Splisense

About:

Splisense is develops antisense oligonucleotide based therapies to target genetic diseases caused by splicing mutations.

Website: https://splisense.com

Top Investors: OrbiMed, Integra Holdings, Cystic Fibrosis Foundation, Israel Biotech Fund, Biotel

Description:

Splisense is develops antisense oligonucleotide based therapies to target genetic diseases caused by splicing mutations. The company has developed a compound to cure the lung disease in cystic fibrosis patients carrying a specific splicing mutation. It is also developing a targeted modulation approach to correct the splicing pattern of CFTR transcripts according to the specific mutation carried.