Data show that SPL23-ASO, targeting the W1282X nonsense mutation, enables production of a functional cystic fibrosis transmembrane conductance regulator protein
JERUSALEM, Israel I June 11, 2021 I SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases, today announced preclinical data for SPL23-ASO, an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of the CF, showing that the ASO enables production of a functional Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein. The data were presented at an invited oral presentation delivered by Batsheva Kerem, Professor of Genetics at the Hebrew University of Jerusalem, and scientific founder and Chief Scientific Officer of SpliSense, in a talk titled “Novel approaches to nonsense mutations” as part of the Clinical trials on CFTR modulation – What lies beyond? symposium at the 44th European Cystic Fibrosis Digital Conference, June 9-12, 2021.
SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA. In particular, the ASO binds to the mutated CFTR RNA in the desired spot, leading to the elimination of the mutated region from the mRNA and allowing the cell to produce functional CFTR proteins. The ASOs are administered directly and preferentially to the lungs via inhalation where it is taken up by the lung cells and drive the production of corrected CFTR mRNA and eventually functional CFTR proteins. SpliSense utilizes proprietary algorithms to support the design of optimized ASOs, thereby maximizing efficiency and reducing the potential for undesired effects.
“We are pleased to add another promising preclinical candidate to our pipeline,” said Gili Hart, PhD, CEO, SpliSense. “CF is a debilitating disease, leading to frequent lung infections, breathing difficulties and reduced life expectancy. Drugs that modulate the activity of CFTR, the mutated protein in CF, significantly advanced the treatment of CF patients, but they are effective only for a small number of mutations. People carrying the W1282X nonsense mutation, for example, do not produce a CFTR protein at all and are therefore not candidates for CFTR modulating drugs. We are therefore excited to see that our technology has the potential to treat people with such mutations and look forward to advancing both SPL23-ASO and SPL84-23, which is designed to treat the 3849+10kb C->T CFTR mutation, into the clinic next year.”
About Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disease that leads to respiratory infections and disabilities and affects over 90,000 people worldwide. It is caused by mutations in the CFTR gene, leading to dysfunctional CFTR proteins. Disease-causing CFTR protein variants create irregularities in the transport of ions, generating mucus obstructions, chronic infection, inflammation and even respiratory failure.
Over 2000 variants in the CFTR gene have been identified, and over 400 are known to cause disease. The past decade has seen a dramatic change in CF care. However, approved CFTR modulators do not support all patients with CF or offer a cure for the disease. Thus, new strategies of medication development are essential to address non-responsive mutations.
About SpliSense
SpliSense is a biopharmaceutical company focused on transformative mRNA-altering treatments targeting unmet needs in genetic pulmonary diseases. The Company’s pioneering platform harnesses ASOs for treatment of genetic diseases such as CF. SpliSense’ pipeline includes innovative therapies in various development stages, from discovery to IND enabling studies. For additional information, please visit our website at www.splisense.com.
SOURCE: SpiSense
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Data show that SPL23-ASO, targeting the W1282X nonsense mutation, enables production of a functional cystic fibrosis transmembrane conductance regulator protein
JERUSALEM, Israel I June 11, 2021 I SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases, today announced preclinical data for SPL23-ASO, an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of the CF, showing that the ASO enables production of a functional Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein. The data were presented at an invited oral presentation delivered by Batsheva Kerem, Professor of Genetics at the Hebrew University of Jerusalem, and scientific founder and Chief Scientific Officer of SpliSense, in a talk titled “Novel approaches to nonsense mutations” as part of the Clinical trials on CFTR modulation – What lies beyond? symposium at the 44th European Cystic Fibrosis Digital Conference, June 9-12, 2021.
SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA. In particular, the ASO binds to the mutated CFTR RNA in the desired spot, leading to the elimination of the mutated region from the mRNA and allowing the cell to produce functional CFTR proteins. The ASOs are administered directly and preferentially to the lungs via inhalation where it is taken up by the lung cells and drive the production of corrected CFTR mRNA and eventually functional CFTR proteins. SpliSense utilizes proprietary algorithms to support the design of optimized ASOs, thereby maximizing efficiency and reducing the potential for undesired effects.
“We are pleased to add another promising preclinical candidate to our pipeline,” said Gili Hart, PhD, CEO, SpliSense. “CF is a debilitating disease, leading to frequent lung infections, breathing difficulties and reduced life expectancy. Drugs that modulate the activity of CFTR, the mutated protein in CF, significantly advanced the treatment of CF patients, but they are effective only for a small number of mutations. People carrying the W1282X nonsense mutation, for example, do not produce a CFTR protein at all and are therefore not candidates for CFTR modulating drugs. We are therefore excited to see that our technology has the potential to treat people with such mutations and look forward to advancing both SPL23-ASO and SPL84-23, which is designed to treat the 3849+10kb C->T CFTR mutation, into the clinic next year.”
About Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disease that leads to respiratory infections and disabilities and affects over 90,000 people worldwide. It is caused by mutations in the CFTR gene, leading to dysfunctional CFTR proteins. Disease-causing CFTR protein variants create irregularities in the transport of ions, generating mucus obstructions, chronic infection, inflammation and even respiratory failure.
Over 2000 variants in the CFTR gene have been identified, and over 400 are known to cause disease. The past decade has seen a dramatic change in CF care. However, approved CFTR modulators do not support all patients with CF or offer a cure for the disease. Thus, new strategies of medication development are essential to address non-responsive mutations.
About SpliSense
SpliSense is a biopharmaceutical company focused on transformative mRNA-altering treatments targeting unmet needs in genetic pulmonary diseases. The Company’s pioneering platform harnesses ASOs for treatment of genetic diseases such as CF. SpliSense’ pipeline includes innovative therapies in various development stages, from discovery to IND enabling studies. For additional information, please visit our website at www.splisense.com.
SOURCE: SpiSense
Post Views: 27
