Larimar Therapeutics’ proprietary protein replacement therapy platform has the potential to provide disease-modifying therapies that are intended to deliver missing proteins inside the machinery of cells to treat devastating rare diseases that currently have ineffective or no treatments available.
(Above) CTI-1601 is similar to frataxin, except it has a Cell Penetrating Peptide (CPP) attached on the end to allow it to move into the cell and mitochondria.
Endogenous Frataxin Production and Processing
Mitochondrial Targeting Sequence (MTS) and Frataxin are produced in the cytoplasm of the cell. Receptors allow the MTS to carry Frataxin through the mitochondrial membrane.
- 1 MTS and Frataxin coded in nuclear genes and produced in cytoplasm of the cell.
- 2 Receptors allow the MTS to carry Frataxin through the mitochondrial membrane.
- 3 Mitochondrial Processing Peptide (MPP) cleaves off MTS.
- 4 Mature human frataxin remains trapped within the mitochondria to function in electron transport chain.
Friedreich’s Ataxia Patients Have Low Levels of Endogenous Frataxin
Patients with FA only produce 10-15% of normal Frataxin levels.
Treatment with CTI-1601 to Supplement Endogenous Frataxin
- CTI-1601 is based on our proprietary platform technology and is designed to act as follows:
- 1 CTI-1601 crosses the cell membrane into cytoplasm.
- 2 CTI-1601 crosses the mitochondrial membrane.
- 3 Mitochondrial Processing Peptide (MPP) cleaves CTI-1601. MTS and CPP will leave cell mitochondria.
- 4 Mature human frataxin remains within the mitochondria to function.