TDI-01

摘要：药明康德在国防部军工名单诉讼中暂胜、Epicrispr获9000万美元融资推进FSHD表观遗传药、FDA受理慢性丁肝新药Lonafarnib、Regeneron 7500万美元押注抗体新贵 1️⃣ CellProthera签署在日本分销ProtheraCytes的谅解备忘录 — The Pharma Letter 法国CellProthera与日本伙伴签署谅解备忘录，计划在当地分销其心脏修复细胞疗法ProtheraCytes，具体条款和上市时间表未披露。这是欧洲细胞治疗公司向东扩张的最新一步，日本再生医学审评通道对这类产品相对友好，商业化路径值得观察。 2️⃣ 前传奇生物CEO掌舵生物技术初创公司，全球寻找药物 — BioPharma Dive 一位前传奇生物首席执行官出任一家生物技术初创公司掌门人，任务是在全球范围内搜寻和引进创新药物。传奇生物靠CAR-T出海跑出标杆，其高管的经验与网络本身就是稀缺资产，这轮人才流动折射出中国生物技术管理者正在成为全球抢手资源。 3️⃣ 抗体药物偶联物：为什么制造它需要一个村庄 — 化学与工程新闻 C&EN深度解读ADC制造为何难：抗体、连接子、细胞毒性药物三段都要精密控制，涉及化学合成、生物工程和质量控制的跨学科协作。中国已是ADC出海主力，读懂这种"村庄式"制造门槛，就明白为什么BD交易里CMC能力越来越值钱。 4️⃣ 药明康德在国防部中国军工名单诉讼中取得暂时胜利 — Fierce Pharma 美国法院裁定药明康德可暂时免于被列入国防部中国军工企业清单，案件仍在审理中。这是药明康德在1260H清单诉讼中的阶段性胜利，为在美业务争取到缓冲期；对出海的中国CXO和生物医药公司而言，这类司法反制路径的示范意义不亚于个案结果。 5️⃣ 生物技术手册细分：谁将冒险开发明天的治疗方法 — 生命科学领导者 行业媒体探讨药企对高风险疗法的态度分化：基因编辑、细胞疗法等前沿赛道需要长周期重投入，企业必须在创新与回报之间做取舍。观点类内容放进见闻，是提醒读者关注行业整体风向，不必纠结单点结论。 6️⃣ 科尔钦斯基警告：禁止中国生物技术交易会把管线交给诺华 — Finance.biggo.com 生物医药投资人科尔钦斯基警告，若禁止美国公司与中国生物技术交易，受伤的是美国自己的管线，获益者反而会是诺华等欧洲大厂。这是在生物安全法案反复拉锯的背景下，行业内公开反对脱钩的又一声音，值得出海企业关注华盛顿风向。 7️⃣ 4只生物科技股下半年将成为主要催化剂 — TradingView TradingView筛选出4只下半年可能迎来重大催化剂（临床读出、审批节点）的生物科技股，具体标的多未在报道中详述。催化剂交易是美股biotech常见玩法，这类列表的可读性大于投资建议，读者更值得关注的是下半年行业整体的数据读出密度。 8️⃣ FDA受理Lonafarnib治疗慢性丁型肝炎的新药申请 — 美通社 EIT Pharma宣布美国FDA已受理Lonafarnib治疗慢性丁型肝炎的NDA。慢性丁肝是肝炎里最难治、最罕见的一种，目前全球没有获批的专门疗法，受理意味着审评正式启动。中国是乙肝大国，丁肝共感染人群的存在让这款药的进展值得国内读者留意。 9️⃣ 制药企业自己做零售商 — The Pharma Letter 制药公司正在从"卖给批发商"转向自建零售渠道直接触达患者，以缩短供应链、掌控患者数据、提升依从性。礼来、诺和诺德都在美国做直营和订阅式服务，这一趋势对国内创新药企的启示是：商业化能力不只是销售团队，还包括数字化患者运营。 1️⃣0️⃣ 印度OPPI推动监管数据保护框架 — The Pharma Letter 印度制药协会（OPPI）游说政府建立监管数据保护机制，防止仿制药直接引用原研的临床试验数据，以激励本土创新研发。数据保护期正是诺和诺德在中国挡住国产仿制药一年的关键机制，印度若跟进，全球仿制药的"数据搭便车"模式将面临重塑。 1️⃣1️⃣ Regeneron 7500万美元A轮投资抗体新贵Infinimmune — Fierce Biotech Regeneron向抗体研发公司Infinimmune投资7500万美元A轮，支持其早期抗体药物开发与平台建设。大药企在早期轮次用真金白银锁定创新抗体平台，是过去一年最稳定的BD动作之一；对国内抗体公司来说，这类"产业资本+技术平台"的合作窗口依然敞开。 🧬 CGT+mRNA+罕见病专题 1️⃣2️⃣ Graviton BioScience的GV101治疗弗里德赖希共济失调IND获批 — 美通社 Graviton BioScience宣布FDA批准GV101的IND申请，用于治疗弗里德赖希共济失调（一种罕见神经退行性疾病）。IND获批意味着可进入临床试验，这类"小患者人群+明确遗传靶点"的罕见病管线，正是基因治疗和靶向药最活跃的试验田。 1️⃣3️⃣ 政府加强再生医学的监管和研究 — BW Healthcare World 多国政府正在加强再生医学的监管框架和科研投入，规范干细胞治疗、组织工程等前沿技术的临床应用，同时推动基础研究向转化医学发展。对CGT行业来说，监管清晰化是双刃剑：门槛提高，但合规企业的赛道反而被划清，长期利好真创新。 1️⃣4️⃣ FDA更新PRV计划后美国罕见病生物技术投资增加 — 制药高管 FDA更新优先审评券（PRV）政策后，美国罕见病生物技术领域投资明显增加。PRV可以转让变现，等于给罕见病研发发了一张可交易的"加速券"，为中小生物技术公司提供了额外融资工具。国内罕见病药物开发可借鉴这一机制设计思路。 1️⃣5️⃣ Silence Therapeutics的siRNA在真性红细胞增多症中取得成功 — The Pharma Letter Silence Therapeutics公布其siRNA疗法在真性红细胞增多症（一种骨髓增殖性肿瘤）中的积极结果，具体数据尚未披露。这是RNAi疗法在罕见血液病领域的又一实证，说明"沉默致病基因"的平台可以横向扩展到更多慢性和罕见疾病。 1️⃣6️⃣ Epicrispr获9000万美元融资推进表观遗传FSHD疗法 — Endpoints News Epicrispr在公布表观遗传药物EPI-321治疗面肩肱型肌营养不良症（FSHD）的早期积极数据后，完成9000万美元C轮融资。FSHD目前无有效疗法，EPI-321通过表观遗传调控干预疾病进程，是"不剪基因、只调开关"路线的代表，融资印证了资本对表观遗传药物的兴趣。 #创新药出海 #基因编辑 #RNAi #GLP1减肥药 #生物医药 #刘肖说CGT

Graviton received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for a novel capsule formulation of GV101 on August 7, 2026. FDA clearance marks a critical regulatory milestone in advancing this formulation of GV101, a capsule with improved PK and oral availability, into human trials for Friedreich's ataxia (FA). GV101 is a best-in-class selective ROCK2 inhibitor that increases frataxin protein levels, a therapeutic approach with potential disease-modifying effects in Friedreich's ataxia. NEW YORK, Aug. 11, 2026 /PRNewswire/ -- Graviton BioScience Corporation, a privately held, clinical-stage biotechnology company developing a diverse pipeline of therapeutics, including selective ROCK2 inhibitors for metabolic, inflammatory, fibrotic, and central nervous system disorders, announced that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for a novel capsule formulation of GV101, a proprietary oral ROCK2 inhibitor being developed for Friedreich's ataxia (FA). This new formulation of GV101 was specifically optimized for development in FA and other potential orphan indications. Preclinical and clinical studies have demonstrated that GV101 increases frataxin protein levels, which is a potentially disease modifying therapeutic approach that addresses the root cause of Friedreich's ataxia (FA). Following the FDA's 30-day review period, Graviton received IND clearance on August 7, 2026, enabling the initiation of a Phase 2 clinical study in individuals living with FA. "Our preclinical work has suggested that ROCK2 inhibition in cells derived from FA patients increased frataxin significantly. ROCK2 inhibition also increased frataxin in PBMCs in patients receiving GV101 in an obesity study. We believe, therefore, that this induction of frataxin by GV101 may become an orally available disease modifying agent." Rui Wu, Head of Research and Preclinical Planned Phase 2 Study in Friedreich's Ataxia GV101 is one of Graviton's proprietary investigational oral ROCK2 inhibitors advancing within the Company's broader development pipeline. The planned Phase 2 study will utilize a novel capsule formulation of GV101 that demonstrated improved pharmacokinetic (PK) properties and oral bioavailability in healthy volunteers in a Phase 1 study. This formulation of GV101 is being developed as a potential treatment for Friedreich's ataxia, a rare genetic disease driven by deficient frataxin production. Graviton now plans to initiate a 12-week, randomized, placebo-controlled, dose-finding Phase 2 trial enrolling up to 48 participants at multiple sites in the United States and internationally. The study is designed to evaluate the efficacy, safety, and tolerability of a range of GV101 capsule doses in individuals living with Friedreich's ataxia. The primary endpoint is an increase from baseline in frataxin levels measured in patients' cells. The Phase 2 study will be followed by an open-label extension, offering active drug to all participants who complete the 12-week trial. Participants will be able to continue receiving their existing FDA-approved standard-of-care treatment throughout the study while taking GV101. Enrollment is expected to begin in the first quarter of 2027. GV101 Development Highlights GV101 has demonstrated a favorable safety and tolerability profile in preclinical and clinical studies. More than 500 participants have received GV101 in clinical studies for durations of up to 24 weeks, with extension studies exceeding one year. In a Phase 1 multiple ascending dose study in healthy volunteers, GV101 achieved clinically relevant exposure and demonstrated a favorable safety and tolerability profile. Preclinical and clinical studies have also demonstrated that GV101 increases both frataxin mRNA and protein levels in patient-derived B-lymphocytes and fibroblasts. Consistent with these findings, oral ROCK2 inhibition with GV101 increased frataxin levels in vivo in an obesity study that evaluated patient-derived peripheral blood mononuclear cells (PBMCs). Collectively, these results provide evidence that ROCK2 inhibition may increase frataxin expression across multiple experimental systems. Increasing frataxin protein levels has the potential to move patients toward levels observed in asymptomatic carriers, supporting GV101's potential as a disease-modifying therapy aimed at addressing the underlying cause of Friedreich's ataxia. About Friedreich's Ataxia Friedreich's ataxia (FA) is a rare, inherited, progressive neurodegenerative disease affecting approximately 5,000 people in the United States and an estimated 15,000 people worldwide, qualifying it as an orphan disease. Friedreich's ataxia is caused by GAA trinucleotide repeat expansions in the FXN gene that result in reduced production of frataxin, a protein essential for mitochondrial function and cellular energy production. Symptoms typically begin in childhood or adolescence and worsen over time, affecting coordination, balance, speech, muscle strength, and mobility. Many individuals eventually become wheelchair-dependent and may develop serious complications, including cardiomyopathy, cardiac arrhythmias, diabetes, and difficulties with swallowing and speech. Significant unmet need remains for treatments that address the underlying frataxin deficiency that drives the disease. About GV101 GV101 is a clinical-stage, best-in-class, selective inhibitor of Rho/Rho-associated coiled-coil containing protein kinase 2 (ROCK2). ROCK2 is a key regulator of multiple cellular pathways involved in inflammation, fibrosis, metabolism, and gene expression. Through selective inhibition of ROCK2, GV101 has demonstrated the potential to modulate disease-relevant biological processes, including increasing frataxin production, a key factor in Friedreich's ataxia. About Graviton BioScience Graviton BioScience Corporation is a clinical-stage drug discovery and development company dedicated to engineering and developing best-in-class therapeutics for treating metabolic, CNS, inflammatory, fibrotic, and other disease indications. Graviton has developed a portfolio of selective ROCK2 inhibitors among other safe, innovative, and novel therapies for patients with fibrotic, autoimmune, and CNS diseases. GV101 is in clinical studies, with additional assets advancing through the pipeline. Leading the Company is Dr. Samuel Waksal, the founder and former Chairman and CEO of Kadmon Pharmaceuticals (acquired by Sanofi). Dr. Waksal is also the founder and former CEO of ImClone Systems (acquired by Eli Lilly) and a founder of MeiraGTx (NASDAQ: MGTX). Forward-Looking Statements This press release contains forward-looking statements regarding Graviton's development plans, regulatory strategy, and potential future clinical applications for its selective ROCK2 inhibitors. Such statements are subject to risks and uncertainties, including, but not limited to, biological, clinical, regulatory, financial, and operational risks. Actual results may differ materially. Graviton BioScience Contact Melanie Glickman Senior Associate, Operations Strategy [email protected] SOURCE Graviton Bioscience Corporation 21% more press release views with Request a Demo