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BioPharma

Services

Precision NanoSystems’ BioPharma Services accelerates the development of mRNA-lipid nanoparticle (LNP) formulations to create genomic medicines by using NanoAssemblr® instruments, ionizable lipids and off-the-shelf reagents offering, end-to-end LNP Formulation Services and cGMP manufacturing. 

Leverage Deep Expertise to Expedite Preclinical and Clinical RNA-LNP Manufacturing

Accelerate formulation development, optimization, and process scale-up by collaborating with our BioPharma Services team that includes payload design, analytical development services, quality control and quality assay (QA/QC) services. If you are looking for a reliable CDMO partner, our contract services team is here to deliver customized solutions for successful LNP drug manufacturing.  


Our experienced team of scientists specializes in optimizing the use of limited materials in scaling drug products from discovery to cGMP manufacturing for a broad range of drug carriers such as Lipid Nanoparticles, Liposomes, and Polymeric Nanoparticles (poly(lactic-co-glycolic acid) PLGA). We have the expertise in handling a wide range of drug payloads, including mRNA, siRNA, saRNA, Replicon RNA, Peptides, Small Molecules, and Viruses with the NanoAssemblr® and GenVoy™ technology platforms. Collaborate with us to develop a comprehensive project plan with customized milestones, deliverables, timeline and budget that aligns with your goals. 

26


Clinical Stage Programs

>44


GMP Batches

2


Approved Drugs

*Precision NanoSystem’s BioPharma Services team is made up of experts that bring in their cumulative experience of working with 26 clinical stage programs producing over 44 GMP batches and have contributed to two approved drug products.
BioPharma Services team
Complete Solutions for Every Step of Pharmaceutical Development 

Partner with BioPharma Services on projects that span the entire drug development process, including proof-of-concept studies in early discovery, lead candidate development in scale-up and manufacturing processes for cGMP production. 

 

 

Formulation Development Icon

Formulation Development  

  • Optimize physicochemical properties and biological activity 
  • Identify critical quality attributes (CQAs) and draft drug product specifications 

Process Development icon

Process Development 

  • Establish critical process parameters (CPPs) for downstream processing, including tangential flow filtration (TFF) and sterile filtration 

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Scale-Up  

  • Confirm equivalency with demonstration batches   
  • Prepare and test batches to support GLP-Tox studies

Tech Transfer Icon

GMP Manufacturing 

  • Prepare GMP Master Batch Records
  • Manufacture GMP batches for Phase I and II clinical trials 

CMC

Chemistry Manufacturing & Controls (CMC) Support 

  • Provide oversight for the manufacture and testing of engineering and GMP batches  
  • Assist with CMC Regulatory Submissions

Screening Icon

Bioassay Development and Screening 

  • Develop robust bioassays to evaluate the potency and in vitro gene expression of any drug payload  
  • Develop a workflow to screen large numbers of variables to support formulation and process development  

Analytical Services Icon

Analytical Services 

  • Develop analytical methods for nanoparticle formulations and raw materials 

 

Analytical Services

 

Developing the right analytical assays for each stage of your project is critical to ensure success in the path from preclinical to clinical drug development. With deep expertise and technical knowledge of genomic medicines, BioPharma Services develops and applies advanced analytical methodologies to support the characterization of the drug substance, raw materials and/or nanoparticle formulation. Our analytical development team works across the preclinical, clinical, process development, manufacturing and quality teams from selecting lead candidates during formulation development to ensuring specifications of the drug product remain consistent as production is scaled.

 

 

Analytical Capabilities

Analytical services for non-viral genomic medicines include drug product identity confirmation, physical characterization, acceptance testing, stability studies, GMP release testing, toxicology testing and raw material testing.

 

Lipid Nanoparticles

 

  • Particle Size Distribution (PDI)
  • Osmolality
  • Zeta-Potential
  • pH
  • Encapsulation Efficiency
  • Total Drug Substance Content
  • In Vitro Potency

 

Lipids

 

  • Multi-Lipid Content
  • Multi-Lipid Identity/Integrity
  • Raw Material Testing
  • Stability/Forced Degradation
  • Residual Solvents
  • Solubility
  • Trace Impurity Analysis

 

Nucleic Acids

 

  • Capping Efficiency
  • PolyAdenylation
  • Residual DNA/Protein
  • dsRNA
  • Unnatural Ribonucleotides
  • Identify & Integrity 
  • Large RNA or saRNA Characterization

 

 

 

 

Analytical Instrumentation

Analytical instrumentation and technologies to assess drug substances and products include particle size analyzers, bioanalyzers, mass spectrometers, liquid chromatographic systems, cryogenic electron microscopes and flow cytometers.

 

Nanoparticles 


  • Dynamic Light Scattering (DLS) 
  • Osmometer 
  • Endotoxin* 
  • Bioburden* 
  • Cryo-TEM* 

 

 

 

 

Lipids

 

  • LC-MS/MS 
  • UHPLC-UV 
  • HPLC-ELSD 
  • UHPLC-CAD 

 

 

 

 

 

Payloads 

 

  • LC-MS/MS 
  • UHPLC-UV 
  • UHPLC-FLR 
  • CE-UV/LIF 
  • Bioanalyzer 

 

 

 

 

Bioassays 

 

  • Cytation 7 cell imaging multi-mode reader, Biospa, and MultiFlo Fx  
  • SDS-PAGE and Jess Automated Western blot  
  • Biotek plate readers (Colorimetric + Luminescence-based assays)  
  • Ella automated ELISA  
  • CytoFLEX Flow Cytometer  
  • RT-qPCR 

     

    *Available through third-party 

    Our BioPharma Services Team

    Experienced: Leverage comprehensive knowledge, insights and expertise in formulation development, process development, analytical development, quality, and GMP manufacturing.

    Transparent: Prioritize direct communication and access to protocols and processes during the development of your drug product. 

    Integrated: Optimize the use of limited materials with NanoAssemblr and GenVoy technology platforms, enabling seamless scaling of drug products from preclinical to clinical development. 

    Flexible: Continue development and manufacturing through the late stages of drug development or transfer protocols to continue development in-house. 

    Streamlined: Collaborate across functional teams from proof-of-concept studies to cGMP manufacturing for Phase I and II clinical trials for an end-to-end workflow. 

    Drug Payloads, Drug Carriers and Research Applications

     

     

    BioPharma Services has broad technical expertise in payload design and lipid-based delivery systems, including lipid nanoparticle and lipopolymeric formulations, for a wide range of disease applications.

     

     

     

    Drug Payloads

    RNA Icon

     

    mRNA, siRNA, saRNA, Replicon RNA 

     

     

    pDNA Icon

     

    pDNA, dsDNA 

     

     

     

    Small Molecule Icon

     

    Small Molecule 

     

     

     

    Peptide icon

     

    Peptide 

     

     

     

    Virus icon

     

    Virus

     

     

     

    Drug Carriers

    LNP

     

    Lipid Nanoparticles

     

     

     

    LIPOSOME

     

    Liposomes

     

     

     

    POLYMER

     

    Polymer Nanoparticles

     

     

     

    EMULSION

     

    Emulsions

     

     

     

    Exosome

     

    Exosomes

     

     

     

    Research Applications 

    Vaccine icon

     

    Vaccine Development 

     

     

     

    Oncology icon

     

    Oncology 

     

     

     

    Liver Delivery

     

    Liver Delivery

     

     

     

    Cardiovascular Disease icon

     

    Cardiovascular Disease

     

     

     

    Lung Delivery icon

     

    Lung Delivery

     

     

     

    Partner with BioPharma Services
    Learn how our team can help accelerate your project
    BioPharma Services Brochure
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    Resource Center

    Publication - Summary

    September 24, 2018

    Nucleic Acid Therapeutics

    MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct

    R. Jain, J.P. Frederick, E.Y. Huang, K.E. Burke, D.M. Mauger, E.A. Andrianova, S.J. Farlow...

    Read More 阅读更多

    Poster

    November 09, 2021

    Accelerated Development of Self-amplifying mRNA (saRNA) Vaccines using Microfluidics

    Read More 阅读更多 PDF

    Poster

    August 31, 2022

    Solving Challenges in Manufacturing of mRNA Drug Products: Rapid Development and Scale-Up of a Model mRNA Ther...

    Read More 阅读更多 PDF

    Publication - Summary

    April 04, 2019

    The Journal of Neuroscience

    PTCD1 is Required for Mitochondrial Oxidative-phosphorylation: Possible Genetic Association with Alzheimer's D...

    D. Fleck, L. Phu, E. Verschueren, T. Hinkle, M. Reichelt, T. Bhangale, B. Haley, Y. Wang, ...

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    Poster

    April 01, 2018

    A Scalable Microfluidics Platform for the Development of Nanoparticles

    Read More 阅读更多 PDF

    Publication - Summary

    February 27, 2018

    Cell Reports

    A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editi...

    J. Finn, A. Smith, M. Patel, L. Shaw, M. Youniss, J. Heteren, T. Dirstine, C. Ciullo, R. L...

    Read More 阅读更多
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