Why It Matters
The cluster demonstrates Barcelona’s capacity to attract capital and accelerate breakthrough therapies, positioning the region as a competitive alternative to traditional biotech centers in Europe and North America.
Key Takeaways
- •Accure's ACT-01 reaches Phase 2 safety success
- •Oryzon's iadademstat advances in AML trials
- •SpliceBio raises $135M Series B for gene therapy
- •Peptomyc's OMO-103 first pan-Myc inhibitor in Phase 2
- •Ona Therapeutics secures over $47M for ADC pipeline
Pulse Analysis
Barcelona’s biotech renaissance is anchored by a dense network of research institutes, university hospitals, and the Barcelona Science Park, the country’s first dedicated science hub. The city accounts for 7.6 % of Catalonia’s GDP and has attracted a €1.7 billion health‑campus investment, aiming to generate up to 50,000 jobs. This ecosystem, coupled with strong public‑private funding, creates a fertile ground for innovative drug development and draws multinational interest to Southern Europe.
The six highlighted companies illustrate the breadth of therapeutic approaches emerging from the region. Accure Therapeutics is moving its neuroprotective small‑molecule ACT‑01 through Phase 2, while Oryzon Genomics pushes LSD1 inhibitors like iadademstat into AML and lung‑cancer trials. SpliceBio’s protein‑splicing platform secured a $135 million Series B to advance SB‑007 for Stargardt disease, and Peptomyc’s mini‑protein OMO‑103 became the first pan‑Myc inhibitor to enter Phase 2 for osteosarcoma. Ona Therapeutics bolsters its ADC pipeline with over $47 million in funding, and Integra Therapeutics leverages CRISPR‑Cas and PiggyBac technology, backed by €10.7 million and a €40 million licensing deal, to expand gene‑writing therapies.
For investors and stakeholders, Barcelona’s rising biotech profile signals a shift in the global innovation landscape. The convergence of cutting‑edge science, sizable venture capital, and supportive governmental grants reduces the time to clinic for high‑risk modalities. As these companies progress through pivotal trials, the region is poised to deliver novel treatments for neuro‑degeneration, cancer, and rare genetic disorders, reinforcing Europe’s competitive edge in next‑generation therapeutics.
Six biotechs to know in Barcelona
Known for its architecture, coasts and vibrant culture, Barcelona is a premier European life sciences hub, ranking sixth in scientific output, according to the Barcelona City Council, and contributes 7.6 % to Catalonia’s gross domestic product.
In this article, we take a look at six biotech companies in Barcelona developing various kinds of therapies that you should know about.
Accure Therapeutics
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Founded: 2020
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Technology: small molecules
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Lead candidate: ACT‑01 for optic neuritis and glaucoma
Barcelona‑based Accure Therapeutics has three major candidates in its pipeline. The clinical candidate ACT‑01 is a small molecule designed to activate trophic factor pathways such as IGF‑1 and BDNF to promote cell survival and endogenous repair of damaged neurons. It is linked to neuroprotective activity and is tuned to treat neurodegenerative disorders, specifically optic neuritis (inflammation of the optic nerve that causes blurred vision) and multiple sclerosis, a chronic autoimmune disease of the central nervous system.
Licensed to Oculis in 2022, ACT‑01 had positive preclinical safety results and is currently in Phase 2 trials, where it met the primary safety endpoint and key secondary efficacy endpoints with no serious adverse events measured.
Next in the lineup is ACT‑02, which is in preclinical studies for Parkinson’s disease. It is an oral, brain‑penetrant inhibitor of the enzyme prolyl oligopeptidase. These enzymes are involved in the catabolism of neurotransmitters and the modulation of certain protein‑protein interactions that can affect cognition and neurodegeneration. It has been found to improve cognition in preclinical models of brain disorders.
Accure also has the small‑molecule ACT‑03, which is in the discovery phase to potentially treat epilepsy.
Accure plans to file an investigational new drug (IND) application for ACT‑02 to possibly reach the clinic this year. For this, it was awarded $1.6 million in grants from the Michael J. Fox Foundation and the Fonds National de Recherche de Luxembourg last year. The financing followed a €1 million ($1.19 million) grant from the Spanish government to evaluate ACT‑03 for epilepsy.
Oryzon Genomics
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Founded: 2000
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Technology: epigenetics
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Lead candidate: iadademstat for cancer
Epigenetics is the study of how behaviors and environmental factors cause changes to DNA that turn genes on and off. Regulating gene transcription and translation could help address a host of diseases. Barcelona‑based Oryzon Genomics focuses on modulating the activity of certain enzymes linked to disease development, such as cancer, CNS, inflammatory, and metabolic disorders.
The enzymes they target are histone‑modifying enzymes. Histone modifications are chemical changes on the ends of amino acids that silence or activate genes in the region.
Oryzon has two drugs in the clinic:
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iadademstat – a selective LSD1 inhibitor for cancer. A first‑in‑human Phase 1/2a trial in patients with refractory and relapsed acute leukemia demonstrated safety. Iadademstat is currently in Phase 1b and Phase 2 trials for blood cancers, including a Phase 1b study combined with gilteritinib for AML and another combined with azacitidine and venetoclax for first‑line AML. The first patient in a Phase 1b study for small‑cell lung cancer was dosed last week; the study is sponsored and conducted by Yale University.
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vafidemstat – an LSD1 inhibitor for CNS and psychiatric disorders. It has been found to reduce cognitive impairment, decrease neuroinflammation, and exert neuroprotective effects. In animal models, vafidemstat improves memory, reduces aggressive behavior, curbs social avoidance, and enhances sociability. Phase 2 trials are ongoing.
SpliceBio
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Founded: 2020
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Technology: protein engineering
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Lead candidate: SB007 for Stargardt disease
A spin‑out of the Muir Lab at Princeton University, SpliceBio has created a Protein Splicing platform that underpins its pipeline. The most advanced candidate is SB007 for Stargardt disease, a rare inherited eye disease caused by mutations in the ABCA4 gene. Because the ABCA4 gene is too large for a single adeno‑associated virus (AAV) vector, SB‑007 splits the gene into two parts, delivering them via two separate AAV8 vectors.
Once inside target eye cells, SB‑007 uses engineered split‑inteins to join the two protein halves into a functional ABCA4 protein. By restoring full‑length ABCA4 expression, the therapy aims to stop or reverse the accumulation of toxic vitamin‑A by‑products, preventing further vision loss.
The Barcelona‑based biotech launched the dose‑expansion portion of Phase 1/2 studies for SB‑007 last month after the FDA granted fast‑track and Orphan Drug designations. A $135 million Series B financing round was secured to advance SB‑007 and expand the pipeline of genetic medicines.
Peptomyc
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Founded: 2014
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Technology: mini‑proteins
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Lead candidate: OMO‑103 for bone cancer
Spun out of the Vall d’Hebron Institute of Oncology (VHIO) in Barcelona, Peptomyc employs mini‑proteins to directly inhibit the MYC gene, whose dysregulation drives 50 %–70 % of cancers. Its candidate OMO‑103 is a MYC inhibitor based on Omomyc, designed by co‑founder and CSO Laura Soucek.
The first patient in a Phase 2 trial for children and adults with advanced osteosarcoma (the most common primary bone cancer) was successfully treated with OMO‑103, making it the first direct pan‑Myc inhibitor to complete a Phase 1 trial.
The company previously received more than €5 million ($5.92 million) from the Horizon Europe EIC Accelerator program to support clinical development and manufacturing scale‑up.
Ona Therapeutics
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Founded: 2019
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Technology: antibody‑drug conjugates (ADCs)
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Lead candidate: ONA‑255 for breast cancers and solid tumors
ADCs combine a monoclonal antibody with a cytotoxic chemotherapy payload via a chemical linker, acting as “biological missiles.” Located in the Barcelona Science Park, Ona Therapeutics has two ADCs in IND‑enabling stages:
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ONA‑255 – in the clinic for breast cancer and solid tumors. It binds a tumor antigen that is up‑regulated in resistant HR‑positive/HER2‑negative breast cancer, especially after CDK4/6 inhibitor failure.
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ONA‑389 – an investigational ADC targeting microsatellite‑stable colorectal cancer and hepatocellular carcinoma.
Ona Therapeutics raised €10 million ($11.84 million) from SICC Innvierte of CDTI Innovation to boost ADC development, following a €30 million ($35.54 million) Series A round in 2020. Total funding now exceeds $47 million.
Integra Therapeutics
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Founded: 2020
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Technology: genomics
Integra Therapeutics, a spin‑off of Pompeu Fabra University, creates gene‑writing tools to improve the scope, efficiency, and safety of therapies for high‑unmet‑need diseases such as cancer and autoimmune conditions. Its platform merges CRISPR‑Cas–modified proteins with PiggyBac transposase to program precise insertions of DNA fragments into the genome. Engineered PiggyBac domains increase on‑target integration while reducing off‑target events.
The company’s Find and Cut‑And‑Transfer (FiCAT) technology can be used for both in‑vivo and ex‑vivo therapies targeting cancer and autoimmunity. Integra secured €10.7 million ($12.67 million) to advance the gene‑writing platform and preclinical validation of CAR‑T therapies, following an earlier €10.5 million ($12.44 million) grant from the European Commission.
Integra also signed a licensing deal with Lithuanian CRISPR pioneer Caszyme to combine FiCAT with Caszyme’s Cas12l editor, a partnership valued at up to €40 million ($47.37 million).
Barcelona: a rising star in biotech?
Barcelona boasts thriving research institutes, university hospitals, and a diverse science and investment ecosystem that make it an attractive hub for research and innovation. Cancer therapeutics are a particular focus, exemplified by companies such as Ona Therapeutics, Peptomyc, and Nuage Therapeutics.
The Barcelona Science Park—the first science park in Spain—hosts three research institutes and more than 70 companies, reinforcing the city’s role in biopharma research. Catalonia is the densest pharma environment in Europe, with nearly 1,400 life‑sciences companies. It is also the birthplace of the five largest Spanish pharmaceutical firms. A €1.7 billion ($2.01 billion) investment underpins the new Clínic–University of Barcelona Health Campus to accelerate R&D, and the upcoming BioCluster of Innovation and Health could generate up to 50,000 new jobs over the next two decades.
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