Tigem

What Tigem does

Tigem (Telethon Institute of Genetics and Medicine, legally Istituto Telethon Di Genetica E Medicina) is a non-profit multidisciplinary research institute headquartered in Pozzuoli (Naples), Italy, founded in 1994 and owned by Fondazione Telethon. The institute is dedicated to understanding the mechanisms underlying rare genetic diseases and to developing innovative therapies, with core research programs in AAV-mediated gene therapy (including approaches for large genes and N-of-1 personalized therapies), drug repurposing via systems biology, bioinformatics, and label-free 3D imaging for lysosomal storage diseases. Its technical surface includes proprietary bioinformatics tools such as TRIMBASE (multi-species TRIM gene family database), Gene2Drug for drug repositioning, exome and NGS analysis pipelines, and structured diagnostics through the Telethon Undiagnosed Disease Program. TIGEM also operates three PhD training programs (Open University, SEMM, Scuola Superiore Meridionale) and the new RAREFIND MSCA-COFUND programme, alongside numerous clinical trials and collaborative projects with universities and EU consortia.

The institute does not generate revenue from commercial products or services; its operations are funded primarily by Fondazione Telethon, the European Community, and competitive grants including ERC awards (most recently a €2.5M ERC Grant to Diego Di Bernardo in June 2025 and an ERC Proof of Concept Grant to Carmine Settembre for the LEAD-ER project). Its customer base is the rare disease research and clinical community — scientific researchers, clinicians, and patients with limited therapeutic options — served indirectly through publications, clinical trials, conferences, and educational programs rather than direct commercial relationships. Notable achievements include the world's first gene therapy intervention for a rare retinal disease performed in Naples, and recognition of senior faculty such as Prof. Andrea Ballabio (Beth Levine Prize in Autophagy Research, 2025) and Prof. Alberto Auricchio (Italian Knowledge Leader 2025).

Name

Tigem

Legal name

Istituto Telethon Di Genetica E Medicina (TIGEM)

Website

https://tigem.it

Company type

Private

Founded year

1994

Operating status

Operating

Headcount range

251–500 employees

Short description

Tigem is a non-profit Italian research institute in Pozzuoli (Naples), owned by Fondazione Telethon, that investigates mechanisms of rare genetic diseases and develops gene therapies, bioinformatics tools, and clinical trials. It is funded by Telethon, the European Community, and ERC grants, and serves the rare disease scientific and clinical community.

Ownership category

akta.pro rank

Product category

Genetic Disease Research

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731), Services-Engineering, Accounting, Research, Management (8700)

akta.pro industry

PRIMARYHLAAACAE

Gene Therapy for Rare/Monogenic Diseases

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAIAIAA

Genetic & Genomic Rare Disease Therapeutics

Healthcare & Life Sciences/Pharmaceuticals/Orphan & Rare Disease Pharmaceuticals

SECONDARYHLAAACAM

Gene Therapy Regulatory, Pharmacovigilance & Clinical Operations Services

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

Keywords

• Rare disease research
• Gene therapy development
• Bioinformatics databases
• Clinical trials research
• PhD scientific training

Headquarters

HQ city

Pozzuoli

HQ country

Italy

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations, Infrastructure

Revenue model

1. Foundation and Public Funding: TIGEM operates as a non-profit research institute primarily funded through support from Fondazione Telethon, the European Community, and various other funding agencies. Revenue is not generated from commercial products or services.
2. Research Grants and Awards: The institute receives competitive research grants including ERC (European Research Council) grants such as the €2.5M ERC Grant awarded to Diego Di Bernardo for systems biology and gene therapy research.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

TIGEM is a multidisciplinary non-profit research institute that studies the mechanisms underlying rare genetic diseases and develops innovative therapies, with a core focus on AAV-mediated gene therapy, drug repurposing, and bioinformatics tools for genomic analysis. It also runs clinical trials, hosts specialized PhD training programs, and maintains bioinformatics databases (such as TRIMBASE) and NGS analysis platforms for the research community.

Product overview

TIGEM (Telethon Institute of Genetics and Medicine) is a multidisciplinary research institute focused on rare genetic diseases and innovative therapy development. The organization operates as a research institution rather than a commercial product company, offering gene therapy research programs, PhD training, and clinical trials. Its technical infrastructure includes bioinformatics tools (TRIMBASE database, gene analysis platforms, NGS analysis tools), which are made available as research resources. The institute's core activities center on clinical trials for rare diseases, PhD training programs in genomic medicine, and development of gene therapy approaches for conditions such as Pompe disease, Fabry disease, Usher Syndrome, and lysosomal storage diseases.

Differentiator

Problem solved

Functional benefit

Products and services

• Gene Therapy Research Programs Multidisciplinary research programs focused on understanding mechanisms underlying rare genetic diseases and developing innovative therapies, including AAV gene therapy approaches. Targeted at academic and clinical collaborators in rare genetic disease research.
• Clinical Trials Clinical trials run by TIGEM's research groups in collaboration with clinical centers for gene therapy treatments of rare genetic diseases. Targeted at patient populations with rare genetic conditions and clinical collaborators.
• PhD Training Programs (GEM, SEMM, SSM) Three structured PhD training programs for students pursuing careers in medical science focused on rare genetic disorders, delivered in partnership with Open University, SEMM, and Scuola Superiore Meridionale. Targeted at prospective doctoral students in life sciences.
• TRIMBASE Genomic analysis database of the TRIM gene family revealing evolutionary properties across multiple species. Targeted at genetic researchers and bioinformaticians.
• Bioinformatics Tools Suite Suite of bioinformatics web tools including gene2drug (drug repositioning), genes-and-drugs networks, exome pipeline, and genome browser tools for genetic research. Targeted at researchers conducting rare disease and genomic studies.
• Next Generation Sequencing Analysis Platform NGS analysis platform hosted at ngs.analysis.tigem.it for processing and analyzing next-generation sequencing data. Targeted at genomic researchers and clinical genomics users.

Quantifiable outcome

• World's first gene therapy intervention for a rare retinal disease performed in Naples

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Integration3 records

AI capability5 records

Feature4 records

Partnerships

Five partnerships are on record, tiered core, major and minor.

• Various UniversitiescoreStrategic or Co-development PartnerTIGEM maintains alliances with various universities that offer additional resources and provide opportunities for excellent students. Specific partnerships mentioned include Scuola Superiore Meridionale (SSM) for PhD programs.
• Open University (OU)majorStrategic or Co-development PartnerPartner institution for one of TIGEM's three PhD programmes, contributing to the training of next-generation scientists.
• SEMM (European School of Molecular Medicine)majorStrategic or Co-development PartnerPartner institution for PhD training, offering programmes in medical science for students pursuing careers in rare genetic disorder research.
• Scuola Superiore Meridionale (SSM)majorStrategic or Co-development PartnerPartner institution for PhD training, collaborating on the PhD Program in Genomic and Experimental Medicine (GEM) and other joint initiatives.
• ABE ItalyminorStrategic or Co-development PartnerCollaboration on the 'Let's Be a Scientist' program promoting science education in collaboration with SSM and TIGEM.

Scale indicators4 records

Recent moves6 records

Expansion highlights6 records

Broad incumbents

• Institut Pasteur: Large French non-profit research foundation with global operations covering genetics, rare disease research, and translational therapeutics. Broader scope than TIGEM but comparable as a publicly funded European research organization generating scientific publications and training the next generation of biomedical scientists.
• Wellcome Sanger Institute: UK-based, Wellcome Trust-funded genomic research institute. Highly comparable as a publicly funded, non-profit genomics and rare disease research organization with similar publication and training outputs, though operating at significantly larger scale.
• Broad Institute of MIT and Harvard: Large US biomedical and genomic research organization with deep capabilities in genome sequencing, rare disease research, and gene therapy discovery. Comparable as a mission-driven, non-profit genomic research organization; broader scale and richer funding base than TIGEM.
• St. Jude Children's Research Hospital: US non-profit pediatric research hospital with extensive gene therapy programs for rare childhood genetic diseases (e.g., SCID-X1, sickle cell). Comparable focus on pediatric rare genetic disease treatment via gene therapy and translational clinical research in a non-commercial setting.

Direct peers

• European Molecular Biology Laboratory (EMBL): Intergovernmental research institute with multiple sites across Europe conducting fundamental molecular biology and genetics research, including disease-gene research. Highly comparable as a publicly funded, non-commercial basic research organization operating across European sites.
• Whitehead Institute for Biomedical Research: MIT-affiliated non-profit research institute focused on fundamental biomedical research including genetics, gene regulation, and disease mechanisms. Comparable as a non-commercial basic science institute with similar PI-driven structure and translational research into rare diseases.
• The Jackson Laboratory: US-based non-profit biomedical research institute focused on mammalian genetics, genomic research, and rare disease model development. Comparable because it operates in a similar niche (model organism genetics, rare disease research), is non-commercial, and combines research with training programs.
• San Raffaele Telethon Institute for Gene Therapy (SR-Tiget): Direct Italian Telethon-affiliated sister institute in Milan that develops gene and cell therapies for rare genetic diseases. It is the most comparable peer because it shares the same parent foundation (Fondazione Telethon) and overlapping gene therapy mission, including AAV and HSC-based therapies; the two are complementary within Telethon's research network.

Regional players

• Istituto Italiano di Tecnologia (IIT): Italian research foundation operating across robotics, computational sciences, and biomedical sciences including genomics. Comparable as an Italian publicly funded research institute with international collaborations and similar institutional/governance structure.
• IGBE - Institute of Genetic and Biomedical Research (CNR): Italian National Research Council (CNR) institute with primary genomics and population genetics research programs. Most relevant Italian regional comparable; similar institutional setting, talent pipeline, and basic-to-translational genetics research focus.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/tigem

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Tigem