AskBio和信念医药将共同努力，通过肝脏靶向治疗，针对医疗需求未被满足的疾病开发潜在基因疗法。 拜耳集团和由拜耳全资拥有并独立运营的基因治疗公司Asklepios BioPharmaceutical（AskBio）近日宣布，与信念医药（Belief BioMed）建立新的战略合作，探索创新基因疗法潜力。 根据合同条款，双方将结合各自在基因治疗技术方面的能力和经验，探索针对可治愈疾病的肝脏靶向治疗。 AskBio首席科学官Mansuo Shannon表示： “ AskBio在开发创新基因疗法时采取协作的方式，在全球范围内寻找最有前景的技术实现我们的目标。信念医药的下一代衣壳技术与AskBio开展的工作相结合将带来光明的前景。我们期待与信念医药团队密切合作。” 信念医药的全球总部位于中国上海，集基因治疗产品研发、生产和临床应用为一体，通过病毒载体技术治疗严重遗传疾病和慢性疾病。 该公司开发了先进的载体技术，并在中国建立了基因治疗药物的商业化生产平台。今年7月，信念医药宣布，中国国家药品监督管理局（NMPA）已受理其治疗乙型血友病的核心产品的新药申请（NDA）。针对先天性遗传疾病，

9月27日，中国生物研究院（新型疫苗国家工程研究中心）在国际顶级学术期刊《Science》杂志以研究论文形式在线发表了最新科研成果《基于柔性区域改造的RSV F蛋白融合前构象稳定性突变设计》（Mutating a flexible region of the RSV F protein can stabilize the prefusion conformation）。该项研究利用计算结构生物学交叉学科技术，提出了基于变构调控的呼吸道合胞病毒（RSV）F蛋白融合前构象稳定性突变设计新策略，为更多疑难病原体疫苗的抗原优化设计提供了具有前景的新思路和新技术。中国生物研究院为该论文唯一完成单位和通讯单位。 呼吸道合胞病毒（RSV）是导致婴幼儿、老年人严重呼吸道疾病的主要感染因素之一。RSV表面的F蛋白介导病毒与宿主细胞的膜融合过程，是疫苗研发的重要靶抗原，但是RSV F蛋白存在两种构象状态，中和活性强的融合前构象（pre-F）不稳定，会自发转变为中和活性弱的融合后构象（post-F）状态。因此，如何将RSV F蛋白稳定到pre-F构象状态，阻止构象转变过程的发生是其作为靶抗原进行疫苗开发的关

Sept. 26, 2024 -- Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces dosing of the first subjects in the multiple-ascending dose (MAD) portion of the Phase 1 study with CDI-988, its potent, broad-spectrum, oral pan-viral protease inhibitor. Topline study results are expected in late 2024 or early 2025. CDI-988 was specifically designed and developed using Cocrystal’s proprietary structure-based drug discovery platform technology and is being developed as the first-in

Orion’s collaboration partner Bayer today announced the submission of a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for the oral androgen receptor inhibitor (ARi) darolutamide. Bayer is seeking approval for the use of darolutamide in combination with androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer (mHSPC). The submission is based on positive results from the pivotal Phase III ARANOTE trial which showed t

MNV-201 is a mitochondrial cell therapy product composed of autologous hematopoietic stem cells enriched with allogeneic mitochondria In pre-clinical studies, MNV-201 demonstrated improved engraftment and bone marrow reconstitution potential of patient derived hematopoietic stem cells In vitro data also demonstrated improved ability to differentiate to erythroid cells, supporting potential for improvement in biomarkers of anemia Sept. 26, 2024 -- Minovia Therapeutics Ltd, a clinical stage biop

The CD19/CD70 Dual CAR is Specifically Designed to Address Both the B-cell and T-cell Dysfunction Implicated in Autoimmune Diseases ALLO-329, is an Investigational Product Built on a New Gene Editing Platform that Features Site-Specific Integration, Intended to Reduce the Risk of Secondary Malignancies, and Leverage the Clinically Validated Dagger® Effect Aimed at Reducing or Eliminating Lymphodepletion, a Potential Barrier in the Adoption of CAR T in Autoimmune Indications Sept. 26, 2024 -- Al

Positive phase III results for Roche’s Gazyva/Gazyvaro show superiority to standard therapy alone in people with lupus nephritis The REGENCY study met its primary endpoint, demonstrating statistically significant and clinically meaningful treatment benefits in people with active lupus nephritis Gazyva/Gazyvaro is designed to target an underlying cause of lupus nephritis, aiming to prevent or delay progression to end-stage kidney disease1,2 Lupus nephritis is a potentially life-threatening manife

September 25, 2024 – Medigene AG (Medigene or the “Company”, FSE: MDG1, Prime Standard), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, today announced the filing of a new patent application protecting MDG2021, a T cell receptor engineered T cell (TCR-T) therapy targeting Kirsten rat sarcoma viral oncogene homologue (KRAS) G12D with human leukocyte antigen (HLA)-A*11, for the TCR-T therapy alone as well

9月26日，中国国家药监局药品审评中心（CDE）官网最新公示，君实生物申报的WJ47156片获得临床试验默示许可，拟开发治疗晚期恶性肿瘤。根据君实生物此前公告介绍，WJ47156片（项目代号“JS125”）是其与微境生物医药合作开发的1类创新药，为一款靶向组蛋白去乙酰化酶（HDACs）抑制剂，属于表观遗传调控剂类药物。 表观遗传学指的是一种可逆的遗传机制，它可以在不改变DNA序列的情况下，通过对DNA与组蛋白的修饰来影响基因表达，这是控制细胞分化和胚胎发育等关键生物过程的重要机制。HDACs参与组蛋白和非组蛋白赖氨酸残基乙酰化和去乙酰化动态平衡过程的调节，其表达已知与多种恶性肿瘤的发生与发展密切相关。 据君实生物此前公告介绍，HDACs具有多种亚型，JS125可选择性对HDAC1、HDAC2、HDAC3产生抑制，并通过诱导细胞周期阻滞、抑制血管生成、调节免疫反应和促进癌症细胞衰老凋亡等来发挥抑制肿瘤作用，达到肿瘤治疗目的。 此外有研究表明，HDACs还是癌症转移和转移相关的疗法耐药性之间的一个重要联系。HDACs在癌细胞中由于MAPK和JNK信号通路的激活而提高表达水平，控制与侵

Genespire raises €46.6 million (~$52 million) in a Series B round to advance its first pediatric in-vivo gene therapy into the clinic One of the largest private Italian Biotech company financings to date Financing co-led by Sofinnova Partners, XGEN Venture and CDP Venture Capital forming a strong syndicate with Indaco SGR Funds dedicated to advancing off-the-shelf pediatric gene therapy for Methylmalonic Acidemia (MMA) into the clinic, and to bolstering the Company’s gene therapy pipeline 25 Sep