Our goal is to develop innovative therapies to help patients
suffering from rare genetic disorders
Our goal is to develop innovative therapies to help patients
Pluvia is a spin-out from the University of Bergen (Norway) working to commercialize decade long research on protein structure and protein misfolding. In particular we are working to develop small molecule pharmacological chaperones with the potential to stabilize and correct the misfolding of proteins involved in selected loss-of-function diseases, thereby rescuing the enzymatic activity needed to modify the disease.
Our lead development program is focusing on phenylketonuria (PKU), a rare genetic disorder caused by mutations in the enzyme phenylalanine hydroxylase that result in defective degradation and accumulation of the amino acid phenylalanine, which becomes toxic to the brain.
We intend to leverage our unique drug discovery platform to target multiple rare disease caused by protein misfolding.
Developing small molecule pharmacological chaperones
– a novel therapeutic approach to protein
Mutations in the DNA sequence of a gene may impact the structure and function of the encoded protein, resulting in genetic disorders. To date more than 6.000 genetic disorders have been identified, affecting an estimated 350 million individuals. Increasing knowledge on the pathogenic mechanisms has revealed that a large number of genetic disorders are associated with destabilization and misfolding of the coded proteins.
Pervasive unstable or misfolded proteins are associated with a variety of diseases. The harmful effect of the misfolded protein may be due to: (a) ‘loss-of-function’, due to effective degradation of the mutant proteins by the quality control system, as observed in many genetic disorders, such as PKU, porphyria, and cystic fibrosis, or (b) ‘gain-of-function’, as seen in many neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease and Huntington’s disease, caused by formation of toxic amyloid aggregates.
Developing effective therapies for rare genetic- or degenerative diseases has proven to be challenging. One particularly promising strategy is the use of pharmacological chaperones that are able to prevent and/or correct misfolding by selectively binding to key sites of the proteins. Recently efforts to develop pharmacological chaperones have been successful in both cystic fibrosis, a life-threatening disease affecting lung function and in Fabry disease, a rare genetic lysosomal storage disorder.
The research group at the University of Bergen (UiB) was the first to envision and prove the potential of a pharmacological chaperone approach for the treatment of PKU. The research of this group on pharmacological chaperones, and on PKU in particular, has been highlighted in a number of top impact journals including Nature Reviews Drug Discovery (7-2008) and Science-Business eXchange (1 2008), among others.
The company intends to develop a proprietary technology platform
and leverage our unique drug discovery platform to target multiple
rare disease caused by protein misfolding.
Our lead project aims at giving PKU patients
a shot at a normal life
Pluvia is currently focusing on developing pharmacological chaperones as potential treatment of PKU, the most common inborn error of metabolism.
PKU is a rare inborn metabolic disorder affecting approximately 1:10,000 newborns. The disease is caused by mutations in the liver enzyme phenylalanine hydroxylase (PAH), resulting in a decreased capacity to convert the amino acid phenylalanine into tyrosine and the build-up of neurotoxic concentrations of phenylalanine. Testing for the disease is included in the newborn screening program throughout the Western world
If left untreated, classical PKU symptoms are severe mental, psychomotor and growth retardation, seizures, and psychiatric disorders. Within the first week of life patients need to follow restrictive low-protein diet for life. Even then, patients experience severe problems resulting in a low quality of life and large social burden. It has become increasingly clear that the outcome of the diet-treatment is suboptimal leading to neuropsychiatric and cognitive problems, growth retardation, nutritional deficiencies, and bone pathology, in addition to reduced quality of life. The only existing non-diet treatment for the condition, Kuvan®, allows for a slightly lighter diet regime for 20 – 30% of the PKU patients. This lack of optimal therapy means that very few of the patients can live a normal life today. There is clearly an unmet medical demand for this patient group.
Our vision is to develop a small-molecule chaperone to prevent the PAH mutation-associated misfolding and thereby restoring the native enzymatic activity of the patient’s own dysfunctional PAH.
A small, international team with a burning ambition
to make a difference
Torgeir Vaage; CEO
Extensive experience from the financial sector in Norway. For the last ten years been involved in a number of early stage biotechnology companies in the Nordic region in the capacity as CEO and CFO. He holds a MSc from Norges Handelshøyskole (Norway) and a PhD from UC Berkeley.
Prof Aurora Martinez; CTO
PhD in Biochemistry. Professor at Dept. of Biomedicine, University of Bergen. Expertise in biophysics, structural biology, drug design and cellular biology, investigating how structure determines molecular recognition, stability and function in selected biomolecular networks. Expertise in compound screening and early stage drug discovery, with special focus on PKU and development of novel therapies based on pharmacological chaperones.
Dr. Mikael Thomson; Drug Development Consultant
PhD in pharmacology and toxicology), Mikael Thomson has worked in the pharmaceutical area for close to 20 years, and comes with a broad background from drug development, extending from preclinical development through phase 2 clinical work, having worked with both large pharma and biotech start-ups in the Nordic region. He is a member of several pharmaceutical societies and has authored or co-authored more than 50 publications/abstracts related to early drug development as well co-author of several patents.
Dr. Anne-Sophie Schillinger; Project Director
PhD in structural bioinformatics and biophysics. Experience in early stage drug development from academic and industry projects. Extensive experience in business development and project management at the Bergen Technology Transfer Office.
Dr. Oscar Aubi Catevilla; Senior Scientist
PhD in Biochemistry, Dept. Biomedicine, University of Bergen. Experience in compound screening and early stage drug discovery. Deep knowledge of the methodological aspects, technologies and assays in the discovery process of pharmacological chaperones and inhibitors.
With us in our quest to help vulnerable patient populations
we have the support from a strong base
of Norwegian investors
We are pround to have attracted a strong base of Norwegian investors to support our ambition to develop ground-breaking new therapies for vulnerable patient populations with unmet medical needs:
Bergen Teknologioverføring AS (BTO) is the technology transfer office in Bergen, owned by University of Bergen, Haukeland University Hospital, the Institute of Marine Research, SIVA, Western Norway University of Applied Sciences and NHH. BTO´s mission is to help its owners and partners to bring their research results out to the market, ensuring that knowledge and inventions benefit individuals, the industry and society as a whole.
Bergen Research Foundation
Bergen Research Foundation gives grants toward research and research supporting activities at the University of Bergen and Haukeland University Hospital, and other Norwegian research institutions if they cooperate with the institutions in Bergen. It also gives grants to support research at the University of Bergen and Haukeland University Hospital at the interface between basic research and clinical research. Since 2017 Bergen Research Foundation is also selectively investing in promising early-stage life science companies.
Sarsia Seed AS is a Norwegian Seed Capital Fund which invests in Norwegian early phase technology companies within the energy/cleantech and biotechnology/life science sectors.
Investinor is an evergreen investment company funded by the Norwegian government. Investinor invests in promising unlisted (private) companies aiming for international growth and expansion, and has a long-term perspective on its ownership in it´s portfolio companies.