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Vector lentiviral episomal no integrativo replicativo

Resumen

Tipo:
Oferta Tecnológica
Referencia:
TOES20170908001
Publicado:
27/09/2017
Caducidad:
27/09/2018
Resumen:
Una pyme española ha desarrollado un vector lentiviral episomal no integrativo replicativo que ofrece una expresión duradera, estable y consistente del transgén en células divisorias y no divisorias. La tecnología se ha diseñado para permitir una administración segura de genes terapéuticos a tejidos bajo proliferación sin integración, convirtiéndola en una tecnología única. La empresa busca compañías biotecnológicas/farmacéuticas o centros de investigación aplicada con una línea de terapia genética interesados en probar y desarrollar fármacos para terapia genética en cualquier tratamiento en el que puedan ser útiles los vectores lentivirales.

Details

Tittle:
Non-integrative replicative episomal lentiviral vector technology
Summary:
A Spanish SME has developed an episomal non-integrative lentiviral vector that provides the lasting, stable and consistent expression of the transgene in dividing and non-dividing cells without modifying the cell genome through integration.
The company is interested in collaborating with Biotech/Pharma Companies or Applied Research Centres with a genetic therapy pipeline, in testing and developing genetic therapy drugs in any therapy where lentiviral vectors may be of use.
Description:
This technology is designed to enable the safe delivery of therapeutic genes to tissues under proliferation without integration, which makes the technology unique. The innovation takes as start point the enormous knowledge and previous long experience in retroviral vectors but is engineered with genetic elements aimed at providing the unmet properties of gamma-retrovirus and self-inactivating lentiviral vectors (SIN-LV). The technology will minimize the patients monitoring costs, because it avoids transgene integration, while maintaining the efficiency of the treatment during the life of the patient.

To better understand the scenario, we have recorded a snapshot of previous gene therapy studies with gamma-retrovirus, a story of partial clinical recovery, transient benefits to patients and unsuccessful approaches. Despite the success curing several monogenic, rare primary congenital immunodeficiency diseases like X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), Wiskott-Aldrich syndrome (WAS) or chronic granulomatous disease (CGD), random insertion of the vector occasionally altered endogenous gene function. Due to this major setback, 5 out of 20 paediatric patients treated for SCID-X1 developed leukaemia one which died as direct consequence of the treatment ; 7 out of 10 treated patients for WAS developed leukaemia, of which two patients died; and some patients treated for CGD resulted in monosomy 7 and myelodisplasic syndrome1. Despite SIN-LV also displays random integration, there are certain preferences in the selection of the integration site that diminish but do not abolish their potential genotoxicity. Moreover, the record of patients treated with SIN-LV is still low and long-term studies are still required to ensure absolute free of genotoxicity.

To overcome the risk of aberrant gene activation or disruption by insertional mutagenesis, the only solution is to obtain a technology that prevents integration in the dividing cells. This has been the breakthrough achievement obtained by the company, that is trying to position as a global leader, already collaborating with Companies in USA and Europe.

Some of the already tested conditions that can be disclosed are:
¿ Stability upon freezing and storage (>12mo.)
¿ Identity of episomal forms (seq)
¿ NO integration (Alu-PCR; FISH analysis)
¿ Low number of copies (2-4/cell)
¿ Homogeneous level expression
¿ Independent of cell line (HEK293, K562, MOLT4)
¿ Produced at levels similar to integrative lentis
¿ Early studies on CD34+ cells are promising
Advantages and Innovations:
This technology consists of a new generation of non-integrative lentiviral vectors.

It is designed for stable gene transfer in cells and tissues, regardless the division rate without integration.

This technology provides in a simple and effective way, the lasting, stable and consistent expression of the transgene in dividing and non-dividing cells without modifying the cell genome through integration.

This new vector incorporates specific eukaryotic sequences, which direct both the binding of the viral genome with the nuclear matrix (Scaffold/Matrix Attachment Regions - S/MAR sequences) and its duplication through the replication of cell DNA (ori sequence). It is generated with a class I mutation in the viral integrase, so the intranuclear viral genome forms are circular episomes.

These facts allow to extend lentiviral vectors clinical use, widening their application to indications in which the modification of tissues or cells with high division rates is required.
Stage of Development:
Prototype available for demonstration
IPs:
Patents granted
CommeR Statunts Regarding IPR Status:
Patented technology in several countries (USA, Europa, Japan etc.)

Partner sought

Type and Role of Partner Sought:
The company is looking to collaborate with Biotech/Pharma Companies or Applied Research Centres for the development and/or manufacture of lentiviral vectors.

The company is interested in contacting with colaborators with a genetic therapy pipeline developing drugs using lentiviral vectors that do not integrate into DNA, willing to test their genetic therapy in pre-clinical phase.

Client

Type and Size of Client:
Industry SME 11-49
Already Engaged in Trans-National Cooperation:
Si
Languages Spoken:
English
Spanish

Keywords

Technology Keywords:
06002002 Biología celular y molecular
06002001 Bioquímica / biofísica
06002005 Ingeniería genética
06001013 Tecnología médica / ingeniería biomédica