Ready-Edit LNP Formulations
CRISPR LNP Gene Editing Solutions

Key Benefits and Advantages of Ready-Edit LNP Platform

Targeted Delivery

LNPs can be engineered to enhance the delivery efficiency of CRISPR system payloads

Reduced Off-Target Effects

mRNA provides transient expression of the gene-editing tool, reducing the likelihood of off-target effects

Scalability and Production

synthesized quickly and in large quantities using in vitro transcription

Safety Profile

mRNA does not integrate into the host genome, reducing the risk of insertional mutagenesis

ReadyEdit LNP Solutions Offerings

1. Start your pilot project with ReadyEdit LNP Solutions

• Cas9 system
• Cas12 system
• Base and Prime Editing

2. Build your CRISPR components including the synthesis of mRNA, sgRNA, ssDNA, and more!

3. LNP Formulations validated and optimized for specific CRISPR applications, featuring recommended ratios for each CRISPR component to significantly enhance your project's success rate

4. Validation with robust screening platforms to validate your gene-editing LNP designs

Case Studies

• Gene Knock out- TRAC Editing in primary T cells with Cas9 mRNA-LNP System

  Result: More than 80% editing observed at the TRAC locus

  

  Method: espCas9-mRNA : SafeEdit TRAC sgRNA were co-encapsulated in LNP at a molar ratio of 1:10 , 2.4 ug of RNA loaded LNP were incubated with cells for 72 hours, the gene editing efficiency for knocking out TRAC locus was sanger sequenced and editing efficiency was analyzed by ICE Analysis tool Software

• Gene Knock out- TRAC Editing in Mouse T cells & Jurkat Cells with Cas9 mRNA-LNP System

  Result: More than 70% editing and 80% editing observed in 3A9 and Jurkat Cells

  

  Method: espCas9-mRNA : SafeEdit TRAC sgRNA were co-encapsulated in LNP at a molar ratio of 1:10 , 2.4 ug of RNA loaded LNP were incubated with cells for 72 hours, the gene editing efficiency for knocking out TRAC locus was sanger sequenced and editing efficiency was analyzed by ICE Analysis tool Software. sgRNA-1 to sgRNA-6 were designed to target different spacer sequence in TRAC gene in mice.

• Gene Knock In- Cas9 mRNA-LNP system

  Result: 27% knock in observed with the LNP formulation

  
• Gene Knock out- High Gene Editing Efficiency in Cells with Cas12a mRNA-LNP System

  Result: 80% editing efficiency observed in HEK Cells

  

  Method: Cas12a mRNA/ SafeEdit TRAC sgRNA (GenScript) were packaged in LNP at 5:1 mass ratio and a serum dependent effect on editing was observed, RNA with SM102-LNP or ALC0315-LNP formulation were incubated with HEK293T cells, cell were lysed at day3 for NGS

• Prime Editing- Good Functional Performance Observed

  Result: >60% editing observed with the PEmax mRNA using electroporation

  

  Method: PEmax mRNA + HEK3 pegRNA with or without nicking guide RNA were encapsulated in SM102 modified LNP, LNP loaded with 2ug of RNA were incubated with 2×10⁵ HEK293T cells. 48h after transfection, cells were collected to extract gDNA. Gene editing were verified by NGS

• Validated ReadyEdit CRISPR LNP Solutions

  NO	LNP Formulations	mRNA	sgRNA	INDEL efficiency in test Cell lines
  1	SM102-LNP	espCas9	TRAC	HEK293T 97%, Jurkat 82%, T cells72% , HepG2 86%
  2	ALC0315-LNP	espCas9	TRAC	HEK293T 87%, Jurkat 80%, T cells 61%
  3	LP01-LNP	espCas9	TRAC	HEK293T 63%, Jurkat 75%
  4	20DODAP-LNP	espCas9	PCSK9	HepG2 87%
  5	SM102-SOPC LNP	espCas9	PCSK9	HepG2 90%
  6	SM102-LNP	HifiCas9	TRAC	HEK293T 97%, T cells 61% , HepG2 88%
  7	SM102-SOPC LNP	espCas9	TRAC	HepG2 90%
  8	SM102 LNP	Cas12a Ultra	TRAC	HEK293T 63%
  9	SM102-Mod LNP	PEmax	HEK3 pegRNA Nicking guide RNA	HEK293T 62%
  10	CXM2-LNP	CBE	TRAC	HEK293T 34%
  11	SM102-Mod LNP	CBE	TRAC	HEK293T 42%