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EDINBURGH, Scotland--(BUSINESS WIRE)--Lario Therapeutics (‘Lario Tx’, or ‘the Company’), a biopharmaceutical company developing first-in-class precision medicines that are targeting disease-modifying treatments for severe neurological disorders, has received the “CDLK5 Forum Award for Excellence - Company Making a Difference 2023 Pre-clinical” from the Loulou Foundation at the annual CDKL5 Forum.
苏格兰爱丁堡-(BUSINESS WIRE)-Lario Therapeutics('Lario Tx'或'the Company'),一家生物制药公司,开发一流的精密药物,针对严重神经系统疾病的疾病缓解治疗,在年度CDKL5论坛上,Loulou基金会收到了“CDLK5卓越论坛奖-公司在2023临床前有所作为”。
The award is in recognition of its development of a validated, precision medicine approach in genetic epilepsies. Lario Tx’s first-in-class, orally active, CNS-penetrant CaV2.3 ion channel inhibitors hold promise as novel anti-seizure therapeutics for multiple epilepsy subtypes, including CDKL5 Deficiency Disorder..
该奖项是对其在遗传性癫痫中开发经过验证的精确医学方法的认可。Lario Tx一流的口服活性CNS渗透性CaV2.3离子通道抑制剂有望成为多种癫痫亚型(包括CDKL5缺乏症)的新型抗癫痫药。。
The CDKL5 Forum, established by the Loulou Foundation, is the largest annual conference on CDKL5 Deficiency Disorder (CDD). CDD is the most prevalent form of genetic Developmental Epileptic Encephalopathy (DEE), affecting approximately 1:40K live births. Caused by loss-of-function mutations in the CDKL5 protein kinase expressed in the brain, the rare neurological disease leads to seizures, developmental delay and severe intellectual disability in most patients.
由Loulou基金会建立的CDKL5论坛是关于CDKL5缺乏症(CDD)的最大年会。CDD是遗传性发育性癫痫性脑病(DEE)的最普遍形式,影响约1:40K活产。由大脑中表达的CDKL5蛋白激酶的功能丧失突变引起,罕见的神经系统疾病导致大多数患者癫痫发作,发育迟缓和严重智力障碍。
The leading hypothesis is that CDKL5 regulates the function of the CaV2.3 channel, which can explain the devastating seizures characteristic of the disorder. CaV2.3 is now being investigated as a key target for CDD and CaV2.3 inhibitors hold great promise as a precision medicine approach..
主要假设是CDKL5调节CaV2.3通道的功能,这可以解释该疾病的破坏性癫痫发作特征。CaV2.3目前正在作为CDD的关键目标进行研究,CaV2.3抑制剂作为精准医学方法具有很大的前景。。
Henning Steinhagen, Ph.D., Co-Founder and CEO of Lario Therapeutics, commented: “We are honored and thank the Loulou Foundation for their recognition of our approach which we hope will have a positive impact on patients affected by CDKL5 Deficiency Disorder. Our target, CaV2.3, represents one of the few newly validated precision medicine approaches in neurology and has a significant potential in severe epileptic conditions and other neurological disorders including Parkinson’s disease.
Henning Steinhagen博士,Lario Therapeutics的联合创始人兼首席执行官,评论:“我们很荣幸并感谢Loulou基金会对我们的方法的认可,我们希望这种方法将对受CDKL5缺乏症影响的患者产生积极影响。我们的目标CaV2.3代表了神经系统中为数不多的经过新验证的精密医学方法之一,并且在严重的癫痫病和其他神经系统疾病(包括Parkins)中具有显着的潜力对的病。
We are planning to progress our therapeutic programs swiftly towards IND and beyond supported by our partners and investors.”.
我们计划在我们的合作伙伴和投资者的支持下,迅速推进我们的治疗计划。
Dan Lavery, Ph.D., CSO of the Loulou Foundation, stated: “The CDKL5 Forum ‘Company Making a Difference Award (Pre-clinical)’ recognises the industry partner who has demonstrated the greatest commitment to pre-clinical therapeutic development for CDD over the past year. At the CDKL5 Forum 2023, the Loulou Foundation is pleased to recognise the team at Lario Therapeutics, a dynamic group of experienced scientists who are developing a novel therapeutic for our patients living with CDD.
Dan Lavery博士,Loulou基金会CSO,表示:“CDKL5论坛”公司“差异奖”(临床前)'认可过去一年对CDD临床前治疗开发表现出最大承诺的行业合作伙伴。在CDKL5 2023论坛上,Loulou基金会很高兴认识Lario Therapeutics的团队,Lario Therapeutics是一个充满活力的有经验的科学家团队,他们正在为我们的CDD患者开发一种新的治疗方法。
The Lario team has shown amazing dedication and engagement with our community, and we are excited to have them join our CDKL5 community as they advance their CaV2.3 pre-clinical development program.” Ana Mingorance, Ph.D., CDO of the Loulou Foundation, added: 'One of the major discoveries about CDD last year was that CaV2.3 is a key target for CDKL5, which is deregulated in the absence of CDKL5.
Lario团队与我们的社区表现出了惊人的奉献精神和参与度,当他们推进CaV2.3临床前开发计划时,他们加入CDKL5社区非常兴奋。”Loulou基金会CDO的Ana Mingorance博士补充说:“去年CDD的主要发现之一是CaV2.3是CDKL5的关键靶标,而CDKL5在没有CDKL5的情况下被放松。
This means that CaV2.3 inhibitors hold promise as a precision medicine approach to treat CDD.”.
这意味着CaV2.3抑制剂有望成为治疗CDD的精准医学方法。
Sinclair Dunlop, Investor Director, Lario Therapeutics, said: “This is an exciting time for Lario Therapeutics, and Epidarex Capital is pleased to have supported Henning and the team to this significant point. The Company’s approach to developing novel disease-modifying precision medicines for the management of severe neurological disorders is potentially transformative, places Lario in a unique position to address unmet clinical needs, and represents a significant market opportunity.”.
Sinclair Dunlop,Lario Therapeutics投资者总监,说:“对于Lario Therapeutics来说,这是一个激动人心的时刻,Epidarex Capital很高兴能够支持Henning和团队这一重要点。该公司开发用于治疗严重神经系统疾病的新型疾病缓解精确药物的方法具有潜在的变革性,Lario处于解决未满足的临床需求的独特位置,并代表了一个重要的市场机会。”。
Lario Tx was founded in 2021 as a spin-out from Epidarex Exeed (epidarex-exeed.com), the therapeutic discovery engine of Epidarex Capital, with seed investment from Epidarex Capital and Axxam which transferred its participation into Golgi Neurosciences. The Company is rapidly advancing, led by Dr Henning Steinhagen, Co-founder and CEO, together with a highly motivated and experienced team..
Lario Tx成立于2021年,是Epidarex Capital的治疗发现引擎Epidarex Exeed(Epidarex Exeed.com)的分拆产品,Epidarex Capital和Axxam的种子投资将其参与转移到高尔基神经科学领域。由共同创始人兼首席执行官Henning Steinhagen博士领导,以及一支积极进取,经验丰富的团队,该公司正在迅速发展。。
For more information about Lario Therapeutics please visit lariotx.com.
有关Lario Therapeutics的更多信息,请访问lariotx.com。