Israel’s SpliSense (see here previously) has begun a first Phase 1/2 human trial of its SPL84 treatment for a specific genetic mutation that causes cystic fibrosis. SpliSense utilizes proprietary RNA therapies called ASOs (antisense oligonucleotides) to correct mutations. https://www.prnewswire.com/news-releases/splisense-initiates-phase-12-study-of-spl84-rna-based-therapy-for-the-treatment-of-cystic-fibrosis-301702837.html

Israel’s SpliSense (see here previously) uses mRNA to fix the root cause of cystic fibrosis – genetic mutations that create a non-functioning protein (CFTR). It not only treats patients with no current treatment but is also much cheaper than existing … Continue reading →

Israel’s SpliSense (see here), is developing mRNA-altering therapies for cystic fibrosis (CF) and other genetic pulmonary diseases. SpliSense founder Professor Batsheva Kerem was part of the Hebrew University team that identified the CFTR gene and developed the CF therapy. https://www.calcalistech.com/ctech/articles/0,7340,L-3907693,00.html

Five Israeli bio-techs at Israel’s MIXiii-Biomed 2018 are developing new therapies for “orphan” (rare) genetic diseases. They include ART Bioscience (Duchenne’s), Eloxx (cystinosis), SpliSense (Cystic Fibrosis), NewStem (Fragile X Syndrome) and Minovia (mitochondrial diseases). https://www.israel21c.org/5-israeli-startups-seeking-cures-for-rare-genetic-diseases/ https://www.artbioscience.com/ http://www.eloxxpharma.com/ http://www.integra-holdings.com/portfolio-items/splisense/ http://www.iati.co.il/company/3793/newstem http://www.iati.co.il/company/3522/minovia-therapeutics … Continue reading →