Companies to follow

The most clinically advanced name in the field. Peptide elamipretide (SS-31) targets cardiolipin on the inner mitochondrial membrane to shore up energy, furthest along in dry AMD and primary mitochondrial myopathy.

Swedish, spun out of NeuroVive. Lead drug KL1333 is an NAD+ modulator meant to restore cellular energy in genetically defined mitochondrial disease (the FALCON program in MELAS-type patients).

Dutch clinical-stage biotech founded by a mitochondrial-medicine physician. Sonlicromanol (KH176) is an oral redox modulator tested in mitochondrial disease (MELAS) and now post-COVID.

A larger rare-disease company with a real mitochondrial footprint, vatiquinone targets oxidative stress (15-lipoxygenase) in Friedreich's ataxia and inherited mitochondrial disease.

The one I watch most for VDAC-adjacent reasons: they inhibit the deubiquitinase USP30 to unleash mitophagy, clearing damaged mitochondria, with programs in kidney disease and Parkinson's.

Protein-replacement play: nomlabofusp delivers frataxin, the protein missing in Friedreich's ataxia, into mitochondria to restore iron–sulfur cluster biology.

Israeli pioneer of mitochondrial augmentation therapy (MAT), enriching a patient's own stem cells with healthy mitochondria, in Pearson syndrome and other primary mitochondrial diseases.

Betting on mitochondrial transplantation, delivering isolated, functional mitochondria as a therapeutic, starting with ischemia–reperfusion injury and transplant medicine.

Japanese biotech developing stabilized functional mitochondria and mitochondria-derived therapeutics for acute organ injury and energy-starved tissues.

Big pharma in the space via its 2017 acquisition of Mitobridge, PPARδ agonism to drive mitochondrial biogenesis, explored in primary mitochondrial myopathy and acute kidney injury.

Well-funded newcomer going after mitochondrial DNA replication and quality control, tuning how cells make and maintain their mitochondrial genome. One to watch.

UK biotech designing small molecules that protect mitochondria from oxidative damage, with programs pointed at Parkinson's, Friedreich's ataxia and muscular dystrophy.

Developing controlled mitochondrial uncouplers (MP101/MP201), gently 'leaking' the membrane to cut oxidative stress, for ALS, MS and traumatic brain injury.

Omaveloxolone (Skyclarys), an Nrf2 activator that reboots antioxidant and mitochondrial gene programs, is the first FDA-approved drug for Friedreich's ataxia (via the Reata acquisition).

Mavodelpar (REN001) is a PPARδ agonist meant to boost mitochondrial biogenesis and fatty-acid oxidation in primary mitochondrial myopathies.

Gene-targeted chimeras (GeneTACs) aimed at the repeat expansion that silences frataxin, trying to fix Friedreich's ataxia at the DNA level.

AAV gene therapy delivering frataxin to the heart, targeting the cardiomyopathy that's the leading cause of death in Friedreich's ataxia.

AAV gene therapies for severe monogenic CNS disease, including Rett syndrome and other conditions where mitochondrial and metabolic failure drive neuronal loss.

Blarcamesine activates the sigma-1 receptor at the ER–mitochondria interface (the MAM), aiming to restore cellular homeostasis in Alzheimer's, Parkinson's and Rett syndrome.

CNM-Au8 is a suspension of gold nanocrystals that catalytically supports the NAD+/NADH balance to keep neurons energized, in late-stage trials for ALS and multiple sclerosis.

Deuterium-stabilized fatty acids that resist lipid peroxidation, protecting mitochondrial membranes, in Friedreich's ataxia, ALS and infantile neuroaxonal dystrophy.

Broad neurodegeneration player whose programs touch mitochondrial and lysosomal quality control (e.g. LRRK2) in Parkinson's and ALS.

Small molecules that stimulate mitochondrial biogenesis to rebuild muscle and nerve energy capacity, aimed at neuromuscular disease.

Best known for Duchenne gene therapy, now expanding into gene therapies for inherited cardiac conditions, including mitochondrial cardiomyopathies.

Swiss biotech (EPFL spinout) that discovered and holds the IP on urolithin A, a gut-metabolite that induces mitophagy; ran the human clinical trials (published in Nature Metabolism and JAMA Network Open) establishing its muscle and mitochondrial effects before licensing it as Mitopure.

David Sinclair-founded, developing next-gen NAD+ boosters (MIB-626) as actual drugs, in clinical testing for metabolic and age-related decline.

Partial epigenetic reprogramming to restore youthful cell function, with mitochondrial and metabolic rejuvenation central to the thesis; lead program in optic neuropathy.

Mines human ageing data for targets in muscle and metabolic decline, where mitochondrial function is a recurring theme, and runs clinical metabolic programs.

A 'rejuvenation' holding company with a pipeline of ageing-biology programs, several centered on mitochondrial and metabolic health.

Runs in-vivo pooled screens (many perturbations in one animal) to find drivers of age-related decline, mitochondrial dysfunction squarely in scope.

Developing 'mitochondrial enhancers', mild uncoupling and redox tuning, to raise baseline mitochondrial resilience in ageing and neurodegeneration.

Early-stage UK biotech developing small-molecule inhibitors of the mitochondrial permeability transition pore, the structure whose prolonged opening drives neuronal death in ALS and Parkinson's.

Early-stage biotech developing small-molecule activators of PINK1, the kinase that kicks off mitophagy, as a disease-modifying approach to Parkinson's rather than a symptom treatment.

Swiss biotech spun out of EPFL, developing small molecules that activate mitophagy directly, targeting neurodegeneration and other diseases where clearing damaged mitochondria could help.

Biotech built around activating autophagy, including mitophagy, with small molecules; a broader cellular-quality-control platform with programs touching neurodegeneration.

Uses an AI-driven mitochondrial-biology platform to find drugs that target cancer metabolism; lead candidate BPM31510 (a CoQ10-based therapy) has been tested in glioblastoma and pancreatic cancer.

Clinical-stage biotech developing an optimized formulation of dichloroacetate, a pyruvate dehydrogenase kinase inhibitor, for Leigh syndrome and other primary mitochondrial diseases.

Early-stage biotech working to restore coenzyme A levels for PKAN, a genetic disorder that causes mitochondrial iron accumulation and neurodegeneration.

Clinical-stage biotech developing PrimeC, a combination therapy for ALS that includes an iron-chelation component thought to help protect motor-neuron mitochondrial function.

Gene-therapy company with a late-stage program for Danon disease, caused by defective autophagy (including mitophagy) that leads to severe cardiomyopathy.

Early-stage UK biotech targeting cellular senescence, a state in which dysfunctional mitochondria are a defining hallmark, aiming to ease age-related tissue decline.

Swiss longevity biotech with multiple subsidiaries working on senescence, the gut-brain axis and other aging biology, several with mitochondrial-health angles.

Longevity biotech working on partial cellular reprogramming, plasma-based therapeutics and autophagy induction, with mitochondrial rejuvenation as part of the underlying biology.