Breakthrough Clinical Results
Sartorius Stedim Biotech and Sensible Biotechnologies have signed a Memorandum of Understanding (MoU) to collaborate on the industrialization and scale-up of Sensible's cell-based platform for mRNA therapeutics. The partnership will focus on process development, facility design, and technical support to enable clinical-grade production of cell-based mRNA by 2026. Sartorius Stedim Biotech will provide advanced equipment and consumables. This collaboration aims to expand Sensible's production capabilities from Slovakia to the US, UK, and broader Europe, unlocking the next generation of mRNA medicines.
Key Highlights
- Partnership between Sartorius Stedim Biotech and Sensible Biotechnologies to scale up cell-based mRNA manufacturing.
- Goal to achieve clinical-grade production of cell-based mRNA by 2026.
- Collaboration includes process development, facility design, and technical support.
- Expansion of Sensible's production capabilities to the US, UK, and broader Europe.
Company Mechanism of Action
Mechanisms of Action of Sensible Biotechnologies' Drugs
Based on the available information, there are no specific details about Sensible Biotechnologies or the mechanisms of action of their drugs. The context does not provide any information about:
- The company's therapeutic agents or their pharmacological mechanisms
- Molecular pathways or drug targets in their pharmaceutical compounds
- Receptor interactions, enzymatic inhibition, or signal transduction pathways of their drugs
- Any details about their investigational drugs or therapeutic portfolio
Without specific information about Sensible Biotechnologies in the provided context, a comprehensive answer about their drug mechanisms cannot be formulated.
Company drugs in pipeline
Sensible Biotechnologies' Drug Pipeline Indications
Based on the provided context, there is no specific information available about Sensible Biotechnologies' drug pipeline, therapeutic indications, disease targets, medical conditions, clinical-stage candidates, preclinical drug candidates, or product portfolio.
The context does not contain any details about Sensible Biotechnologies' development programs, therapeutic areas, or disease states that their drugs might be targeting.
MoA used in other indications
mRNA Therapeutics: Indications, Intervention Models, and Delivery Systems
Therapeutic Indications
mRNA therapeutics are being trialed for a wide range of diseases, with over 70 current clinical trials identified since the approval of the first mRNA therapeutic (COVID-19 vaccines) in 2021. These therapeutics fall into several major categories:
Vaccines
- Targeting viral antigens
- Targeting bacterial or parasitic antigens
- Targeting general and individualized cancer antigens
- Targeting self-antigens
- Infectious diseases including viruses like EV-A71 (hand, foot, and mouth disease)
Protein Replacement Therapies
- Genetic disorders (maintenance therapeutics)
- Interventional therapeutics where delivering therapeutic proteins could improve outcomes
- Hemophilia A (HemA)
- Ischemic heart disease using VEGF-A
- Cancer using proinflammatory cytokines
- Osteoarthritis using adipose derived stem cells engineered with modified mRNA encoding insulin-like growth factor 1 (IGF-1)
- Bone regeneration using mRNA encoding bone morphogenetic protein (BMP-2)
- Rare diseases through lipid nanoparticle-mRNA therapeutics
Therapeutic Antibodies
- Based on mRNA sequences encoding heavy and light chains of clinically relevant antibodies
- Enables patient cells to produce antibodies directly
Cell and Gene Therapies
- CRISPR with mRNA-mediated delivery of Cas9
- In vivo generation of cells expressing CAR through mRNA
- Cell reprogramming using modified mRNA molecules encoding pluripotent factors to reprogram somatic cells into pluripotent stem cells
- Gene editing using RNA-based technologies like base editor and prime editor CRISPR systems
Intervention Models and Delivery Systems
The effectiveness of mRNA therapeutics relies heavily on their delivery systems and formulations:
Lipid Nanoparticles (LNPs)
- Most common delivery system
- Improve delivery efficiency
- Protect mRNA from extracellular degradation
Chemically Modified mRNAs
- Base modifications (e.g., N-methyl pseudouridine)
- Show 5-12-fold increase in translation efficiency
- Enhance stability and reduce immunogenicity
Targeted Delivery Systems
- Gal-LNPs (galactose-modified lipid nanoparticles) for liver-targeting
- Used in Hemophilia A treatment with N-methyl pseudouridine (mΨ) modified functional FVIII mRNA
Biomaterial Combinations
- Transcript-activated matrices (TAMs) combining biomaterials and chemically modified mRNA (cmRNA)
- Fibrin gel with cmRNA
- Micro-macro biphasic calcium phosphate (MBCP) granules with cmRNA
- Used particularly for bone regeneration with cmRNA encoding bone morphogenetic protein (BMP)-2
Clinical Outcomes and Advantages
The clinical outcomes observed in trials demonstrate several advantages of mRNA therapeutics:
- Precise dosing
- Biocompatibility
- Transient expression
- Minimal risk of genomic integration
- Reduced risk of mutagenesis compared to other nucleic acid therapies
- Ability for repeated dosing and pharmacokinetic control
- Safer profile compared to viral gene therapies with reduced risks of genomic integration and oncogene activation
Specific outcomes include: - mΨ-FVIII mRNA showing high therapeutic efficacy up to 15 days in vivo in HemA mouse model - Gal-LNPs found to be safe for systemic administration - BMP-2 cmRNA inducing osteogenic markers and mineral deposition in bone regeneration models - Adipose derived stem cells with IGF-1 mRNA demonstrating superior therapeutic effect over native stem cells in osteoarthritis