作者: Boye, Shannon E. ; Dizhoor, Alexander M. ; Hauswirth, William W. ; Peshenko, Igor V. ; Peterson, James J. ; Choudhury, Shreyasi ; Ruan, Qing ; McCullough, K. Tyler ; Ding, Xi-Qin ; Min, Seok Hong ; Olshevskaya, Elena V. ; Boye, Sanford L. ; Zhang, Zhonghong

Mutations in GUCY2D are the cause of Leber congenital amaurosis type 1 (LCA1). GUCY2D encodes retinal guanylate cyclase-1 (retGC1), a protein expressed exclusively in outer segments of photoreceptors and essential for timely recovery from photoexcitation. Recent clinical data show that, despite a high degree of visual disturbance stemming from a loss of cone function, LCA1 patients retain normal photoreceptor architecture, except for foveal cone outer segment abnormalities and, in some patients, foveal cone loss. These results point to the cone-rich central retina as a target for GUCY2D replacement. LCA1 gene replacement studies thus far have been conducted in rod-dominant models (mouse) or with vectors and organisms lacking clinical translatability. Here we investigate gene replacement in the Nrl(-/-) Gucy2e(-/-) mouse, an all-cone model deficient in retGC1. We show that AAV-retGC1 treatment fully restores cone function, cone-mediated visual behavior, and guanylate cyclase activity, and preserves cones in treated Nrl(-/-) Gucy2e(-/-) mice over the long-term. A novel finding was that retinal function could be restored to levels above that in Nrl(-/-) controls, contrasting results in other models of retGC1 deficiency. We attribute this to increased cyclase activity in treated Nrl(-/-) Gucy2e(-/-) mice relative to Nrl(-/-) controls. Thus, Nrl(-/-) Gucy2e(-/-) mice possess an expanded dynamic range in ERG response to gene replacement relative to other models. Lastly, we show that a candidate clinical vector, AAV5-GRK1-GUCY2D, when delivered to adult Nrl(-/-) Gucy2e(-/-) mice, restores retinal function that persists for at least 6 months. Our results provide strong support for clinical application of a gene therapy targeted to the cone-rich, central retina of LCA1 patients.

项与 AAV8-GUCY2D gene therapy (Fondazione Telethon) 相关的新闻（医药）

2023-04-25

·BioSpace

Atsena Therapeutics Announces Positive 6-month Data from Ongoing Phase I/II Clinical Trial of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)

ATSN-101 demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious adverse events 6 months post treatment Data presented at ARVO 2023 DURHAM, N.C., April 25, 2023 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced positive 6-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). The data were presented by Christine Nichols Kay, MD, Clinical Ophthalmology Advisor for Atsena, at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting. “We’re encouraged by the clinically meaningful improvements in vision ATSN-101 has demonstrated at the highest dose 6 months post treatment, as well as the favorable safety pro our subretinal gene therapy,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “The latest data reinforce our confidence in the potential of ATSN-101 to improve vision in patients with GUCY2D-associated LCA1, which results in early and severe vision impairment or blindness and lacks an approved treatment. We look forward to reporting 12-month data later this year and are exploring options to advance ATSN-101 into a pivotal trial.” In the Phase I/II trial, 15 patients, including three pediatric patients, received ATSN-101 via subretinal delivery. Three adult cohorts (N=3 each) were treated with three ascending doses. Six additional patients received the high dose in a dose expansion phase. Among patients who received the high-dose treatment (N=9), the mean (SE) change from baseline in retinal sensitivity by dark-adapted full-field stimulus testing (FST) was significantly greater in treated eyes compared with untreated eyes at Day 28 and all subsequent follow-up visits. Two high-dose patients demonstrated best corrected visual acuity (BCVA) improvement greater than 0.3 logMAR, and no treated eyes had a decrease in BCVA. Of the five high-dose patients who were tested with the MLMT mobility test, four patients demonstrated either a maximum MLMT score of 6 or a ≥2 level improvement, compared to baseline when available or to the untreated fellow eye, at one or more post-treatment visits. To date, no drug-related serious adverse events have been reported and ocular inflammation has been infrequent, minimal, and reversible with steroid treatment. About GUCY2D-associated Leber congenital amaurosis (LCA1) LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1. About Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children. Interim safety and efficacy data has demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. The company’s additional pipeline of proprietary assets includes ATSN-201, an investigational gene therapy that leverages one of the company’s novel spreading capsids, AAV.SPR, for the treatment of X-linked retinoschisis (XLRS), and ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com Business Contact: info@atsenatx.com

临床结果基因疗法临床研究

2023-02-08

·BioSpace

Atsena Therapeutics to Present Positive Interim Encore Data from the Phase I/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital Amaurosis (LCA1) at the 46th Annual Macula Society Meeting

DURHAM, N.C., Feb. 08, 2023 (GLOBE NEWSWIRE) --Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that positive interim data from the ongoing Phase I/II clinical trial of ATSN-101 will be presented at the 46th Annual Macula Society Meeting, which is being held February 15-18 in Miami, FL. This is an encore presentation of data that were presented as a late-breaker at the American Academy of Ophthalmology 2022 Annual Meeting. ATSN-101, Atsena’s lead investigational gene therapy product, is being evaluated in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). LCA1 is a monogenic eye disease that disrupts the function of the retina and results in early and severe vision impairment or blindness. “We are pleased to have the opportunity to share the positive interim safety and efficacy data from our ongoing Phase I/II trial of ATSN-101 with the retinal disease community at the upcoming Macula Society Annual Meeting,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “Subretinal delivery of ATSN-101 was well tolerated and demonstrated clinically meaningful improvements in vision in patients treated with the highest dose. With no approved treatments available for patients with LCA1, we are honored to be at the forefront of research for this inherited retinal disease and look forward to reporting additional data from our Phase I/II trial at medical meetings later this year.” Details of the presentation are as follows: Title: Safety and Efficacy of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1) Section: Session III - Inherited Retinal Disorders Date / Time: Wednesday, February 15, 6:16 p.m. EST Location: Fontainebleau Miami Beach Presenter: Christine Nichols Kay, MD, Atsena Therapeutics About GUCY2D-associated Leber congenital amaurosis (LCA1) LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1. About Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for a form of LCA, one of the most common causes of blindness in children. Positive interim data presented at the American Academy of Ophthalmology 2022 Annual Meeting demonstrated that subretinal delivery of ATSN-101 was well tolerated and patients treated with the highest dose saw clinically meaningful improvements in vision. The company’s additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com Business Contact: info@atsenatx.com

基因疗法临床研究

2022-10-16

·药明康德

一周盘点 | 多项基因疗法公布积极数据，鼻内阿尔兹海默病疗法临床试验在即...

▎药明康德内容团队编辑本期看点１. 治疗重症肌无力的小分子疗法NMD670的1/2a期临床试验获积极数据，高达50%的患者达预先指定的响应标准。２. 治疗达农病的基因疗法RP-A501的1期临床试验数据亮眼，所有患者均获持续的心脏功能改善。３. 眼科基因疗法botaretigene sparoparvovec的1/2期临床试验的初步数据积极，多项指标持续改善。4. 靶向IL-17的口服抑制剂DC-806在治疗银屑病的1期临床试验中获积极结果，高剂量组皮损面积及严重程度下降近50%。药明康德内容团队整理NMD670：公布1/2a期临床试验数据NMD Pharma公布了其用于治疗神经肌肉疾病潜在“first-in-class”小分子骨骼肌特异性氯离子通道（ClC-1）抑制剂NMD670的1/2a期临床试验的积极数据。近期，NMD670还被FDA授予了治疗重症肌无力（MG）的孤儿药资格。MG是一种罕见的慢性自身免疫性疾病，其中IgG抗体会破坏神经和肌肉之间的联系，导致虚弱和可能致命的肌肉无力。MG通常会影响控制眼睛和眼睑、面部表情、咀嚼、吞咽和说话的肌肉，在大多数患者中，它最终会影响大多数的骨骼肌。超过85%的MG患者在18个月内会进展为全身性的MG，当影响负责呼吸的肌肉时可能会危及生命。NMD Pharma已证明ClC-1抑制可以增强神经肌肉传递并恢复骨骼肌功能，这种新的治疗方法已在MG和一系列其他神经肌肉疾病的动物模型中证明了令人信服的临床前疗效数据。此次公布的临床数据显示，NMD670在健康志愿者和患者中是安全且耐受性良好的。给予单剂量的NMD670与定量重症肌无力评分的临床显著改善相关，高达50%的患者符合预先指定的响应标准。电生理学终点表明患者神经肌肉传递的恢复与肌肉力量和功能的增加有关。RP-A501：公布1期临床试验数据Rocket Pharmaceuticals公布了其基因疗法RP-A501治疗达农病的1期临床试验的积极更新。来自儿科和成人队列患者的最新安全性和有效性数据显示，RP-A501通常具有良好的耐受性并具有持续的临床益处。达农病是一种罕见的X连锁遗传性疾病，由编码溶酶体相关膜蛋白2（LAMP2）的基因突变引起。LAMP2基因突变将导致自噬体和糖原的积累，特别是在心肌和其他组织中，最终会导致心力衰竭。男性患者通常在青春期或刚成年时期死亡。该病唯一有效的治疗选择是心脏移植，但它会导致大量并发症，因此不被认为是治愈性的。目前尚无可用于治疗达农病的特定疗法。RP-A501是一种针对达农病的研究性基因治疗产品。根据新闻稿，该疗法也是单基因心力衰竭的首个潜在基因疗法，由重组腺相关血清型9（AAV9）衣壳装载着功能性版本的人类LAMP2转基因组成。1期试验结果显示，在6-36个月的随访中，所有接受密切监测免疫调节方案的成人和儿童患者的纽约心功能（NYHA）分级均有改善（从II级到I级），患者在常规活动期间不再出现心脏病症状或在活动中出现与心脏相关的限制。botaretigene sparoparvovec（AAV-RPGR）：公布1/2期临床试验的初步数据强生（Johnson & Johnson）旗下杨森（Janssen）与MeiraGTx共同宣布，其在研基因疗法botaretigene sparoparvovec（曾用名AAV-RPGR），在临床1/2期试验MGT009中获得积极初步结果。数据分析显示此基因疗法具有良好的安全性，并改善遗传性X连锁视网膜色素变性（XLRP）病患的视力。Botaretigene sparoparvovec是一款专一性靶向眼睛RPGR基因变异的疗法。通过一次性的视网膜下注射，由腺相关病毒（AAV）递送具功能性的RPGR基因以弥补视网膜细胞的损失，预计可维持并具潜力恢复XLRP患者的视力。此疗法获得美国FDA的快速通道资格与孤儿药资格，以及欧洲药品管理局（EMA）所颁布的优先药品资格与先进治疗药物资格。在1/2期试验的剂量爬坡与扩增阶段，与随机分配不接受治疗的对照组相比，接受botaretigene sparoparvovec基因疗法6个月后的患者，在视觉功能、视网膜灵敏度与功能性视觉三项指标上，皆呈现功能性显著且持续的增加。DC-806：公布1期临床试验数据DICE Therapeutics公司宣布其靶向IL-17的口服抑制剂DC-806在治疗银屑病的1期临床试验中取得了积极结果。该试验是一项随机、双盲、安慰剂对照临床研究。银屑病是一种常见的慢性炎症性皮肤病，其主要病症包括瘙痒、出血和疼痛，并且患者皮肤时常出现红斑和鳞屑。银屑病的治疗周期长、病情易反复、甚至可能导致容貌受损，给患者造成了沉重的心理负担，然而目前对于这种疾病并没有治愈方法。既往研究表明，白介素-17（IL-17）在银屑病的病理机制中起着关键作用，使其成为了银屑病治疗药物的主要研发靶点之一。数据显示，治疗4周后，高剂量组的银屑病皮损面积和严重程度指数（PASI）比基线时减少的平均百分比为43.7%，而安慰剂组为13.3%，这些数据均具有统计学意义。此外，对生物标志物的分析结果表明，高剂量和低剂量的DC-806都展示出了生物学活性，药物起效迅速，并且其药物靶点作用具有剂量依赖性。在所有的剂量组中，DC-806的安全性和耐受性均良好，所有治疗伴发不良反应（TEAEs）都被归类为轻度或中度，观察到的TEAEs的频率、严重程度或类型没有剂量依赖的趋势。ATSN-101（SAR439483）：公布1/2期临床试验数据Atsena Therapeutics公布了其用于治疗GUCY2D相关的Leber先天性黑蒙（LCA1）的在研基因疗法ATSN-101（SAR439483）的1/2期临床试验的积极结果。LCA1是一种由GUCY2D基因突变引起的单基因眼病，会破坏视网膜的功能，导致早期和严重的视力障碍或失明。目前尚无批准的LCA1治疗方法。数据表明，视网膜下给药ATSN-101具有良好的耐受性，接受最高剂量治疗的患者在视网膜敏感性、最佳矫正视力等视觉功能方面有临床意义的改善。安全性方面，没有报告与药物相关的严重不良事件，大多数治疗中出现的不良事件是轻微和短暂的。SLN124：公布1期临床试验的初步数据Silence Therapeutics公布了其靶向TMPRSS6基因的siRNA疗法SLN124的1期研究的初步结果。SLN124是一种基因“沉默”疗法，旨在暂时“沉默”TMPRSS6基因，一种阻止肝脏产生控制体内铁水平的特定激素——铁调素的基因。随着铁调素的增加，血液中的铁含量会随之降低，然后产生更多健康的红细胞，从而改善贫血。SLN124已获得β地中海贫血的罕见儿科疾病资格和孤儿药资格，以及真性红细胞增多症孤儿药资格。该1期临床研究的主要目标是评估SLN124皮下给药在α/β-地中海贫血患者中的安全性和耐受性。结果显示，在24例患有非输血依赖性地中海贫血的成年人中，单次给药后未发现严重的不良事件、与SLN124相关的严重治疗紧急不良事件（TEAE）或导致退出临床试验的TEAE。此外，未观察到剂量限制性毒性或药物相关的肝损伤。INBRX-101：公布1期临床试验数据Inhibrx公布了其优化的重组人AAT-Fc融合蛋白INBRX-101用于因α-1抗胰蛋白酶缺乏症（AATD）引起的肺气肿患者的1期临床试验的支气管肺泡灌洗液检测结果。结果显示，测试的所有AATD患者的支气管肺泡灌洗液样本中均检测到了INBRX-101。AATD是一种遗传性孤儿病，其特征是α-1抗胰蛋白酶（AAT）蛋白水平不足，这会导致肺组织和功能丧失，并降低预期寿命。使用血浆衍生AAT的增强疗法是目前的护理标准，但不能将患者维持在正常的AAT范围内，需要每周进行一次静脉给药。INBRX-101是一种精准设计的重组人AAT-Fc融合蛋白，旨在使AATD患者安全地达到和维持健康人的AAT水平，与血浆衍生AAT疗法相比，将有机会减少给药频率。目前，INBRX-101已获得了FDA授予的治疗AATD的孤儿药资格。KUR-101：公布1期临床试验的初步数据atai Life Sciences公布了其开发的KUR-101用于治疗阿片类药物使用障碍（OUD）的1期临床试验的初步数据。KUR-101是一种口服的非典型的阿片受体调节剂，具有独特的药理作用，可能使其长期使用更安全。氘化修饰改善了KUR-101的药代动力学（PK）和整体安全性，同时降低了剂量要求。初步结果显示，KUR-101的安全性及耐受性良好，且具有不受食物影响的PK曲线。在单次剂量递增试验中，没有报告严重或严重的不良事件，大多数与治疗相关的不良事件是轻微的。KUR-101治疗后的呼吸频率的变化与接受安慰剂治疗的患者相当，并且在不同剂量之间具有可比性。foralumab（NI-0401）：计划于2023年向FDA提交IND申请Tiziana Life Sciences宣布，将在完成所要求的毒理学研究后于2023年向FDA提交其鼻内给药foralumab用于治疗阿尔茨海默病的IND申请。Foralumab（之前被称为为NI-0401）是一种全人源抗CD3单克隆抗体，在健康志愿者和克罗恩病患者中静脉注射给药后，细胞因子的释放减少。在人源化小鼠模型中，口服foralumab可以调节T细胞的免疫反应并增强调节性T细胞，从而为治疗炎症和自身免疫性疾病提供益处，而通常不会发生与肠外mAb治疗相关的潜在不良事件。BBP-398：1/2期临床试验完成首例患者给药BridgeBio Pharma宣布其所开发的SHP2抑制剂BBP-398，与安进（Amgen）开发的sotorasib联用疗法，完成首例1/2期临床试验患者的给药。这些患者为带有KRAS G12C突变的晚期非小细胞肺癌（NSCLC）患者。SHP2是一种蛋白酪氨酸磷酸酶，连接生长因子、细胞因子、整合素信号与下游RAS/MAPK信号通路，调节细胞增殖和生存。SHP2活性过高是多种癌症发生的重要因素，它也是对多种靶向疗法产生耐药性的机制，并且可以抑制抗肿瘤免疫反应。Sotorasib是首个用于治疗携带KRAS G12C突变的局部晚期或转移性NSCLC患者的靶向治疗。临床前研究结果表明，在KRAS G12C突变的NSCLC细胞系衍生的异种移植模型中，BBP-398与sotorasib的组合具有协同功效。如果该试验成功，该组合疗法有望解决KRAS G12C突变的NSCLC患者严重未满足的需求。Zetomipzomib：IND申请获得FDA许可Kezar Life Sciences宣布，其用于治疗自身免疫性肝炎（AIH）的潜在“first-in-class”选择性免疫蛋白酶体抑制剂zetomipzomib的IND申请获FDA许可。Zetomipzomib是一款潜在“first-in-class”、具选择性的免疫蛋白酶体抑制剂。其具有广泛的疗效潜力可治疗许多种类的自身免疫疾病。在临床前的动物实验中，zetomipzomib被证实可以在许多自身免疫疾病的动物模型中，可以选择性地抑制免疫蛋白酶体，因此可以广泛性地抑制发炎反应。临床1期试验数据显示，此药物在严重、慢性免疫疾病的开发上具有良好的安全性与耐受性。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] Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting. Retrieved October 3, 2022, from https://www.janssen.com/janssen-announces-late-breaking-data-two-gene-therapy-programs-american-academy-ophthalmology-2022[2] Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial for RP-A501 in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022. Retrieved September 30, 2022, from https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-positive-updates-phase-1-0[3] Atsena Therapeutics Announces Positive Results from Phase I/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital Amaurosis (LCA1). Retrieved October 3, 2022, from https://atsenatx.com/press-release/atsena-therapeutics-announces-positive-results-from-phase-i-ii-clinical-trial-of-atsn-101-for-the-treatment-of-gucy2d-associated-leber-congenital-amaurosis-lca1/[4] Freeline Initiates Dosing of Second Cohort in MARVEL-1 Trial of FLT190 Gene Therapy Candidate for People with Fabry Disease. Retrieved October 4, 2022, from https://www.freeline.life/investors/newsroom/freeline-initiates-dosing-of-second-cohort-in-marvel-1-trial-of-flt190-gene-therapy-candidate-for-people-with-fabry-disease/[5] Silence Therapeutics Announces Preliminary Single Dose Results from SLN124 Phase 1 Study in Patients with Thalassemia. Retrieved September 29, 2022, from https://silence-therapeutics.com/investors/press-releases/press-releases-details/2022/Silence-Therapeutics-Announces-Preliminary-Single-Dose-Results-from-SLN124-Phase-1-Study-in-Patients-with-Thalassemia/default.aspx[6] Avidity Biosciences Announces the Phase 1/2 FORTITUDE™ Trial of AOC 1020 in Adults with Facioscapulohumeral Muscular Dystrophy. Retrieved September 29, 2022, from https://www.prnewswire.com/news-releases/avidity-biosciences-announces-the-phase-12-fortitude-trial-of-aoc-1020-in-adults-with-facioscapulohumeral-muscular-dystrophy-301636385.html[7] Alzamend Neuro Submits IND Application for Phase I/IIA Trial for an Immunotherapy (ALZN002) to Treat Mild to Moderate Dementia of the Alzheimer’s Type. Retrieved September 29, 2022, from https://www.businesswire.com/news/home/20220929005339/en[8] Oncternal Therapeutics Receives IND Clearance for ONCT-808, its autologous CAR T Product Candidate Targeting ROR1 for the Treatment of Aggressive B Cell Lymphoma. Retrieved October 3, 2022, from https://investor.oncternal.com/news-releases/news-release-details/oncternal-therapeutics-receives-ind-clearance-onct-808-its[9] Lyell Immunopharma Announces FDA Clearance of its IND for LYL845, a TIL Product Candidate Enhanced with its Novel Epigenetic Reprogramming Technology for Solid Tumors. Retrieved October 6, 2022, from https://ir.lyell.com/news-releases/news-release-details/lyell-immunopharma-announces-fda-clearance-its-ind-lyl845-til[10] Mustang Bio Announces First Patient Treated in Its Multicenter Phase 1/2 Clinical Trial of MB-106, a First-in-Class CD20-targeted, Autologous CAR T Cell Therapy to Treat B-cell Non-Hodgkin Lymphoma and Chronic Lymphocytic Leukemia. Retrieved October 6, 2022, from https://ir.mustangbio.com/news-events/press-releases/detail/149/mustang-bio-announces-first-patient-treated-in-its[11] TeraImmune Receives FDA IND Clearance to Initiate Phase 1/2a Clinical Trial of TI-168 for Treatment of Hemophilia A with Refractory Inhibitors. Retrieved October 11, 2022, from https://www.teraimmune.com/news-media/en-teraimmune-receives-fda-ind-clearance-to-initiate-phase-1-2a-clinical-trial-of-ti-168[12] 에이비엘, ‘사노피 L/O’ 파킨슨병 신약 “美1상 IND 신청“. Retrieved October 4, 2022, from http://biospectator.com/view/news_view.php?varAtcId=17345[13] Inhibrx Announces Opportunity for Accelerated Approval Pathway on Functional AAT Serum Levels for INBRX-101 in AATD and Announces Bronchoalveolar Lavage Fluid Detection Results from the Phase 1 Study. Retrieved October 4, 2022, from https://www.prnewswire.com/news-releases/inhibrx-announces-opportunity-for-accelerated-approval-pathway-on-functional-aat-serum-levels-for-inbrx-101-in-aatd-and-announces-bronchoalveolar-lavage-fluid-detection-results-from-the-phase-1-study-301639680.html[14] AIRWAY THERAPEUTICS OPENS INTERNATIONAL CLINICAL TRIAL SITES AND TREATS FIRST PATIENT IN SPAIN IN PHASE 1B STUDY OF ZELPULTIDE ALFA (AT-100) IN PRETERM INFANTS AT RISK FOR BRONCHOPULMONARY DYSPLASIA (BPD). Retrieved October 3, 2022, from https://www.airwaytherapeutics.com/airway-therapeutics-opens-international-clinical-trial-sites-and-treats-first-patient-in-spain-in-phase-1b-study-of-zelpultide-alfa-at-100-in-preterm-infants-at-risk-for-bronchopulmonary-dysplasia/[15] Prometheus Biosciences Receives FDA Clearance of IND Application for its Second Precision Program, PRA052. Retrieved October 3, 2022, from https://ir.prometheusbiosciences.com/news-releases/news-release-details/prometheus-biosciences-receives-fda-clearance-ind-application[16] KEZAR LIFE SCIENCES RECEIVES FDA CLEARANCE OF IND FOR ZETOMIPZOMIB FOR THE TREATMENT OF AUTOIMMUNE HEPATITIS. Retrieved October 3, 2022, from https://ir.kezarlifesciences.com/news-releases/news-release-details/kezar-life-sciences-receives-fda-clearance-ind-zetomipzomib[17] Abcuro Initiates Phase 1/2 Trial Evaluating ABC008 in Patients with T Cell Large Granular Lymphocytic Leukemia. Retrieved October 4, 2022, from https://abcuro.com/uncategorized/abcuro-initiates-phase-1-2-trial-evaluating-abc008-in-patients-with-t-cell-large-granular-lymphocytic-leukemia/[18] NextCure Announces Initiation of Phase 1b/2 Clinical Trial to Evaluate NC410 in Combination with KEYTRUDA® (Pembrolizumab) in Patients with Immune Checkpoint Refractory or Naïve Solid Tumors. Retrieved October 4, 2022, from https://www.globenewswire.com/news-release/2022/10/04/2528267/0/en/NextCure-Announces-Initiation-of-Phase-1b-2-Clinical-Trial-to-Evaluate-NC410-in-Combination-with-KEYTRUDA-Pembrolizumab-in-Patients-with-Immune-Checkpoint-Refractory-or-Na%C3%AFve-Solid.html[19] Clarametyx Announces FDA Acceptance of Investigational New Drug (IND) Application for Phase 1 Study of Immune-Enabling Antibody Therapy CMTX-101. Retrieved October 5, 2022, from https://clarametyx.com/clarametyx-announces-fda-acceptance-of-investigational-new-drug-ind-application-for-phase-1-study-of-immune-enabling-antibody-therapy-cmtx-101/[20] MBX Biosciences Advances Phase 1 Clinical Trial of Long-Acting Parathyroid Hormone Peptide Prodrug, MBX 2109. Retrieved October 5, 2022, from https://mbxbio.com/news/pr/mbx-biosciences-advances-phase-1-clinical-trial-of-long-acting-parathyroid-hormone-peptide-prodrug-mbx-2109/[21] Peptilogics Announces First Patient Dosed in Phase 1b Study of PLG0206 for Periprosthetic Joint Infection. Retrieved October 6, 2022, from https://www.businesswire.com/news/home/20221006005192/en/Peptilogics-Announces-First-Patient-Dosed-in-Phase-1b-Study-of-PLG0206-for-Periprosthetic-Joint-Infection[22] BridgeBio Pharma Announces First Lung Cancer Patient Dosed in Phase 1/2 Trial and US FDA Fast Track Designation for SHP2 inhibitor BBP-398 in Combination with Amgen’s LUMAKRAS® (sotorasib). Retrieved October 11, 2022, from https://bridgebio.com/news/bridgebio-pharma-announces-first-lung-cancer-patient-dosed-in-phase-1-2-trial-and-us-fda-fast-track-designation-for-shp2-inhibitor-bbp-398-in-combination-with-amgens-lumakras-sotorasib/[23] Tiziana Life Sciences Plans to Submit IND for Phase 1 Trial of Intranasal Foralumab in Alzheimer's Disease Patients. Retrieved October 12, 2022, from https://www.tizianalifesciences.com/news-item?s=2022-10-12-tiziana-life-sciences-plans-to-submit-ind-for-phase-1-trial-of-intranasal-foralumab-in-alzheimers-disease-patients[24] DICE Therapeutics Announces Positive Topline Data from Phase 1 Clinical Trial of Lead Oral IL-17 Antagonist, DC-806, for Psoriasis Retrieved October 12, 2022, from https://investors.dicetherapeutics.com/news-releases/news-release-details/dice-therapeutics-announces-positive-topline-data-phase-1[25] Novo Seeds Portfolio Company NMD Pharma Reports Positive Top-Line Phase I/IIa Data in Myasthenia Gravis. Retrieved October 11, 2022, from https://www.nmdpharma.com/news/nmd670-top-line-data [26] atai Life Sciences Announces Positive Initial Results for Phase 1 Trial of KUR-101, an Oral Formulation of Mitragynine for OUD. Retrieved October 11, 2022, from https://ir.atai.life/news-releases/news-release-details/atai-life-sciences-announces-positive-initial-results-phase-1[27] Immuneering Announces FDA Clearance of IND Application for Phase 1/2a Clinical Trial of IMM-1-104 to Treat Advanced Solid Tumors with RAS Mutations. Retrieved September 30, 2022, from https://www.globenewswire.com/news-release/2022/09/30/2525857/0/en/Immuneering-Announces-FDA-Clearance-of-IND-Application-for-Phase-1-2a-Clinical-Trial-of-IMM-1-104-to-Treat-Advanced-Solid-Tumors-with-RAS-Mutations.html[28] AC Immune Opens New Centers In Phase 1b/2 Trial Evaluating ACI-24 Targeting Abeta In Alzheimer’s Disease And Down Syndrome. Retrieved October 4, 2022, from https://ir.acimmune.com/news-releases/news-release-details/ac-immune-opens-new-centers-phase-1b2-trial-evaluating-aci-24[29] Icosavax Initiates Phase 1 Trial of IVX-A12 Against RSV and hMPV in Older Adults. Retrieved October 4, 2022, from https://investors.icosavax.com/news-releases/news-release-details/icosavax-initiates-phase-1-trial-ivx-a12-against-rsv-and-hmpv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

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