Also called propionic aciduria, propionic acidosis (PA) is a rare autosomal recessive metabolic disorder that is a branched-chain organic acidemia. Propionic acidemia is the result of propionyl-CoA carboxylase deficiency (PCC deficiency), which is akin to methylmalonic acidemia arising from methylmalonyl-CoA mutase deficiency. Patients with propionic acidemia are unable to process certain proteins and fats properly, resulting in a buildup of toxic substances such as propionic acid in the blood/water, causing the signs and symptoms of propionic acidemia.
The PCC enzymes include alpha and beta subunits encoded by the PCCA and PCCB genes; mutations in both copies of the PCCA and PCCB genes result in PCC enzyme deficiency. Possible treatments for propionic acidemia may include functional enzyme replacement therapy or mRNA-based PCC therapy.
Encapsuled by LNP, mRNA-3927, a kind of mRNA medicine, comprises genes encoding the PCCA and PCCB for the expression of active PCC enzymes. mRNA-3927 was designed by Moderna to enable the body to restore the missing or dysfunctional PCC enzyme, which leads to propionic acidemia. Moderna's mRNA-3927 has received granted orphan drugs, rare paediatric disease designations, fast track designation from the FDA and orphan drug designation from the EMA for the treatment of propionic acidemia.
A Phase 1/2 study (first-in-human study) is currently underway to evaluate the drug's safety, pharmacologic activity and therapeutic efficacy of mRNA-3927 in patients with genetically confirmed propionic acidemia. In May 2023, Moderna presented that mRNA-3927 was demonstrated generally well-tolerated at the doses interjected from the interim data of the phase 1/2 study.
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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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mRNA Therapeutics Pipelines for Propionic Acidemia
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Code Name
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Target Protein
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Indications
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Manufacturer
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Latest Stage
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mRNA-3927
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α and β subunits of PCC enzyme (PCCA and PCCB)
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Propionic acidemia (PA)
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Moderna
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Phase I/II
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