SHANGHAI and MIDDLETOWN (DE),

上海和米德尔敦（特拉华州），

April 9, 2025 —

2025年4月9日 —

HuidaGene Therapeutics ('HuidaGene'), a global clinical-stage biotechnology company advancing next-generation genomic medicines, today announced the expansion of its high-fidelity CRISPR-Cas12 platform, hfCas12Max, into new therapeutic areas through sublicensing by Synthego Corporation ('Synthego') to Vita Therapeutics, Inc.

慧达基因治疗公司（HuidaGene），一家全球临床阶段的生物技术公司，致力于推进下一代基因组药物，今天宣布通过Synthego公司（Synthego Corporation）向Vita Therapeutics公司转授权，将其高保真CRISPR-Cas12平台hfCas12Max扩展到新的治疗领域。

('Vita')..

('生命')..

Under the sublicense agreement, Vita gains access to HuidaGene's engineered hfCas12Max nuclease and optimized guide RNAs for use in preclinical and clinical applications, including the development of hypoimmunogenic iPSC-based cell therapies targeting neuromuscular disorders.

根据分许可协议，Vita获得了HuidaGene的工程化hfCas12Max核酸酶和优化的引导RNA的使用权，用于包括开发针对神经肌肉疾病的低免疫原性iPSC细胞疗法在内的临床前和临床应用。

This sublicensing milestone underscores the significant value and broad applicability of HuidaGene's proprietary hfCas12Max platform. Initially licensed to Synthego in 2024 for manufacturing, commercialization, and sublicensing, hfCas12Max continues to gain industry recognition for its superior on-target editing efficiency, reduced off-target effects, and compact size, ideal for therapeutic delivery..

这一转授权里程碑突显了惠达基因公司专有的hfCas12Max平台的巨大价值和广泛适用性。该平台最初于2024年授权给Synthego用于制造、商业化和转授权，hfCas12Max凭借其卓越的靶向编辑效率、减少的脱靶效应以及适合治疗递送的小巧尺寸，持续获得行业认可。

'The expansion of hfCas12Max into clinical cell therapy programs underscores the growing influence of HuidaGene's genome editing innovations across various therapeutic areas,' stated Alvin Luk, Ph.D., M.B.A., Chief Executive Officer and Co-founder of HuidaGene. 'We are proud to witness our high-fidelity CRISPR technologies facilitating pioneering treatments beyond our own pipeline, reinforcing our mission to deliver transformative, one-time cures to patients worldwide.'.

“hfCas12Max在临床细胞治疗项目中的扩展应用，彰显了辉大基因的基因编辑创新在各个治疗领域的影响力日益增强，”辉大基因首席执行官兼联合创始人郑洪飞博士（Alvin Luk, Ph.D., M.B.A.）表示，“我们很自豪看到我们的高保真CRISPR技术正在推动超出我们自身研发管线的开创性疗法，进一步巩固我们为全球患者提供变革性一次性治愈方案的使命。”

hfCas12Max was developed through HuidaGene's proprietary AI-powered, CRISPR-based HG-PRECISE

hfCas12Max 是通过辉大基因专有的 AI 驱动、基于 CRISPR 的 HG-PRECISE 开发的。

Ò

Ò

platform, a leading technology engine for engineering novel DNA editors, RNA editors, and epigenetic regulators. Its broader adoption through partnerships further strengthens HuidaGene's leadership position in the field of genome editing.

平台，一个领先的技术引擎，用于设计新型DNA编辑器、RNA编辑器和表观遗传调控因子。通过合作更广泛地采用该平台，进一步巩固了辉达基因在基因组编辑领域的领导地位。

For more information about the strategic licensing agreement for hfCas12Max CRISPR nuclease between Synthego and Vita, please visit

有关Synthego与Vita之间关于hfCas12Max CRISPR核酸酶的战略许可协议的更多信息，请访问

www.synthego.com.

www.synthego.com

About HuidaGene

关于慧达基因

HuidaGene utilizes its proprietary AI-driven, CRISPR-based

HuidaGene利用其专有的AI驱动、基于CRISPR的

HG-PRECISE

HG-PRECISE

Ò

Ò

platform to develop potentially curative genome medicine. The company's current clinical programs include:

开发潜在治愈性基因组药物的平台。公司目前的临床项目包括：

HG004gene replacement therapy (ODD & RPDD from the US FDA and ODD from EMA) for

HG004基因替代疗法（获得美国FDA的ODD和RPDD以及EMA的ODD认证）

RPE65

RPE65

-associated retinal disease (IRD)

-相关视网膜疾病 (IRD)

'LIGHT'

'光'

first-in-human trial (NCT06088992)

首次人体试验 (NCT06088992)

China's first

中国的第一个

'STAR'

‘星星’

Phase 1/2 multi-national, 'master protocol' clinical trial (NCT05906953)

第1/2阶段多国、“主协议”临床试验（NCT05906953）

HG202 CRISPR/RNA-editing therapy for neovascular age-related macular degeneration (AMD)

HG202 CRISPR/RNA编辑疗法用于新生血管性年龄相关性黄斑变性（AMD）

'SIGHT-I'

'视线-I'

first-in-human trial (NCT06031727)

首次人体试验 (NCT06031727)

World's first CRISPR/RNA editing therapy

世界上首个CRISPR/RNA编辑疗法

'BRIGHT'

'明亮'

Phase 1 clinical trial (NCT06623279)

第一阶段临床试验 (NCT06623279)

HG204 RNA-editing therapy (ODD & RPDD from FDA and ODD by EMA) for

HG204 RNA编辑疗法（获得FDA的ODD和RPDD以及EMA的ODD）用于

MECP2

MECP2

duplication syndrome

重复综合征

World's first CRISPR/RNA-editing

世界上首个CRISPR/RNA编辑

'HERO'

'英雄'

for neurodevelopment disorders first-in-human trial (NCT06615206)

用于神经发育障碍的首次人体试验 (NCT06615206)

HG302 DNA-editing therapy (ODD & RPDD from FDA) for Duchenne muscular dystrophy (DMD)

HG302 DNA编辑疗法（获得FDA的ODD和RPDD资格）用于杜氏肌营养不良症（DMD）

World's first CRISPR/DNA-editing

世界上第一个CRISPR/DNA编辑

'MUSCLE'

'肌肉'

first-in-human trial (NCT06594094)

首次人体试验 (NCT06594094)

The preclinical pipelines are advancing simultaneously in HG303 CRISPR/DNA-editing for ALS and CRISPR/RNA-editing therapy for Alzheimer's and Huntington's Disease. With an extensive intellectual property portfolio, HuidaGene is a leader in genome medicines for neurology and ophthalmology. Learn more at .

临床前管线正在HG303 CRISPR/DNA编辑治疗ALS（肌萎缩侧索硬化症）和CRISPR/RNA编辑治疗阿尔茨海默病及亨廷顿病方面同时推进。凭借广泛的知识产权组合，HuidaGene是神经学和眼科基因组药物的领导者。欲了解更多信息，请访问。

huidagene.com

辉达基因.com

or on

或在

LinkedIn.

领英。

Previous

上一步

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不再

Next

下一步

HuidaGene Therapeutics to Present at Multiple Key Conferences Highlighting Advances in CRISPR-based Therapies

惠达基因治疗公司将在多个重要会议上展示基于CRISPR疗法的进展。

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