唯可生物构建了面向基因编辑治疗产品的综合安全性评估平台，重点聚焦于靶向编辑事件的精准检测以及脱靶风险的系统性评估。该一体化解决方案融合先进的生物信息学分析与高通量测序技术，全面支持 IND 申报前研究及临床开发阶段的安全性评价需求。
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Target
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DNA
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Editing Method
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Double-strand break (DSB)
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Features
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Recognizes NGG PAM; high efficiency; widely used
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Advantages
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Easy to operate; well-established; mature applications (e.g., CAR-T, genetic disease research)
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Limitations
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May cause unintended large deletions and rearrangements; requires cellular DNA repair pathways; off-target risks
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Target
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DNA
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Editing Method
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Single-strand staggered cut (5' overhang)
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Features
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Recognizes T-rich PAM; creates sticky ends; cutting site is shifted
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Advantages
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More flexible PAM; suitable for A/T-rich regions; enables multiplex editing
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Limitations
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Off-target risk exists; efficiency may be lower than Cas9 in some systems
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Target
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RNA
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Editing Method
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RNA-guided cleavage
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Features
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Specifically targets RNA without affecting the genome
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Advantages
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Suitable for RNA editing and pathogen detection (e.g., SHERLOCK); no permanent genomic changes
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Limitations
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Limited to RNA; cannot introduce stable DNA edits; risk of collateral RNA cleavage
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Target
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DNA (single-base level)
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Editing Method
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Single-base conversion without DSB
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Features
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Cas fused to deaminase; enables C→T or A→G precise base substitutions
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Advantages
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High precision; low indel rate; ideal for correcting point mutations
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Limitations
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Limited to specific base conversions; editing window is restricted
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Target
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DNA (small fragment insertion, deletion, replacement)
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Editing Method
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Cas fused to reverse transcriptase + pegRNA; edits DNA without DSB
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Features
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Most versatile; supports targeted insertions, deletions, and all base substitutions
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Advantages
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Broad editing scope; fewer by-products; minimal DSB-related risks
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Limitations
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System is complex; delivery is challenging; editing efficiency varies; potential off-target
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唯可生物可依据合作伙伴的研发管线，定制专属专业解决方案
基于专业解决方案，唯可生物科学运用成熟的技术平台或开发新方法，以满足每个项目的具体需求。
凭借丰富的成功合作经验，唯可生物科学在病毒整合位点分析与基因编辑安全性评估领域处于领先地位。
源自德国癌症研究中心（DKFZ）与GeneWerk GmbH，在细胞与基因治疗（CGT）安全性评估领域拥有超过20年的专业经验。
唯可生物的质量管理体系全面符合 GCP 与 GLP 标准，覆盖临床与非临床研究全流程
通过严格的质量控制与全球标准遵循，唯可生物为每个研发项目保障数据的可靠性与完整性
国内CGT业务咨询
电话:021-68409257
+86 177 5118 1335
国内农业咨询
电话:+86 173 1002 4557
欧美CGT业务咨询
电话:+49 151 1246 3684
