Our goal is to develop innovative therapies to help patients
suffering from rare genetic disorders
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.
News
August 2026
Pluvia welcomes our new CEO, Deborah Ramsdell, to our team.
Deborah Ramsdell brings more than three decades of biotechnology leadership, regulatory and clinical development experience, with a strong focus on rar diseases.
“Deborah’s appointment comes at an important point in Pluvia’s evolution. Her extensive rare disease experience, her network in the US, and practical development expertise make her ideally suited to lead Pluvia through its next stage of development.” – Sveinung Hole, Chairman of the Board of Pluvia Biotech
“I am delighted to join the company as we prepare to move PBAS499 into the clinic and look forward to working with the team, Board, investors and PKU community to bring a meaningful new treatment option to people living with PKU.” – Deborah Ramsdell, CEO of Pluvia Biotech
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May 2026
Pluvia Biotech welcomes new investors supporting final steps
for PKU therapy candidate towards clinical development
Pluvia Biotech today announced that Mid Atlantic Bio Angels (BioAngels) and Tidligfasekapital Vestland are investing in Pluvia’s drug development program. These new investors from the United States and Norway are joining a seed-extension round by Pluvia’s existing investors, Sarsia and Investinor, to support final preclinical development steps towards First-in-Human studies for Pluvia’s therapeutic candidate PBAS499. This follows the recently announced investment from the US PKU patient advocacy organization, the NPKUA.
“After significant due diligence by our membership base of experienced life science and pharmaceutical professionals, we are pleased to support Pluvia as it advances toward clinical development with the goal of bringing a meaningful new treatment option to PKU patients”, says Yaniv Sneor, Founder of Mid Atlantic Bio Angels.
Espen Hundsnes Grøvlen, Fund Manager of Tidligfasekapital Vestland adds “Pluvia is an excellent example of how world-class research originating from Western Norway can be translated into innovative companies with global potential. We are pleased to support Pluvia in this important phase of the company”.
February 2026
National PKU Alliance invests in Pluvia Biotech to support development of potential novel PKU treatment
National PKU Alliance (NPKUA), the US patient advocacy organization for PKU, is investing in Pluvia’s drug development program.
The support from NPKUA will strengthen Pluvia’s scientific basis and contributes to the completion of preclinical activities to progress PBAS499 towards an Investigational New Drug (IND) application for clinical studies in PKU patients.
“After careful evaluation by our Scientific Advisory Board, NPKUA will support development of PBAS499 as a potential oral therapy option for PKU,” says Catherine Warren, Executive Director of NPKUA “Financial support of this program aligns with our mission to improve the lives of people with PKU by expanding treatment options to ultimately allow for more natural protein intake.”
November 2025
Pluvia Biotech receives grant from the Research Council of Norway to advance PKU therapy candidate.
Pluvia has been awarded an Innovation Project for the Industrial Sector (IPN) grant of NOK 15.6 million from the Research Council of Norway. The funding will support the last preclinical development steps for PBAS499, Pluvia`s lead therapeutic candidate, and enable the company to progress the program to the IND-ready stage.
“PBAS499 has demonstrated efficacy and safety in in vitro and in vivo models for PKU,” says Dr. Ann Kari Grindheim, Director Non-clinical Development of Pluvia Biotech. “This grant allows us to progress our PKU R&D program and
move efficiently through the final stages of IND-enabling development.”
February 2025
Pluvia Biotech Announces Publication of Key Patent for PKU lead product PBAS499
Pluvia today announced the publication by the World Intellectual Property Organization (WIPO) of a key Composition of Matter patent application for its lead compound PBAS499.
The invention relates to compounds for use in the treatment of hyperphenylalaninemia (HPA), in particular phenylketonuria (PKU), acting as pharmacological chaperones of the enzyme phenylalanine hydroxylase.
Willem van Weperen, Pluvia’s CEO says: “The Pluvia team is proud to execute on another milestone to protect our innovative therapy candidate PBAS499, after having secured Orphan Drug Designations in the US and Europe last year. We are working hard to move this innovation to the clinic to ultimately make a difference for PKU patients.”
September 2024
Pluvia Biotech Presents Preclinical Data of potential PKU treatment PBAS499 at SSIEM Annual Symposium
This is the first time that PBAS499 data will be shared with the scientific community during the SSIEM conference, where rare genetic disease expert clinicians and scientists from all over the world will come together.
Ann Kari Grindheim, PhD, Director of Preclinical Development and presenter of the data, says “We are proud to share recent data that show the ability of our lead compound PBAS499 to stabilize a majority of the most frequent missense variants of the PAH enzyme. Furthermore, PBAS499 shows convincing efficacy in a PKU mouse model, as well as a clean safety profile, both in vitro and in vivo.”
May 2024
Pluvia Biotech Receives European Orphan Drug Designation
The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designaton (ODD) for Pluvia`s lead compound PBAS499.
“The direct burden of the disease, as well as the burden of the diet, can have significant medical, psychological, and social impacts on these patients. Pluvia’s approach to treat the underlying cause of PKU by stabilizing the deficient PAH enzyme is a promising way to address this.” says Prof. Francjan van Spronsen, MD, PhD, Prof. of Pediatric Metabolic Disease, University Medical Center Groningen, The Netherlands.
April 2024
Pluvia Announces Research Collaboration with PTC Therapeutics.
A research collaboration has been established between Pluvia and PTC Therapeutics.
“We are very proud to be preparing global regulatory submissions for sepiapterin, our promising and differentiated therapy being developed for children and adults with PKU,” said Matthew B. Klein, M.D., PTC Chief Executive Officer. “A collaboration with the expert team at Pluvia allows us to continue to pursue our interest in building on our inborn errors of metabolism platform.”
Jan. 2024
Pluvia Receives Orphan Drug and Rare Pediatric Disease Designations from FDA.
The U.S. Food and Drug Administration has granted both the Orphan Drug Designation and the Rare Pediatric Disease Designation for Pluvia’s lead compound PBAS499 for the treatment of Phenylketonuria.
“We are excited about these valuable designations by the FDA to support Pluvia’s mission to develop a new oral therapy option for PKU patients. We look forward to continue our momentum to realize a Series A round early 2024 and to move PBAS499 to clinical Proof of Concept.” – Willem van Weperen, Pluvia’s CEO
Oct. 2023
We welcome our new CEO, Willem van Weperen, to the Pluvia team.
“We are delighted to welcome Willem as our new CEO. His relevant expertise and network align perfectly with Pluvia’s stage of development. Under his guidance, we believe Pluvia Biotech will make significant strides in developing a new therapeutic option for patients with PKU.” – Sveinung Hole, Chairman of the Board at Pluvia Biotech
OUR SCIENCE
Developing small molecule pharmacological chaperones
– a novel therapeutic approach to protein
misfolding diseases
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 PROJECTS
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.
OUR TEAM
A small, international team with a burning ambition
to make a difference
Deborah Ramsdell; CEO
Deborah Ramsdell has extensive experience in bringing treatments for rare diseases into patients, and partnering with companies to bring them to market. For the last 14 years she has been involved with several early stage biotechnology companies in the capacity of CEO and/or COO. Based in the US, she brings an extensive network across US rare disease and biotechnology communities.
Torgeir Vaage; CFO
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. Ann Kari Grindheim; Director of non-clinical development
Ann Kari Grindheim is a pharmacist by training and has extensive experience from cell biology and biotechnology research and development from the University of Bergen. Ann Kari is in charge of all cell culture work in Pluvia as well as managing the preclinical development.
Dr. Karina Skjervheim Prestegård; Senior Scientist
Karina S. Prestegård has a Master of Science in Biology and PhD in Biomedicine from the University of Bergen. She has several years of experience from working with transgenic mouse models, and is in charge of planning and executing the in vivo experiments.
Altanchimeg Altankhuyag; Researcher
Altanchimeg Altankhuyag has a Master of Science in Molecular Biology (University of Bergen). She is doing the day-to-day maintenance of our mouse colonies, with genotyping as her main task. She is also involved in the planning and execution of the in vivo experiments.
Trond-André Kråkenes; Research Technician
Trond-André Kråkenes has a Master of Science in Nanoscience from the University of Bergen. He is in charge of analysing phenylalanine levels in preclinical samples. He also performs enzyme activity assays and other in vitro research activities.
Sebastián González Rodríguez, Research Technician
Sebastián González Rodríguez has a Master of Scinence in Biomedicine from the University of Bergen. He is responsible for protein purification and small-molecule drug screenings targeting protein–protein interactions. He also supports the processing of blood samples from mouse experiments.
OUR INVESTORS
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 investors to support our ambition to develop ground-breaking new therapies for vulnerable patient populations with unmet medical needs:
Mid Atlantic Bio Angels
The Mid Atlantic Bio Angels Group is a NY-based life science angel investor group, formed in 2012, that invests exclusively in life science companies, with no geographic limitations (www.bioangels.net).
Tidligfasekapital Vestland
Tidligfasekapital Vestland is an early-stage venture fund manager based in Bergen, Norway, currently deploying capital through its first fund, Tidligfasefond Vestland 1. The fund invests in early-stage companies across a variety of industries with ties to Western Norway. (www.tidligfasekapitalvestland.no).
National PKU Alliance
National PKU Alliance (NPKUA) is the only national organization based in the United States dedicated solely to supporting individuals and families affected by phenylketonuria (PKU). NPKUA’s mission is to improve the lives of individuals with PKU, pursue a cure by expanding research and provide education and support to individuals living with PKU and their caregivers. NPKUA has selected Pluvia as their first investment in a PKU therapy. To learn more about NPKUA, please visit www.npkua.org.
Research Council of Norway
Pluvia is supported by the Research Council of Norway through the Innovation Project for the Industrial Sector (IPN) programme.
Bergen Teknologioverføring
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
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
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.
