Gene therapy medicines deliver recombinant genes into target cells to achieve therapeutic, and in some cases prophylactic or diagnostic, effects. They are investigated and used in oncology, monogenic diseases and various chronic indications.

Gene Design and Vector Development

Biomedica provides tools and services to support gene construct design and vector engineering, including:

• Lentiviral products and services, including ORF clones and plasmids 
   
• AAV products and services 
   
• CRISPR associated proteins and nucleases 
   
• CRISPR Cas9 gene editing kits
   

These solutions enable efficient and targeted design of gene therapy vectors and transgenes for research and preclinical development.

Gene Transfer and Transfection

Efficient gene delivery, particularly into hard to transfect immune cells, is critical for gene and cell therapy workflows. Biomedica offers:

• ProteanFect™ TuffCell Transfection Kit
  Coacervate based delivery platform for reliable gene transfer into primary T cells and NK cells, providing high efficiency with minimal cell stress. 
   
• Additional transfection reagents for a wide range of cell types and applications
   

This enables robust and reproducible expression systems for gene therapy research.

Viral Titer Quantification and Vector Characterization

Comprehensive characterization of viral vectors (e.g. AAV, lentivirus) is essential for process development and quality control:

• Ella™ / Simple Plex™ Microfluidic Cartridge Assays
  Automated immunoassays as a robust and rapid alternative to conventional ELISA or dot blot approaches for AAV titer determination. 
   
• Maurice
  Next generation icIEF and CE SDS platform to assess stability, identity and purity of AAV vectors and lipid nanoparticles (LNPs), including empty versus full capsid analysis. 
   
• Simple Western™ Technology
  Automated capillary western platform providing a multi attribute solution for monitoring critical quality attributes (CQAs) such as purity, titer, empty/full capsid content and capsid protein ratios. 
   
• Milo
  Automated single cell western platform to measure protein expression in individual cells, enabling assessment of gene editing efficiency and expression at single cell resolution.

Functional Assessment, Immune Response and Safety

To optimize therapeutic efficacy and safety of gene therapy products, Biomedica offers solutions for functional assays and immunomonitoring:

• nCounter® Gene Therapy Optimization Panel
  Standardized gene expression panel covering innate and adaptive immune responses, host–vector interactions and toxicity related pathways (≈800 genes across 40 pathways). 
   

• RNAscope™ in situ hybridization (ISH)
  Single molecule, subcellular resolution ISH to visualize 

  • Tissue biodistribution and cellular tropism of AAV and other viral vectors
  • Transgene expression 
   

• ELISpot and FluoroSpot assays
  Sensitive analysis of cellular immune responses to peptide pools derived from vector and transgene in PBMCs (e.g. IFN γ, TNF α, IL 2, granzyme B) as well as innate responses such as type I interferon. 
   
• Lentivirus Titer p24 ELISA Detection Kit
  High sensitivity ELISA for accurate quantification of lentiviral titer in cell culture supernatants, supporting optimization of production and transduction. 
   
• Micro Flow Imaging™ (MFI) systems
  Direct imaging of subvisible particles in solution to detect and characterize AAV capsid aggregates and contaminants based on multiple morphological parameters.
   

Somatic cell therapies use cells or tissues that have been manipulated ex vivo so that their biological properties are changed or they perform a new function in the body. A leading example is CAR T or CAR NK cell therapy, where immune cells are engineered to recognize and destroy tumor cells.
Biomedica supports key steps in these workflows – from cell isolation and genetic modification to expansion, functional testing and quality control.

Targeted Cell Isolation and Enrichment

Efficient and gentle isolation of defined cell populations is fundamental to cell therapy manufacturing:

• Pala Cell Sorter and Single Cell Dispenser
  Benchtop, fluorescence activated platform for gentle single cell isolation into 96 /384 well plates or enrichment into tubes, suitable for clonal expansion and targeted enrichment in minutes. 
   
• MojoSort Magnetic Cell Separation
  Animal component free (ACF), GMP grade, antibody coated magnetic beads for the selection and isolation of cells with consistent lot-to-lot bioactive performance.
   
• MagCellect™ Magnetic Cell Separation and Isolation Kits
  Column free magnetic separation for enrichment of defined cell subsets by positive or negative selection. 

CAR Detection and Quantification

Monitoring expression, trafficking and persistence of CAR constructs is critical for product characterization:

• Anti linker rabbit monoclonal antibodies (G4S, Whitlow/218)
  Recognize commonly used scFv linker sequences and enable universal detection of CAR constructs expressed at physiological levels; full GMP certification expected in 2026.
   
• CAR Enrichment Kits
  Magnetic bead based immunoaffinity kits for targeted enrichment of CAR expressing cells, using releasable beads to obtain bead free, viable CAR+ cells; full GMP certification expected in 2026.
   
• Posibeads™
  Bead based positive controls that behave like cells and are coated with G4S or Whitlow/218 linker peptides to validate antibody conjugates and ensure specific binding and fluorescence in flow cytometry. 
   
• G4S and Whitlow/218 CAR Antigen Density Quantitation Kits
  Kits designed to quantify surface expression levels of CARs containing G4S or Whitlow/218 linkers.
   

In addition, Biomedica offers Ultra LEAF™ and ACF GMP grade flow cytometry antibodies, minimizing interference from endotoxin or preservatives in sensitive immune and stem cell assays.

Differentiation, Expansion, Activation and Culture Media

For ex vivo expansion, polarization and differentiation of immune and stem cells:

• CellXVivo Immune Cell Differentiation and Expansion Kits
  Optimized combinations of proteins and antibodies with streamlined protocols for dendritic cells, macrophages, T helper subsets, regulatory T cells, B cells, NK cells and cytokine induced killer (CIK) cells. 
   
• ExCellerate Media
  Media formulations tailored for immune and stem cell expansion. 
   
• ProPak™ GMP Cytokines and GMP T Cell Media Bags
  GMP grade reagents and media bags for scalable cell processing. 
   
• PromoExQ GMP grade excipient and cell culture solutions
  Produced at EXCiPACT™ certified sites with extensive documentation packages (CoA, EQAF, GMP and safety statements, certificates of origin and conformity). 
   
• Cancer Cell Culture Portfolio
  Xeno free, serum free media and supplements for expansion of primary human cancer cells and cell lines as 2D monolayers or 3D spheroids under highly standardized conditions. 
   
• Cell Vive™ GMP cell culture and ancillary reagents
  Chemically defined (CD), xeno free (XF) and animal component free (ACF) products manufactured under USP <1043> and Ph. Eur. 5.2.12 guidelines in a dedicated ISO 13485:2016 certified GMP facility.
   
• Genie Color Mycoplasma Detection Kit
  Rapid, one-step colorimetric assay designed to detect mycoplasma contamination with high accuracy and minimal equipment requirements.
   

• GMP grade recombinant proteins and growth factors 
   

  • GMP grade cytokines and growth factors
  • Ultra-low endotoxin proteins

For cleanroom compatible handling, triple bagged (3 B) cell culture plastics are available to help reduce contamination risk when transferring materials into high grade aseptic production areas.

Functional Assessment and Quality Analytics for Cell Therapies

To characterize potency, function and safety of cell therapy products, Biomedica provides:

• Beacon optofluidic platform
  Real time, multimodal single cell functional profiling of T cells, linking phenotypic and functional data from the same live cells to accelerate discovery and candidate selection. 
   
• nCounter® CAR T Characterization Panel
  Gene expression panel developed with CAR T experts to support robust product release assays and QC, covering eight key biological domains with 780 human genes. 
   
• FluoroSpot assays for CAR T functionality
  Multiplexed cytokine secretion assays complementing flow cytometry, aligned with regulatory expectations for comprehensive immune profiling of CGT and ATMP products. 
   
• Simple Plex™ Platform
  Automated immunoassay system for high quality potency and cytokine data with minimal hands on time, suitable for cell therapy development and manufacturing. 
   
• Cell & Gene Therapy Bioprocess Kits
  Highly sensitive ELISA based kits for host cell proteins (HCPs), residual DNA and other process related impurities, supporting quality and safety throughout development and production. 
   
• Human CAR T Cytokine Release Syndrome (CRS) 18 Plex Panel
  Simultaneous, quantitative detection of key CRS mediators from very small sample volumes on standard flow cytometers. 
   
• LumiMAT™ Endotoxin Test
  NF κB based, luciferase reporter cell line assay for sensitive detection of pyrogens via luminescence readout on microplate readers. 
   
• RNAscope™ ISH
  Spatial analysis of AAV vector biodistribution, transgene mRNA and cell markers in tissues; also suitable for assessing antisense oligonucleotide (ASO) or siRNA therapeutics alongside target transcripts.

Tissue engineered medicines aim to replace, repair or regenerate human tissues. They often rely on stem cells and organoids, which require precisely defined, reliable reagents from basic discovery through to translational research.

GMP Grade Reagents for Stem Cells and Organoids

Biomedica offers an extensive portfolio of reagents tailored for stem cell and organoid workflows:

• GMP grade recombinant proteins and growth factors 

• GMP grade cytokines and growth factors
• Ultra-low endotoxin proteins

• Organoid culture proteins and growth factors
  Including niche defining proteins for organoid maintenance and expansion. 
   
• Antibodies for organoid markers and organoid specific reagents
  Covering key markers and signaling pathways involved in organoid biology.

GMP grade small molecules

Designed to support ex vivo stem cell and organoid workflows, enabling controlled modulation of proliferation, differentiation and reprogramming under GMP compatible conditions.
 

Extracellular Matrix proteins

High quality ECM preparations that closely mimic the in vivo niche and support both 2D and 3D culture formats.

Wnt3a Based Organoid Culture 

Essential niche components for maintaining Lgr5 positive intestinal stem cells and generating organoids from the small intestine, colon, stomach, pancreas and liver.
Often combined with Afamin to provide optimal conditions for long term organoid growth. 

The quality and safety of ATMPs are tightly linked to controlled, sterile manufacturing conditions. Biomedica provides a range of solutions for environmental monitoring, microbiological quality control and rapid release testing.

Cleanroom and Particle Monitoring

• TRIO.BAS NVC Particle Counter
  Advanced handheld particle counter measuring particle sizes from 0.3 to 5 µm at 0.1 CFM (2.83 LPM), ideal for monitoring cleanroom conditions during critical, small batch and individualized therapy production.

Automated Plate Reading and Ready to Use Culture Media

• EMMA imaging platform
  Vision AI based system for automated capturing and analysis of Petri dishes (55–90 mm) with various media, enabling reliable classification of positive and negative plates for environmental and microbiological QC. 
   
• Ready to use culture media
  Standard and customized media, USP/EP/JP conform and produced in cleanroom class 5-8 (conform ISO14644), for environmental monitoring, sterility testing and media fills (including bottles, contact plates and media fill infusion bags; lot reservation options available).

Rapid Microbiological and Endotoxin Testing

• LumiMAT™
  Endotoxin/pyrogen test using NF κB activated NOMO 1 cells with luciferase reporter readout; luminescence based detection on plate readers enables sensitive, rapid pyrogen testing. 
   
• IAN
  Ultra rapid microbiological detection system using solid phase cytometry and AI to detect and enumerate bacteria, yeasts and molds in liquids within minutes; designed for industrial QC labs and non growth based results. 
   
• PRENOVA® TCT3
  Technology that differentiates human from microbial ATP, significantly shortening microbial load release testing (e.g. to 2–3 days) – particularly relevant for “just in time” microbiological release in T cell and CAR T therapy workflows.
   
• RiboNat High Speed Sterility Test
  Sterility test that utilizes the Nucleic Acid Amplification Test (NAT) method to rapidly detect bacteria and fungi.