Houston Methodist Academic Institute
LEADING RNA MEDICINE The mission of The Center for RNA Therapeutics at Houston Methodist is: (1) to perform cutting-edge research that generates innovative discoveries in RNA biology
CENTER FOR RNA THERAPEUTICS
(2) to translate our findings into transformative RNA therapeutics (3) to democratize RNA therapeutics by offering academic groups and companies a pathway to take their RNA research from bench to bedside
CENTER FOR RNA THERAPEUTICS The Center for RNA Therapeutics has developed infrastructure to create, manufacture, test, develop and implement first in man clinical trials. The Center for RNA Therapeutics is enriched by scientists with skills in RNA biology that are implementing new research findings into all aspects of RNA therapeutic development to provide the most current products and technologies to all researchers. The Center for RNA Therapeutics along with commitment from Houston Methodist Hospital Research Institute also offer GLP/ GMP manufacturing and GLP animal testing and can generate
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all preclinical data required for IND submissions. FIRST-IN-MAN CLINICAL TRIALS UNIT
In collaboration with the Department of Nanomedicine, we also
RNA BIOINFORMATICS
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offer encapsulation of the RNA into lipid nanoparticles. Our
Houston Methodist Academic Institute Center for RNA Therapeutics houstonmethodist.org/rna-therapeutics houstonmethodist.org/rnacore For more information contact us at
RNAcore@houstonmethodist.org 713.363.8554 6670 Bertner Avenue, Houston, TX 77030 HMRI Office of Communications & External Relations | CORE-RNA-22-00858 | EG | DH | 250 | 02.2023
philosophy is to democratize RNA, and therefore we offer these RNAs at affordable prices and quick turn-around times. Our
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services can be effective research tools for basic studies, or therapeutics for those wanting to develop them. Thus, everyone in research may find benefit from our services. Another service
GLP PRECLINICAL TESTING
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RNA CONSTRUCT DESIGN
Center for RNA Therapeutics
we offer is our expertise in using these tools, and experimental design in both an academic research setting as well as translational projects that are scaled up to move towards the clinic.
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ASSAYS TO VALIDATE INTEGRITY & PURITY
RNA SYNTHESIS & PURIFICATION NANOPARTICLE GENERATION & DELIVERY
cGMP MANUFACTURING TEAM & FACILITIES
CENTER FOR RNA THERAPEUTICS
WT Cells Transfected with HMH nGFP RNA
RNA SERVICES
RNA Therapeutics Supporting Investigators
The RNACore provides services for the development
Within past seven years:
and generation of high-fidelity mRNA, mmRNA, and long
• Manufactured for 10 small biotech companies
use of an error-free plasmid template and efficient, fast construct generation, we can provide a wide range of ready made and custom RNA molecules for any desired species, with various specified modifications. The RNACore produces high-fidelity research and clinical-grade RNA, including: • mRNA • Modified mRNA (mmRNA) • Self amplifying RNA • Encapsulation of Lipid Nanoparticles • Bicistronic constructs
RNA can be produced for any species or any loci of interest, and can incorporate any of the following modifications: • Modified Nucleotides • 5’ ARCA or CleanCap • Alternative polyA tails • Sequence optimization • Custom sequences
• Fulfilled over 300 orders from more than 75 different clients
** • D esigned and developed over 250 unique RNA constructs 10 * • Fulfilled 80 requests for analytical services Neutralizing Antibody Titers
noncoding RNA for any gene/loci of interest. With the
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• C onsulted for 12 companies on RNA design, manufacturing, 10 assessment, and delivery • Published 26 manuscripts
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Unvaccinated Empty LNPs mRNA Commercial Kit (Academic Lab) mRNA CleanCap (HMH) mRNA ARCA (HMH)
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ROADMAP TO THE CLINIC Our roadmap to the clinic can help you translate your ideas and take your product to the clinic. Our Center is enriched by scientists with skills
RNA IDEA
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ENCAPSULATE
RNA DRUG DEVELOPMENT
in RNA construct design, synthesis, purification,
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cGMP PRODUCTION 4 HMH · cGMP
An example of the superiority of mRNA vaccines made by our Center at Houston Methodist and that made in an external academic Unvaccinated lab as assessed by the Empty LNPs immune response of mRNA Commercial Kit (Academic Lab) vaccinated animals. mRNA CleanCap (HMH) mRNA ARCA (HMH) Attention to sequence, structure, base modifications, purification, encapsulation and other factors can improve translational efficiency and efficacy of RNA products.
pre-clinical data for IND submissions; a cGMP grade RNA therapeutics; a first-in-man clinical
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PHASE II · PHASE III · FDA APPROVAL VGXI
trials unit; and faculty collaborators in the Department of Nanomedicine who devise customized solutions for RNA delivery.
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The Houston Methodist Center for RNA Therapeutics received the Vaccine Industry Award for the Best Academic Team Research Award at the 2021 World Vaccine Congress. The Center was recognized for its work as an academic research organization supporting small companies and academic groups in their development and testing of mRNA Therapeutics.
RNA IDEA
Small Biotech · Academic Group
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DESIGN · SYNTHESIZE · PURIFY · LYOPHILIZE · VALIDATE HMH Center · RNA Therapeutics
GLP PRECLINICAL STUDIES HMH · CMP
and validation; a GLP program to generate team with clean rooms to manufacture clinical
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.
Neutralizing Antibody Titers
RNA available from RNAcore
PHASE I (FIRST IN MAN)
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CPRIT FUNDING The RNACore funded by CPRIT, can generate customized RNA constructs for cancer biologist and has allowed the cores researchers to be able to optimize new methods for RNA delivery. The CPRIT RNACore at the Houston Methodist Research Institute will help Texas scientists to make breakthrough discoveries, and will help Texas physician investigators turn these discoveries into transformational therapies for cancer patients.