Being a prokaryotic adaptive immune system, CRISPR/Cas can implant foreign disease-causing DNA into bacterial genomes. The discovery of this system revolutionized genome editing strategies because Cas9 can be directed to any desired target using a guide RNA (gRNA) composed of a customized crRNA sequence and tracrRNA fused together. This powerful technology offers great potential to reverse illnesses by correcting mutations in polygenic diseases such as cardiovascular disease.
The mRNA is neither infectious nor an integration platform, and there is no potential risk of infection or insertion of mutations. It is typically delivered by LNP as a non-virus vector. Several companies, including Intellia Therapeutics and CRISPR Therapeutics, have developed in vivo gene editing technologies based on mRNA and Cas9 gRNA.
Intellia Therapeutics developed NTLA-2001 for the treatment of Transthyretin Amyloidosis (ATTR). The company recently received IND clearance from the U.S. Food and Drug Administration to initiate a Phase III trial of NTLA-2001 for the treatment of ATTR-CM, with a significant international trial starting at the end of 2023.
CRISPR Therapeutics is developing CTX-310 for the treatment of atherosclerotic cardiovascular disease (ASCVD) and dyslipidemia.
Yaohai Bio-Pharma Offers One-Stop Solution for RNA
Custom Deliverables
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Grade
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Deliverables
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Specification
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Applications
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non-GMP
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Drug Substance, mRNA
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0.1~10 mg (mRNA)
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Preclinical research such as cell transfection, Analytical method development, Pre-stability studies, Formulation development
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Drug Product, LNP-mRNA
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GMP, Sterility
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Drug Substance, mRNA
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10 mg~70 g
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Investigational new drug (IND), Clinical trial authorisation (CTA), Clinical trial supply, Biologic license application (BLA), Commercial supply
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Drug Product, LNP-mRNA
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5000 vials or pre-filled syringes/ cartridges
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