Loading organizations...
§ Private Profile · Milan, Lombardia, Italy
Gene therapies for pediatric genetic diseases. Off-the-shelf, immune-shielded lentiviral vectors enable lifelong treatment.
Genespire is a Milan, Italy-based biotechnology company that develops off-the-shelf gene therapies using proprietary immune-shielded lentiviral vectors for pediatric patients with rare genetic diseases. The company's platform enables the lifelong production of therapeutic genes directly from the patient's liver following a single injection, which prevents immune responses and eliminates the need for repeat dosing. Its lead candidate, GENE202, is currently undergoing investigational new drug-enabling studies to treat methylmalonic acidemia, with clinical trials targeted to begin by the end of 2026. To support its clinical advancement, the firm raised $52 million in a Series B funding round in September 2024 led by Sofinnova Partners, alongside institutional backing from Fondazione Telethon and Ospedale San Raffaele. Operating as a spin-out from the SR-Tiget gene therapy research institute, Genespire was founded in 2023 by Luigi Naldini and Alessio Cantore.
Genespire has raised $69.0M across 2 funding rounds.
Genespire has raised $69.0M in total across 2 funding rounds.
Genespire has raised $69.0M across 2 funding rounds. Most recently, it raised $52.0M Series B in September 2024.
| Date | Round | Lead Investors | Other Investors | Status |
|---|---|---|---|---|
| Sep 1, 2024 | $52M Series B | Soffinova Partners, Agostino Scornajenchi, Xgen Ventures | — | Announced |
| Apr 1, 2020 | $17M Series A | Soffinova Partners | — | Announced |
Genespire is a biotechnology company developing off-the-shelf gene therapies using proprietary immune shielded lentiviral vectors (ISLVs) for genetic diseases, particularly in children.[1][2][3][4] These vectors enable one-time treatments by allowing lifelong production of therapeutic genes directly from a patient's liver, targeting conditions like methylmalonic acidemia, hyper-IgM immunodeficiency, and metabolic diseases, all in preclinical stages.[1][4][5] Based in Milan, Italy, Genespire serves pediatric and adult patients with genetic disorders, solving the problem of immune responses that limit traditional lentiviral vectors, while offering stable, long-term gene expression even in growing organs.[1][4]
The company focuses on *in vivo* gene therapy, injecting vectors into peripheral blood for liver targeting, which integrates therapeutic genes into patient DNA for sustained effects.[4] With programs like GENE101 for hyper-IgM syndrome and GENE103/104 for metabolic diseases, Genespire shows early momentum through its innovative platform, though all candidates remain preclinical as of available data.[5]
Genespire was co-founded by gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, in collaboration with Fondazione Telethon and Ospedale San Raffaele.[1] Naldini, a renowned expert, brings deep expertise from advancing lentiviral vector technology, while Cantore contributes biopharmaceutical research experience.[1] The company emerged from efforts to overcome immune barriers in lentiviral vectors, building on over a decade of gene therapy successes and regulatory approvals.[4]
Early traction stems from developing ISLVs, a novel vector class masked from the immune system, tailored for pediatric genetic diseases.[1][3][4] Led by an international management team from top biotech and biopharma organizations, Genespire established operations in Milan, positioning itself to extend gene therapy's reach.[1]
Genespire rides the gene therapy expansion trend, fueled by clinical successes and approvals over the past decade, shifting from ex vivo to *in vivo* approaches for scalability.[4] Timing aligns with growing demand for pediatric treatments, where traditional vectors fail due to dilution in expanding tissues—ISLVs counter this via genomic integration.[1][4] Market forces like rising genetic disease prevalence and immune-evasive tech advancements favor Genespire, influencing the ecosystem by broadening lentiviral applications beyond AAV limitations.[2][4]
As a Milan-based innovator tied to Telethon and San Raffaele, it strengthens Europe's biotech hub, potentially accelerating off-the-shelf therapies and reducing manufacturing costs for rare diseases.[1][3]
Genespire's ISLV platform positions it for pipeline advancement into clinical trials, starting with lead preclinical assets like GENE101 and metabolic programs, potentially yielding first approvals in 3-5 years amid gene therapy's momentum.[4][5] Trends like immune stealth tech and pediatric focus will shape its path, with partnerships (e.g., Telethon) aiding scaling. Its influence may evolve from niche innovator to ecosystem leader, driving accessible, lifelong treatments that transform genetic disease management—echoing its mission to unlock gene therapy's full potential for children.[1][4]
Genespire has raised $69.0M in total across 2 funding rounds.
Genespire's investors include Soffinova Partners, Agostino SCORNAJENCHI, XGen Ventures.