Startups
2017
MITOPATH SLThrough development of a novel gene therapy treatment, MitoPath is dedicated to helping patients living with MNGIE take back their future.
Mitochondrial NeuroGastroIntestinal Encephalomyopathy or MNGIE, is a rare autosomal recessive mitochondrial disease. People living with MNGIE have a mutation in their DNA where they have low to no expression of a protein called thymidine phosphorylase or TYMP gene.
Having low to no TYMP protein indirectly leads to the incorporation of mutations in the mitochondrial DNA. As time goes on, more mutations are incorporated until the mitochondria are unable to carry out their normal functions.
The disease’s symptoms become apparent around puberty, but the accurate diagnosis of the disease is often delayed by 6 – 8 years due to it being commonly misdiagnosed as any of the following: anorexia nervosa, MELAS, MERRF, inflammatory bowel disease, superior mesenteric artery syndrome, Whipple disease, chronic intestinal pseudo-obstruction, chronic inflammatory demyelinating polyneuropathy or Charcot Marie Tooth disease. Individuals with MNGIE have extremely low weight, and their first symptoms tend to be neurologic. As the disease progresses, the function of the gastrointestinal system is impaired, leading to the most severe symptoms and eventually resulting in death in ~85% of cases by age 40.
Mitochondrial NeuroGastroIntestinal Encephalomyopathy or MNGIE, is a rare autosomal recessive mitochondrial disease. People living with MNGIE have a mutation in their DNA where they have low to no expression of a protein called thymidine phosphorylase or TYMP gene.
Having low to no TYMP protein indirectly leads to the incorporation of mutations in the mitochondrial DNA. As time goes on, more mutations are incorporated until the mitochondria are unable to carry out their normal functions.
The disease’s symptoms become apparent around puberty, but the accurate diagnosis of the disease is often delayed by 6 – 8 years due to it being commonly misdiagnosed as any of the following: anorexia nervosa, MELAS, MERRF, inflammatory bowel disease, superior mesenteric artery syndrome, Whipple disease, chronic intestinal pseudo-obstruction, chronic inflammatory demyelinating polyneuropathy or Charcot Marie Tooth disease. Individuals with MNGIE have extremely low weight, and their first symptoms tend to be neurologic. As the disease progresses, the function of the gastrointestinal system is impaired, leading to the most severe symptoms and eventually resulting in death in ~85% of cases by age 40.
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Avinguda de Pablo Picasso 12, 08940 Cornellà de Llobregat, Spain
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DeepTech Area
Spinoff information
Company Description
MitoPath is actively pursuing a gene therapy that uses a modified virus (AAV2/8) to deliver millions of functioning copies of the TYMP gene to liver cells. We believe that this approach has a high likelihood of significantly improving the quality of life of patients with MNGIE. For us to deliver this gene therapy to all affected, we first need to raise funding and demonstrate effective results through a clinical trial. That’s where you come in.
Product description
Patient Impact
Highly derisked opportunity enabled at Columbia Univ. and Vall d’Hebron Research Institute in Barcelona, Spain.
Partners
Looking for strategic partners with whom to complete the pre-IND package of the lead clinical candidate.
Vision
Clinical trial sites identified in US and EU for a run-in, dose escalation trial. Aware of 3 times the number of patients needed for trial based on EMA Protocol Assistance Meeting.
Highly derisked opportunity enabled at Columbia Univ. and Vall d’Hebron Research Institute in Barcelona, Spain.
Partners
Looking for strategic partners with whom to complete the pre-IND package of the lead clinical candidate.
Vision
Clinical trial sites identified in US and EU for a run-in, dose escalation trial. Aware of 3 times the number of patients needed for trial based on EMA Protocol Assistance Meeting.