20240024506. POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA simplified abstract (ModernaTX, Inc.)
Contents
- 1 POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA
- 1.1 Organization Name
- 1.2 Inventor(s)
- 1.3 POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA - A simplified explanation of the abstract
- 1.4 Simplified Explanation
- 1.5 Potential Applications
- 1.6 Problems Solved
- 1.7 Benefits
- 1.8 Original Abstract Submitted
POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA
Organization Name
Inventor(s)
Lei Jiang of Cambridge MA (US)
Lin Tung Guey of Lexington MA (US)
Paolo G. V. Martini of Boston MA (US)
Vladimir Presnyak of Manchester NH (US)
POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA - A simplified explanation of the abstract
This abstract first appeared for US patent application 20240024506 titled 'POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA
Simplified Explanation
The abstract of this patent application describes mRNA therapy for the treatment of propionic acidemia (PA). The mRNA molecules used in this invention encode human propionyl-coa carboxylase alpha (PCCA) and/or human propionyl-coa carboxylase beta (PCCB), as well as functional fragments and fusion proteins comprising PCCA and/or PCCB. These mRNA molecules are preferably encapsulated in lipid nanoparticles (LNPs) for efficient delivery to cells and tissues in subjects. The mRNA therapies increase and/or restore deficient levels of propionyl-coa carboxylase (PCC) expression and/or activity in subjects, while also decreasing levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity.
- mRNA therapy for propionic acidemia (PA)
- Encodes human propionyl-coa carboxylase alpha (PCCA) and/or human propionyl-coa carboxylase beta (PCCB)
- Functional fragments and fusion proteins of PCCA and/or PCCB
- Encapsulated in lipid nanoparticles (LNPs) for efficient delivery
- Increases and/or restores deficient levels of PCC expression and/or activity
- Decreases levels of disease-associated toxic metabolites
Potential Applications
- Treatment of propionic acidemia (PA)
- Gene therapy for genetic disorders affecting propionyl-coa carboxylase (PCC) activity
Problems Solved
- Deficient levels of propionyl-coa carboxylase (PCC) expression and/or activity
- Accumulation of disease-associated toxic metabolites
Benefits
- Improved treatment for propionic acidemia (PA)
- Restoration of normal PCC activity
- Reduction of disease-associated toxic metabolites
Original Abstract Submitted
this disclosure relates to mrna therapy for the treatment of propionic acidemia (pa). mrnas for use in the invention, when administered in vivo, encode human propionyl-coa carboxylase alpha (pcca) and/or human propionyl-coa carboxylase beta (pccb), and isoforms thereof, functional fragments thereof, and fusion proteins comprising pcca and/or pccb. mrnas of the invention are preferably encapsulated in lipid nanoparticles (lnps) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mrna therapies of the invention increase and/or restore deficient levels of propionyl-coa carboxylase (pcc) expression and/or activity in subjects. mrna therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient pcca or pccb activity, in subjects.
