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Off-the-shelf compositions for target genes

We developed advanced gene editing compositions for multiple genes to treat different diseases using our novel proprietary OMNI nucleases. These gene editing compositions were validated using several systems from computational through human cell-line to target cells validations. Each solution was adjusted to the specific editing requirements of the diseases including allele specific knockout (KO), the use of non-NGG nucleases and the use of a variety of nucleases with different sizes to fit different delivery modalities. These compositions are available for out-licensing and can be adjusted to fit your needs.

Target Gene
Organ/Purpose
Computational
In Cell-Line
In Target Cells
AAVS1
Any type
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AAVS1
Any type
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APLP2 
Macrophages 
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APLP2 
Macrophages 
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C3 
Liver
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C3 
Liver
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ROSA26 
Any Type
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ROSA26 
Any Type
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Target Gene
Computational
In Cell-Line
In Target Cells
B2M
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B2M
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CD3E
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CD3E
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CIITA
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CIITA
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CISH
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CISH
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CTLA4
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CTLA4
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FAS
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FAS
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FASLG
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FASLG
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HAVCR2 (TIM3)
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HAVCR2 (TIM3)
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HLAE
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HLAE
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IL15
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IL15
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LAG3
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LAG3
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PDCD1 
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PDCD1 
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TET2 
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TET2 
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TIGIT
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TIGIT
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TRAC 
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TRAC 
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TRBC1 
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TRBC1 
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TRBC2
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TRBC2
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Target Gene
Disease
Computational
In Cell-Line
In Target Cells
ANGPTL3
Dyslipidemia including homozygous familial hypercholesterolemia
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ANGPTL3
Dyslipidemia including homozygous familial hypercholesterolemia
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HBV
Hepatitis
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HBV
Hepatitis
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LDLR
Atherosclerotic cardiovascular disease
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LDLR
Atherosclerotic cardiovascular disease
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SERPINA1 
A1AD 
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SERPINA1 
A1AD 
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Target Gene
Disease
Computational
In Cell-Line
LRRK2 
Parkinson’s disease 
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LRRK2 
Parkinson’s disease 
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Target Gene
Disease
Computational
In Cell-Line
In Target Cells
ELANE 
Severe Congenital Neutropenia 
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ELANE 
Severe Congenital Neutropenia 
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GATA2
Myeloid malignancies
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GATA2
Myeloid malignancies
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RPS19
Diamond Blackfan Anemia
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RPS19
Diamond Blackfan Anemia
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SAMD9
Myeloid malignancies
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SAMD9
Myeloid malignancies
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SAMD9L
Myeloid malignancies
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SAMD9L
Myeloid malignancies
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Target Gene
Disease
Computational
In Cell-Line
BEST1
Autosomal dominant vitreoretinochoroidopathy
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BEST1
Autosomal dominant vitreoretinochoroidopathy
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FLG
Ichthyosis vulgaris
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FLG
Ichthyosis vulgaris
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PRPH2
Retinitis Pigmentosa
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PRPH2
Retinitis Pigmentosa
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RHO
Retinitis Pigmentosa
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RHO
Retinitis Pigmentosa
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RPE65
Retinitis Pigmentosa
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RPE65
Retinitis Pigmentosa
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SARM1
Neuronal and macular degeneration
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SARM1
Neuronal and macular degeneration
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TCF4 
Fuchs Endothelial Corneal Dystrophy 
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TCF4 
Fuchs Endothelial Corneal Dystrophy 
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TGFBi 
Corneal Dystrophies 
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TGFBi 
Corneal Dystrophies 
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Custom nucleases

We use our in-house AI-based computational engineering platform to predict optimal nuclease variants based on the customer’s requirements. These predicted variants are then tested in our high- throughput screening systems to validate their improved efficacy. We offer the optimal variants to our collaborators and licensees via out-licensing agreements. All our validated nucleases are highly active and highly specific.

Consulting services

EmendoBio is an expert in gene editing with vast experience in the discovery of novel nucleases, engineering them, and development of CRISPR based gene editing compositions. We offer our partners consulting services in several areas including gene editing strategy design, gRNA selection, off-target experiments and analysis, and more.

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Partners

EmendoBio’s OMNI platform has expanded the CRISPR nuclease toolbox and currently provides a great variety of CRISPR gene editing based solutions. We believe in the power of partnerships to drive innovation and create a lasting sustainable value and believe that our OMNI platform can contribute greatly to the development of novel CRISPR based therapies. We offer our OMNI nucleases for out licensing and welcome strategic collaborations to integrate OMNI nucleases in the development of novel therapies.

Our Collaborators and Licensees

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Contact Our Business Development Team

Please email us at bd@emendobio.com and we shall get back to you shortly.

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