Project Description

SPLISENSE

At a glance

Established by Integra Holdings in 2016, SpliSense is developing novel drug candidates based on antisense oligonucleotides (ASOs) targeting major splicing mutations in genetic diseases. SpliSense is currently focusing on splicing mutations in the CFTR gene for treating Cystic Fibrosis (CF) patients.

The discovery platform was invented and developed by Prof. Batsheva Kerem from the Department of Genetics at the Hebrew University.

Market Potential

The global market for CF is estimated at $3.5 billion, with increasing market share and sales of disease modifying agents currently developed and marketed by major pharma companies.

Company Highlights

Novel, fully synthetic ASOs have been designed and selected as drug candidates targeting the splicing machinery.  As a significant fraction of CF-causing mutations (10-15%) affects pre-mRNA splicing, leading to the generation of aberrantly spliced CFTR transcripts, the company is now developing a targeted modulation approach aimed to correct the splicing pattern of CFTR transcripts, according to the specific mutation carried. This strategy will interfere with the aberrant CFTR splicing in patients carrying splicing variants/mutations leading to the generation of normal full length protein and restoration of the CFTR function. Prof. Kerem, an expert geneticist, identified and cloned the CFTR gene.

Key Shareholders

Integra Holdings, Yissum.

Funding

Extensive business development activity is ongoing, to support further development of the technology

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