Our Science
Larimar Therapeutics’ proprietary protein replacement therapy platform has the potential to provide disease-modifying therapies that are intended to deliver missing or deficient proteins inside the machinery of cells to treat devastating rare diseases that currently have limited or no treatments available.
(Above) Nomlabofusp is the same as frataxin found in human cells, 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 Peptidase (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
- Nomlabofusp is based on our proprietary platform technology and is designed to act as follows:
- 1 Nomlabofusp crosses the cell membrane into cytoplasm.
- 2 Nomlabofusp crosses the mitochondrial membrane.
- 3 Mitochondrial Processing Peptidase (MPP) cleaves nomlabofusp.
- 4 Mature human frataxin remains within the mitochondria to function. MTS and CPP will leave mitochondria.