San Raffaele Telethon Institute for Gene Therapy
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.
- Company typePrivate
- Founded1971
- HeadquartersMilan, Italy
- Headcount51–100
- GTM typeB2B
- OfferingServices
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.
San Raffaele Telethon Institute for Gene Therapy firmographics
Firmographics- 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
- 51–100 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
San Raffaele Telethon Institute for Gene Therapy industry classification
Industry- 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
Where San Raffaele Telethon Institute for Gene Therapy is headquartered
LocationHeadquarters
- HQ city
- Milan
- HQ country
- Italy
- HQ region
- Europe
Offices1 record
Markets served
San Raffaele Telethon Institute for Gene Therapy business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Infrastructure, Operations
Revenue model
- 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
- Industry Research Collaborations: Sponsored research agreements with pharmaceutical and biotech companies for preclinical validation, strategic alliances, and privileged access to patentable results
- 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
San Raffaele Telethon Institute for Gene Therapy product offering
Product offeringCore 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
Companies that use San Raffaele Telethon Institute for Gene Therapy
Customer profileNamed customers1 record
Segments2 records
Ideal customer profiles3 records
San Raffaele Telethon Institute for Gene Therapy technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
San Raffaele Telethon Institute for Gene Therapy partnerships and signals
Strategic signalPartnerships
Ten partnerships are on record, tiered core and minor.
- Cytiva (Danaher company)coreMulti-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 TelethoncoreLong-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.
- RegulusminorIndustry collaboration for microRNA research and therapeutics development.
- Kite (Gilead)minorCollaboration in cell therapy and CAR-T research.
- BiogenminorIndustry collaboration in gene therapy and neurological disease research.
- Intellia TherapeuticsminorCollaboration in CRISPR gene editing technologies.
- Chroma MedicineminorCollaboration in epigenetic editing technologies.
- AstraZenecaminorIndustry collaboration in pharmaceutical research and development.
- Orchard TherapeuticsminorCollaboration in gene therapy for rare diseases.
- EXTEND - National Technology Transfer Biotech HubcoreNational technology transfer hub supporting biotech innovation and commercialization of research outputs.
Scale indicators5 records
Recent moves6 records
Expansion highlights6 records
San Raffaele Telethon Institute for Gene Therapy competitors and assessment
Company assessmentDirect 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
San Raffaele Telethon Institute for Gene Therapy social profiles
Digital presenceSan Raffaele Telethon Institute for Gene Therapy financial estimates
Financial estimateRevenue estimate
Valuation estimate
San Raffaele Telethon Institute for Gene Therapy leadership team
Management profileNumber of profiles
Profiles16 records
San Raffaele Telethon Institute for Gene Therapy funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
San Raffaele Telethon Institute for Gene Therapy M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about San Raffaele Telethon Institute for Gene Therapy
What does San Raffaele Telethon Institute for Gene Therapy do?
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.
Is San Raffaele Telethon Institute for Gene Therapy a public or private company?
San Raffaele Telethon Institute for Gene Therapy is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was San Raffaele Telethon Institute for Gene Therapy founded?
San Raffaele Telethon Institute for Gene Therapy was founded in 1971. It employs 51 to 100 people.
Where is San Raffaele Telethon Institute for Gene Therapy based?
San Raffaele Telethon Institute for Gene Therapy is headquartered in Milan, Italy, in the Europe region.
How does San Raffaele Telethon Institute for Gene Therapy make money?
Three revenue lines are on record. Licensing and IP Royalties are the primary driver. The others are industry Research Collaborations and spin-off Equity.
Who are San Raffaele Telethon Institute for Gene Therapy's main competitors?
Direct peers on record are Intellia Therapeutics, Orchard Therapeutics, bluebird bio, Genespire and Genenta Science. Others are Fondazione Telethon and Telethon Foundation. Broad incumbents are CRISPR Therapeutics, Spark Therapeutics (Roche) and BioMarin Pharmaceutical.
Does San Raffaele Telethon Institute for Gene Therapy have an API?
No public API is recorded for San Raffaele Telethon Institute for Gene Therapy.
What industry is San Raffaele Telethon Institute for Gene Therapy in?
San Raffaele Telethon Institute for Gene Therapy's product category is Gene Therapy Research. Its primary akta.pro industry code is HLAAACAB, Lentiviral Vector Gene Therapy Developers, with a secondary code of HLAAACAE, Gene Therapy for Rare/Monogenic Diseases. Its NAICS code is 541714 and its SIC code is 8731.