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Breaking the delivery bottleneck · turning nucleic acid therapeutics into products

The delivery layer
of nucleic acid medicine

Viral vectors got the cargo in. LNPs made it manufacturable. The next generation is decided by precision and safety of delivery. Harmony Tao Biotech pairs proprietary ionizable lipids with a ligand library to design targeted delivery for liver, spleen, lung and other non-liver tissues.

Targeted LNP delivery
3靶向递送方向Targeted delivery programs
2在研管线 HT001 / HT002Pipelines HT001 / HT002
4平台核心资产Core platform assets
1即用型旗舰产品 Lipoeasy®Ready-to-use flagship Lipoeasy®
行业背景Industry context

核酸药物的下一站,取决于递送

The next wave of nucleic acid drugs is decided by delivery

mRNA 疫苗完成了核酸药物的产业化验证。基因编辑、体内细胞治疗、蛋白替代疗法正在把核酸药物推向更广阔的治疗场景 —— 而每一种新场景,都对递送的「组织选择性」与「安全性」提出更高要求。

mRNA vaccines industrialised nucleic acid drugs. Gene editing, in vivo cell therapy and protein replacement are now pushing them into far broader indications — and every new indication raises the bar on tissue selectivity and safety of delivery.

基因编辑

Gene editing

遗传病、肿瘤及代谢性疾病

Genetic, oncology & metabolic disease

致病基因修复、沉默或调控

Repair, silence or modulate the disease-causing gene

细胞治疗

Cell therapy

肿瘤、自身免疫病、再生医学

Oncology, autoimmune disease, regenerative medicine

改造免疫细胞或干细胞,增强靶向杀伤或修复功能

Engineer immune or stem cells for targeted killing or repair

蛋白替代疗法

Protein replacement

罕见病、慢性病

Rare and chronic disease

补充缺失或缺陷蛋白

Restore missing or defective proteins

核酸疫苗

Nucleic acid vaccines

传染病预防

Infectious disease prevention

表达抗原并诱导特异性免疫应答

Express antigen and induce specific immune response

两道行业关卡

Two barriers the industry has not cleared

行业痛点一Barrier 01

基因编辑依赖病毒载体,安全性代价高

Gene editing leans on viral vectors — with a safety price

('AAV / LV 载体转导高效、表达持久,是基因编辑的成熟路线,但存在免疫原性、大剂量给药的安全隐患与规模化生产瓶颈。行业迫切需要「非病毒、可重复给药」的递送方案。', 'AAV and lentiviral vectors transduce efficiently and express durably, but carry immunogenicity, dose-related safety concerns and manufacturing bottlenecks. The field needs a non-viral route that can be re-dosed.')

AAV and lentiviral vectors transduce efficiently and express durably, but carry immunogenicity, dose-related safety concerns and manufacturing bottlenecks. The field needs a non-viral route that can be re-dosed.

基因编辑三步:载体递送 · 进入细胞 · 基因编辑
Gene editing in three steps: vector delivery, cell entry, editing
行业痛点二Barrier 02

LNP 递送高度集中于肝脏与肌肉

LNP delivery is still concentrated in liver and muscle

('目前成熟的 LNP 应用集中在静脉肝靶向与肌注疫苗。脾、肺、背根神经节等组织与特定细胞类型的靶向递送,仍是限制核酸疗法边界的关键瓶颈 —— 这正是道一的切入点。', "Today's mature LNP applications are intravenous liver targeting and intramuscular vaccines. Spleen, lung, dorsal root ganglion and defined cell types remain the bottleneck that limits how far nucleic acid therapy can reach — this is exactly where Harmony Tao works.")

Today's mature LNP applications are intravenous liver targeting and intramuscular vaccines. Spleen, lung, dorsal root ganglion and defined cell types remain the bottleneck that limits how far nucleic acid therapy can reach — this is exactly where Harmony Tao works.

已实现肝靶向,更多组织仍在等待
Liver is solved; many organs are still waiting
三大方向Three directions

三个靶向递送方向

Three targeted delivery directions

同一个脂质材料平台,三种给药路径,解决三类临床未满足需求。

One lipid platform, three administration routes, three unmet clinical needs.

肝靶向 LNP 递送

Liver-targeted LNP delivery

基因编辑疗法Gene editing therapeutics
  • High-dose intravenous administration
  • Chronic-disease repeat dosing
  • Addressing: safety of high-dose, repeat dosing

脾靶向 LNP 递送

Spleen-targeted LNP delivery

in vivo CAR-TIn vivo CAR-T
  • Passive spleen targeting + active cell targeting
  • Engineering T cells directly in vivo
  • Addressing: manufacturing cost and access

肺靶向 LNP 递送

Lung-targeted LNP delivery

吸入给药Inhaled administration
  • Inhalation route
  • Chronic respiratory disease
  • Addressing: delivery efficiency and inhalation safety

ADDS:AI Design of Delivery System

A lipid library, a ligand library, the Lipoeasy® validation tool and a T-LNP in vitro / in vivo database are fused into one reusable delivery design engine, with AI in vitro and in vivo models turning formulation screening from trial-and-error into prediction.

Lipid libraryLigand libraryLipoeasy® toolT-LNP databaseAI models
LNP targeting

在研管线

Pipeline in development

HT001

肝靶向 LNP 递送的血友病 A 基因编辑疗法

Liver-targeted LNP delivery for haemophilia A gene editing

基因编辑疗法

Gene editing

HT002

脾靶向 in vivo CAR-T 肿瘤免疫治疗

Spleen-targeted in vivo CAR-T for cancer immunotherapy

体内细胞治疗

In vivo cell therapy

把您的 RNA 货物交给我们,我们负责把它送到正确的组织

Hand us your RNA cargo — we take care of getting it to the right tissue

('一体化递送设计服务:脂质材料定制 · LNP 处方筛选 · 理化表征 · 体内外转染验证 · 动物药效评价。', 'End-to-end delivery design: custom lipid synthesis, LNP formulation screening, physicochemical characterisation, in vitro / in vivo transfection and animal efficacy evaluation.')

End-to-end delivery design: custom lipid synthesis, LNP formulation screening, physicochemical characterisation, in vitro / in vivo transfection and animal efficacy evaluation.