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CAMBRIDGE, Mass., June 25, 2024 (GLOBE NEWSWIRE) -- WAVE Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced positive results from its Phase 1b/2a SELECT-HD clinical trial of WVE-003, which is being developed as a potential disease modifying therapeutic for Huntington’s disease (HD).

馬薩諸塞州劍橋，2024年6月25日（環(huán)球通訊社）——WAVE生命科學(xué)有限公司猖腕。（納斯達(dá)克：WVE）是一家臨床階段生物技術(shù)公司好啸，專注于釋放RNA藥物改變?nèi)祟惤】档膹V泛潛力咱筛，今天宣布了其WVE-003 1b/2a期SELECT-HD臨床試驗(yàn)的積極結(jié)果瓦统，該試驗(yàn)正在開發(fā)中作為亨廷頓舞蹈参饭摹（HD）的潛在疾病改善治療藥物暑苍。

WVE-003 is a first-in-class, allele-selective antisense oligonucleotide (ASO) designed to lower mutant huntingtin (mHTT) protein and preserve healthy, wild-type huntingtin (wtHTT) protein..

WVE-003 是一種首創(chuàng)的等位基因選擇性反義寡核苷酸 (ASO)标康，旨在降低突變型狩獵蛋白 (mHTT) 蛋白砍梁，并保留健康的野生型狩獵蛋白 (wtHTT) 蛋白。

In the multidose portion of the SELECT-HD study (n=23), participants received either every-eight-week (Q8W) intrathecal doses of 30 mg WVE-003 (n=16) or placebo (n=7), with 12 weeks of follow up (total study period of 28 weeks). Key results are as follows:

在SELECT-HD研究的多劑量部分（n=23）愤栽，參與者每八周（Q8W）鞘內(nèi)注射30 mg WVE-003（n=16）或安慰劑（n=7）嗤雪，隨訪12周（總研究期28周）。主要結(jié)果如下：

WVE-003 was generally safe and well-tolerated, with no Serious Adverse Events (SAEs) reported; ventricular volume was in line with natural history.

WVE-003通常安全且耐受性良好齿喧，未報(bào)告嚴(yán)重不良事件（SAE）妥析；心室容積符合自然史。

Significant mHTT protein lowering was observed throughout the 28-week assessment period.

在整個(gè)28周的評(píng)估期間轻欣，觀察到mHTT蛋白顯著降低省咨。

At 24 weeks (8 weeks after last dose), mean mHTT lowering in cerebrospinal fluid (CSF) was 46% versus placebo (p=0.0007).

在24周（最后一次給藥后8周），與安慰劑相比诸迟，腦脊液（CSF）的平均mHTT降低46%（p=0.0007）茸炒。

At 28 weeks (12 weeks after last dose), mean mHTT lowering in CSF was 44% versus placebo (p=0.0002), which supports quarterly or less frequent dosing.

在第28周（最后一次給藥后12周）愕乎，與安慰劑相比阵苇，腦脊液中mHTT的平均降低率為44%（p=0.0002），這支持每季度或不太頻繁的給藥感论。

wtHTT protein was preserved throughout the 28-week assessment period, validating allele-selective silencing. Additionally, statistically significant increases were observed in wtHTT protein versus placebo.

绅项。此外，與安慰劑相比比肄，wtHTT蛋白觀察到統(tǒng)計(jì)學(xué)上顯著的增加快耿。

wtHTT protein supports the health and function of neurons and is crucial for CSF flow in the ventricles. mHTT also has a detrimental effect on wtHTT at the protein level, which further decreases wtHTT’s function. Only selective lowering of mHTT has potential to relieve its negative impact on wtHTT protein function..

wtHTT蛋白支持神經(jīng)元的健康和功能，對(duì)腦室中的腦脊液流動(dòng)至關(guān)重要芳绩。mHTT在蛋白質(zhì)水平上對(duì)wtHTT也有不利影響掀亥，這進(jìn)一步降低了wtHTT的功能。只有選擇性降低mHTT才能緩解其對(duì)wtHTT蛋白功能的負(fù)面影響妥色。搪花。

Most WVE-003-treated participants had neurofilament light protein (NfL) levels that were in the range of placebo or had NfL levels that increased and returned to the range of placebo.

大多數(shù)接受WVE-003治療的參與者的神經(jīng)絲輕蛋白（NfL）水平在安慰劑范圍內(nèi)，或者NfL水平升高并恢復(fù)到安慰劑范圍锉窑。

At 24 weeks (the last MRI assessment), mHTT reduction was correlated with slowing of caudate atrophy (R=-0.50; p=0.047). Caudate atrophy is an imaging biomarker that is predictive of clinical outcomes, including clinically meaningful worsening of Total Motor Score (TMS).

在第24周（最后一次MRI評(píng)估）岛牺，mHTT減少與尾狀萎縮減慢相關(guān)（R=-0.50；p=0.047）袁朗。尾狀萎縮是一種成像生物標(biāo)志物涮愧，可預(yù)測(cè)臨床結(jié)果，包括總運(yùn)動(dòng)評(píng)分（TMS）的臨床意義惡化跃渠。

While not powered for clinical outcomes, a slowing of decline was observed for TMS for WVE-003 versus placebo (4.25 mean difference at 24 weeks, p=not significant).

雖然沒有為臨床結(jié)果提供動(dòng)力订搏，但與安慰劑相比，WVE-003的TMS下降速度減慢（24周時(shí)平均差異為4.25沛四，p=不顯著）框辞。

“We are very proud to have demonstrated mHTT lowering of 46%, with preservation of wtHTT, and are encouraged to see these reductions in mHTT significantly correlating with a slowing in caudate atrophy after just 28 weeks. These results represent a significant achievement for Wave, for the oligonucleotide field, and most importantly, for the HD community,” said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Chief Development Officer at Wave Life Sciences.

Wave生命科學(xué)首席開發(fā)官Anne Marie Li Kwai Cheung說：“我們非常自豪地證明了mHTT降低了46%，保留了wtHTT，并且很高興看到mHTT的降低與僅28周后尾狀萎縮的減緩顯著相關(guān)涂嫡。這些結(jié)果代表了Wave秒牙，寡核苷酸領(lǐng)域，最重要的是HD社區(qū)的重大成就抬泛」ネ危”。

“Alongside the HD community, we have been working diligently to establish caudate volume as a biomarker for clinical development due to its association with clinical outcomes. We believe these strong data compel a case for accelerated approval for WVE-003, which we plan to discuss with regulators. We would like to express our immense gratitude to the HD community, the study participants, their families, and study site staff for their trust, support, and engagement that have helped us reach this important milestone.”.

“與HD社區(qū)一起纱昧，我們一直在努力建立尾狀核體積作為臨床開發(fā)的生物標(biāo)志物刨啸，因?yàn)樗c臨床結(jié)果有關(guān)。我們相信這些強(qiáng)大的數(shù)據(jù)迫使我們加速批準(zhǔn)WVE-003识脆，我們計(jì)劃與監(jiān)管機(jī)構(gòu)討論设联。我們要對(duì)HD社區(qū)，研究參與者灼捂，他們的家人和研究現(xiàn)場(chǎng)工作人員的信任离例，支持和參與表示衷心的感謝，他們的信任悉稠，支持和參與幫助我們實(shí)現(xiàn)了這一重要里程碑宫蛆。”的猛。

“Wild-type huntingtin plays such a critical role in the central nervous system, and it’s very exciting to finally have an opportunity to evaluate mHTT lowering in the context of allele-selectivity and to see positive signals emerging,” said Ralf Reilmann, MD, founder of the George-Huntington Institute, Muenster, Germany, as well as the Primary Investigator and member of the Advisory Committee for SELECT-HD.

“野生型亨廷頓蛋白在中樞神經(jīng)系統(tǒng)中起著至關(guān)重要的作用耀盗，最終有機(jī)會(huì)在等位基因選擇性的背景下評(píng)估m(xù)HTT的降低并看到積極的信號(hào)出現(xiàn)，這是非常令人興奮的卦尊，”德國明斯特喬治亨廷頓研究所（GeorgeHuntington Institute）創(chuàng)始人猎之，醫(yī)學(xué)博士拉爾夫·雷爾曼（RalfReilmann）以及SELECT-HD咨詢委員會(huì)的主要研究者和成員說。

“Additionally, these data have arrived at an opportune time when the HD community is coalescing around rapid, efficient registrational trial design utilizing sensitive clinical endpoints to detect early treatment effects, and Wave is well positioned to take advantage of this momentum with WVE-003. These data provide hope and a compelling path forward as the community continues to drive toward a long-awaited therapy to treat this devastating disease.”.

“此外乐跺，這些數(shù)據(jù)已經(jīng)到達(dá)了一個(gè)合適的時(shí)機(jī)鹃远，HD社區(qū)正在圍繞快速，有效的注冊(cè)試驗(yàn)設(shè)計(jì)進(jìn)行整合弓并，利用敏感的臨床終點(diǎn)來檢測(cè)早期治療效果笤簸，Wave很好地利用了WVE-003的這一勢(shì)頭。隨著社區(qū)繼續(xù)推動(dòng)人們期待已久的治療方法來治療這種毀滅性疾病远丸，這些數(shù)據(jù)提供了希望和令人信服的前進(jìn)道路沛愕。”喜毅。

HD is a debilitating and ultimately fatal autosomal dominant neurological disorder. The HD population is significant – in the United States alone, approximately 30,000 people have clinical symptoms of HD and more than 200,000 are at risk of inheriting HD. WVE-003 is expected to address approximately 40% of individuals with HD, and up to 80% of HD may be addressed in the future with other SNP-targeted candidates..

HD是一種使人衰弱并最終致命的常染色體顯性神經(jīng)系統(tǒng)疾病姑享。HD人口非常多-僅在美國，就有大約30000人有HD的臨床癥狀苇皂，超過200000人有遺傳HD的風(fēng)險(xiǎn)罪褒。WVE-003有望解決大約40%的HD患者耙肖，未來可能會(huì)與其他針對(duì)SNP的候選人一起解決多達(dá)80%的HD患者。婿着。

“With these results, we have delivered the first-ever clinical demonstration of allele-selective silencing in any disease target. This was only possible due to the specificity, potency and durability enabled through our PRISM platform and it is validating of more than 10 years of chemistry innovation pioneered at Wave, including PN chemistry and stereochemistry,” said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences.

Wave Life Sciences總裁兼首席執(zhí)行官授瘦、MBA醫(yī)學(xué)博士保羅·博爾諾（Paul Bolno）表示：“有了這些結(jié)果，我們首次在任何疾病靶標(biāo)中進(jìn)行了等位基因選擇性沉默的臨床證明竟宋。這只有通過我們的PRISM平臺(tái)才能實(shí)現(xiàn)特異性提完、效力和耐久性，并且它驗(yàn)證了Wave十多年來率先進(jìn)行的化學(xué)創(chuàng)新丘侠，包括PN化學(xué)和立體化學(xué)徒欣。”蜗字。

“The translation of genetic insights and preclinical data in the clinic is also highly encouraging and reinforces the broader value of our pipeline. We are looking forward to our Duchenne muscular dystrophy and Alpha-1 antitrypsin deficiency data this year, the continued advancement of our INHBE program for obesity, and new targets to be shared at R&D Day this Fall, which together will open up a substantial total addressable market for Wave.

“臨床上遺傳見解和臨床前數(shù)據(jù)的翻譯也非常令人鼓舞打肝，并增強(qiáng)了我們管道的更廣泛價(jià)值。我們期待著今年我們的杜興氏肌營養(yǎng)不良癥和α-1抗胰蛋白酶缺乏癥數(shù)據(jù)挪捕，我們針對(duì)肥胖的INHBE計(jì)劃的持續(xù)推進(jìn)粗梭，以及今年秋季研發(fā)日將共享的新目標(biāo)，這將共同為Wave開辟一個(gè)可觀的總目標(biāo)市場(chǎng)担神。

We are at a very exciting point in Wave’s history as we advance our mission to unlock the broad potential of RNA medicines.”.

隨著我們推進(jìn)我們的使命楼吃，以釋放RNA藥物的廣泛潛力始花，我們正處于Wave歷史上一個(gè)非常激動(dòng)人心的時(shí)刻入驮。”茴辈。

Cash Runway

現(xiàn)金跑道

Wave continues to expect that its current cash and cash equivalents will be sufficient to fund operations into the fourth quarter of 2025.

Wave繼續(xù)預(yù)計(jì)其目前的現(xiàn)金和現(xiàn)金等價(jià)物將足以為2025年第四季度的運(yùn)營提供資金抚送。

Investor Conference Call and Webcast

投資者電話會(huì)議和網(wǎng)絡(luò)廣播

Wave will host an investor conference call today at 8:30 a.m. ET to review the SELECT-HD clinical trial results. A webcast of the conference call can be accessed by visiting “Investor Events” on the investor relations section of the Wave Life Sciences website: https://ir.wavelifesciences.com/events-publications/events.

Wave將于美國東部時(shí)間今天上午8:30召開投資者電話會(huì)議，審查SELECT-HD臨床試驗(yàn)結(jié)果绎噩。通過訪問Wave Life Sciences網(wǎng)站投資者關(guān)系部分的“投資者活動(dòng)”帕卦，可以訪問電話會(huì)議的網(wǎng)絡(luò)廣播：https://ir.wavelifesciences.com/events-publications/events.

Analysts planning to participate during the Q&A portion of the live call can join the conference call at the audio-conferencing link available here. Once registered, participants will receive the dial-in information. Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website..

計(jì)劃參加現(xiàn)場(chǎng)電話問答部分的分析師可以通過此處提供的音頻會(huì)議鏈接參加電話會(huì)議。注冊(cè)后缨诱，參與者將收到撥入信息÷樱現(xiàn)場(chǎng)活動(dòng)結(jié)束后，Wave生命科學(xué)網(wǎng)站將提供網(wǎng)絡(luò)廣播的存檔版本纳倒。躁盗。

About SELECT-HD

關(guān)于SELECT-HD

The SELECT-HD trial (NCT05032196) was a global, multicenter, randomized, double-blind, placebo-controlled Phase 1b/2a clinical trial to assess the safety and tolerability of single- and multiple-ascending intrathecal doses of WVE-003 in people with a confirmed diagnosis of HD who are in the early stages of the disease and carry SNP3 in association with their cytosine-adenine-guanine (CAG) expansion.

SELECT-HD試驗(yàn)（NCT05032196）是一項(xiàng)全球性，多中心苟暗，隨機(jī)拗酌，雙盲，安慰劑對(duì)照的1b/2a期臨床試驗(yàn)韭寸，用于評(píng)估單次和多次鞘內(nèi)注射WVE-003的安全性和耐受性春哨。確診為HD的患者處于疾病的早期階段荆隘，并攜帶SNP3與胞嘧啶腺嘌呤鳥嘌呤（CAG）擴(kuò)增相關(guān)。

Additional objectives include assessing pharmacokinetics and exploratory pharmacodynamic and clinical endpoints..

其他目標(biāo)包括評(píng)估藥代動(dòng)力學(xué)和探索性藥效學(xué)和臨床終點(diǎn)赴背。椰拒。

About WVE-003

關(guān)于WVE-003

WVE-003 is a first-in-class, allele-selective antisense oligonucleotide that selectively lowers mutant huntingtin (mHTT) protein by targeting a single nucleotide polymorphism (SNP3) located on the mHTT messenger RNA that is not present on the wild-type huntingtin (wtHTT) mRNA. Wave’s approach to Huntington’s disease (HD) is guided by the recognition that, in addition to a gain of function of the mHTT protein, people with HD have lost one copy of the healthy wtHTT allele, leaving them with a smaller protective reservoir of wtHTT protein than unaffected individuals.

WVE-003是一種一流的等位基因選擇性反義寡核苷酸，它通過靶向位于野生型亨廷頓蛋白（wtHTT）mRNA上不存在的mHTT信使RNA上的單核苷酸多態(tài)性（SNP3）來選擇性降低突變亨廷頓蛋白（mHTT）蛋白凰荚。Wave對(duì)亨廷頓菜嗜（HD）的方法是通過認(rèn)識(shí)到，除了獲得mHTT蛋白的功能外浇揩，HD患者還失去了一個(gè)健康的wtHTT等位基因拷貝仪壮，使他們比未受影響的個(gè)體擁有更小的wtHTT蛋白保護(hù)庫。

wtHTT protein is critical for neuronal function, and suppression may have detrimental long-term consequences. For more information, please refer to Wave’s published manuscript of WVE-003 preclinical data here..

wtHTT蛋白對(duì)神經(jīng)元功能至關(guān)重要胳徽，抑制可能會(huì)產(chǎn)生有害的長期后果积锅。有關(guān)更多信息，請(qǐng)參閱Wave發(fā)布的WVE-003臨床前數(shù)據(jù)手稿养盗。缚陷。

About Huntington’s Disease

關(guān)于亨廷頓氏癥

Huntington’s disease (HD) is a debilitating and ultimately fatal autosomal dominant neurological disorder, which is passed down from generation to generation within affected families. The symptoms of HD have been compared to having Alzheimer’s disease, Amyotrophic lateral sclerosis, and Parkinson’s disease all at once.

亨廷頓舞蹈病（HD）是一種使人衰弱并最終致命的常染色體顯性神經(jīng)系統(tǒng)疾病必痢，在受影響的家庭中代代相傳拱矫。HD的癥狀被比作同時(shí)患有阿爾茨海默氏病、肌萎縮側(cè)索硬化癥和帕金森氏病涤朴。

HD is caused by an expanded cytosine-adenine-guanine (CAG) triplet repeat in the huntingtin (HTT) gene that results in production of mutant HTT (mHTT) protein. Accumulation of mHTT causes progressive loss of neurons in the brain, affecting thinking ability, emotions, and movement. Clinical symptom onset typically occurs during adulthood, between the ages of 30 and 50.

牌聋。mHTT的積累會(huì)導(dǎo)致大腦中神經(jīng)元的逐漸喪失，從而影響思維能力茁升，情緒和運(yùn)動(dòng)嫂前。臨床癥狀發(fā)作通常發(fā)生在成年期，年齡在30至50歲之間鹰泡。

It is characterized by cognitive decline, psychiatric illness, and chorea, and patients ultimately succumb to pneumonia, heart failure or other complications. There are no disease modifying treatments for HD..

萝渐。HD沒有改善疾病的治療方法。叛冠。

About Wave Life Sciences

關(guān)于波浪生命科學(xué)

Wave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave’s RNA medicines platform, PRISM?, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders.

Wave Life Sciences（納斯達(dá)克：WVE）是一家生物技術(shù)公司磨慷，專注于挖掘RNA藥物在改變?nèi)祟惤】捣矫娴膹V泛潛力。Wave的RNA藥物平臺(tái)PRISM?結(jié)合了多種模式把奢、化學(xué)創(chuàng)新和對(duì)人類遺傳學(xué)的深刻見解薇痛，實(shí)現(xiàn)了治療罕見和流行疾病的科學(xué)突破。

Its toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave’s diversified pipeline includes clinical programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity.

其RNA靶向模式工具包包括編輯弧岳，剪接凳忙，RNA干擾和反義沉默，為Wave提供了無與倫比的能力禽炬，用于設(shè)計(jì)和可持續(xù)地提供最佳解決疾病生物學(xué)問題的候選藥物涧卵。Wave的多元化渠道包括杜興氏肌營養(yǎng)不良癥勤家，α-1抗胰蛋白酶缺乏癥和亨廷頓舞蹈病的臨床計(jì)劃，以及肥胖癥的臨床前計(jì)劃柳恐。

英文鏈接：https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-announces-positive-results-phase-1b2a-select