San Raffaele Telethon Institute for Gene Therapy

What San Raffaele Telethon Institute for Gene Therapy does

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) is a non-profit research institute established as a joint venture between IRCCS Ospedale San Raffaele and Fondazione Telethon, and operating within the IRCCS Ospedale San Raffaele campus in Milan, Italy. SR-TIGET focuses on translational and clinical research in gene therapy for rare genetic diseases — including metabolic disorders, hemophilia, methylmalonic acidemia (MMA) and lysosomal storage diseases — combining laboratory science with in-house early-phase clinical trial capability. The institute employs more than 1,800 researchers (roughly half physician-scientists) across 100+ high-tech laboratories and publishes at high volume, with 2,625 papers in 2023 at a mean impact factor of 8.05.

Its core technology centers on lentiviral vector production and homology-directed repair-based gene editing in hematopoietic stem cells, including a proprietary immune-shielded lentiviral vector platform for in-vivo gene therapy. The institute protects and exploits this technology through a portfolio of 959 granted patents across 139 patent families (521 actively valorized as of December 31, 2024) and through a growing portfolio of spin-off companies including Genenta Science, Genespire, nChromaBio, Biorek, and Repron Therapeutics.

SR-TIGET's business model is not commercial product sales. Instead, it monetizes its research through a combination of public grant funding (PNRR, ERC Consolidator Grants, Ricerca Finalizzata), industry-sponsored research and preclinical collaborations with pharma and biotech (e.g., Intellia, Chroma Medicine, Kite/Gilead, Biogen, AstraZeneca, Merck, Orchard Therapeutics), intellectual property licensing and royalties managed by a dedicated Business Development Division, and equity participation in spin-off companies. Major recent commercial milestones include a multi-year Cytiva (Danaher) collaboration announced in March 2026 — the first Danaher Beacon in the EU — and a €46.6M Series B for spin-off Genespire in September 2024 to advance the first pediatric in-vivo gene therapy (GENE202) for MMA into clinical trials.

Name

San Raffaele Telethon Institute for Gene Therapy

Legal name

San Raffaele Telethon Institute for Gene Therapy

Website

https://research.hsr.it

Company type

Private

Founded year

1971

Operating status

Operating

Headcount range

101–250 employees

Short description

SR-TIGET is a non-profit gene therapy research institute within IRCCS Ospedale San Raffaele in Milan, developing lentiviral vector and gene editing therapies for rare genetic diseases and monetizing outputs via licensing, industry collaborations, public grants, and spin-off companies.

Ownership category

akta.pro rank

Product category

Gene Therapy Research

NAICS

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

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Lentiviral Vector Gene Therapy Developers (HLAAACAB)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN), Gene Therapy Regulatory, Pharmacovigilance & Clinical Operations Services (HLAAACAM)

Keywords

• Gene therapy research
• Lentiviral vectors
• Hematopoietic stem cells
• Genetic disease treatments
• Translational research

Headquarters

HQ city

Milan

HQ country

Italy

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Technology or R&D, Infrastructure, Operations

Revenue model

1. Licensing and IP Royalties: The Business Development Division manages licensing of intellectual property rights and know-how developed by researchers, generating revenue through patent licensing agreements with biotech and pharma companies
2. Industry Research Collaborations: Sponsored research agreements with pharmaceutical and biotech companies for preclinical validation, strategic alliances, and privileged access to patentable results
3. Spin-off Equity: Creation and management of spin-off companies in the life science sector, including Genenta Science, Genespire, nChromaBio, Biorek, and Repron Therapeutics, which generate economic returns through equity participation

Go-to-market motion1 record

Distribution channels3 records

Marketing channels3 records

Core offering

SR-TIGET is a translational research institute that develops gene and cell therapies for rare genetic diseases, with core capabilities in lentiviral vector production and homology-directed repair-based gene editing in hematopoietic stem cells. It conducts preclinical development and early-phase clinical trials, licenses intellectual property to biotech and pharmaceutical partners, and creates spin-off companies to commercialize research outputs. The institute operates within IRCCS Ospedale San Raffaele, enabling bench-to-bedside translation of gene therapy discoveries.

Product overview

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) is a premier research institute within IRCCS Ospedale San Raffaele specializing in gene therapy development. The institute conducts translational and clinical research focused on lentiviral vector production, homology-directed repair-based gene editing in hematopoietic stem cells, and advanced therapies for genetic diseases. SR-TIGET operates as part of a broader institutional ecosystem that includes multiple research divisions (Immunology, Experimental Oncology, Genetics and Cell Biology, Neuroscience), core facilities, and spin-off companies. The institute's research activities span from basic discovery to preclinical development and early-phase clinical trials, with a focus on treating rare genetic diseases through innovative gene therapy approaches.

Differentiator

Problem solved

Functional benefit

Products and services

• Industry Research Collaborations
• Patent Licensing and IP Transfer
• Spin-off Company Creation
• Clinical Trial Services

Quantifiable outcome

• 2,625 scientific papers published in 2023 with mean impact factor of 8.05
• +2 more outcomes

Named customers1 record

Segments2 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Ten partnerships are on record, tiered core and minor.

• Cytiva (Danaher company)coreStrategic or Co-development Partner · 23 March 2026Multi-year collaboration to advance next-generation genomic medicine platforms, marking the first Danaher Beacon in the European Union. Combines SR-TIGET's scientific expertise with Cytiva's manufacturing capabilities, focusing on lentiviral vector production and homology-directed repair-based gene editing in hematopoietic stem cells.
• Fondazione TelethoncoreStrategic or Co-development PartnerLong-standing partnership supporting gene therapy research and development, including co-founding of SR-TIGET. Fondazione Telethon is a key supporter of genetic disease research in Italy.
• RegulusminorStrategic or Co-development PartnerIndustry collaboration for microRNA research and therapeutics development.
• Kite (Gilead)minorStrategic or Co-development PartnerCollaboration in cell therapy and CAR-T research.
• BiogenminorStrategic or Co-development PartnerIndustry collaboration in gene therapy and neurological disease research.
• Intellia TherapeuticsminorStrategic or Co-development PartnerCollaboration in CRISPR gene editing technologies.
• Chroma MedicineminorStrategic or Co-development PartnerCollaboration in epigenetic editing technologies.
• AstraZenecaminorStrategic or Co-development PartnerIndustry collaboration in pharmaceutical research and development.
• Orchard TherapeuticsminorStrategic or Co-development PartnerCollaboration in gene therapy for rare diseases.
• EXTEND - National Technology Transfer Biotech HubcoreStrategic or Co-development PartnerNational technology transfer hub supporting biotech innovation and commercialization of research outputs.

Scale indicators5 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Intellia Therapeutics: Clinical-stage gene editing company developing CRISPR/Cas9-based therapies; SR-TIGET partner with overlapping gene editing programs in hematopoietic stem cells.
• Orchard Therapeutics: Commercial gene therapy company developing lentiviral-based ex vivo therapies for rare genetic diseases including immunodeficiencies and metabolic disorders; directly comparable in indication space and platform approach.
• bluebird bio: Pioneer in lentiviral vector gene therapy for rare genetic diseases (beta-thalassemia, sickle cell disease, cerebral adrenoleukodystrophy); directly comparable in platform and rare disease focus.
• Genespire: SR-TIGET spin-off developing in vivo gene therapies for rare metabolic diseases including GENE202 for MMA; directly comparable as a lentiviral-based gene therapy developer emerging from the same institute.
• Genenta Science: SR-TIGET spin-off developing lentiviral vector-based cell therapies for solid tumors; directly comparable as a gene therapy company originating from the same institute and using its lentiviral platform.

Others

• Fondazione Telethon: Core strategic partner and joint-venture co-founder of SR-TIGET; comparable as the financial and mission backbone enabling SR-TIGET's research programs.
• Telethon Foundation: Italian non-profit funder and co-founder of SR-TIGET; comparable as a sibling non-profit organization funding genetic disease research and enabling gene therapy translation.

Broad incumbents

• CRISPR Therapeutics: Leading commercial gene editing company (Casgevy approval with Vertex); broader incumbent competing across multiple indications where SR-TIGET also operates.
• Spark Therapeutics (Roche): Roche subsidiary developing AAV-based gene therapies (Luxturna); established commercial gene therapy player overlapping in rare disease gene therapy.
• BioMarin Pharmaceutical: Established rare disease biotech with a gene therapy program (Roctavian for hemophilia A); broad incumbent in the same rare genetic disease commercial space.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• Facebookfacebook.com/SR-Tiget-San-Raffaele-Telethon-Institute-For-Gene-Therapy
• LinkedInlinkedin.com/company/sr-tiget

Revenue estimate

Valuation estimate

Number of profiles

Profiles16 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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