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从效率插件到自动化研发闭环 AI制药范式迭代首先,是“预测工具”。代表能力包括虚拟筛选、结构预测、性质计算,AI主要是科研人员的效率插件。其次,是“生成平台”。模型开始从头生成分子和蛋白,商业模式从软件订阅升级到按项目交付、管线授权和联合研发。最后,是“实验闭环+临床验证”。模型与自动化实验室连接、连续产生高质量数据,自动试错迭代;候选物接受II、III期临床检验。平台还要证明复购、里程碑和持续交付能力。当前AIDD正处于2.0向3.0切换的早期。技术可用性与商业合作已经得到验证,Rentosertib进入III期是重要节点,但“AIDD能否系统性提高中后期临床成功率”仍没有准确答案。把阶段说得太成熟,会高估短期利润;把AI只当概念,又会低估研发范式的变化。AI可以在远超人工枚举能力的化学与蛋白空间中寻找新结构,并同时处理多个成药性目标。把失败前置,实现反向筛选。分子动力学、自由能计算、ADMET预测和表型模型,能在合成、动物和临床之前排除一部分低质量候选物。英矽智能公开研究显示,Rentosertib从靶点发现到临床前候选物提名约18个月;2025年发表的IIa期研究中,最高剂量组12周肺活量指标出现积极趋势,但样本量有限,仍需更大规模试验验证。让研发从“线性接力”变成“高频循环”。机器人实验室连续完成合成、纯化、检测和数据回传,科研智能体据此提出下一轮方案。真正的效率提升,是每轮迭代之间少等待、少丢数据。
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