Genespire
Genespire is an Italian clinical-stage biotech developing in-vivo gene therapies using its proprietary Immune Shielded Lentiviral Vectors (ISLV) platform, with lead program GENE202 targeting pediatric methylmalonic acidemia and other rare genetic diseases.
- Company typePrivate
- Founded2020
- HeadquartersMilan, Italy
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
Genespire firmographics
Firmographics- Name
- Genespire
- Legal name
- GeneSpire S.r.l.
- Website
- https://genespire.com
- Company type
- Private
- Founded year
- 2020
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Genespire is an Italian clinical-stage biotech developing in-vivo gene therapies using its proprietary Immune Shielded Lentiviral Vectors (ISLV) platform, with lead program GENE202 targeting pediatric methylmalonic acidemia and other rare genetic diseases.
- Ownership category
- akta.pro rank
Genespire industry classification
Industry- Product category
- Gene Therapy
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- In Vitro & In Vivo Diagnostic Substances (2835)
- akta.pro primary industry
- Lentiviral Vector Gene Therapy Developers (HLAAACAB)
- akta.pro secondary industries
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)
Keywords
Where Genespire is headquartered
LocationHeadquarters
- HQ city
- Milan
- HQ country
- Italy
- HQ region
- Europe
Offices1 record
Markets served
Genespire business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations
Marketing channels3 records
Genespire product offering
Product offeringCore offering
Genespire develops off-the-shelf, in vivo gene therapies using its proprietary Immune Shielded Lentiviral Vector (ISLV) platform. The lead product candidate GENE202 is an ISLV-based gene therapy for methylmalonic acidemia (MMA), a rare pediatric metabolic disease, and the company is also advancing programs for additional inherited metabolic diseases and bleeding disorders.
Product overview
Genespire develops off-the-shelf gene therapies using its proprietary Immune Shielded Lentiviral Vector (ISLV) platform. The company's lead product is GENE202, an ISLV-based gene therapy targeting methylmalonic acidemia (MMA). The ISLV platform enables in vivo liver-directed gene therapy, allowing permanent therapeutic gene expression even in growing organs and young patients. The company also develops programs for additional inherited metabolic diseases and bleeding disorders.
Differentiator
Problem solved
Functional benefit
Products and services
- GENE202 An ISLV-based gene therapy for treating methylmalonic acidemia (MMA), a rare genetic metabolic disease primarily affecting children. Designed as a one-time, off-the-shelf treatment accessible to pediatric patients, providing permanent therapeutic gene expression by inserting the corrective gene into patient liver cells.
Quantifiable outcome
- GENE202 received Orphan Drug Designation from FDA and EC for MMA treatment
Companies that use Genespire
Customer profileSegments1 record
Ideal customer profiles1 record
Genespire technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature1 record
Genespire partnerships and signals
Strategic signalPartnerships
Two partnerships are on record, tiered core.
- San Raffaele Telethon Institute for Gene Therapy (SR-Tiget)coreStrategic alliance for development of transformative gene therapies for genetic diseases. The institute, led by Prof. Luigi Naldini, is internationally recognized for gene therapy research and has been involved in the co-founding of Genespire.
- Fondazione TelethoncoreOne of the main Italian biomedical charities focused on curing rare genetic diseases. Co-founded Genespire and provides ongoing institutional support as a board member representative (Dr. Francesca Pasinelli serves as Director).
Scale indicators3 records
Recent moves6 records
Expansion highlights5 records
Genespire competitors and assessment
Company assessmentDirect peers
- Orchard Therapeutics: Commercial-stage lentiviral gene therapy developer (Libmeldy, Strimvelis) targeting rare monogenic diseases. Directly comparable on platform modality (lentiviral vectors), indication space (pediatric rare metabolic/immune diseases), and clinical/CMC execution model. Genespire's VP Clinical (Simon Hawkins) and CMC leads previously led ex vivo lentiviral programs at Orchard.
- AVROBIO: Clinical-stage lentiviral gene therapy company developing one-time treatments for rare lysosomal storage diseases and other monogenic conditions. Direct peer on lentiviral platform, ex vivo hematopoietic target, and rare-disease commercial strategy. Genespire's VP Clinical led ex vivo lentiviral programs at AVROBIO through pivotal regulatory submissions.
- MolMed S.p.A. Italian cell and gene therapy company developing lentiviral-based therapies (Zalmoxis, CAR-T). Direct peer on geography (Milan-based), lentiviral modality, and rare-disease focus. Genespire's Head of Project Management previously held senior regulatory and program leadership roles at MolMed.
Emerging players
- Cellectis S.A. Clinical-stage gene editing company using lentiviral-based delivery for allogeneic CAR-T and gene editing applications. Comparable on lentiviral vector expertise and translational gene therapy development; Genespire's CDO Julianne Smith previously led translational sciences at Cellectis.
- Verve Therapeutics: Clinical-stage in-vivo gene editing company using lipid nanoparticle delivery to the liver for cardiometabolic diseases (e.g., familial hypercholesterolemia). Emerging competitor validating the in-vivo liver-directed approach that ISLV also targets, though using base-editing rather than lentiviral gene insertion.
- Intellia Therapeutics: Clinical-stage in-vivo CRISPR/Cas9 gene editing company with liver-directed programs (transthyretin amyloidosis, hereditary angioedema). Emerging peer on the broader in-vivo liver gene therapy modality and rare-disease focus; could compete with ISLV on next-generation liver indications.
Broad incumbents
- bluebird bio: Established commercial lentiviral gene therapy developer (Zynteglo, Skysona, Lyfgenia) for rare genetic diseases including metabolic and neurological indications. Broad incumbent on lentiviral modality, regulatory experience with FDA/EMA, and ultra-rare disease commercial model.
- uniQure N.V. Clinical and commercial gene therapy company with AAV-based platform targeting liver and CNS indications (Hemgenix for hemophilia). Broad incumbent on rare-disease liver-directed gene therapy, though using AAV rather than lentiviral vectors.
- BioMarin Pharmaceutical: Large rare-disease biotech with AAV gene therapy programs (Roctavian for hemophilia A) and metabolic disease franchise. Broad incumbent on rare metabolic and hematologic disease commercialization, regulatory pathways, and pricing dynamics directly relevant to GENE202's MMA opportunity.
Others
- Fondazione Telethon / SR-Tiget (Transgene/Telethon spin-out ecosystem): Institutional co-founder and strategic partner of Genespire. Comparable as the originating research institution for multiple gene therapy companies in Italy; Ospedale San Raffaele/Telethon spin-outs (including the Strimvelis heritage) form the ecosystem in which Genespire is positioned.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat3 records
Key highlights7 records
Customer concentration
Genespire social profiles
Digital presenceGenespire compliance and trust
Trust signalCompliance4 records
Genespire financial estimates
Financial estimateRevenue estimate
Valuation estimate
Genespire leadership team
Management profileNumber of profiles
Profiles11 records
Genespire funding detail
Funding detailFunding overview
Funding rounds2 records
Investors4 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Genespire 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 Genespire
What does Genespire do?
Genespire develops off-the-shelf, in vivo gene therapies using its proprietary Immune Shielded Lentiviral Vector (ISLV) platform. The lead product candidate GENE202 is an ISLV-based gene therapy for methylmalonic acidemia (MMA), a rare pediatric metabolic disease, and the company is also advancing programs for additional inherited metabolic diseases and bleeding disorders.
Is Genespire a public or private company?
Genespire is a private company. It is classified as venture growth investor backed and is currently operating.
When was Genespire founded?
Genespire was founded in 2020. It employs 1 to 10 people.
Where is Genespire based?
Genespire is headquartered in Milan, Italy, in the Europe region.
Who are Genespire's main competitors?
Direct peers on record are Orchard Therapeutics, AVROBIO and MolMed S.p.A.. Emerging players are Cellectis S.A., Verve Therapeutics and Intellia Therapeutics. Broad incumbents are bluebird bio, uniQure N.V. and BioMarin Pharmaceutical. Fondazione Telethon / SR-Tiget (Transgene/Telethon spin-out ecosystem) is listed as an others.
Does Genespire have an API?
No public API is recorded for Genespire.
What industry is Genespire in?
Genespire's product category is Gene Therapy. 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 2835.