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From bench to bedside guide RNA Manufacturing
From pre-clinical to commercial therapeutic quality

guide RNA Manufacturing

First discovered in 1990 in a single-celled organism, guide RNA is a type of RNA (ribonucleic acid) that serves as a “guide” for proteins and enzymes to reach the target sequences and perform an edit. Through the formation of complexes between the guide RNA and enzymes, the genetic alteration of a target RNA or DNA is possible.

BioSpring is the world-leading expert in guide RNA manufacturing and analysis. We are here to guide your programs to clinical and commercial readiness, providing you with unmatched quality and technical support to ensure your success. We offer customizable quality grades, including preclinical-grade, pre-GMP quality and GMP grade. Whether you need µgs for preclinical programs or large quantities of guide RNA for commercial applications, our proven and scalable manufacturing platform has you covered. In addition to manufacturing, we offer the full suite and customization of release tests, analytical method development, and validation for guide RNAs – early-stage to commercial.

To maintain our leadership in clinical and commercial manufacturing of guide RNA, we tripled our guide RNA capacity in 2023. For clients with programs in advanced stages, we offer process validation and commercial readiness support.



                gmp certified         fda inspected

Ex vivo and in vivo guide RNAs for: 

  • Genome Editing 
  • Base Editing
  • Next Generation Genome Editing 

~ 40 - 140 (+)mers*

*longer lenghts available upon request

 

Customizable grades, always the highest quality

quality grades gRNA

 

Accelerating your path to clinic and beyond

With two decades of GMP RNA manufacturing experience

  • No lead time to initiate GMP programs
  • Continuous capacity increase & development of new technologies
  • Cutting-edge solutions: MS/MS & NGS for ID confirmation & sequencing, high-resolution LC-MS for impurity characterization, in vitro activity assays
  • Fully-customizable release & stability scopes, tailored to your specific application

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Filling Services

We offer batches in liquid, frozen liquid, and lyophilized form. Our non-sterile filling for GMP and GLP/Tox is carried out in a Class C (ISO 7/8) non-sterile environment. We also offer the option for low endotox and bioburden filling. In the coming years, we look forward to implementing sterile fill and finish services for guide RNA.

Have a question or need a quote? Contact us

     
     

Need mRNA for your gene editing program?

Now offering IVT mRNA

Knowledge Corner

What is gene editing?

Gene editing is a process that takes advantage of the natural repair system of DNA strands in order for specific changes to be made in the DNA. Possibly the most well-known platform of gene editing is the Cas9 system that uses sgRNA (single guide RNA). This system was pioneered by Emmanuelle Charpentier and Jennifer A. Doudna, who won the Nobel Prize in Chemistry in 2020 for their work in recreating the “genetic scissors” from bacteria Streptococcus pyogenes’ CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas immune system. The CRISPR-Cas9 system is able to precisely cut DNA under direction of a sgRNA approximately 100 nucleotides long. The sgRNA is made up of two main sections: the “scaffold” region, also known as “direct repeat region” which provides structure; and the “spacer” region, which is specific to the target DNA sequence. This combination allows for a precise double-strand cut to be made.

CRISPR_Cas.jpeg

What is base editing?

Another application of guide RNA-mediated editing is base editing. In base editing, a single base pair is targeted and transformed from C-G to T-A, or A-T to G-C. For editing to occur, guide RNA recruits the modified Cas9 protein and deaminase protein to the target sequence. Base editing allows for targeted editing of the DNA strand, while only having to nick (cut) one strand of DNA.

Bases_crop.jpeg 

 

Ex vivo vs in vivo gene editing

There are many applications of gene editing. In ex vivo gene editing, cells from a patient are extracted, edited, and then reintroduced to the patient (autologous cell therapy), or similarly using the cells from a healthy donor (allogeneic cell therapy). A challenge to highlight in ex vivo gene editing is that the edited cell must be able to survive the extraction, manipulation, and transplantation process, as well as potentially replicate when returned to the body.

In_Vitro_Editing.jpeg

In contrast, in vivo gene editing is used in cases where the patient’s cells cannot be removed and reintroduced, as in the case of some diseases that target the central nervous system and the musculoskeletal system. Ensuring safe and efficient delivery of the essential ingredients for editing the target tissue, not to mention controlled cell editing within the patient, are potential limitations in in vivo gene editing.

The field of gene editing is rapidly diversifying and evolving. CRISPR Car-T cells designed to target cancerous leukemia and lymphoma cells are in clinical trials, as are CRISPR-based treatments of sickle cell disease. The therapeutic potential of guide RNA in developing novel therapies across a broad spectrum of disease states is vast.

References

Rohner, E., Yang, R., Foo, K.S. et al. Unlocking the promise of mRNA therapeutics. Nat Biotechnol 40, 1586–1600 (2022). https://doi.org/10.1038/s41587-022-01491-z
Foster, J. B., Barrett, D. M., & Karikó, K. The emerging role of in vitro-transcribed mRNA in adoptive T cell immunotherapy. Mol Ther, 27(4), 747-756 (2019). https://doi.org/10.1016/j.ymthe.2019.01.018
Sahin, U., Karikó, K. & Türeci, Ö. mRNA-based therapeutics — developing a new class of drugs. Nat Rev Drug Discov 13, 759–780 (2014). https://doi.org/10.1038/nrd4278

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