A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single-Ascending Dose and Multiple-Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral FB-101 in Healthy Subjects

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of oral doses of FB-101 in healthy subjects.

开始日期2019-10-14

申办/合作机构

1st Biotherapeutics, Inc.初创企业

100 项与 FB-101 相关的临床结果

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100 项与 FB-101 相关的转化医学

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100 项与 FB-101 相关的专利（医药）

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项与 FB-101 相关的新闻（医药）

2023-04-20

·PRNewswire

Parkinson's Disease Clinical Trial Pipeline Space Brims With Novel Emerging Therapies with Over 120 Key Companies Working in the Domain | DelveInsight

Parkinson's disease is a progressive disorder that is caused by the degeneration of nerve cells in the part of the brain called the substantia nigra, which controls movement. These nerve cells die or become impaired, losing the ability to produce an important dopamine chemical. Studies have shown that symptoms of Parkinson's develop in patients with an 80% or greater loss of dopamine-producing cells in the substantia nigra. Most Parkinson's patients are treated with medications to relieve the symptoms of the disease. These medications work by stimulating the remaining cells in the substantia nigra to produce more dopamine (levodopa medications) or by inhibiting some of the acetylcholine that is produced (anticholinergic medications), therefore restoring the balance between the chemicals in the brain. LAS VEGAS, April 19, 2023 /PRNewswire/ -- DelveInsight's ' Parkinson's Disease Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline Parkinson's disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Parkinson's disease pipeline domain. Key Takeaways from the Parkinson's Disease Pipeline Report DelveInsight's Parkinson's disease pipeline report depicts a robust space with 120+ active players working to develop 140+ pipeline therapies for Parkinson's disease treatment. Key Parkinson's disease companies such as Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, Inc., Modag, Annovis Bio Inc., BioVie Inc., United Neuroscience Ltd., Luye Pharma Group, AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., Nobilis Therapeutics Inc., BlueRock Therapeutics, Taiwan Mitochondrion Applied Technology Co., Ltd., NeuroDerm Ltd., Pharma Two B, Serina Therapeutics, Sun Pharma Advanced Research Company Limited, CuraSen Therapeutics, Inc., Takeda, Xoc Pharmaceuticals, Sumitomo Pharma Co., Ltd., XWPharma, Athira Pharma, Aptinyx, VistaGen Therapeutics, Inc., 1ST Biotherapeutics, Inc., Novo Nordisk A/S, AstraZeneca, MedImmune, Anavex Life Sciences Corp., Living Cell Technologies, Intra-Cellular Therapies, Inc., Alkahest, Inc., Kissei Pharmaceutical Co., Ltd., and others are evaluating new Parkinson's disease drugs to improve the treatment landscape. Promising Parkinson's disease pipeline therapies in various stages of development include Tavapadon, IkT-148009, NLY01, PT320, BIIB122, Prasinezumab, KDT-3594, AAV2-GDN, AKST4290, anle138b, ITI-214, NTCELL, Buntanetap, ANAVEX2-73, ATH-1017, NE3107, MEDI1341, AZD0328, Liraglutide, UB-312, LY03003, FB-101, AV-101, ABBV-951, NYX-458, DSP-9632P, Valiloxybate, SER-214, UCB7853, TAK-071, XC130-A10H, K0706, Talineuren, Pirepemat, Lu AF28996, WD-1603, CVN424, CST-103, NBTX-001, MSK-DA01, Aadipose-Derived Mesenchymal Stem Cells, ND0612, P2B001, 1ST-103, 1ST-105, and others. In January 2023, Neurocrine Biosciences and Voyager Therapeutics announced the formation of a new strategic collaboration to advance multiple gene therapies for the treatment of neurological diseases. The collaboration includes Voyager's preclinical, intravenously administered GBA1 gene therapy program for Parkinson's and other GBA1-mediated diseases, combining a GBA1 gene replacement payload with novel capsids from Voyager's TRACERTM (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform. In addition, Neurocrine Biosciences and Voyager have agreed to collaborate on three new gene therapy programs directed to rare CNS targets, each also leveraging Voyager's novel TRACER capsids. In October 2022, Biogen Inc. and Denali Therapeutics Inc. announced that dosing has commenced in the global Phase 3 LIGHTHOUSE study to evaluate the efficacy and safety profile of BIIB122 (DNL151), as compared to placebo in approximately 400 participants with Parkinson's disease and a confirmed pathogenic mutation in the leucine-rich repeat kinase 2 (LRRK2) gene. In September 2022, Neuron23, Inc. an early-stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases, and QIAGEN announced a collaboration to develop a companion diagnostic for Neuron23's LRRK2 inhibitor for Parkinson's disease. Request a sample and discover the recent advances in Parkinson's disease drug treatment @ Parkinson's Disease Pipeline Report The Parkinson's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Parkinson's disease drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Parkinson's disease clinical trial landscape. Parkinson's Disease Overview Parkinson's disease is a progressive disorder caused by nerve cell degeneration in the substantia nigra, a part of the brain that controls movement. These nerve cells die or become impaired, losing the ability to produce dopamine, an important chemical. According to research, symptoms of Parkinson's disease develop in patients who have an 80% or greater loss of dopamine-producing cells in the substantia nigra. Dopamine works typically in tandem with other neurotransmitters to help coordinate the millions of nerve and muscle cells involved in the movement. This balance is disrupted when there is insufficient dopamine, resulting in tremors (trembling in the hands, arms, legs, and jaw), rigidity (stiffness of the limbs), slowness of movement, and impaired balance and coordination - the hallmark Parkinson's disease symptoms. The cause of Parkinson's disease is largely unknown. However, as potential causes of the disease, theories involving oxidative damage, environmental toxins, genetic factors, and accelerated aging have been discussed. The majority of Parkinson's patients are given medications to alleviate their symptoms. These Parkinson's disease medications work by either stimulating the remaining cells in the substantia nigra to produce more dopamine (levodopa medications) or inhibiting some of the acetylcholine produced (anticholinergic medications), restoring the balance of chemicals in the brain. Find out more about drugs for Parkinson's disease @ New Parkinson's Disease Drugs A snapshot of the Parkinson's Disease Pipeline Drugs mentioned in the report: Learn more about the emerging Parkinson's disease pipeline therapies @ Parkinson's Disease Clinical Trials Parkinson's Disease Therapeutics Assessment The Parkinson's disease pipeline report proffers an integral view of Parkinson's disease emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Parkinson's Disease Pipeline Report Coverage: Global Parkinson's Disease Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Parkinson's Disease Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Parkinson's Disease Therapeutics Assessment By Route of Administration: Inhalation, Intranasal, Intravenous, Oral, Parenteral, Subcutaneous Parkinson's Disease Therapeutics Assessment By Molecule Type: Antibody, Antisense oligonucleotides, Immunotherapy, Monoclonal antibody, Peptides, Protein, Recombinant protein, Small molecule, Stem Cell, Vaccine Parkinson's Disease Therapeutics Assessment By Mechanism of Action: Dopamine D1 receptor agonists, Dopamine D5 receptor agonists, Bcr-abl tyrosine kinase inhibitors, Glucagon like peptide 1 receptor agonists, LRRK2 protein inhibitors, Alpha-synuclein inhibitors, Dopamine receptor agonists Key Parkinson's Disease Companies: Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, Inc., Modag, Annovis Bio Inc., BioVie Inc., United Neuroscience Ltd., Luye Pharma Group, AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., Nobilis Therapeutics Inc., BlueRock Therapeutics, Taiwan Mitochondrion Applied Technology Co., Ltd., NeuroDerm Ltd., Pharma Two B, Serina Therapeutics, Sun Pharma Advanced Research Company Limited, CuraSen Therapeutics, Inc., Takeda, Xoc Pharmaceuticals, Sumitomo Pharma Co., Ltd., XWPharma, Athira Pharma, Aptinyx, VistaGen Therapeutics, Inc., 1ST Biotherapeutics, Inc., Novo Nordisk A/S, AstraZeneca, MedImmune, Anavex Life Sciences Corp., Living Cell Technologies, Intra-Cellular Therapies, Inc., Alkahest, Inc., Kissei Pharmaceutical Co., Ltd., and others. Key Parkinson's Disease Pipeline Therapies: Tavapadon, IkT-148009, NLY01, PT320, BIIB122, Prasinezumab, KDT-3594, AAV2-GDN, AKST4290, anle138b, ITI-214, NTCELL, Buntanetap, ANAVEX2-73, ATH-1017, NE3107, MEDI1341, AZD0328, Liraglutide, UB-312, LY03003, FB-101, AV-101, ABBV-951, NYX-458, DSP-9632P, Valiloxybate, SER-214, UCB7853, TAK-071, XC130-A10H, K0706, Talineuren, Pirepemat, Lu AF28996, WD-1603, CVN424, CST-103, NBTX-001, MSK-DA01, Aadipose-Derived Mesenchymal Stem Cells, ND0612, P2B001, and others. Dive deep into rich insights for new drugs for Parkinson's disease treatment; visit @ Parkinson's Disease Medications Table of Contents For further information on the Parkinson's disease pipeline therapeutics, reach out @ Parkinson's Disease Drug Treatment Related Reports Parkinson's Disease Market Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease companies, including Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, among others. Parkinson's Disease Epidemiology Forecast Parkinson's Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and Parkinson's disease epidemiology trends. Dementia with Lewy Bodies Pipeline Dementia with Lewy Bodies Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key dementia with Lewy bodies companies, including Eli Lilly and Company, CuraSen Therapeutics, Inc., Athira Pharma, Aptinyx Inc., among others. Dementia with Lewy Bodies Epidemiology Forecast Dementia with Lewy Bodies Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted dementia with Lewy bodies epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Dementia Market Dementia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key dementia companies, including Hoffmann-La Roche, Forest Laboratories, Inc., Janssen Pharmaceuticals, Inc., Merck and Co., Inc., Novartis, among others. Parkinson's Disease-Related Dementia Market Parkinson's Disease-Related Dementia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease-related dementia companies, including AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., among others. 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It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

临床3期

2022-10-21

·药明康德

一周盘点 | 利用“合成致死”的蛋白降解剂、治疗卵巢功能不全的外泌体疗法临床试验在即...

▎药明康德内容团队编辑本期看点1. 卡纳万病（Canavan）基因疗法BBP-812的1/2期临床试验的初步数据积极。2. 杜氏肌营养不良症基因疗法SGT-001在用药一年后显著改善患者的行走功能。3. 首个用于治疗原发性卵巢功能不全的同种异体外泌体疗法EV-Pure提交IND申请获FDA批准。4. 利用“合成致死”效应的潜在“first-in-class”强效SMARCA2蛋白降解剂PRT3789提交IND申请获FDA批准。药明康德内容团队整理BBP-812：公布1/2期临床试验数据BridgeBio Pharma公布了其用于治疗卡纳万病的研究性静脉注射AAV9基因疗法BBP-812的1/2期临床试验数据。BBP-812能够将ASPA基因的功能拷贝递送到全身并进入大脑，从而可能根治疾病。卡纳万病是一种由编码天冬氨酸酶的ASPA基因突变引起的超罕见、可致残或致命的疾病。大多数儿童无法爬行、行走、坐着或说话，在幼时就会面临死亡的威胁。天冬氨酸酶活性的缺乏会导致N-乙酰天冬氨酸（NAA）的积累，并最终导致对髓鞘的毒性。目前，该病尚无获批的疗法。此次所公布3例患者的药效学数据显示，接受BBP-812治疗3-6个月后，患者脑脊液、脑白质及尿液中的NAA持续且大幅度的减少，这表明此在研疗法能够产生功能性的ASPA酶。新闻稿指出，如果临床试验成功，BBP-812将成为出生时患有卡纳万病的儿童的首个治疗选择。此前，BBP-812已被FDA授予快速通道资格、罕见儿科疾病认定和孤儿药资格。BBP-812还被欧洲药品管理局（EMA）授予孤儿药资格。SGT-001：公布1/2期临床试验数据Solid Biosciences公布了其基因疗法SGT-001在1/2期临床试验中的更新的数据。SGT-001能通过新型腺相关病毒（AAV）载体，将合成微抗肌萎缩蛋白（microdystrophin）的转基因递送至患者体内，治疗杜氏肌营养不良症（DMD）的遗传根源。该临床试验结果显示，给药一年后，接受SGT-001治疗患者的行走功能得到了改善。SV95C是一项由可穿戴设备测量的指标，用于评估患者日常生活中最快的自发进行步伐，被EMA认定为五岁及以上DMD患者临床试验中可用的次要终点。在该临床试验中，接受SGT-001的患者的SV95C水平与基线时相比改善了8.8%-9.5%，与疾病自然史数据相比改善了23.9%-24.6%，与对照组患者相比改善了26.0%-26.7%。此前，SGT-001已被FDA授予罕见儿科疾病认定、快速通道资格，并被FDA和EMA授予孤儿药资格。EV-Pure：IND申请获得FDA许可EV-Pure是一种同种异体外泌体疗法，适用于原发性卵巢功能不全的女性。原发性卵巢功能不全导致约1%的40岁以下的女性不孕，这种疾病的并发症还包括早期骨质疏松症、早发性心脏病，以及无法怀孕引起的抑郁症和焦虑症等严重问题，目前尚无获批的疗法。根据新闻稿，该疗法是在该适应症中首个提交IND申请获得FDA批准的同种异体外泌体疗法。PRT3789：IND申请获得FDA许可PRT3789是一种潜在“first-in-class”的强效选择性SMARCA2蛋白降解剂。SMARCA2和SMARCA4参与多个致癌过程，包括允许DNA转录为RNA的过程。在某些特定的癌症（包括非小细胞肺癌）中，约10%-20%的患者携带SMARCA4突变。由于SMARCA2或SMARCA4的活性是肿瘤细胞生长所必需的，因此具SMARCA4缺陷的癌细胞高度依赖SMARCA2生存。在SMARCA4缺陷的癌症中降解SMARCA2蛋白被认为会产生很强的“合成致死”效应，导致癌细胞死亡，同时不伤害拥有SMARCA4表达的正常细胞。INO-3107：公布1/2期临床试验数据INOVIO公布了DNA疗法INO-3107临床1/2期试验数据。INO-3107用于治疗成人HPV 6和HPV 11相关的复发性呼吸道乳头状瘤病（RRP）。此疗法旨在引发针对HPV 6和HPV 11的靶向T细胞反应，以杀死受感染的细胞，使乳头状瘤消退，并可能清除或降低病毒水平，从而防止或减缓新乳头状瘤生长。此次公布的研究结果显示，与前一年相比，使用INO-3107治疗的21名受试者中，有16名（76%）手术干预次数降低，6名受试者在试验期间无需手术干预，该临床终点的改善具有统计学意义。此外，INO-3107具有良好的耐受性和免疫原性。2020年，FDA授予INO-3107孤儿药资格，用于治疗RRP。KIO-100：公布1期临床试验数据Kiora Pharmaceuticals公布了其二氢乳清酸脱氢酶（DHODH）抑制剂KIO-100用于治疗葡萄膜炎的1期试验结果。葡萄膜炎是一种T细胞介导的眼内炎症性疾病。结果显示，玻璃体内注射KIO-100能以剂量依赖性方式减少眼内炎症，并在研究期间显著提高视力。最高剂量组在第14天时在ETDRS视力表字母评估视力方面的改善达三行（相当于分辨率加倍），并保持稳定直到第28天。此外，该药物耐受性良好，对眼内组织没有严重的副作用或其他不良事件。FB-101：公布1期临床试验数据1ST Biotherapyatetics公布了其c-Abl抑制剂FB-101的1期临床数据。FB-101正在被开发用于治疗帕金森病，并可能用于与α-突触核蛋白病相关的其他神经退行性疾病。FB-101的1期临床试验的单剂量递增部分在健康的成人受试者中完成。结果显示，口服FB-101在所有队列中均具有良好的耐受性和安全性。FB-101的血液浓度呈剂量依赖性，未观察到年龄或性别差异。值得注意的是，从受试者身上收集的脑脊液证实了FB-101具有强烈的血脑屏障渗透性。OTT166：公布1b期临床试验数据OcuTerra Therapeutics公布了其OTT166眼药水用于治疗糖尿病视网膜病变的1b期临床试验数据。OTT166为一在研选择性小分子RGD序列抑制剂。研究结果显示，OTT166的耐受性良好，没有眼部或全身毒性的证据，没有报告药物相关的严重不良事件，并且患者在整体试验中所展现的最佳矫正视力（BCVA）稳定。在研究结束时，两组的平均和中位视网膜厚度（CRT）都有临床意义的减少（分别为-22/-28微米），37%的患者被认为获得了临床缓解。FAP-2286：公布1期临床试验数据Clovis Oncology公布了其靶向成纤维细胞活化蛋白（FAP）的放射治疗候选药物FAP-2286在实体肿瘤中的1期临床试验数据。FAP在多种肿瘤类型中表达，是具有前景的癌症治疗靶点。此次公布的数据显示，未观察到与177Lu-FAP-2286相关的严重不良事件、治疗中断或死亡。目前已在1名患者中确认了部分缓解（PR），并在1名患者中确认了疾病稳定（SD）。此外，FAP-2286在一系列实体肿瘤中展现出高肿瘤摄取率，并能长时间留存。DB-OTO：IND申请获得FDA许可DB-OTO是针对由编码otoferlin的OTOF基因突变引起的严重先天性听力损失的基因疗法。正常的otoferlin使耳朵的感觉细胞响应声音刺激而释放神经递质，从而激活听觉神经元。无功能性的otoferlin则无法将听觉信号传递到大脑。DB-OTO使用AAV载体将OTOF基因的健康拷贝传递至耳蜗毛细胞，以此恢复长期的生理听力。2021年，DB-OTO获得了FDA的孤儿药资格和罕见儿科疾病认定。TK-8001：1/2期临床试验完成首例患者给药TK-8001是一种TCR-T细胞疗法，对黑色素瘤相关抗原基因A1（MAGE-A1）具有特异性，适用于实体瘤患者。MAGE-A1是侵袭性癌症的标志，与不良的临床预后有关，且有大量证据表明MAGE-A1是肿瘤发生的潜在驱动因素。与使用来自人类供体的TCR-T细胞相比，TK-8001在体外表现出连续杀伤活性，并具有增强的体内抗肿瘤活性。ITIL-306：1期临床试验完成首例患者给药ITIL-306是一种自体肿瘤浸润淋巴细胞（TIL）疗法，采用Instil Bio专有的CoStAR分子设计，该分子被叶酸受体α（FRα）激活，在肿瘤微环境中提供强大的共刺激信号。CoStAR建立在天然TIL的关键优势之上，以增强肿瘤微环境中TIL的细胞因子释放，细胞溶解活性和TIL的增殖。先前发表的临床前数据表明，CoStAR-T细胞能够在没有外源性IL-2的情况下增强体内肿瘤控制，能够支持ITIL-306的1期研究中不含IL-2的治疗方案。KYV-101：向FDA提交IND申请KYV-101是一款处于临床阶段、靶向CD19的CAR-T细胞疗法，其特性尤其适用于治疗B细胞驱动的自身免疫性疾病，如狼疮性肾炎、系统性硬化症和炎性肌病。临床前研究证明，该疗法可在疾病模型的血液循环和组织中，诱导深度和完全的B细胞耗竭，产生显著疗效。AT-02：1期临床试验完成首例患者给药AT-02是泛淀粉样蛋白去除（PAR）肽技术与IgG1抗体的融合蛋白，正在开发用于治疗系统性淀粉样变性（SA）。AT-02能够以亚摩尔亲和力与ATTR和AL淀粉样蛋白结合，促进巨噬细胞介导的淀粉样蛋白吞噬作用。体内临床前数据显示，在人AL淀粉样瘤和系统性AA淀粉样变性小鼠模型中，淀粉样蛋白显著减少。与未经治疗的动物相比，AT-02治疗患有进行性系统性AA淀粉样变性的小鼠，可将局部心脏淀粉样蛋白减少约50%，并显著减少肾和肝淀粉样蛋白和器官损伤。LB-100：在西班牙开启1b/2期临床试验LB-100是一种抑制丝氨酸/苏氨酸磷酸酶的临床候选药物。丝氨酸/苏氨酸磷酸酶无处不在，并一直被认为是抗癌药物的潜在重要靶点。然而，由于这些酶的多功能性，人们普遍认为抑制这些磷酸酶的药物会产生极大毒性，无法将它们发展为抗癌治疗。但Lixte Biotechnology Holdings的临床前研究表明，LB-100单药可抑制一系列人类癌症，当与标准细胞毒杀药物和/或放射疗法结合使用时，LB-100可增强其对血液学和实体瘤癌症的有效性，且不会增强毒性。此外，在癌症动物模型中以非常低的剂量给药时，LB-100显著提高了PD-1阻断剂的有效性。这项临床试验旨在检视LB-100与阿霉素联合治疗软组织肉瘤的疗效和安全性。药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息参考资料：[1] Decibel Therapeutics Receives FDA Clearance of IND Application for DB-OTO, a Gene Therapy Product Candidate Designed to Provide Hearing to Individuals with Otoferlin-Related Hearing Loss. Retrieved October 17, 2022, from https://ir.decibeltx.com/news-releases/news-release-details/decibel-therapeutics-receives-fda-clearance-ind-application-db[2] Kiora Pharmaceuticals Announces Publication of Phase 1 Trial Results for KIO-100 as a Treatment for Ocular Inflammatory Disease. Retrieved October 17, 2022, from https://ir.kiorapharma.com/news-events/press-releases/detail/165/kiora-pharmaceuticals-announces-publication-of-phase-1[3] Solid Biosciences Reports Additional Preclinical Data Demonstrating that its Novel Capsid, AAV-SLB101, Provides Superior Transduction Efficiency and Enhanced Distribution to Skeletal Muscle. Retrieved October 17, 2022, from https://www.solidbio.com/about/media/press-releases/solid-biosciences-reports-additional-preclinical-data-demonstrating-that-its-novel-capsid-aav-slb101-provides-superior-transduction-efficiency-and-enhanced-distribution-to-skeletal-muscle[4] BridgeBio Pharma Presents Updated Positive Data from its BBP-812 Canavan Disease Gene Therapy Program at the 51st Annual Meeting of the Child Neurology Society. Retrieved October 13, 2022, from https://bridgebio.com/news/bridgebio-pharma-presents-updated-positive-data-from-its-bbp-812-canavan-disease-gene-therapy-program-at-the-51st-annual-meeting-of-the-child-neurology-society/[5] INOVIO Reports Positive Interim Phase 1/2 Results for INO-3107 for the Treatment of Recurrent Respiratory Papillomatosis. Retrieved October 13, 2022, from https://ir.inovio.com/news-releases/news-releases-details/2022/INOVIO-Reports-Positive-Interim-Phase-12-Results-for-INO-3107-for-the-Treatment-of-Recurrent-Respiratory-Papillomatosis/default.aspx[6] T-knife Therapeutics Announces Dosing of First Patient with TK-8001 in the IMAG1NE Phase 1/2 Clinical Trial. Retrieved October 20, 2022, from https://www.t-knife.com/news/press-releases/10-20-22/[7] Instil Bio Announces First Patient Dosed with ITIL-306, our First Engineered TIL Therapy Using the CoStAR Platform Targeting Folate Receptor Alpha (FRα), in Non-Small Cell Lung Cancer. Retrieved October 18, 2022, from https://ir.instilbio.com/news-releases/news-release-details/instil-bio-announces-first-patient-dosed-itil-306-our-first[8] NKGen Biotech Receives IND Clearance from FDA for SNK02 Allogeneic Natural Killer Cell Therapy for Solid Tumors. Retrieved October 17, 2022, from https://nkgenbiotech.com/nkgen-biotech-receives-ind-clearance-from-fda-for-snk02-allogeneic-natural-killer-cell-therapy-for-solid-tumors/[9] Kyverna Therapeutics Submits IND for Novel CAR T-Cell Therapy to Treat Lupus Nephritis. Retrieved October 18, 2022, from https://kyvernatx.com/press-releases/kyverna-therapeutics-submits-ind-for-novel-car-t-cell-therapy-to-treat-lupus-nephritis/[10] FDA Approves First IND of its Kind for Allogenic Exosomal Treatment of Female Reproductive Disorder - Primary Ovarian Insufficiency. Retrieved October 18, 2022, from https://www.prnewswire.com/news-releases/fda-approves-first-ind-of-its-kind-for-allogenic-exosomal-treatment-of-female-reproductive-disorder---primary-ovarian-insufficiency-301651645.html[11] Attralus Announces First Subject Enrolled in Phase 1 Clinical Trial of AT-02, Its Lead Pan-Amyloid Removal Therapeutic Candidate. Retrieved October 17, 2022, from https://attralus.com/press-releases/attralus-announces-first-subject-enrolled-in-phase-1-clinical-trial-of-at-02-its-lead-pan-amyloid-removal-therapeutic-candidate[12] Prelude Therapeutics Receives FDA Clearance of IND for PRT3789, a Potent and Selective First-in-Class SMARCA2 Protein Degrader. Retrieved October 18, 2022, from https://investors.preludetx.com/news-releases/news-release-details/prelude-therapeutics-receives-fda-clearance-ind-prt3789-potent[13] 1ST Biotherapeutics, Inc., Announces Positive Phase I Data of c-Abl Inhibitor in Neurodegeneration. Retrieved October 18, 2022, from https://www.globenewswire.com/news-release/2022/10/18/2536020/0/en/1ST-Biotherapeutics-Inc-Announces-Positive-Phase-I-Data-of-c-Abl-Inhibitor-in-Neurodegeneration.html[14] OcuTerra Reports Publication of OTT166 Safety and Biological Activity Data from Phase 1b Study in Diabetic Eye Disease. Retrieved October 19, 2022, from https://www.businesswire.com/news/home/20221019005275/en/OcuTerra-Reports-Publication-of-OTT166-Safety-and-Biological-Activity-Data-from-Phase-1b-Study-in-Diabetic-Eye-Disease[15] LIXTE BIOTECHNOLOGY ANNOUNCES APPROVAL OF A PHASE 1B/2 RANDOMIZED TRIAL OF DOXORUBICIN +/-LB-100 IN ADVANCED SOFT TISSUE SARCOMAS TO BE CONDUCTED BY THE SPANISH SARCOMA GROUP. Retrieved October 13, 2022, from https://ir.lixte.com/news-events/press-releases/detail/83/lixte-biotechnology-announces-approval-of-a-phase-1b2[16] Clovis Oncology Highlights Updated LuMIERE Phase 1 Data Of Targeted Radiotherapy Candidate FAP-2286 At The 35th Annual EANM Congress. Retrieved October 17, 2022, from https://ir.clovisoncology.com/investors-and-news/news-releases/press-release-details/2022/Clovis-Oncology-Highlights-Updated-LuMIERE-Phase-1-Data-of-Targeted-Radiotherapy-Candidate-FAP-2286-at-the-35th-Annual-EANM-Congress/default.aspx免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

蛋白降解靶向嵌合体基因疗法小分子药物细胞疗法免疫疗法

100 项与 FB-101 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
帕金森病	临床1期	美国	1st Biotherapeutics, Inc.初创企业	2019-10-14
肌萎缩侧索硬化	临床1期	美国	1st Biotherapeutics, Inc.初创企业	-

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04165837 (PipelineReview) 人工标引	临床1期	神经变性	-	FB-101	憲網鹽簾選憲鹹網窪選(繭網積淵鑰繭鬱觸蓋構) = The SAD portion of the Phase I clinical trial of FB-101 was completed in Baltimore, MD, USA, and demonstrated that oral administration of FB-101 was well-tolerated across all cohorts with favorable safety results. No clinically significant findings were observed on safety measures including laboratory findings, vital signs, and ECGs at any dose. 構範齋鬱網鬱襯夢選膚 (範窪簾窪鹹憲構鬱願蓋 )	积极	2022-10-18
				Placebo