SpliSense Presents Preclinical Data for SPL23-ASO, mRNA-Altering Drug Candidate for the Treatment of Cystic Fibrosis, at the European Cystic Fibrosis Conference

在欧洲囊性纤维化会议上,SpliSense提供了SPL23-ASO的临床前数据,SPL23-ASO是治疗囊性纤维化的mRNA改变药物候选

2021-06-11 18:00:07 BioSpace

本文共1582个字,阅读需4分钟

Data show that SPL23-ASO, targeting the W1282X nonsense mutation, enables production of a functional cystic fibrosis transmembrane conductance regulator protein JERUSALEM, June 11, 2021 /PRNewswire/ -- 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. Company contact: Tsipi Haitovsky Global Media Liaison SpliSense +972-52-5989-892 Tsipihai5@gmail.com View original content:http://www.prnewswire.com/news-releases/splisense-presents-preclinical-data-for-spl23-aso-mrna-altering-drug-candidate-for-the-treatment-of-cystic-fibrosis-at-the-european-cystic-fibrosis-conference-301310187.html SOURCE SpliSense
数据表明,靶向W1282X无义突变的SPL23-ASO能够产生功能性囊性纤维化跨膜电导调节蛋白 耶路撒冷2021年6月11日电/PRNewswire/--为囊性纤维化(CF)和其他肺部疾病开发改变mRNA疗法的生物制药公司SpliSense今天公布了SPL23-ASO的临床前数据,该反义寡核苷酸(ASO)靶向与严重形式的CF相关的W1282X无义突变,显示ASO能够生产功能性囊性纤维化跨膜电导调节因子(CFTR)蛋白。这些数据是在耶路撒冷希伯来大学遗传学教授,SpliSense的科学创始人兼首席科学官Batsheva Kerem受邀发表的口头报告中发表的,该报告题为“无厘头突变的新方法”,作为CFTR调制的临床试验的一部分----还有什么?第44届欧洲囊性纤维化数字会议专题讨论会,2021年6月9-12日。 SpliSense利用短的、精确靶向的、称为ASOs的专有RNA延伸来纠正CFTR mRNA的各种突变。特别地,ASO在所需的位点与突变的CFTR RNA结合,导致从mRNA中消除突变的区域,并允许细胞产生功能性CFTR蛋白。ASO通过吸入被肺细胞吸收并驱动经校正的CFTR mRNA和最终功能化的CFTR蛋白的产生。SpliSense利用专有算法来支持优化ASO的设计,从而最大限度地提高效率并减少潜在的不良影响。 Splisense公司首席执行官Gili Hart博士说:“我们很高兴在我们的管道中再增加一个有前途的临床前候选人。“CF是一种使人衰弱的疾病,会导致肺部频繁感染、呼吸困难和预期寿命缩短。调节CF中突变蛋白CFTR活性的药物显著提前了CF患者的治疗,但仅对少量突变有效。例如,携带W1282X无义突变的人根本不产生CFTR蛋白,因此不是CFTR调节药物的候选者。因此,我们很兴奋地看到我们的技术有可能治疗这种突变的人,并期待着将SPL23-ASO和SPL84-23这两种设计用于治疗3849+10kb的C->T CFTR突变的技术在明年推向临床。“ 关于囊性纤维化 囊性纤维化(Cystic fibrosis,CF)是一种遗传性疾病,可导致呼吸道感染和致残,影响全球9万多人。它是由CFTR基因突变引起的,导致CFTR蛋白功能失调。致病性CFTR蛋白变异体在离子运输中造成不规则,产生粘液阻塞、慢性感染、炎症甚至呼吸衰竭。 在CFTR基因中有超过2000个变异体已被鉴定,其中超过400个已知可导致疾病。在过去的十年中,CF护理发生了巨大的变化。然而,批准的CFTR调节剂并不支持所有的CF患者,也不能为该病提供治愈方法。因此,药物开发的新策略对于解决无反应突变至关重要。 关于SpliSense SpliSense是一家生物制药公司,专注于针对遗传性肺部疾病未满足需求的改变mRNA治疗。该公司的开创性平台利用ASOs治疗遗传疾病,如CF。Splisense的管道包括处于不同发展阶段的创新疗法,从发现到IND使能研究。欲了解更多信息,请访问我们的网站:www.splisense.com。 公司联系人: 济皮·海托夫斯基 全球媒体联络 斯普利塞塞 +972-52-5989-892 @gmail.com tsipihai5@gmail.com 查看原文内容:http://www.prnewswire.com/news-releases/splisense-presents-preclinical-data-for-spl23-aso-mrna-alterning-drug-candidate-for-the-treatment-of-cystic-fibrosis-at-the-european-cystic-fibrosis-conference-301310187.html 源SpliSense

以上中文文本为机器翻译,存在不同程度偏差和错误;偶尔因源网页结构局限,内容无法一次完整呈现。请理解并参考原站原文阅读。

阅读原文