Therapeutic IVT RNA
VectorBuilder offers a comprehensive platform for the development of RNA therapeutics including vaccines, gene replacement, chimeric antigen receptor (CAR), and gene editing. Leveraging our extensive design and production experience with premade and custom RNAs, our team can support in vitro transcription (IVT) RNA synthesis and lipid nanoparticle (LNP) encapsulation for a variety of RNA-based expression systems. Researchers can accelerate the development of their RNA therapeutics with our CRO services, offering in vitro and in vivo candidate screening and functional testing.
Highlights
Comprehensive Platform Offering research and clinical grades of RNA, a variety of RNA systems, customizable IVT RNA production and LNP encapsulation, and a full suite of CRO and CDMO services.
Experts in RNA Development Our team comprises scientists with expertise of the full scope of considerations for therapeutic IVT vector design, sequence optimization and screening, and process development.
Highest Quality and Consistency State-of-the-art facilities and equipment providing the highest purity and consistency, ensured by a full panel of quality control assays.Services Offered
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Featured Citations
Phase Separation Enhanced PROTAC for Highly Efficient Protein Degradation
Yu, et al.
Biomacromolecules, 2024, doi: 10.1021/acs.biomac.4c00424
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Abstract: Biomacromolecular condensates formed via phase separation establish compartments for the enrichment of specific compositions, which is also used as a biological tool to enhance molecule condensation, thereby increasing the efficiency of biological processes. Proteolysis-targeting chimeras (PROTACs) have been developed as powerful tools for targeted protein degradation in cells, offering a promising approach for therapies for different diseases. Herein, we introduce an intrinsically disordered region in the PROTAC (denoted PSETAC), which led to the formation of droplets of target proteins in the cells and increased degradation efficiency compared with PROTAC without phase separation. Further, using a nucleus targeting intrinsically disordered domain, the PSETAC was able to target and degrade nuclear-located proteins. Finally, we demonstrated intracellular delivery of PSETAC using lipid nanoparticle-encapsulated mRNA (mRNA-LNP) for the degradation of the endogenous target protein. This study established the PSETAC mRNA-LNP method as a potentially translatable, safe therapeutic strategy for the development of clinical applications based on PROTAC.
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