What We Offer

Expert Scientific Services

Six specialized service areas designed and executed by industry experts to advance drug discovery, gene therapy and gene editing programs toward the clinic.

Functional Genomics Strategy

End-to-end design and execution of genome-wide and targeted CRISPR screens. Services include sgRNA library selection and synthesis, viral transduction or electroporation optimization, pooled screen execution, NGS-based library deconvolution, and MAGeCK/BAGEL2 analysis. Applicable in cancer models, primary HSCs, iPSC-derived cell types, and organoid systems.

Genome-wide KO CRISPRa/CRISPRi Pooled & Arrayed Library Design MAGeCK Analysis

Translational Biology Support

Scientific leadership across the IND-enabling spectrum — from disease biology assessment and proof-of-concept study design to CRO selection and management, protocol development, and data interpretation. Specialized expertise in gene therapy, base editing, and prime editing programs in hematopoietic stem cells and liver-targeted therapeutics.

IND Strategy HSC Engineering CRO Management Protocol Development Data Interpretation

Biomarker Development Strategy

Identification, selection, and analytical strategy for protein and genetic biomarkers in gene therapy clinical programs. Services include context-of-use definition, biomarker qualification roadmap development, and analytical framework for Phase 1 trial design. Expertise in pharmacodynamic biomarkers, patient stratification markers, and safety biomarkers specific to gene editing modalities.

Pharmacodynamic Patient Stratification Safety Markers Context-of-Use

Assay Development & Qualification

Full lifecycle assay development from exploratory research to GLP-aligned validation. Specialized in ddPCR editing efficiency assays, NGS-based off-target detection panels, CRISPR screen readout qualification, and multiplex biomarker panels. All deliverables include SOPs, analytical validation reports, and raw data packages suitable for IND filing.

ddPCR Assays NGS Off-Target Analytical Validation SOP Development GLP-Aligned

AI/ML Applications in Biology

Large-scale Perturb-seq dataset generation specifically designed for training gene expression foundation models and AI-driven drug target discovery platforms. Services include experimental design for perturbation coverage, cell line engineering, sgRNA library construction, 10x Genomics Chromium single-cell RNA-seq execution, preliminary QC and analysis, and structured data delivery in AI/ML-compatible formats.

Perturb-seq scRNA-seq Foundation Models 10x Chromium Data Engineering

Gene Therapy Research Support

Specialized support for gene therapy and gene editing programs advancing through preclinical and IND-enabling stages. Includes CRISPR/Cas9 and base editing target validation, CD34+ hematopoietic stem cell engineering and culture optimization, electroporation and RNP delivery protocols, and integration with FDA pre-IND guidance and regulatory pathway strategy.

CRISPR/Cas9 Base Editing CD34+ HSC RNP Delivery FDA Pre-IND

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