Compound Screening and Drug Discovery Core

Platform Capabilities

• Cystic Fibrosis (CF): Our fully established, flagship platform. It is widely recognized as a preclinical gold standard for CF drug development.
• Other Disease Indications: Our technology is highly adaptable to disease areas such as pulmonary, gastrointestinal, dermatological, renal, and metabolic. We welcome the opportunity to develop and optimize new functional assays in these areas.

Therapeutic Modalities We Test

We can evaluate cellular function after treatment with a wide variety of modalities including: mRNA, circRNA, ASOs, CRISPR-edited cells, small molecules, and peptides.

Equipment

• 2-Plate BREATHE-Z TECC-24 System: allows for transepithelial resistance (Rt) and potential difference (PD) measurements from two 24-well Transwell filter plates (48 separate membranes). The TECC is surrounded by a chamber that allows for analysis with or without CO2.
• 5-Plate MTECC-24 with Impedance Spectra Analysis: allows Rt and PD measurements from five 24-well Transwell filter plates (120 separate membranes). This system integrates impedance spectra analysis for a deeper, more comprehensive investigation of epithelial barrier functions.

Model Systems and Biobank Resources

• Biobank: Houses a number of patient-derived primary airway cells, immortalized 16HBEge cells, and FRT cells.
• CFTR cDNA Library: Consisting of over 600 CFTR variants.
• Custom FRT Lines: Custom generation of stable FRT cell lines expressing target constructs.
• Specialized Media: Optimized media formulated to support primary airway cell growth and differentiation.

New Exploratory Assay Development

The TECC assay equipment is compatible with a wide range of cell lines. We are equipped to onboard, test, and optimize assays using the following cell lines (either client-provided or core-sourced):

• Cell Lines: human bronchial epithelial (HBE) derived from people with CF, 16HBEge, nasal epithelial, Fisher rat thyroid (FRT), T84, Calu-3, Caco-2, HEK293, and NCI-H441
• Potential Target Indications: COPD, asthma, non-CF bronchiectasis, idiopathic pulmonary fibrosis (IPF), interstitial lung diseases, ulcerative colitis, Crohn’s disease, celiac disease, pancreatic insufficiency, eczema, psoriasis, polycystic kidney disease, nephropathic cystinosis, diabetic nephropathy, Liddle syndrome.

Downstream Analysis

• qPCR(including CFTR analysis)
• Western Blot(including CFTR)

Note: Additional targets may require optimization.

Menu of Services

Established CF Services (Ready to Run)

• High-throughput functional testing (TECC assays) for CFTR
• Provision of established CF cell lines (FRT, CFBE, etc.)
• Molecular cloning and stable transfection of CFTR variants
• Validated downstream molecular analysis:
  • qPCR(specifically optimized for CFTR analysis)
  • Western Blot(specifically optimized for CFTR profiling)
• Specialized media supply (Airway cell differentiation media / 3T3 cell-conditioned media)

Pilot & Custom Assay Development (New Indications)

• Collaboration on protocol design for non-CF epithelial models
• Onboarding and baseline barrier integrity testing for new cell lines

Note: All non-CF targets and downstream analyses will require a collaborative optimization phase.

Whether you want to utilize our gold-standard Cystic Fibrosis screening pipeline or pioneer a new epithelial barrier assay for another disease area, we are ready to collaborate.

For more information and to discuss your project, please contact: Jessica Centa, PhD | Assistant Director | jessica.centa@rosalindfranklin.edu