng is a leading expert in ALS research in China, having treated over 10,000 patients and established the world's largest ALS patient database with over 5,000 entries [2][3]. - He emphasizes the importance of providing hope to patients while being honest about their conditions, often reassuring them about their overall health status [3][4]. - Dr. Fan has been involved in the development of new drug trials, which have become a source of hope for patients who have exhausted other treatment options [5][8]. Group 2: New Drug Trials and Research - Recently, Dr. Fan has led multiple clinical trials for new ALS drugs at Peking University Third Hospital, including a drug developed in collaboration with Tsinghua University, which received approval for clinical trials in August 2023 [8][9]. - There are currently nearly 10 ALS drug trials underway at the hospital, including both international and domestic drug development efforts [10][11]. - The article notes that ALS drug development is particularly challenging, with only a few drugs successfully reaching the market in the past decades, highlighting the need for continued research and investment in this area [10][11]. Group 3: Patient Involvement and Data Collection - Dr. Fan has established a new data platform that allows ALS patients to upload their health information, significantly increasing the amount of real-time data available for research [21][24]. - The platform has collected over ten thousand data points, doubling the previous data collected over two decades, which aids in the rapid recruitment of patients for clinical trials [21][24]. - The article emphasizes the importance of precise patient selection for clinical trials, as different ALS subtypes may respond differently to treatments, thus improving the success rates of trials [12][14]. Group 4: Community and Advocacy - Many of Dr. Fan's patients have become advocates for ALS awareness and research, contributing to community support and fundraising efforts [17][20]. - The article highlights the role of patients like Liu Jijun and Cai Lei in promoting ALS research and support networks, showcasing the impact of patient-led initiatives [17][20]. - Dr. Fan's approach to patient care includes not only medical treatment but also fostering a supportive community among ALS patients, which is crucial for their mental and emotional well-being [16][18].

Core Insights - The report highlights the approval of semaglutide (Wegovy) for the treatment of metabolic dysfunction-associated steatotic liver disease (MASH) by the FDA, which is expected to enhance the market penetration of related products and diagnostic tools [7][10][15] - The report suggests that companies like Furuya Co., Ltd. will benefit from the growth opportunities in the MASH indication market due to the increasing prescription rates of semaglutide and the rising demand for diagnostic instruments [15][5] Industry Performance Review - As of August 15, 2025, the pharmaceutical and biotechnology sector's TTM-PE stands at 51.31 times, which is 110% higher than its historical low of 24.38 times [16] - The healthcare sector has shown a relative premium of 281% compared to the CSI 300 index, which is significantly above the average premium rate of 241% over the past decade [16] - The pharmaceutical and biotechnology sector experienced a 3.08% increase in the week from August 11 to August 15, 2025, ranking 10th among 27 sub-industries [20][24] Company Dynamics - Furuya Co., Ltd. is noted for its FibroScan series of liver fibrosis diagnostic instruments, which are the first globally to utilize transient elastography technology for non-invasive liver stiffness measurement [15] - The report emphasizes the strategic collaborations between Furuya and leading pharmaceutical companies to promote the clinical application and commercialization of new drugs [15] - The report identifies several companies that could be key players in the MASH treatment landscape, including innovative drug and device companies such as Furuya, Anglikon, and others [15]

Company Overview - SineuGene, established at the end of 2021, focuses on gene therapy for neurological diseases, leveraging over a decade of foundational research from Professor Jia Yichang's laboratory at Tsinghua University [6][7] - The company has developed various animal disease models, including fruit flies, mice, and Bama pigs, to identify reliable drug targets [7] - SineuGene has established a platform for AAV screening targeting the central nervous system, aiming to create highly targeted, efficient, and low-immunogenic AAV serotypes [7] Product Development - SNUG01, the first-in-class gene therapy product developed by SineuGene, targets TRIM72 and is the first gene therapy product globally to do so [4] - The therapy utilizes recombinant adeno-associated virus type 9 (rAAV9) for precise delivery of the human TRIM72 gene to neurons via intrathecal injection [4] - Preclinical studies indicate that TRIM72 may protect neurons through multiple mechanisms, potentially delaying the degenerative progression of motor neurons in ALS patients [4] Regulatory Milestones - SNUG01 received Orphan Drug Designation (ODD) from the FDA for the treatment of amyotrophic lateral sclerosis (ALS), which is a significant milestone following its clinical trial approval [3][4] - The ODD status provides several benefits, including a 25% tax credit on clinical research costs, exemption from up to $3 million in Biologics License Application fees, and 7 years of market exclusivity post-approval [3] - This designation is expected to accelerate the development and market entry of SNUG01, ultimately benefiting ALS patients in need of effective treatments [3] Clinical Research - The upcoming I/IIa phase international multicenter registration clinical trial aims to assess the safety, tolerability, and preliminary efficacy of SNUG01 in adult ALS patients [3][4] - The trial is designed to ensure that patients in both China and the U.S. can benefit from the treatment simultaneously [3] Disease Context - ALS is a progressive and fatal neurodegenerative disease affecting both upper and lower motor neurons, leading to muscle weakness and atrophy, with a median survival of only 3-5 years [5] - Currently, there are no curative treatments available, and existing therapies only provide limited disease progression delay [5]