成都先导2021年半年度业绩说明会-m6米乐网页版

      成都先导药物开发股份有限公司是一家从事新药研发的生物技术公司，总部位于中国成都，在英国剑桥、美国休斯顿设有子公司，并于2020年4月在上海证券交易所科创板挂牌上市（股票名称：成都先导，股票代码：688222.sh）。成都先导为小分子及核酸新药发现与优化建立了一个国际领先的，以dna编码化合物库的设计、合成和筛选（del），以及基于分子片段和三维结构信息的药物设计（fbdd/sbdd）为核心的技术平台。目前，公司基于数千种不同的骨架结构，已经完成超过万亿种结构全新、具有多样性和类药性的dna编码化合物的合成，并且已有多个案例证实了其针对已知生物靶点和新兴生物靶点筛选苗头化合物的能力及有效性。

      2020年底前，成都先导并购了坐落于英国剑桥的vernalis公司，该公司是fbdd/sbdd技术的领先者。现在，成都先导拥有约500人的科学家团队，并且能够提供一整套从靶基因到新药临床试验申请阶段的研发服务，覆盖范围包括重组蛋白表达纯化、结构生物学、计算化学与药物化学、生物化学和生物物理学、细胞生物学、体内药理学、药代动力学、药学研究等。

      成都先导核酸药物研发平台包括生物信息学，核酸药物化学，rna 生物学，分子与细胞生物学，转化研究，以及临床医学等。该核酸药物研发平台不仅能提供高质量的定制化的rnai技术服务，还能提供高质量的sirna设计、sirna的快速平行合成与化学修饰，并进行基因敲除活性测试、稳定性测试、脱靶风险评估及核酸药物体内分布及其他体内外生物功能评价等。目前，公司已在恶性实体肿瘤和免疫性疾病领域建立了小核酸新药产品管线，旨在开发符合临床需求的小核酸新药。目前项目处于早期阶段。

      成都先导业务模式灵活，范围包括基于单一功能的服务（ffs，如，蛋白表达与纯化，结构生物学，生物信息学，计算化学，药物化学，核酸及有机合成，分析化学，生物物理，细胞生物学，药代，药效等），del 筛选，del设计，合成及表征，整合式药物发现项目，风险分担项目，合资企业到项目转让许可。成都先导建立了自主研发新药管线，拥有大约20个内部新药项目，分别处于临床及临床前不同阶段，目前已有4个项目获得临床试验批件并进入临床试验。成都先导业务遍布北美、欧洲、亚洲、非洲及澳大利亚等，现已与多家国际著名制药公司、生物技术公司、化学公司、基金会以及科研机构建立合作，致力于新药的发现与应用。

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      hitgen inc. is a biotech company headquartered in chengdu, china, with subsidiaries in cambridge, uk and houston, usa. it became a publicly listed company in shanghai stock exchange in april 2020 (ticker code 688222.sh).  hitgen has established a drug discovery research platform for small molecules and nucleic acid drug centered on the design, synthesis and screening of dna encoded chemical libraries (dels), fragment-based drug discovery (fbdd) and structure-based drug design (sbdd) technologies. hitgen's dels currently contains more than 1 trillion novel, diverse, drug-like small molecules and macrocyclic compounds. these compounds are members of dels synthesized from many thousands of distinct chemical scaffolds, designed with tractable chemistry, and have yielded proven results for the discovery of small molecule leads against precedented and unprecedented classes of biological targets.

      through its acquisition of cambridge uk based vernalis r&d ltd before the end of 2020, a leader in fbdd/sbdd, hitgen now has a research team of over 500 scientists and offers a full set of research capabilities from recombinant protein expression and purification, structural biology, assay development, screening, del synthesis, nucleic acid and small molecule chemical synthesis, computational and medicinal chemistry, biochemistry and biophysics, cell biology, in vivo pharmacology, dmpk, cmc, etc., to enable drug discovery research from target gene to ind filing.

      hitgen rna therapeutics r&d platform includes key capabilities such as bioinformatics, organic chemistry and medicinal chemistry，rna biology, molecular biology and cell biology, translational research and clinical research. the platform not only provides high quality services to support internal and external rna therapeutics r&d projects, but also carries out sirna sequence design, sirna parallel synthesis and chemical modification/conjugation, mrna and protein knock-down assays in both engineered cell lines and primary cell lines, stability test, off-target assessment, bio-distribution and other relate in vitro/in vivo assays for activity and toxicity study, etc. hitgen has also established its own pipelines in early stages to discover novel rna therapeutics, aiming for providing new treatment for unmet medical needs in oncology and immunology.

      hitgen operates a flexible business model, ranging from a single capability-based fee for services (ffs，e.g., protein expression and purification, structural biology, bioinformatics, computational chemistry, medicinal chemistry, nucleic and organic chemistry, analytical chemistry， biophysics, pk, pd, etc.), del screening, del design, synthesis and characterization, integrated drug discovery projects, risk sharing projects, collaborative ventures to program out-licensing. hitgen has approximately 20 in-house drug discovery programs at different stages of research & development. at present, 4 programs have obtained ind approvals by nmpa and entered into clinical trials. hitgen is collaborating with pharmaceutical, biotech and chemical companies, foundations and research institutes in north america, europe, asia, africa and australia to enable the discovery and development of novel medicines and agrochemicals.

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