Seven companies transforming the biotech industry in Italy 

Famed for its architecture, cuisine, art, and fashion, Italy is a notable player in the pharmaceutical industry, with a steady biotech industry, and a constantly growing number of companies.

More than 90% of the biopharma industry’s revenue originates from the regions of Lombardy, Latium, Tuscany, and Piedmont. As of 2023, the Italian biotech industry is made up of over 800 companies, employing around 13,700 people, and generating more than €13 billion in turnover​. 

In addition to healthcare, there has been a notable expansion in biotechnology applications related to the bioeconomy, including agriculture, environmental sustainability, and industrial processes. These areas have shown significant growth, particularly in the last few years, with some sectors experiencing growth rates exceeding 30%.

Today, we take a closer look at seven biotech companies leading the charge in Italy. 

AAVantgarde Bio

AAVantgarde Bio, based in Milan, Italy, is a biotech company focused on the development of gene therapies, specifically targeting inherited retinal disorders. These conditions, often genetic, can lead to progressive vision loss or complete blindness.

Why the double A in AAVantgarde Bio? Well, simply because the company’s primary scientific approach involves the use of adeno-associated virus (AAV) vectors. These vectors are engineered viruses that deliver functional copies of genes into the patient’s cells. The company is leveraging these AAV vectors to target specific mutations that cause retinal diseases and correct defective genes responsible for the diseases to halt or even reverse the progression of vision loss.

The AAV vectors are particularly suited for retinal therapy due to their ability to efficiently transduce, meaning introduce genetic material, into retinal cells with minimal immune response, making them a promising tool for long-term treatment.

AAVantgarde Bio is currently advancing a pipeline of gene therapies aimed at various inherited retinal diseases. The company’s most advanced candidate, AAVB-081 is focused on type 1 Usher syndrome, often characterized by night blindness followed by a progressive decay of peripheral vision.

Last year, the Italian biotech completed a series A funding round, raising $67.7 million. 

Alia Therapeutics

Founded in 2018 as a spin-off from the University of Trento, the Italian biotech company is dedicated to creating treatments for rare genetic diseases by leveraging CRISPR-Cas systems to correct genetic mutations at their source.

Alia Therapeutics is focused on improving the specificity and safety of CRISPR-based therapies, particularly through the development of highly specific Cas9 variants. These enhanced versions of the CRISPR-Cas system are designed to reduce off-target effects, which are a major concern in gene-editing therapies. 

Its lead candidate is aimed at treating autosomal dominant retinitis pigmentosa (ADRP), a genetic disorder that affects the retina. It is one form of retinitis pigmentosa (RP), a group of inherited disorders that cause progressive vision loss due to the degeneration of photoreceptor cells in the retina.

In 2023, Alia Therapeutics secured $4.9 million in seed financing, led by Sofinnova Partners. This funding will support the development of its CRISPR platform and the advancement of its lead candidate towards clinical trials. 

Contraria Biotech

The concept of reverse vaccinology has been documented since the 1990s. Relying on the principle of cloning and protein expression in an organism’s genome sequence, the technology has been used in the creation of a vaccine against meningococcus – a bacterium that causes meningitis. Now, Contraria, previously known as Achilles Vaccines, is employing this technology to develop vaccines against malaria. 

Located in the “vaccine hub” of Siena in Italy, the biotech company leverages bioinformatics and artificial intelligence in its next-generation reverse vaccinology (NGRV) platform. Additionally, the company is developing vaccines based on modified outer membrane vesicles (mOMV).  Outer membrane vesicles (OMV) contain phospholipids, lipopolysaccharide, outer membrane proteins, and entrapped soluble periplasmic components, and have demonstrated immunogenicity as vaccines in animal and human models.

The company is advancing several vaccine candidates in its pipeline. Among them is a vaccine for malaria, which has received significant attention and funding, including $12.2 million from the EU Malaria Fund. Another prominent candidate is MAbCo19, a monoclonal antibody developed to combat SARS-CoV-2, the virus responsible for COVID-19. This antibody is derived from B cells of recovered COVID-19 patients, selected for their ability to produce neutralizing antibodies.

Genenta Science

Genenta Science, founded in 2014 and based in Milan, Italy, is a clinical-stage biotechnology company specializing in immuno-oncology. The company is pioneering the development of a cell-based platform that leverages hematopoietic stem cells to treat solid tumors. 

Genenta’s approach involves the use of autologous hematopoietic stem and progenitor cells (HSPCs) that are genetically modified ex vivo to express interferon-alpha (IFN-α) under the control of tumor-specific promoters. These modified cells are designed to home in on the tumor microenvironment and deliver the immunomodulatory protein directly within the tumor, thereby avoiding the systemic toxicity typically associated with IFN-α therapy. This targeted delivery is intended to enhance the immune response against the tumor while minimizing adverse effects​.

The company’s lead product, Temferon, is in phase 1/2 clinical trials for treating glioblastoma multiforme (GBM), a highly aggressive brain tumor with limited treatment options. Temferon has shown promising preliminary results, with no dose-limiting toxicities observed in the trial thus far, and evidence of immune cell activation within the tumor microenvironment.

In 2024, Genenta announced that it had dosed the first patients in the final cohort of its trial for GBM, and preliminary data suggested a potential survival benefit in these patients. Furthermore, Genenta expanded its clinical focus to include renal cell cancer.

Kither Biotech

The Turin-based biotech specializes in the development of treatments for chronic inflammatory diseases, particularly respiratory conditions such as asthma and chronic obstructive pulmonary disease (COPD). The company’s approach is to modulate intracellular signaling pathways involved in inflammation, aiming to provide more effective and targeted therapies.

More specifically, Kither’s focus is on modulating the phosphoinositide 3-kinase (PI3K) pathway, a signaling pathway involved in cell growth, proliferation, and survival. Dysregulation of this pathway is linked to various inflammatory diseases, including asthma and COPD. Kither’s approach involves the development of small molecule inhibitors that target specific components of the PI3K pathway, aiming to reduce inflammation and improve respiratory function.

Its lead technology platform centers around the inhibition of PI3K gamma (PI3Kγ), a specific isoform of the PI3K enzyme that plays a key role in the inflammatory response within the lungs. By selectively inhibiting PI3Kγ, Kither Biotech aims to reduce chronic inflammation without compromising the body’s ability to respond to infections.

The Italian biotech’s lead candidate, KIT2014, is designed for the treatment of cystic fibrosis (CF). KIT2014 has shown promise in preclinical studies, where it demonstrated the ability to increase cAMP levels in pulmonary cells. The company is advancing KIT2014 towards clinical trials, with plans to evaluate its safety and efficacy in human patients. 

In 2022, the company completed a $20.5 million series B round.

Resalis Therapeutics

The Italian biotech focuses on the development of therapeutics based on non-coding RNA (ncRNA). The company’s research and development efforts are concentrated on addressing metabolic disorders, such as metabolic associated steatohepatitis (MASH), and various cancers. 

MASH is often asymptomatic in its early stages, making it difficult to diagnose until it has progressed. When symptoms do appear, they might include fatigue, weakness, abdominal discomfort, and signs of liver dysfunction. If left untreated, MASH can progress to liver fibrosis, cirrhosis, and even hepatocellular carcinoma.

Unlike messenger RNA (mRNA), which codes for proteins, ncRNAs play a regulatory role in gene expression. The company targets specific ncRNAs that are involved in disease pathways, with the goal of modulating these pathways to achieve therapeutic effects. This approach is seen as a promising avenue for treating conditions where traditional therapies may be less effective.

The company’s lead candidate, RES-010, targets MASH and obesity. The therapy is designed to modulate ncRNAs involved in lipid metabolism and inflammatory processes, aiming to reduce liver fat and inflammation. This candidate has shown positive results in preclinical studies, and Resalis is preparing to advance it into clinical trials for both indications. To support these next steps, Resalis Therapeutics recently secured $11.1 million in a series A funding round. 

Sibylla Biotech

This biotech company, headquartered in Milan, Italy, develops small molecule therapies targeting protein folding pathways. The company’s approach centers on folding interfering degraders (FIDs), a novel class of therapeutics designed to target misfolded proteins, which are implicated in various diseases, including cancer and neurodegenerative disorders.

Proteins must fold into specific three-dimensional shapes to function correctly. The company’s FIDs are small molecules that selectively bind to and stabilize certain misfolded proteins, leading to their degradation by the cellular quality control system. This process effectively removes the harmful proteins, potentially halting the progression of the disease.

The company’s technology is particularly relevant for diseases where protein misfolding plays a critical role, such as certain cancers and neurodegenerative diseases like Huntington’s and Parkinson’s. 

The Italian biotech’s pipeline includes several FID-based candidates targeting various therapeutic areas but its pipeline is still preclinical. Its lead candidate is a cyclin D1 degrader, cyclin D1 is often overexpressed in various types of cancer, including breast cancer, lymphoma, and melanoma. Overexpression can lead to uncontrolled cell proliferation because the regulation of the cell cycle is disrupted.

Sibylla Biotech is in the process of advancing this candidate towards clinical trials, and the $25.5 million series A it completed in 2022 will help the company along the way.

Italy, a bit behind its European counterparts

While the Italian biotech sector is experiencing steady growth and the companies we mentioned earlier are definitely worth keeping an eye on in the years to come, it seems Italy is lagging behind other European countries. 

Compared to other European biotech powerhouses like Germany, France, and the UK, Italy’s biotech sector struggles with a less developed funding landscape, particularly in late-stage financing. Italian biotech companies often face challenges in attracting the large-scale investment needed to scale operations and compete on a global stage​. 

Italy’s biotech ecosystem is marked by a strong focus on healthcare and a growing bioeconomy which could drive a more diversified landscape. However, to fully capitalize on its strengths, Italy will need to address funding challenges, scale up its operations, and enhance its global visibility. With room for improvement, Italy has the potential to strengthen its position in the European biotech landscape​.