Houston Methodist RNAcore Brochure

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Houston Methodist Research Institute 6670 Bertner Avenue Houston, TX 77030 houstonmethodist.org/RNAcore

As part of the Center for RNA Therapeutics, the RNAcore:

RNAcore RNAcore@houstonmethodist.org 713.441.7283

• performs rigorous research that reveals fundamental insights in RNA biology.

• translates new findings into novel RNA-based products.

• assists other academic groups and small companies in developing their great ideas into transformative RNA therapeutics.

The Houston Methodist Center for RNA Therapeutics received the Vaccine Industry Award for the Best Academic Team Research Award at the 2021World Vaccine Congress.

HMRI Office of Communications & External Relations | 100 | 05.2024

Houston Methodist Research Institute

RNAcore

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FIRST-IN-MAN CLINICAL TRIALS UNIT

RNAcore

RNA BIOINFORMATICS

RNA INNOVATION H2N

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REGULATORY STRATEGY

The RNAcore at the Houston Methodist GLP PRECLINICAL TESTING

Research Institute is as a leader in RNA

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RNA CONSTRUCT DESIGN

Center for RNA Therapeutics

cGMP MANUFACTURING TEAM & FACILITIES

synthesis, generating RNA constructs for the scientific and medical communities in the

ASSAYS TO VALIDATE INTEGRITY & PURITY

Texas Medical Center and across the world.

RNA SYNTHESIS & PURIFICATION NANOPARTICLE GENERATION & DELIVERY

The RNAcore is supported by the Cancer Prevention Research Institute of Texas.

Analytical Services

This Core also works with the government,

The RNAcore also provides services for assessing the purity, integrity and identity of in vitro transcribed RNA, following GLP (Good Laboratory Practices) guidelines. The following assays are provided on a fee-for-service basis:

of cutting-edge RNA technologies.

RNA Services The RNAcore is staffed by experienced scientists who design, synthesize, validate, test and encapsulate high-fidelity RNA to provide a wide range of generic or customized RNA molecules, for any desired species, with various specified modifications for research and clinical-grade including: • mRNA • modified mRNA (mmRNA) • self-amplifying RNA • long noncoding RNA • customized bicistronic constructs • constructs with reporter genes • lyophilization

Encapsulation Services We provide Lipid Nanoparticle (LNP) encapsulation of RNA as an integral service from idea to delivery.

• nucleic acid purity and concentration by UV Spectrophotometry • nucleic acid integrity by Bioanalyzer/TapeStation (microfluidic electrophoresis) • integrity and purity assessment by HPLC • impurity assessment of residual template DNA by qPCR • impurity assessment of residual E. coli DNA by qPCR • endotoxin testing of mRNA samples with the Nexgen-MCS • analysis of size, charge and encapsulation efficiency of lipid nanoparticles Neutralizing Antibody Titers

NGOs and industry to further the development

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Unvaccinated Empty LNPs mRNA Commercial Kit (Academic Lab) mRNA CleanCap (RNAcore) mRNA ARCA (RNAcore)

An example of the superiority of mRNA vaccines made by our RNAcore in comparison to the RNA made in an external academic lab as assessed by the immune response of vaccinated animals. Attention to sequence, structure, base modifications, purification, encapsulation and other factors can improve translational efficiency and efficacy of RNA products.

Specialized Services RNA molecules can be produced for any species, any loci of interest, and incorporate any of the following: • Modified nucleotides • 5’ CleanCap AU/AG and M6 cap • Long 150 nt poly-A tail • Sequence-optimized • Nucleotide fluorophore tagging

RNA can be generated as research or clinical grade. The RNAcore is now offering UltraPure RNA which has been HPLC purified to remove immunogenic byproducts and impurities.