Genespire Srl
Genespire develops off-the-shelf gene therapies using proprietary immune shielded lentiviral vectors (ISLV) designed for permanent therapy production directly from the liver, aiming to provide a one-time treatment for adults and children with genetic diseases. Co-founded by leading experts and supported by renowned institutions, the company focuses on innovative gene therapy solutions to treat rare genetic disorders.
Industries
Nr. of Employees
small (1-50)
Products
Off-the-shelf liver-directed integrating vector gene therapy candidates
Preclinical-stage gene therapy candidates based on immune-evasive integrating lentiviral vectors intended for systemic delivery to the liver to provide durable therapeutic gene expression.
Ex vivo gene-edited T-cell and hematopoietic stem cell candidates
Programs using improved gene editing technologies applied ex vivo to T cells and hematopoietic stem/progenitor cells for therapeutic applications.
Off-the-shelf liver-directed integrating vector gene therapy candidates
Preclinical-stage gene therapy candidates based on immune-evasive integrating lentiviral vectors intended for systemic delivery to the liver to provide durable therapeutic gene expression.
Ex vivo gene-edited T-cell and hematopoietic stem cell candidates
Programs using improved gene editing technologies applied ex vivo to T cells and hematopoietic stem/progenitor cells for therapeutic applications.
Services
In-house design and execution of preclinical efficacy, safety and dosing studies to support translation to clinical trials.
Process development, quality and tech transfer activities supporting clinical manufacturing readiness for gene and cell therapy products.
Preparation of regulatory engagement plans and clinical development strategies for in vivo and ex vivo gene therapy programs.
Establishing strategic collaborations with academic institutes, research hospitals and investors to advance programs.
In-house design and execution of preclinical efficacy, safety and dosing studies to support translation to clinical trials.
Process development, quality and tech transfer activities supporting clinical manufacturing readiness for gene and cell therapy products.
Preparation of regulatory engagement plans and clinical development strategies for in vivo and ex vivo gene therapy programs.
Establishing strategic collaborations with academic institutes, research hospitals and investors to advance programs.
Expertise Areas
- Liver-directed in vivo gene therapy
- Lentiviral vector engineering and immune evasion
- Ex vivo gene editing for T cells and hematopoietic stem cells
- Preclinical development and translational research
Key Technologies
- Immune-evasive lentiviral vectors
- Integrating viral vector platforms
- Systemic liver-targeted vector delivery
- Ex vivo nuclease-based gene editing