Divesiran

LONDON--( BUSINESS WIRE )--Silence Therapeutics plc, Nasdaq: SLN (“Silence” or “the Company”), an experienced and innovative biotechnology company committed to transforming people’s lives by silencing diseases through precision engineered medicines, today reported its financial results for the second quarter ended June 30, 2024 and reviewed recent business highlights. “The first half of this year has been marked by strong execution across Silence, including outstanding clinical results in both our wholly owned clinical programs and advancing our first partnered program into the clinic under our AstraZeneca collaboration,” said Craig Tooman, President and Chief Executive Officer at Silence. “Silence’s siRNA technology is continuing to demonstrate itself in the clinic and we expect that positive momentum to continue into the second half of this year and beyond.” “With the $120 million equity raise and milestones from our collaborations, we are well positioned to execute on key clinical objectives for both our zerlasiran and divesiran programs,” said Rhonda Hellums, Chief Financial Officer at Silence. “We will continue to evaluate partnering opportunities for our proprietary mRNAi GOLD™ platform and zerlasiran program with the ultimate goal of delivering maximum value to Silence shareholders and patients.” Second Quarter 2024 & Recent Business Highlights Zerlasiran for cardiovascular disease Announced positive topline 48-week data from the ALPACAR-360 Phase 2 study of zerlasiran in patients with elevated lipoprotein(a) (Lp(a)). Zerlasiran produced highly significant and sustained reductions in Lp(a) to week 48 (end of treatment period) and was well tolerated. Data support advancing zerlasiran into Phase 3. Divesiran for hematological diseases Announced positive results from the ongoing SANRECO Phase 1 study of divesiran in polycythemia vera (PV) patients. Divesiran eliminated the need for phlebotomy in all well-controlled patients following infrequent dosing and was well tolerated. Data support advancing divesiran into Phase 2. Collaborations Achieved a $2.0 million research milestone payment under our collaboration with Hansoh Pharmaceutical Group Company Limited (Hansoh). Organizational updates Promoted Rhonda Hellums, Silence’s Chief Financial Officer, and Steven Romano, MD, Silence’s Chief R&D Officer, to Executive Vice President. Hired Gianine Esposito as Chief Human Resources Officer (CHRO). Gianine brings 25 years of Human Resources experience with over 15 years of pharmaceutical experience and joins Silence from Shionogi Inc. where she was the SVP and CHRO. In her time at Shionogi, Gianine assisted in building out its US-based clinical and commercial organizations through different stages of growth. Hired Marianna Anesti as Vice President, Head of Business Development. Marianna brings 18 years of diverse Biotech & Pharmaceutical experience and joins Silence from Siga Technologies where she was the VP, Business Development & Corporate Strategy. In her time at Siga, Marianna built a pipeline of M&A opportunities, strategic partnerships, and potential in-licensing deals. Prior to Siga, Marianna was Director, R&D Strategy & Consulting at Pfizer where she partnered with Senior R&D leaders across the business to help shape new BD strategies. Financial Highlights for the Period Ended June 30,2024 Cash Position: Cash, cash equivalents and U.S. Treasury Bills of £149.6 million ($189.2 million) as of June 30, 2024, compared with £50.2 million ($63.4 million) as of June 30, 2023. Collaboration Revenue: Collaboration revenue for the three and six months ended June 30, 2024 was £0.6 million (June 30,2023: £9.0 million) and £12.9 million (June 30, 2023: £20.2 million), respectively. Revenue from the current year period primarily related to our ongoing AstraZeneca and Hansoh collaborations and from our Mallinckrodt collaboration we completed in March 2024. R&D Expenses: Research and development (R&D) expenses for the three and six months ended June 30, 2024 was £11.0 million (June 30,2023: £12.6 million) and £20.2 million (June 30, 2023: £25.2 million), respectively. The decrease compared to the prior year period primarily related to the timing of clinical studies and manufacturing activities for our divesiran clinical program. G&A Expenses: General and administrative (G&A) expenses for the three and six months ended June 30, 2024 was £5.3 million (June 30,2023: £5.1 million) and £10.5 million (June 30, 2023: £11.6 million), respectively. The decrease from the prior year six-month period was mainly due to decreased payroll costs and equity-based compensation. Net Loss: Net loss for the three and six months ended June 30, 2024 was £15.6 million, or 11.1 pence basic and diluted net loss per share, (June 30,2023: £10.4 million or 9.6 pence) and £17.4 million, or 12.8 pence basic and diluted net loss per share (June 30, 2023: £20.6 million or 19.0 pence), respectively. Total ADSs outstanding were approximately 46,797,823, as of June 30, 2024. About Silence Therapeutics Silence Therapeutics is developing a new generation of medicines by harnessing the body's natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet need. Silence's proprietary mRNAi GOLD™ platform can be used to create siRNAs (short interfering RNAs) that precisely target and silence disease-associated genes in the liver, which represents a substantial opportunity. Silence's wholly owned product candidates include zerlasiran (SLN360) designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of lipoprotein(a) and divesiran (SLN124) designed to address rare hematological diseases including polycythemia vera. Silence also maintains ongoing research and development collaborations with AstraZeneca and Hansoh Pharma, among others. For more information, please visit https://www.silence-therapeutics.com/ . Forward-Looking Statements Certain statements made in this announcement are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and other securities laws, including with respect to the Company’s clinical and commercial prospects, potential partnerships or collaborations under new and existing collaborations and the initiation, advancement or completion of the Company’s clinical trials. These forward-looking statements are not historical facts but rather are based on the Company's current assumptions, beliefs, expectations, estimates and projections about its industry. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements, including those risks identified in the Company’s most recent Admission Document and its Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission on March 13, 2024. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority. Condensed consolidated income statement (unaudited) Three months ended Three months ended Six months ended Six months ended June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023 £000s (except per share information) £000s £000s £000s £000s Revenue 598 9,104 13,004 20,478 Cost of sales (2,638 ) (2,831 ) (4,851 ) (7,365 ) Gross profit (2,040 ) 6,273 8,153 13,113 Research and development costs (10,995 ) (12,615 ) (20,174 ) (25,154 ) General and administrative expenses (5,288 ) (5,115 ) (10,458 ) (11,565 ) Operating loss (18,323 ) (11,457 ) (22,479 ) (23,606 ) Finance and other expenses (183 ) (893 ) (25 ) (1,753 ) Finance and other income 1,065 340 1,698 676 Loss for the period before taxation (17,441 ) (12,010 ) (20,806 ) (24,683 ) Taxation 1,879 1,609 3,368 4,078 Loss for the period after taxation (15,562 ) (10,401 ) (17,438 ) (20,605 ) Loss per ordinary share (basic and diluted) (11.1) pence (9.6) pence (12.8) pence (19.0) pence Condensed consolidated balance sheet (unaudited) June 30, 2024 December 31, 2023 £000s £000s Non-current assets Property, plant and equipment 1,686 1,813 Goodwill 7,662 7,840 Other intangible assets 267 284 Other long term assets 2,361 2,580 Financial assets at amortized cost 284 284 12,260 12,801 Current assets Cash and cash equivalents 109,482 54,031 Financial assets at amortized cost 40,112 - R&D tax credit receivable 12,852 17,627 Other current assets 12,244 9,135 Trade receivables 1,686 228 176,376 81,021 Non-current liabilities Contract liabilities (58,187 ) (58,910 ) Lease liability - (93 ) (58,187 ) (59,003 ) Current liabilities Contract liabilities (2,766 ) (5,161 ) Trade and other payables (14,992 ) (12,429 ) Lease liability (184 ) (179 ) (17,942 ) (17,769 ) Net assets 112,507 17,050 Capital and reserves attributable to the owners of the parent Share capital 7,019 5,942 Capital reserves 423,831 313,769 Translation reserve 1,807 1,951 Accumulated losses (320,150 ) (304,612 ) Total shareholders' equity 112,507 17,050

▎药明康德内容团队编辑 本期看点 1. 干细胞疗法VX-880治疗1型糖尿病（T1D）患者在早期临床试验中获得积极数据，在末次随访时12名患者中有11名减少或停止使用外源性胰岛素。 2. 下一代寡核苷酸疗法RGLS8429用于治疗常染色体显性多囊肾病的早期临床试验结果积极，接受3 mg/kg剂量的患者中有70%的肾脏总体积缩小。 3. 通过抗耗竭技术增强的CAR-T细胞疗法LYL797在临床试验中被证明可以浸润到实体瘤中，治疗ROR1阳性复发/难治性三阴性乳腺癌患者的临床获益率（CBR）为60%。 药明康德内容团队整理 VX-880：公布1/2期临床试验的新数据 Vertex Pharmaceuticals公司公布了其在研细胞疗法VX-880治疗1型糖尿病的1/2期临床试验的最新数据。VX-880是一种在研干细胞来源、完全分化、产生胰岛素的同种异体胰岛细胞疗法。VX-880有可能通过恢复胰岛细胞功能，包括葡萄糖反应性胰岛素产生，恢复机体调节葡萄糖水平的能力。VX-880通过肝门静脉输注给药，需要长期免疫抑制治疗以保护输入的胰岛细胞免受免疫排斥。VX-880已获得美国FDA授予的再生医学先进疗法（RMAT）认定和快速通道资格，以及欧洲药品管理局（EMA）授予的PRIME资格。 公布的结果显示，在接受单次全剂量VX-880输注后90天内，所有T1D患者均显示出胰岛细胞移植成功和对葡萄糖反应的胰岛素生成。在最后一次随访时，12名患者中有11名减少或停止使用外源性胰岛素。Vertex预计增加入组患者数量，并将该疗法推进关键试验阶段。 RGLS8429：公布1b期临床试验的新数据 Regulus Therapeutics公司公布了其开发的新型下一代寡核苷酸疗法RGLS8429用于治疗常染色体显性多囊肾病的1b期临床试验的新数据。常染色体显性多囊肾病是由PKD1或PKD2基因突变引起的，也是最常见的人类单基因疾病之一，同时是终末期肾病的主要原因。RGLS8429旨在抑制miR-17并优先靶向肾脏，在临床前研究中已被证明能够明显改善肾脏功能、大小和其他疾病严重程度指标，并具有优越的药理学特征。此前，该公司已公布1期单次递增剂量临床试验的结果，结果显示，RGLS8429具有良好的安全性、耐受性和药代动力学（PK）特征。 此次公布的是1b期研究中第三个队列的结果，患者对RGLS8429的耐受性良好，未发现安全性问题。PC1和PC2是PKD1和PKD2基因的蛋白质产物，已被证明与疾病严重程度呈负相关。接受RGLS8429治疗患者的PC1和PC2与基线相比的百分比变化在所有测试剂量中均显示出缓解，与安慰剂相比，3 mg/kg剂量组具有统计学意义。探索性成像分析表明，3 mg/kg剂量组的患者中，70%的患者肾脏总体积缩小。 LYL797：公布1期临床试验的初步数据 Lyell Immunopharma公司公布了其第一代重编程ROR1靶向CAR-T细胞候选产品LYL797的1期临床试验的初步数据。LYL797采用了Lyell公司的抗耗竭基因重编程技术（c-Jun）和其专有的体外制造方法——表观遗传重编程技术（Epi-R）。LYL797过度表达c-Jun可以纠正耗竭T细胞中AP-1家族转录因子的失衡。在临床前研究中，c-Jun的过度表达使T细胞能够抵抗耗竭、浸润实体瘤并保持其功能。Epi-R技术旨在生成干样T细胞群，减少耗竭，提高增殖和抗肿瘤活性。 该研究的初始数据集主要由ROR1阳性复发/难治性三阴性乳腺癌患者组成。结果显示，LYL797对这些患者具有剂量依赖性的抗肿瘤临床活性，患者的客观缓解率为40%，CBR为60%。新闻稿指出，该试验首次证明了通过抗耗竭技术增强的CAR-T细胞既可以增殖又可以浸润到实体瘤中。在无肺部受累的患者中未观察到与LYL797相关的显著安全信号；在肺转移性疾病患者中观察到可治疗的肺炎。根据现有的积极数据，该公司计划扩大开发范围，包括ROR1阳性复发/难治性铂类耐药卵巢癌、子宫内膜癌、多发性骨髓瘤和慢性淋巴细胞白血病等新肿瘤类型。 NTLA-2001：公布1期临床试验的新数据 Intellia Therapeutics公司公布了其CRISPR/Cas9基因编辑疗法NTLA-2001的最新临床数据。NTLA-2001是Intellia与再生元（Regeneron）开展的多靶点发现、开发和商业化合作的一部分，Intellia负责主导NTLA-2001的开发和商业化。之前新闻稿指出，NTLA-2001以CRISPR/Cas9技术为基础，有望成为治疗转甲状腺素蛋白（ATTR）淀粉样变性的首个单次给药疗法。此外，NTLA-2001是首个通过全身给药或静脉注射来编辑人体内基因的候选CRISPR基因编辑疗法，基于Intellia公司专有的非病毒平台，利用脂质纳米颗粒（LNP）向肝脏输送由两部分组成的基因编辑系统：靶向血清转甲状腺素（TTR）基因的特异性gRNA和编码Cas9酶的mRNA。 此次公布的结果显示，三位先前在1期剂量递增研究中接受最低剂量NTLA-2001治疗的ATTR淀粉样变性患者，在接受后续55 mg NTLA-2001治疗后，其TTR蛋白水平中位减少90%。根据新闻稿，这是首次临床数据显示体内CRISPR/Cas9基因编辑疗法可有效重复给药，成功完成临床概念验证。 WVE-003：公布1b/2a期临床试验的新数据 Wave Life Sciences公司宣布，在研疗法WVE-003在治疗亨廷顿病（HD）的1b/2a期临床试验SELECT-HD中取得了积极成果。WVE-003是一款等位基因选择性反义寡核苷酸，旨在降低突变亨廷顿蛋白（mHTT）表达并保留健康的野生型亨廷顿蛋白（wtHTT）。 数据显示，WVE-003总体安全性和耐受性良好，没有报告严重不良事件。在28周评估期间观察到WVE-003组患者显著的mHTT蛋白水平降低：在24周（最后一次给药后8周），脑脊液（CSF）中mHTT与安慰剂组患者相比平均降低46%（p=0.0007）；在28周（最后一次给药后12周），CSF中mHTT平均降低44%（p=0.0002），支持每季度或更少频率的给药。尾状核萎缩是一个影像学生物标志物，可预测患者的临床结果，包括总运动评分（TMS）的临床显著恶化。在24周（最后一次影像评估）时，mHTT的减少与尾状核萎缩的减缓相关（R=-0.50；p=0.047）。 ENTR-601-44：公布1期临床试验的初步数据 Entrada Therapeutics公司公布了其用于治疗杜氏肌营养不良（DMD）的候选疗法ENTR-601的1期临床试验的积极初步数据。ENTR-601-44是一种专有的内泌体逃逸载体（EEV）偶联磷酸二酰胺吗啉寡聚物（PMO），旨在解决由于DMD基因突变或缺失外显子而导致的DMD的根本原因。ENTR-601-44可用于治疗DMD患者中可跳过44号外显子的患者，目前正在评估其恢复mRNA阅读框的潜力，并允许翻译略有缩短但仍有功能的肌营养不良蛋白。 此次公布的结果显示，ENTR-601-44在健康受试者中耐受性良好，没有严重的不良事件，没有与药物相关的不良事件，也没有在生命体征、心电图、体格检查或实验室评估中观察到临床显著的变化或趋势。研究人员在接受ENTR-601-44的患者的肌肉中检测到了药物浓度。与接受安慰剂的患者相比，ENTR-601-44组患者的外显子跳跃水平具有统计学意义。 Divesiran（SLN124）：公布1期临床试验数据 Silence Therapeutics公司宣布，其正在进行的1期临床试验SANRECO获得积极结果。该研究评估了divesiran（SLN124），一种针对TMPRSS6的siRNA，用于治疗真红细胞增多症（PV）患者的安全性和疗效。PV是一种罕见的骨髓增生性肿瘤，其特征是红细胞过量生成，常常导致血细胞比容升高。Divesiran是一款靶向TMPRSS6的siRNA。TMPRSS6是铁调素（hepcidin）的负调节因子，而铁调素是体内铁代谢的主要调节因子，负责铁的吸收、分布和储存。通过在PV患者中沉默TMPRSS6的表达，divesiran旨在增加肝细胞铁调素的生成和释放，从而限制骨髓中的铁含量，减少依赖铁供应的红细胞过度生成。 截至2024年3月29日的数据，在16名可评估患者中，8名患者血细胞比容控制良好。这是一项检测血液中红细胞比例的指标。截至目前，divesiran的耐受性良好，没有发现重大安全问题。 Ficerafusp alfa（BCA101）：公布1/1b期临床试验数据 Bicara Therapeutics公司公布了其双功能抗体ficerafusp alfa（BCA101）的1/1b期开放标签剂量扩增研究的最新中期数据。Ficerafusp alfa旨在抑制EGFR并直接在肿瘤部位使TGF-β失效。这种作用机制使得BCA101能够抑制肿瘤增殖，同时恢复局部免疫细胞的细胞毒活性。 此次公布的结果显示，ficerafusp alfa与PD-1抑制剂帕博利珠单抗联用，在随访至少12个月的头颈部鳞状细胞癌（HNSCC）患者中显示出有临床意义的抗肿瘤活性，并显著优于标准疗法。在HPV阴性HNSCC患者中，总缓解率为64%（18/28），完全缓解率为18%（5/28），中位无进展生存期为9.8个月，尚未达到中位缓解持续时间和中位总生存期。安全性方面，该联合疗法的耐受性良好，最常见的治疗相关不良事件包括痤疮样皮疹（76%，大多数为1/2级）、疲劳（43%）和低磷血症（38%）。 Lunresertib（RP-6306）：公布1期临床试验的初步数据 Repare Therapeutics公司报告了其PKMYT1抑制剂lunresertib联用FOLFIRI治疗晚期实体瘤患者的1期临床试验的积极数据。Lunresertib是一种潜在“first-in-class”的精准肿瘤学小分子PKMYT1抑制剂，靶向实体瘤中的CCNE1扩增、FBXW7和PPP2R1A变异。 截至2024年5月2日的数据，该联合疗法在曾接受过多线治疗、具有CCNE1扩增和FBXW7突变的肿瘤患者中展现出可喜的疗效。不同肿瘤类型患者的总缓解率为18.2%（n=33），包括4例确认和2例未确认的部分缓解，无论既往是否暴露于伊立替康。结直肠癌患者的临床获益延长，40%的伊立替康初治患者接受治疗超过9个月，而该比例的临床基准为20%。安全性方面，lunresertib联合治疗耐受性良好，没有超过单独使用lunresertib或标准FOLFIRI化疗方案的预期毒性。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料（可上下滑动查看） [1] Vertex Announces Positive Results From Ongoing Phase 1/2 Study of VX-880 for the Treatment of Type 1 Diabetes Presented at the American Diabetes Association 84th Scientific Sessions. Retrieved June 21, 2024 from https://www.businesswire.com/news/home/20240621506971/en [2] Wave Life Sciences Announces Positive Results from Phase 1b/2a SELECT-HD Trial with First Clinical Demonstration of Allele-Selective Mutant Huntingtin Lowering in Huntington’s Disease. Retrieved June 25, 2024, from https://www.globenewswire.com/news-release/2024/06/25/2903685/0/en/Wave-Life-Sciences-Announces-Positive-Results-from-Phase-1b-2a-SELECT-HD-Trial-with-First-Clinical-Demonstration-of-Allele-Selective-Mutant-Huntingtin-Lowering-in-Huntington-s-Dise.html [3] Silence Therapeutics Announces Positive Results from Ongoing SANRECO Phase 1 Study of Divesiran in Polycythemia Vera Patients. Retrieved June 27, 2024, from https://www.businesswire.com/news/home/20240627515407/en [4] Intellia Announces Positive Clinical Proof-of-Concept Data for Redosing a CRISPR-Based Therapy with its Proprietary LNP-Based Delivery Platform. Retrieved June 26, 2024 from https://ir.intelliatx.com/news-releases/news-release-details/intellia-announces-positive-clinical-proof-concept-data-redosing [5] Repare Therapeutics Announces Positive Initial Data at ESMO GI from Phase 1 MINOTAUR Trial Evaluating Lunresertib in Combination with FOLFIRI. Retrieved June 28, 2024, from https://www.businesswire.com/news/home/20240626903583/en [6] Lyell Immunopharma Reports Dose-dependent Clinical Activity from Phase 1 Trial of LYL797, a ROR1-targeted CAR-T Cell Product Candidate Enhanced with its Proprietary Anti-exhaustion Technology. Retrieved June 28, 2024, from https://ir.lyell.com/news-releases/news-release-details/lyell-immunopharma-reports-dose-dependent-clinical-activity [7] Entrada Therapeutics Reports Positive Preliminary Data in Healthy Volunteers from Phase 1 ENTR-601-44-101 Trial for Duchenne Muscular Dystrophy. Retrieved June 28, 2024, from https://www.globenewswire.com/news-release/2024/06/24/2902819/0/en/Entrada-Therapeutics-Reports-Positive-Preliminary-Data-in-Healthy-Volunteers-from-Phase-1-ENTR-601-44-101-Trial-for-Duchenne-Muscular-Dystrophy.html [8] Regulus Therapeutics Announces Positive Topline Data from the Third Cohort of Patients in its Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). Retrieved June 28, 2024, from https://www.prnewswire.com/news-releases/regulus-therapeutics-announces-positive-topline-data-from-the-third-cohort-of-patients-in-its-phase-1b-multiple-ascending-dose-mad-clinical-trial-of-rgls8429-for-the-treatment-of-autosomal-dominant-polycystic-kidney-disease-adp-302179857.html [9] Bicara Therapeutics Reports Updated Interim Phase 1/1b Data of Ficerafusp Alfa (BCA101) in 1L HPV-negative Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (HNSCC). Retrieved June 28, 2024, from https://www.businesswire.com/news/home/20240626900045/en [10] Elicio Therapeutics Announces Preliminary Disease-Free Survival analysis from the Ongoing AMPLIFY-7P Phase 1a Study of ELI-002 7P. Retrieved June 28, 2024, from https://www.globenewswire.com/news-release/2024/06/27/2905517/0/en/Elicio-Therapeutics-Announces-Preliminary-Disease-Free-Survival-analysis-from-the-Ongoing-AMPLIFY-7P-Phase-1a-Study-of-ELI-002-7P.html [11] FSD Pharma Receives Ethics Committee Approval for a Phase-1 Multiple Ascending Doses Clinical Trial for Lucid-21-302 (Lucid-MS) in Australia. Retrieved June 28, 2024, from https://webfiles.thecse.com/FSD_Pharma_HREC_Ethics_-_6.27.2024_-_FINAL.pdf?T19KUkcoQLzWZRlxkwkgXY7TVuhIrMDo= [12] Quanta Announces Dosing of First Patient in Phase 1/1b Clinical Trial of QTX3046, an Oral KRASᴳ¹²ᴰ-Selective Dual-State Allosteric Inhibitor, and Other Program Updates. Retrieved June 28, 2024, from https://www.globenewswire.com/news-release/2024/06/27/2905223/0/en/Quanta-Announces-Dosing-of-First-Patient-in-Phase-1-1b-Clinical-Trial-of-QTX3046-an-Oral-KRAS%E1%B4%B3-%E1%B4%B0-Selective-Dual-State-Allosteric-Inhibitor-and-Other-Program-Updates.html [13] First gene therapy for prevalent pathologies in neurology receives FDA IND clearance - EG 427 initiating first-in-human clinical study. Retrieved June 28, 2024, from https://www.globenewswire.com/news-release/2024/06/24/2902739/0/en/First-gene-therapy-for-prevalent-pathologies-in-neurology-receives-FDA-IND-clearance-EG-427-initiating-first-in-human-clinical-study.html [14] 4DMT Announces FDA Clearance of IND Application for 4D-175 Genetic Medicine for the Treatment of Geographic Atrophy. Retrieved June 28, 2024, from https://www.globenewswire.com/news-release/2024/06/24/2902932/0/en/4DMT-Announces-FDA-Clearance-of-IND-Application-for-4D-175-Genetic-Medicine-for-the-Treatment-of-Geographic-Atrophy.html [15] Adicet Bio Announces FDA Clearance of IND Application for ADI-270 in Renal Cell Carcinoma. Retrieved June 28, 2024, from https://www.businesswire.com/news/home/20240624689420/en [16] KBio Announces FDA Clearance of IND Application for EV68-228-N for the Treatment of Acute Flaccid Myelitis and the First Patient Dosed in the Phase 1 Study. Retrieved June 28, 2024, from https://www.globenewswire.com/news-release/2024/06/28/2905938/0/en/KBio-Announces-FDA-Clearance-of-IND-Application-for-EV68-228-N-for-the-Treatment-of-Acute-Flaccid-Myelitis-and-the-First-Patient-Dosed-in-the-Phase-1-Study.html [17] GT Biopharma Announces FDA Clearance of Investigational New Drug (IND) Application for GTB-3650, an NK Cell Engager for Treatment of CD33+ Leukemia. Retrieved June 28, 2024, from https://www.globenewswire.com/news-release/2024/06/27/2905285/0/en/GT-Biopharma-Announces-FDA-Clearance-of-Investigational-New-Drug-IND-Application-for-GTB-3650-an-NK-Cell-Engager-for-Treatment-of-CD33-Leukemia.html [18] Synaptogenix Announces FDA Authorization of IND Application for Clinical Trial for Bryostatin-1 in Multiple Sclerosis. Retrieved June 28, 2024, from https://www.prnewswire.com/news-releases/synaptogenix-announces-fda-authorization-of-ind-application-for-clinical-trial-for-bryostatin-1-in-multiple-sclerosis-302182860.html [19] TRexBio Announces Collaboration Partner Lilly Has Initiated a Phase 1 Study of TRB-051 for Autoimmune and Inflammatory Diseases. Retrieved June 28, 2024, from https://www.businesswire.com/news/home/20240625989975/en [20] ENCell Presents the Phase 1 Clinical Trial Results of their Mesenchymal Stem Cell Therapy, EN001, for Charcot-Marie-Tooth Disease at the PNS conference. Retrieved June 26, 2024 from https://www.businesswire.com/news/home/20240624485930/en [21] OrsoBio to Present Phase 1 and Preclinical Data for Liver-Targeted Mitochondrial Protonophore TLC-6740 at the American Diabetes Association’s 84th Scientific Sessions. Retrieved June 26, 2024 from https://www.businesswire.com/news/home/20240621611137/en [22] Biond Biologics Announces First Patient Dosed with BND-35 in Phase 1 Clinical Trial. Retrieved June 26, 2024 from https://www.prnewswire.com/news-releases/biond-biologics-announces-first-patient-dosed-with-bnd-35-in-phase-1-clinical-trial-302180049.html [23] Enlivex Announces the Dosing of the First Patient in a Randomized, Placebo-Controlled Phase I/II Trial Evaluating AllocetraTM in up to 46 Patients with Thumb Osteoarthritis. Retrieved June 26, 2024 from https://www.globenewswire.com/news-release/2024/06/24/2902937/0/en/Enlivex-Announces-the-Dosing-of-the-First-Patient-in-a-Randomized-Placebo-Controlled-Phase-I-II-Trial-Evaluating-AllocetraTM-in-up-to-46-Patients-with-Thumb-Osteoarthritis.html [24] IDEAYA Biosciences Announces First-Patient-In for Phase 1 Clinical Trial Evaluating IDE397 and Trodelvy® Combination in MTAP-Deletion Bladder Cancer. Retrieved June 26, 2024 from https://www.prnewswire.com/news-releases/ideaya-biosciences-announces-first-patient-in-for-phase-1-clinical-trial-evaluating-ide397-and-trodelvy-combination-in-mtap-deletion-bladder-cancer-302181366.html [25] Allakos Announces Positive Results from its Ongoing Phase 1 Trial of AK006 in Healthy Volunteers, with AK006 Demonstrating High Receptor Occupancy on Mast Cells and a Favorable Safety Profile. Retrieved June 26, 2024 from https://www.globenewswire.com/news-release/2024/06/25/2904125/0/en/Allakos-Announces-Positive-Results-from-its-Ongoing-Phase-1-Trial-of-AK006-in-Healthy-Volunteers-with-AK006-Demonstrating-High-Receptor-Occupancy-on-Mast-Cells-and-a-Favorable-Safe.html [26] Werewolf Therapeutics Highlights Initial Safety and Efficacy Data from its Ongoing Phase 1 Clinical Trial Evaluating WTX-330 in Patients with Advanced or Metastatic Solid Tumors or Non-Hodgkin Lymphoma. Retrieved June 26, 2024 from https://www.globenewswire.com/news-release/2024/06/25/2903639/0/en/Werewolf-Therapeutics-Highlights-Initial-Safety-and-Efficacy-Data-from-its-Ongoing-Phase-1-Clinical-Trial-Evaluating-WTX-330-in-Patients-with-Advanced-or-Metastatic-Solid-Tumors-or.html [27] Ethris Announces Positive Initial Phase 1 Data Demonstrating Nasal Delivery of mRNA Lead Candidate ETH47. Retrieved June 26, 2024 from https://www.businesswire.com/news/home/20240624540176/en [28] Revelation Biosciences Announces Phase 1 Clinical Study of Gemini Met the Primary Safety Endpoint and Showed Statistically Significant Biomarker Activity. Retrieved June 26, 2024 from https://www.businesswire.com/news/home/20240624937640/en [29] TiumBio Presents Promising Clinical Data from Phase 1 of its Hemophilia Treatment Candidate TU7710 at ISTH 2024. Retrieved June 26, 2024 from https://www.prnewswire.com/news-releases/tiumbio-presents-promising-clinical-data-from-phase-1-of-its-hemophilia-treatment-candidate-tu7710-at-isth-2024-302180104.html [30] GenVivo Announces Initiation of Patient Dosing in a US Phase I/Ib Clinical Trial Evaluating GEN2, a Personalizing, Gene Vector Immunotherapy, in Patients with Advanced Solid Tumors. Retrieved June 28, 2024, from https://www.globenewswire.com/NC/news-release/2024/06/27/2905012/0/en/GenVivo-Announces-Initiation-of-Patient-Dosing-in-a-US-Phase-I-Ib-Clinical-Trial-Evaluating-GEN2-a-Personalizing-Gene-Vector-Immunotherapy-in-Patients-with-Advanced-Solid-Tumors.html 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