JERUSALEM, April 3, 2024 /PRNewswire/ -- SpliSense, a clinical-stage biotechnology company focused on the development of transformative RNA-based therapies for pulmonary diseases including cystic fibrosis (CF), muco-obstructive diseases like COPD, asthma, non-CF bronchiectasis (NCFB) and idiopathic pulmonary fibrosis (IPF), today announced that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for the initiation of a Phase 2 study for SPL84 in CF. SPL84 is the Company's lead antisense oligonucleotide (ASO) product for the treatment of people with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
CF Foundation Provides Up to $8.5M to SpliSense to Support a Clinical Trial for a Potential Treatment for Splicing Mutations
JERUSALEM, Sept. 6, 2023 /PRNewswire/ -- SpliSense, a biopharmaceutical company developing transformative RNA-based therapies for pulmonary diseases including cystic fibrosis (CF), muco-obstructive diseases and idiopathic pulmonary fibrosis (IPF), today announced that it has successfully completed a first-in-human, Phase 1 clinical trial of SPL84, the Company's lead inhaled anti sense oligonucleotide (ASO) product for the treatment of patients with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene. SPL84 was shown to be safe and well tolerated.
SpliSense has commenced a Phase I/II clinical trial of its lead anti sense oligonucleotide (ASO) product, SPL84, to treat patients with cystic fibrosis (CF) carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.
JERUSALEM, Dec. 14, 2022 /PRNewswire/ -- SpliSense, a biopharmaceutical company developing transformative RNA-based therapies for pulmonary diseases including cystic fibrosis (CF), muco-obstructive diseases and idiopathic pulmonary fibrosis (IPF), today announced that it has initiated a first-in-human, Phase 1/2 clinical trial of SPL84, the Company's lead anti sense oligonucleotide (ASO) product for the treatment of patients with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.
JERUSALEM, Jan. 4, 2022 /PRNewswire/ -- SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation (ODD) to SPL84-23-1, an antisense oligonucleotide (ASO) targeting the 3849 mutation in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene associated with a severe form of CF lung disease.