Pipeline

Our interest is focused on DNA stability in stem cells maintaining different tissues. DNA becomes unstable, causing stem cell death, due to low DNA repair activity (mainly because of mutations in ATM), low telomerase activity leading to abnormally short telomeres (mainly caused by mutations in telomerase genes) and high sensitivity to oxidative stress due to low SOD and catalase function. Some of these aspects are more important than others in different pathologies but typically all of them concur.
We work with short synthetic peptides as active molecules, up to 11 amino acids. Typically we encapsulate them in PLGA, which ensures a very good distribution in all the body with no toxicity

Idiopathic Pulmonary Fibrosis (IPF)

In our pipeline, we are very interested in idiopathic pulmonary fibrosis (IPF) and our peptide Neumomir has already demonstrated very clear and strong results in IPF animal models, recovering lung tissue and function, also increasing animal lifespan, weight, etc. This drug might work both in patients where the disease is already developed (reverting the symptoms) even if these are well advanced and also as preventive in patients prone to suffer from the disase. Neumomir might be indicated be indicated to all IPF patients and not only those with low telomerase activity. In addition to the animal model, we have also tested this drug in biopsies from many different patients, with extraordinary results.

Blood Diseases

Blood diseases like dyskeratosis congenita (DC) and aplastic anemias (AA) are caused in many cases by low telomerase activity. It is very interesting that many of these patients also develop lung fibrosis, which might be addressed by our drugs too. Gestelmir and Anemir have been already tested in biopsies from patients, reverting the symptoms, increasing cell lifespan, etc.

Ataxia Telangiectasia

Ataxia telangiectasia is caused by low capacity of human cells to repair DNA, needed daily to tackle aggressions breaks in the double helix caused by external aggression, including oxidative stress. We are developing a drug that will address the main problem these patients have: death of Purkinje cells in their brains. We are developing a strategy able to make our drug cross the Blood Brain Barrier (BBB), so the drug could be administered as I.V. once every 2 or 4 weeks. In addition, these patients suffer bone marrow problems and also lung fibrosis, that our drugs could address too.

Down Syndrome

Down Syndrome is a very complex pathology but today we know that oxidative stress is a main factor because it causes neuronal cell death in early stages of the baby development. We are working on a new drug able to protect the baby brain even at very early stages of development, so the mom could take the drug during pregnancy and protect the baby’s brain; the drug would also be taken after birth, for life

Muscle Dystrophies

Muscle dystrophies have similar biochemical and cellular pathways with fibrosis because it is fibrosis what mainly end up preventing the sick muscle to keep on functioning. In our antifibrotic program, we have peptide it potential preventing this problem to occur, currently under development in animal models that suffer these diseases too.

Parkinson

Other diseases like Parkinson’s are known to be clearly aggravated by oxidative stress, so we are developing short peptides able to increase cell defense in these cases

Rare and Aggressive Tumors

Muscle dystrophies have similar biochemical and cellular pathways with fibrosis because it is fibrosis what mainly end up preventing the sick muscle to keep on functioning. In our antifibrotic program, we have peptide it potential preventing this problem to occur, currently under development in animal models that suffer these diseases too.

Fragile X

Fragile X is a disease causing mental retardation and cognitive problems to children and adults, X-Tocomir has passed Clinical Phase III demonstrating a clear improvement in cognitive functions of patients taking the drug, versus those only treated with the placebo. Our company plans to bring worldwide this drug to patients by the end of 2015. Also, we are investigating this drug in other diseases like Down Syndrome.

CONTACT US

  • IIB – CSIC
  • C/ ARTURO DUPERIER 4.
  • 28.029 MADRID, SPAIN
  • +34 91 830 59 50