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小分子开发商Satellos宣布SAT-3247治疗杜氏肌营养不良症(DMD)犬模型可实现肌肉修复和再生

Satellos Presents Preliminary Data Showing Muscle Repair and Regeneration from SAT-3247 Treatment in Canine Model of Duchenne Muscular Dystrophy (“DMD”)

businesswire 等信源发布 2024-07-02 17:00

可切换为仅中文


TORONTO--(BUSINESS WIRE)--Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced preliminary results from a single case study of SAT-3247 treatment in a canine model of Duchenne muscular dystrophy (DMD or Duchenne).

多伦多--(商业新闻短讯)--Satellos Bioscience Inc.(“Satellos”或“公司”)(TSX:MSCL,OTCQB:MSCLF),一家开发新的小分子治疗方法以改善肌肉疾病和紊乱治疗的公共生物技术公司,今天宣布了在杜兴氏肌营养不良症(DMD或杜兴氏)犬模型中进行SAT-3247治疗的单一病例研究的初步结果。

This model represents a severe dystrophy that closely reflects disease progression observed in people with DMD. SAT-3247 is in development as a novel regenerative medicine approach to treating DMD. These data were presented at the Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference this past weekend..

该模型代表了一种严重的营养不良,与DMD患者的疾病进展密切相关。SAT-3247正在开发作为治疗DMD的新型再生医学方法。这些数据在上周末的家长项目肌营养不良症(PPMD)第30届年会上公布。。

'We are encouraged by these initial data showing treatment with SAT-3247 improved regeneration and muscle force in a canine model of Duchenne,' said Frank Gleeson, Co-founder and CEO of Satellos Bioscience. 'As Satellos' first results in a large animal study, we are pleased that these findings continue to validate the improvement in muscle repair and regeneration that we have consistently seen in the mdx mouse model.

Satellos Bioscience联合创始人兼首席执行官弗兰克·格雷森(FrankGleeson)说:“我们对这些初步数据感到鼓舞,这些数据显示SAT-3247治疗可以改善杜兴犬模型的再生和肌肉力量。”作为Satellos在大型动物研究中的第一个结果,我们很高兴这些发现继续验证了我们在mdx小鼠模型中一直看到的肌肉修复和再生的改善。

This pilot study offers further support that SAT-3247 treatment may be capable of restoring muscle repair and regeneration that is impaired in people living with Duchenne.'.

这项初步研究提供了进一步的支持,即SAT-3247治疗可能能够恢复杜兴氏病患者受损的肌肉修复和再生。”。

After treatment with SAT-3247 the animals showed an increase in Regenerative Index (RI), a measure of the number of newly regenerated muscle fibers versus the number of damaged and dying muscle fibers.

用SAT-3247处理后,动物的再生指数(RI)增加,RI是衡量新再生肌纤维数量与受损和死亡肌纤维数量的指标。

“Increases in the Regenerative Index suggest that muscle repair and regeneration is occurring,” commented Phil Lambert, Ph.D., Chief Scientific Officer of Satellos Bioscience. “While preliminary, these results further build and support our understanding of the unique mechanism of action of SAT-3247.

Satellos Bioscience首席科学官Phil Lambert博士评论道:“再生指数的增加表明肌肉修复和再生正在发生。”。。

We continue to work diligently to advance this novel small molecule drug candidate into a first-in-human clinical trial this quarter.”.

我们继续努力将这种新型小分子候选药物推进本季度首次人体临床试验。”。

The highlighted results released at the PPMD conference and available on the Company’s website here are from one of two dystrophic animals in a pilot study in which each animal was treated for four months with a daily oral dose of SAT-3247. A summary of the results for the single case study animal reported is as follows:.

。报告的单例研究动物的结果总结如下:。

After four months of SAT-3247 treatment, skeletal muscle displayed an approximate four hundred and fifty percent (450%) increase in Regenerative Index; and

经过四个月的SAT-3247治疗后,骨骼肌的再生指数增加了约450%;和

After two months of SAT-3247 treatment, skeletal muscle displayed up to a one hundred percent (100%) increase in muscle force.

经过两个月的SAT-3247治疗后,骨骼肌的肌力增加了100%。

About SAT-3247

关于SAT-3247

SAT-3247 is designed as a once-daily, oral small molecule drug that targets the root cause of muscle loss in degenerative diseases, initially in Duchenne. SAT-3247 presents a novel mechanism of action to restore impaired muscle regeneration caused by the absence of functional dystrophin.

SAT-3247是一种每日一次的口服小分子药物,主要针对退行性疾病中肌肉损失的根本原因,最初是在杜兴。。

About Duchenne Muscular Dystrophy

关于杜兴氏肌营养不良症

Duchenne muscular dystrophy is an inherited disease caused by mutations in the dystrophin gene that no longer allow the dystrophin protein to function properly. Consequently, as discovered by Satellos, muscle repair and regeneration are impaired. Satellos designed SAT-3247 to restore the process of muscle repair and regeneration by regulating a dystrophin-independent pathway with the goal of increasing muscle function.

杜兴氏肌营养不良症是一种遗传性疾病,由肌营养不良蛋白基因突变引起,该突变不再使肌营养不良蛋白正常发挥功能。因此,正如Satellos所发现的那样,肌肉修复和再生受到损害。Satellos设计了SAT-3247,通过调节不依赖肌营养不良蛋白的途径来恢复肌肉修复和再生过程,目的是增加肌肉功能。

SAT-3247 is intended to work as a standalone therapeutic without regard to a patient’s genetic mutation or ambulatory status. Our approach has the potential to complement approaches designed to restore dystrophin production..

SAT-3247旨在作为独立的治疗药物,而不考虑患者的基因突变或门诊状态。我们的方法有可能补充旨在恢复肌营养不良蛋白产生的方法。。

About Satellos Bioscience Inc.

关于Satellos Bioscience Inc。

Satellos is a publicly traded biotechnology company dedicated to developing life-improving medicines to treat degenerative muscle diseases. Satellos has incorporated breakthrough research in muscle stem cell polarity into a proprietary discovery platform, called MyoReGenX™, to identify degenerative muscle diseases where deficits in this process affect muscle regeneration and are amenable to therapeutic intervention.

。Satellos将肌肉干细胞极性的突破性研究纳入了一个专有的发现平台,称为MyoReGenX™,以识别退行性肌肉疾病,其中该过程中的缺陷会影响肌肉再生,并且可以进行治疗干预。

With this platform, Satellos is building a pipeline of novel therapeutics to correct muscle stem cell polarity and promote the body’s innate muscle repair and regeneration process. The Company’s lead program is an oral, small molecule drug candidate in development as a potential disease-modifying treatment for Duchenne muscular dystrophy.

有了这个平台,Satellos正在建立一系列新的治疗方法,以纠正肌肉干细胞的极性,促进身体先天性肌肉修复和再生过程。该公司的领先项目是一种口服小分子候选药物,正在开发中,作为杜兴氏肌营养不良症的潜在疾病缓解疗法。

Satellos is headquartered in Toronto, Ontario. For more information, visit www.satellos.com..

Satellos总部位于安大略省多伦多。有关更多信息,请访问www.Satellos.com。。

Notice on Forward-Looking Statements

关于前瞻性声明的通知

This press release includes forward-looking information or forward-looking statements within the meaning of applicable securities laws regarding Satellos and its business, which may include, but are not limited to, statements regarding the anticipated benefits to patients from a small molecule treatment for Duchenne; the advancement of our lead drug candidate into clinical trials; our belief that assessing regenerative status in skeletal muscle could potentially provide a meaningful way to predict functional outcomes in future clinical trial,, the pharmacodynamic properties and mechanism-of-action of our lead drug candidate; the potential of our approach in other degenerative muscle diseases or in muscle injury or trauma; the general benefits of modulating stem cell polarity by administering small molecule drugs; its/their prospective impact on Duchenne patients, patients with other degenerative muscle disease or muscle injury or trauma, and on muscle regeneration generally; the utility of regenerating muscle by modulating polarity; adoption of Satellos’ approach by the medical community; and Satellos’ technologies and drug development plans.

本新闻稿包括适用证券法所指的有关Satellos及其业务的前瞻性信息或前瞻性声明,其中可能包括但不限于有关Duchenne小分子治疗对患者的预期益处的声明;我们的主要候选药物进入临床试验的进展;我们相信,评估骨骼肌的再生状态可能为预测未来临床试验的功能结果提供一种有意义的方法,即我们的主要候选药物的药效学特性和作用机制;我们的方法在其他退行性肌肉疾病或肌肉损伤或创伤中的潜力;通过施用小分子药物调节干细胞极性的一般益处;;通过调节极性来再生肌肉的效用;医学界采用Satellos的方法;以及Satellos的技术和药物开发计划。

All statements that are, or information which is, not historical facts, including without limitation, statements regarding future estimates, plans, programs, forecasts, projections, objectives, assumptions, expectations or beliefs of future performance, occurrences or developments, are “forward-looking information or statements.” Often but not always, forward-looking information or statements can be identified by the use of words such as “shall”, “intends”, “anticipate”, “believe”, “plan”, “expect”, “intend”, “estimate”, “anticipate”, “potential”, “prospective” , “assert” or any variations (including negative or plural variations) .

所有非历史事实的陈述或信息,包括但不限于关于未来业绩、事件或发展的未来估计、计划、计划、预测、预测、目标、假设、期望或信念的陈述,都是“前瞻性信息或陈述”。通常但不总是,前瞻性信息或陈述可以通过使用“应该”、“打算”、“预期”、“相信”、“计划”、“预期”、“打算”、“估计”、“预期”、“潜在”、“预期”、“断言”或任何变体(包括负面或复数变体)来识别。