GRP

New research has identified hundreds of proteins that might contribute to the onset of common, chronic metabolic diseases such as type 2 diabetes, and consequently pathways to potential treatments. New research has identified hundreds of proteins that might contribute to the onset of common, chronic metabolic diseases such as type 2 diabetes, and consequently pathways to potential treatments. The study, published in Nature Metabolism, was led by an international research team from the Medical Research Council (MRC) Epidemiology Unit, University of Cambridge, the Precision Healthcare University Research Institute (PHURI) at Queen Mary University of London, and the Berlin Institute of Health at Charité (BIH) at Universitätsmedizin Berlin in Germany. The researchers successfully linked more than 900 regions in the human genome to almost 3000 proteins in our blood, with many of these not previously identified. The team then applied these findings to existing genetic studies for hundreds of diseases and found more than 500 gene-protein-disease links. For example, the team showed for the first time that people with high levels of a hormone called GRP are less likely to develop type 2 diabetes, most likely because it decreases the chances of becoming overweight. This 'proteogenomic' evidence supports GRP as a potential target for the prevention and/or treatment of diabetes. The study has led to a greater scientific understanding of hundreds of genome regions, which open the door to more targeted and ultimately successful treatment options in the future as proteins are essential functional units of the human body and the most common target of drugs that exist today. Senior author Professor Claudia Langenberg, director of the Precision Healthcare University Research Institute (PHURI) at Queen Mary University of London and MRC Investigator and program lead at the MRC Epidemiology Unit at the time of the study, said: "Thousands of regions in our genome have been identified to increase our risk for developing different diseases, but for most of them we have a poor understanding of why this is. "By measuring and integrating information on thousands of proteins in human plasma, we were able to create robust links between the genes that encode these proteins to many different diseases and demystify about 200 regions. This really narrows down the potential therapeutic targets at each genomic region, often a bottleneck for translation of genomic discoveries." Senior author Professor Maik Pietzner, Professor at the PHURI and group leader at the Berlin Institute of Health at Charité (BIH), said "In an additional promising example, we identified a protein, called DKKL1, to be involved in multiple sclerosis that reinforces the depletion of certain immune cells -- B-cells -- as intervention. "These early-stage results are exciting and show the potential of such technologies for drug discovery, not just for metabolic diseases." Mine Koprulu lead author of the study, Gates scholar and PhD student at the MRC Epidemiology Unit, said: "The biological mechanisms underlying diseases are not always very well understood. To address this issue, we systematically linked genetic variation, blood protein levels and disease risks in this study to be able to differentiate proteins which are likely to cause a disease, for example diabetes, from those which may only be a result of diseases. Identifying causal proteins is important because only interventions on causal proteins will lead to safe and effective treatments. We are hugely grateful to the EPIC Norfolk volunteers and team who made this research possible."

SYDNEY, Nov. 21, 2022 /PRNewswire/ -- Highlights Clarity's fifth successful Investigational New Drug (IND) application with the United States Food and Drug Administration (US FDA), opening up therapeutic applications for SAR-Bombesin A total of six products with both the diagnostic and therapeutic applications for SARTATE, SAR-bisPSMA and SAR Bombesin are now under IND for US clinical trials First therapeutic clinical program for SAR-Bombesin will be in PSMA negative metastatic castrate resistant prostate cancer, an area of high unmet need Clarity Pharmaceuticals (ASX: CU6) ("Clarity"), a clinical-stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, announces the approval of its Investigational New Drug (IND) application by the United States Food and Drug Administration (US FDA) to evaluate its SAR-Bombesin product for identification and treatment of metastatic castrate-resistant prostate cancer (mCRPC). Clarity's Executive Chairman, Dr Alan Taylor, commented, "This trial with SAR-Bombesin marks our third therapy program, and sixth program overall, in the Targeted Copper Theranostic pipeline that is progressing through clinical trials in the US. Receiving clearance from the FDA is yet another significant milestone for the Company as it continues to showcase the capabilities of Clarity's small but high performing team in developing next-generation theranostics from the benchtop, through preclinical studies and into clinical trials in the largest pharmaceutical market in the world. This is Clarity's fifth IND, highlighting our strategy of developing radiopharmaceuticals for the very large US market." This IND gives Clarity clearance to proceed with a US-based Phase I/IIa 64Cu/67Cu SAR-Bombesin theranostic trial for identification and treatment of mCRPC that is expressing the Gastrin-Releasing Peptide receptor (GRPr). COMBAT (Copper-67 SAR Bombesin in metastatic castrate resistant prostate cancer) is a dose escalation study with a cohort expansion. The aim for the study is to determine the safety and efficacy of 67Cu-SAR-Bombesin in participants with GRPr-expressing mCRPC in patients who are ineligible for therapy with 177Lu-PSMA-617. Approximately 25% of mCRPC patients have low or no uptake of a PSMA-targeting tracer.1 These patients are therefore unlikely to show uptake of PSMA-targeted products, such as 68Ga-PSMA-11 for imaging or 177Lu-PSMA-617 for therapy, and currently have few radiopharmaceutical treatment options available to them. Given prostate cancer is one of the largest indications in oncology, there is a significant unmet medical need in this segment. The SAR-Bombesin product targets the GRP receptor found on prostate and many other cancers. As such, the SAR-Bombesin could offer valuable imaging and therapeutic options for not only PSMA negative patients, but also the large number of patients that have the target receptor on their cancers. Clarity's Executive Chairman, Dr Alan Taylor, commented, "We look forward to further progressing the development of SAR-Bombesin as a theranostic under this trial, as well as a stand-alone diagnostic agent under the SABRE and BOP clinical trials that are currently progressing in the US and Australia respectively. The diagnostic SAR-Bombesin product has already shown utility in improving the management of disease for PSMA-negative prostate cancer patients and, given the mounting clinical and preclinical data to date, we believe that it has potential to provide this large patient population with more accurate and precise detection and treatment of disease. SAR-Bombesin is a pan-cancer product, and the open IND offers exciting opportunities for exploring new theranostic indications with this versatile product as Clarity pursues our ultimate goal of improving treatment outcomes for children and adults with cancer." About SAR-Bombesin SAR-Bombesin is a highly targeted pan-cancer radiopharmaceutical with broad cancer application. It targets the gastrin-releasing peptide receptor (GRPr) present on cells of a range of cancers, including but not limited to prostate, breast and ovarian cancers. GRPr is found in approximately 75-100% of prostate cancers, including prostate cancers that don't express PSMA (PSMA-negative)2-6. The product utilises Clarity's proprietary sarcophagine (SAR) technology that securely holds copper isotopes inside a cage-like structure, called a chelator. Unlike other commercially available chelators, the SAR technology prevents copper leakage into the body. SAR-Bombesin is a Targeted Copper Theranostic (TCT) that can be used with isotopes of copper-64 (Cu-64 or 64Cu) for imaging and copper-67 (Cu-67 or 67Cu) for therapy. About Prostate Cancer Prostate cancer is the second most common cancer diagnosed in men globally and the fifth leading cause of cancer death worldwide7. The National Cancer Institute estimates in 2022 there will be 268,490 new cases of prostate cancer in the US and around 34,500 deaths from the disease8. About Clarity Pharmaceuticals Clarity is a clinical stage radiopharmaceutical company focused on the treatment of serious disease. The Company is a leader in innovative radiopharmaceuticals, developing targeted copper theranostics based on its SAR Technology Platform for the treatment of cancer in children and adults. References Vlachostergios PJ, Niaz MJ, Sun M, et al. Prostate-Specific Membrane Antigen Uptake and Survival in Metastatic Castration-Resistant Prostate Cancer. Frontiers in oncology. 2021;11. Markwalder R, Reubi JC. Gastrin-releasing peptide receptors in the human prostate: relation to neoplastic transformation. Cancer research. 1999;59(5):1152-1159. Fleischmann A, Waser B, Reubi JC. High expression of gastrin-releasing peptide receptors in the vascular bed of urinary tract cancers: promising candidates for vascular targeting applications. Endocrine-related cancer. 2009;16(2):623-633. Ananias HJ, van den Heuvel MC, Helfrich W, de Jong IJ. Expression of the gastrin-releasing peptide receptor, the prostate stem cell antigen and the prostate-specific membrane antigen in lymph node and bone metastases of prostate cancer. The Prostate. 2009;69(10):1101-1108. Reubi JC, Wenger S, Schmuckli-Maurer J, Schaer JC, Gugger M. Bombesin receptor subtypes in human cancers: detection with the universal radioligand (125)I-[D-TYR(6), beta-ALA(11), PHE(13), NLE(14)] bombesin(6-14). Clin Cancer Res. 2002;8(4):1139-1146. Sun B, Halmos G, Schally AV, Wang X, Martinez M. Presence of receptors for bombesin/gastrin-releasing peptide and mRNA for three receptor subtypes in human prostate cancers. The Prostate. 2000;42(4):295-303. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries < American Cancer Society, Cancer Statistics Center, < For more information, please contact: This announcement has been authorised for release by the Executive Chairman. Company Codes: Australia:CU6

强强联手，CRISPRTherapeutics和Nkarta达成全球合作伙伴关系吉凯基因科创板IPO申请获受理，拟募资12亿元1.85亿美元！Athenex重金买下NKT细胞领先公司TalarisIPO募集资金1.5亿美元，开发针对自身免疫疾病的细胞疗法恒润达生完成C轮数亿元融资加速布局CAR-T细胞病毒载体生产基地用AI开发基因治疗，DynoTherapeutic完成1亿美元A轮融资武田10.4亿美元达成合作的RNAi疗法最新临床结果公布生物技术新锐公司想用miRNA药物治疗糖尿病 重大事件 强强联手，CRISPR Therapeutics和Nkarta达成全球合作伙伴关系近日，基因编辑巨头CRISPR Therapeutics和CAR-NK细胞疗法领域领先公司Nkarta共同宣布，双方将达成战略合作伙伴关系，以进行基于CRISPR/Cas9基因编辑技术的癌症免疫细胞治疗的研究、开发和商业化。根据协议，双方将共同开发和商业化两款CAR-NK细胞，分别靶向CD70和未知靶点。此外，双方还将促进新型“NK+T”候选药物的开发。 吉凯基因科创板IPO申请获受理，拟募资12亿元近日，据上交所披露，上市委会议已受理了吉凯基因的上市申请。吉凯基因本次IPO拟募资12亿元，用于靶标筛选、平台升级以及创新药项目研发。吉凯基因成立于2002年，设立至今，一直利用 RNAi 等技术，开展药物靶标发现及其衍生业务。目前该公司已经建立了GRP 平台、CHAMP平台和细胞治疗平台。这三大平台可为研究型医生提供标准化研究服务，并可自主开展药物靶标的筛选和创新药物的开发。通过自主创新研究，目前该公司已有15个新药研发项目进入IND研究阶段，其中7个已许可或转让，研发生产基地总面积达11000平方米。 细胞与基因疗法 1.85亿美元！Athenex重金买下NKT细胞领先公司 近日，全球生物制药公司Athenex，Inc.宣布以1.85亿美元收购Kuur Therapeutics，Inc.。Kuur是一家主要从事开发“通用型”CAR-NKT细胞免疫疗法的公司，其目前的研究重点是通过改造NKT细胞，以增强它们的抗肿瘤功效。2021年1月，Kuur的CAR-NKT细胞免疫疗法在治疗实体瘤和血液瘤的I期临床研究中显示出良好的安全性和耐受性。此次收购Kuur将拓展Athenex公司在细胞疗法开发方面的能力。 生物技术公司Talaris IPO募集资金1.5亿美元，开发针对自身免疫疾病的细胞疗法近日，细胞疗法公司Talaris Therapeutics登陆纳斯达克，并募集资金1.5亿美元。这是一家专注于开发治疗因器官移植带来的免疫排斥疾病细胞疗法公司。目前，该公司主要开发的候选产品是FCR001，用于治疗器官移植导致的免疫排斥疾病。这是“通用型”干细胞疗法，通过健康供体的干细胞和免疫细胞在器官移植患者体内构建一套新的免疫系统，让患者免受因器官移植而需要终生服用免疫抑制剂药物的痛苦。 恒润达生完成C轮数亿元融资加速布局CAR-T细胞病毒载体生产基地 近日，恒润达生完成数亿元C轮融资，盈科资本为本轮投资领投方。恒润达生方面表示，本轮融资将主要用于3个非实体瘤项目的II期临床试验及新一代细胞免疫疗法的研发，全面布局上游CAR-T细胞病毒载体生产基地以及打造服务全行业的CDMO平台。 用AI开发基因治疗，Dyno Therapeutic完成1亿美元A轮融资 近日，生物技术公司Dyno Therapeutics宣布完成1亿美元（约6.5亿元）A轮融资。本轮融资由Andreessen Horowitz领投，Casdin Capital，GV，Obvious Ventures等投资机构跟投。Dyno是一家利用AI技术开发基因治疗的生物技术公司，拥有独特的CapsidMap平台可指导设计AAV衣壳。本轮融资主要用于加速CapsidMap平台的建设，以设计出针对肝脏、肌肉、眼睛和中枢神经系统（CNS）疾病的改良载体，从而治疗相关领域疾病。 核酸药物 武田10.4亿美元达成合作的RNAi疗法最新临床结果公布近日，武田RNAi疗法领域的合作伙伴Arrowhead Pharmaceuticals宣布，其RNAi候选疗法ARO-AAT针对α-1抗胰蛋白酶缺乏症相关肝病（AATLD）的临床研究取得了积极结果。ARO-AAT是Arrowhead与武田共同开发的第二代皮下给药RNAi疗法，旨在抑制突变的α-1抗胰蛋白酶（Z-AAT）蛋白在肝脏的产生，以治疗AATLD。最新公布的数据显示，ARO-AAT可以持续减少肝内炎症性Z-AAT蛋白、Z-AAT单体和Z-AAT聚合物；减轻组织学小球（histological globule）负担；改善纤维化及其他与肝脏健康相关的生物标志物。 生物技术新锐公司想用miRNA药物治疗糖尿病近日，生物技术公司Nexturn Bio inc.宣布，为扩大其新的生物业务，将在母公司的支持下投资生物技术公司RosVivo Therapeutics,Inc.。RosVivo公司是一家利用miRNA技术开发下一代RNA药物的公司，其候选产品RSVI-301是一种尝试治愈糖尿病的miRNA新药物。