Mitochondrial Disease Therapeutic Pipeline
Several treatments for mitochondrial diseases are already making their way along this path. Soon mito patients and their families will be able to see the treatment approaches being developed, where they are in the development pipeline, and what mitochondrial diseases they target.
Conditions in the Pipeline
- Barth Syndrome
- Inherited Mitochondrial Disorders
- KSS-CPEO
- Leigh Syndrome
- LHON
- MELAS
- MERRF
- Mitochondrial Myopathies
- MIDD
- Pearson Syndrome
- PDCD
- Seizures
- TK2 Deficiency

Pre-Clinical Development

Phase I

Phase II

Phase III

Regulatory Approval
Phase II/III tests how well a new treatment works and compares the new treatment with a standard treatment.
Barth Syndrome
Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics

KSS-CPEO
KL1333
Abliva AB

Leigh Syndrome
MNV-BM-BLD
PTC Therapeutics

NV354
Abliva AB

Leigh Syndrome


NV354
Abliva AB

Leigh Syndrome
MNV-BM-BLD
PTC Therapeutics
NV354
Abliva AB
LHON
LUMEVOQ (GS010)
GenSight Biologics
Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics
MELAS
Vatiquinone (PTC-743)
PTC Therapeutics
Study Name:
MIT-E Study
IW-6463
Cyclerion Therapeutics
Sonlicromanol (KH176)
Khondrion
KL1333
Abliva AB
MERRF
Vatiquinone (PTC-743)
PTC Therapeutics
Study Name:
MIT-E Study
KL1333
Abliva AB
Mitochondrial Myopathies/PMM
Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics
Bocidelpar (ASP0367)
Astellas Pharmaceuticals
REN001
Reneo Pharmaceuticals
MIDD
Sonlicromanol (KH176)
Khondrian
KL1333
Abliva AB
Pearson Syndrome
MNV-BM-BLD
Minovia Therapeutics
PDCD
Dichloroacetate
Seizures
Vatiquinone (PTC-743)
PTC Therapeutics
Study Name:
MIT-E Study
TK2 Deficiency
MT1621
Modis Therapeutics