Monogenic loss-of-function diseases affect millions with few curative treatments, and mRNA vaccines' clinical success validates mRNA as a therapeutic platform. Creative Biolabs' Genetic Disease related mRNA Development Service delivers therapeutic protein blueprints rapidly, enabling safe transient protein replacement or precise gene editing via a non-integrating mutation-agnostic approach. Specializing in turning theoretical constructs into validated candidates, the end-to-end service uses advanced LNP formulation, nucleoside modification, and proprietary in silico optimization to solve key genetic medicine challenges and accelerate pipelines.
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Our therapeutic platform involves packaging In Vitro Transcribed (IVT) mRNA into highly engineered Lipid Nanoparticles (LNPs). Upon systemic or local administration, the LNPs are taken up by target cells via endocytosis. The ionizable lipids in our formulation are designed to facilitate endosomal escape (the process of releasing the mRNA from the endosome before it is degraded), allowing the intact mRNA to enter the cytoplasm. Here, the host cell's ribosome machinery immediately translates the mRNA into the desired therapeutic protein. Expression is transient, offering a highly controlled therapeutic window.
The service is highly effective for conditions resulting from the absence or deficiency of a single protein:
Fig.1 The strategy of treating rare genetic diseases with modified mRNA.1
We combine molecular biology with nanomedicine to create a best-in-class service platform:
Creative Biolabs delivers fully customized mRNA solutions, leveraging our decades of experience in nucleic acid chemistry and advanced delivery vectors. We translate complexity into efficacy, ensuring your genetic medicine program is positioned for clinical success.
Target Gene Sequence (cDNA), Disease Model Information, and Desired Target Cell Type or Organ.
Conduct in silico optimization, including codon usage adjustment and UTR engineering, then create the linearized DNA template.
Perform In Vitro Transcription with custom-selected modified nucleosides (e.g., Pseudouridine) and incorporate Cap 1 structure, yielding highly stable, low-immunogenic mRNA.
Precisely formulate ionizable lipids, helper lipids, and customized targeting moieties (e.g., high-affinity binding molecules for CNS penetration) to prepare mRNA-encapsulated LNPs with tailored tissue tropism.
Test endosomal escape, translational efficiency in target cells, and systemic efficacy and safety in relevant animal models to provide proof-of-concept for targeted delivery.
Adjust formulation and synthesis protocols to achieve scalable, reproducible production suitable for clinical trials.
Custom LNP Engineering
Precision-engineered proprietary ionizable lipids and targeting ligands enable extra-hepatic delivery, with validated formulations supporting central nervous system (CNS) penetration across the blood-brain barrier (BBB).
Proprietary Sequence Optimization
Specialized in silico algorithms drive untranslated region (UTR) engineering and codon optimization, effectively enhancing translational efficiency and protein expression yields in target cell populations.
Immunogenicity Mitigation Mastery
Tailored selection and incorporation of advanced nucleoside modifications (e.g., pseudouridine) and cap structures significantly attenuate innate immune responses, enabling safe repeat dosing critical for managing chronic genetic disorders.
Seamless Scale-Up & Quality Assurance
Integrated service portfolio covering initial research-grade in vitro transcription (IVT) synthesis, with a well-documented transition to good manufacturing practice (GMP)-compliant production for clinical materials—delivering a single, controlled platform throughout the entire development lifecycle.
Agnostic Therapeutic Design
Rapid development of a single mutation-agnostic mRNA construct, capable of addressing all loss-of-function defects within a target gene, substantially accelerating the translational path to clinical application.
Comprehensive QC/QA Protocols
Rigorous multi-stage quality control leveraging high-standard analytical techniques to quantify and assess the purity, stability, and encapsulation efficiency of the final mRNA-lipid nanoparticle (LNP) product.
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While mRNA expression is transient, we significantly extend its duration through superior nucleoside modifications and UTR engineering to maximize stability. For chronic conditions, we optimize the regimen for effective repeat dosing, utilizing our low-immunogenicity LNP formulation to ensure patient safety and compliance.
mRNA offers two major advantages: non-integration safety and the ability to target non-dividing cells (like neurons) immediately without needing to enter the nucleus. Furthermore, our LNP delivery system bypasses the limitations of pre-existing anti-AAV immunity, allowing for potential repeat dosing.
Yes. Since our approach is to deliver the wild-type, healthy gene product, a single mRNA construct can potentially treat all patients suffering from loss-of-function due to various point mutations in that single gene. This drastically accelerates the development timeline compared to variant-specific therapies.
We provide comprehensive in vivo biodistribution and expression studies, often using highly sensitive luciferase reporter mRNA, followed by quantitative PCR and immunohistochemistry to confirm expression levels and localization within specific brain regions or muscle tissues post-administration.
We offer both. Our service begins with research-grade materials for rapid in vitro and early in vivo testing. Once validation is complete, we seamlessly transition to producing GMP-compliant materials suitable for filing and advancing your therapeutic into clinical trials. Contact us for our detailed GMP production timeline.
Creative Biolabs offers a complete, integrated end-to-end service, providing the scientific rigor and technical expertise necessary to navigate the complexities of mRNA development for genetic diseases. Our focus on targeted delivery, reduced immunogenicity, and high-yield synthesis ensures your therapeutic asset is optimized for clinical success.
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| Cat. No | Product Name | Promoter |
|---|---|---|
| GTTS-WQ005MR | IVTScrip™ mRNA-Fluc, 5-Methoxy-U modified (Cap 1, 30 nt-poly(A)) | Reporter |
| GTTS-WQ006MR | IVTScrip™ mRNA-Fluc, 5-Methoxy-U, Cyanine 5-U modified (Cap 1, 30 nt-poly(A)) | Reporter |
| GTTS-WQ007MR | IVTScrip™ mRNA-mCherry, 5-Methoxy-U modified (Cap 1, 30 nt-poly(A)) | Reporter |
| GTTS-WQ008MR | IVTScrip™ mRNA-RLuc, 5-Methoxy-U modified (Cap 1, 30 nt-poly(A)) | Reporter |
| GTTS-WQ009MR | IVTScrip™ mRNA-β gal (Cap 1, 30 nt-poly(A)) | Reporter |
| Cat. No | Product Name | Type |
|---|---|---|
| GTVCR-WQ005MR | IVTScrip™ pT7-VEE-mRNA-β gal Vector | T7 |
| GTVCR-WQ006MR | IVTScrip™ pT7-VEE-mRNA-CCL1 Vector | T7 |
| GTVCR-WQ007MR | IVTScrip™ pT7-VEE-mRNA-IL1A Vector | T7 |
| GTVCR-WQ008MR | IVTScrip™ pT7-VEE-mRNA-TNF Vector | T7 |
| GTVCR-WQ009MR | IVTScrip™ pT7-VEE-mRNA-IFNG Vector | T7 |
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