Vés enrere

Uni-large is a new generation of gene editing techniques combining the precision of CRISPR/cas9 and the efficiency of viral vectors established in clinic, which allows the insertion of multi-kilobase genome fragments.

 

BACKGROUND

Congenital muscular dystrophy Type 1A (MDC1A) is an early onset and life-threatening disease that affects 1-9 in 1,000,000 people worldwide. It is caused by mutations in the laminin alpha-2 gene, a large gene that exceeds the size limit of adeno-associuated viral vectors (AAV), used for in vivo gene therapy. Therefore, it is necessary to develop alternative therapies. Uni-large will provide a safe and therapeutic opportunity.

 

THE TECHNOLOGY

Uni-Large combines the precision of modern programmable nucleases and specific DNA binding proteins (CRISPR/Cas9, Zinc Finger Proteins) and the efficiency and efficacy of classic vectors for integrative gene delivery (lentivirus, transposases). This technology will allow for targeted integration of large payloads (6-30kb) into the human genome, which constitute its differential value. The therapeutic effect does not depend on HDR (often limiting efficacy), and is potentially safer than competing technologies (absence of double-strand breaks, reduction of insertion mutagenesis).

 

ADVANTAGES

  • Safe and efficient, novel and specific treatment
  • MDC1A is life-threatening, no treatment available
  • Versatile strategy, applicable to other diseases
  • Efficient for large genes

 

STATE OF DEVELOPMENT

The technology has been tested both in vitro in cells and in vivo using a mouse model of MDC1A with positive results. Further steps should be the development of regulatory preclinical assays and the study of the use of UniLarge to treat other indications.

 

INTELLECTUAL PROPERTY

A US patent application has been filed, obtaining a very positive patentability report.

 

MARKET OPPORTUNITY

UniLarge can be extended to other diseases, giving a valuable competitive advantage to the gene therapy industry, one of the most attractive markets in the pharmaceutical and technological sector ($ 7,500 million in 2024).

 

COMMERCIAL OPPORTUNITY

Technology available for licensing with technical cooperation.

 

CONTACT

Dr Cinta Diez

Technology Transfer Unit

Pompeu Fabra University

T. +34.93 316 09 13

[email protected]

 

KEYWORDS

Gene editing, genome editing, MDC1A, congenital muscular dystrophy, large genes, HDR.

 

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