Viruses are formidable adversaries, possessing an uncanny ability to mutate into stronger forms each time one strain was defeated, necessitating new interventions like booster shots. This ongoing struggle against viruses has led to the elusive quest for a <keyword id="21392555" type="General" weightage="20" keywordseo="universal-vaccine" source="keywords">universal vaccine</keyword> since the advent of <keyword id="21392556" type="General" weightage="20" keywordseo="antiviral-research" source="keywords">antiviral research</keyword>.<br /><br />However, there's hope on the horizon as scientists pioneer a novel <keyword id="19959412" type="General" weightage="100" keywordseo="vaccine" source="keywords">vaccine</keyword> platform with <keyword id="21392554" type="General" weightage="20" keywordseo="RNA" source="keywords">RNA</keyword> at its core. RNA, acting as the messenger between DNA and protein synthesis, plays a pivotal role. When the body is infected, it produces RNA molecules, known as RNAis, to combat the <keyword id="19994619" type="General" weightage="20" keywordseo="virus" source="keywords">virus</keyword>. Viruses counteract by producing proteins that thwart this <keyword id="20082133" type="General" weightage="20" keywordseo="immune-response" source="keywords">immune response</keyword>.<br /><br />The innovative technique aims to manipulate the virus's protein production within the vaccine. Unlike conventional vaccines that rely on the body's immune response, this approach activates <keyword id="21392557" type="General" weightage="20" keywordseo="RNAi" source="keywords">RNAi</keyword>, providing a fresh strategy for viral defence. By weakening the virus initially, its ability to obstruct RNAi is impaired, rendering it vulnerable to the host's immune response. This weakened virus can then be used to bolster the RNAi immune system through vaccination.<br /><br />Moreover, this method addresses the challenge of viral mutation by targeting thousands of sections of the viral genome, ensuring efficacy against future variants. It also offers potential benefits for vulnerable groups such as infants and those with compromised immune systems, circumventing the limitations of traditional vaccines.<br /><br />Early trials on mice, including those lacking B and T cells, have shown promising results, demonstrating the vaccine's effectiveness and potential durability. Future plans include adapting this technology to combat influenza, potentially through a nasal spray vaccine, providing an alternative to injections.<br /><br />While obstacles such as extensive human trials and regulatory approvals remain, the prospect of universal protection against a range of viruses is within reach. This breakthrough could revolutionise our approach to combating infectious diseases, offering hope for a future with fewer health threats.<br /><br />The findings of this research have been published in <em>Proceedings of the National Academy of Sciences</em> and can be accessed <a href="https://doi.org/10.1073/pnas.2321170121" data-type="tilCustomLink">here</a>.<br />

Pioneering RNA vaccines could tackle all future virus mutations, eliminate need for booster doses

<p><div class="KonaFilter image-container display-table float_right"><div><div class="image on-image"><img src="https://static-ssl.businessinsider.com/image/57f6840c9bd9781c008b5405-1111/pre-mrna-1ysv-tubes.png" alt="RNA" data-mce-source="Vossman on Wikipedia" data-mce-caption="Computer generated image of what a single strand of RNA looks like." data-link="http://en.wikipedia.org/wiki/RNA#/media/File:Pre-mRNA-1ysv-tubes.png" /><p class="source"><span><a href="http://en.wikipedia.org/wiki/RNA#/media/File:Pre-mRNA-1ysv-tubes.png">Vossman on Wikipedia</a></span></p><p class="caption">Computer generated image of what a single strand of RNA looks like.</p></div><p></p></div></div>Biotech company Alnylam Pharmaceuticals is down almost 50% Thursday after stopping a late-stage clinical trial.</p><p>The drug, revusiran, was being developed to treat a condition called hereditary ATTR amyloidosis with cardiomyopathy. It's a rare disease in which amyloid proteins build up in the body, in <a href="http://www.mayoclinic.org/diseases-conditions/amyloidosis/basics/definition/con-20024354">particular the heart</a>.</p><p>Alnylam discontinued the trial after 18 patients died who were part of the trial.</p><p>"Patient safety comes first. We have stopped all dosing and are actively monitoring patients across revusiran studies to ensure their safety," Alnylam CEO John Maraganore said in a release issued Wednesday evening, adding that the company would be looking into what caused these deaths.</p><p>Revusiran is a type of treatment called an RNA interference (RNAi). These types of drugs aim to go in and silence the gene that's making a certain dysfunctional gene. This isn't the <a href="https://www.statnews.com/2016/09/29/nobel-prize-rnai-biotech/">first time RNAi has faltered</a>: it originally won the Nobel Prize back in 2006, and it's taken longer than expected to get it to work in humans.</p><p>However, Alnylam said in the release, this shouldn't affect patisiran, another RNAi drug the company has in late-stage clinical trials for a hereditary ATTR amyloidosis with polyneuropathy, a condition that's fairly similar, but affects the nervous system more.</p><p>Even so, the news hit the company hard. Its market cap dropped from around $6 billion to around $3.27 billion.</p><p><div class="KonaFilter image-container display-table"><div><div class="image on-image"><img src="https://static-ssl.businessinsider.com/image/57f67a569bd97820008b547b-800/alnylam stock chart.jpg" alt="Alnylam stock chart" data-mce-source="Google Finance" /><p class="source"><span>Google Finance</span></p></div><p></p></div></div></p>    <div class="margin-top">        <h3>NOW WATCH: <a href="http://www.businessinsider.com/exercise-and-diet-regime-to-get-six-pack-abs-2016-9">An exercise scientist reveals how to get six-pack abs</a></h3>        <div>            <div>    <script src="http://player.ooyala.com/v3/6e12e8b3387a44daacfb73afba25a76e"></script>    <div class="ooyala-video-player" data-video-autoplay="false" data-video-id="VpazNiNDE63KBq5y6Gwk_Op1wXaaHQeK" data-player-variable="ooyalapopvideo" id="ooyalaplayer_popvideo" style="width:1280px;height:720px"></div>    <script>    if (typeof RunFunctions === 'undefined') { var RunFunctions = []; }     RunFunctions.push(function()    {         var ooyalapopvideo = OO.Player.create('ooyalaplayer_popvideo', 'VpazNiNDE63KBq5y6Gwk_Op1wXaaHQeK',{ autoplay: 'false' });     });    </script>    <noscript><div>Please enable Javascript to watch this video</div></noscript></div>        </div>        <script>(function() { function createPlayer() { OO.ready(function() { RunFunctions.forEach(function(ooyalaFunction) { ooyalaFunction(); }); RunFunctions = [];}); } createPlayer(); }()); </script>    </div>

A biotech company lost $3 billion in value overnight after one of its drugs failed a key trial

<p><div class="KonaFilter image-container display-table"><div><div class="image"><img src="https://static-ssl.businessinsider.com/image/5ad493e7146e711f008b4bbb-1000/shutterstock453339415.jpg" border="0" alt="surgeon surgeons operating medical operation doctors" data-mce-source="lenetstan/Shutterstock" /></div></div></div></p><ul><li><strong>The initial-public-offering market suffered through a slow start to the year due to the partial government shutdown and the brutal fourth-quarter for financial markets, but public debuts are ratcheting back up.</strong></li><li><strong>While a host of high-profile unicorns and decacorns like Lyft, Uber, Airbnb, and Slack are all set to debut on the public market this year, healthcare has so far dominated the US IPO slate.</strong></li><li><strong>It's still early to compare the 2019 performance to prior years, but healthcare is the most active sector to debut on US exchanges this year, according to Renaissance Capital.</strong></li><li><strong>The group's showing in the first-quarter suggests a strong year ahead for biotech and healthcare</strong>,<strong> some analysts say. </strong></li></ul><hr /><p>Investors in private and public companies know there's a <a href="https://www.businessinsider.com/tech-startups-going-public-2019-rumored-ipos-2019-2">massive slate</a> of highly anticipated initial public offerings coming this year.</p><p>High-profile technology and transportation unicorns and decacorns like <a href="https://www.businessinsider.com/inside-lyft-ipo-roadshow-stop-in-nyc-2019-3">Lyft</a>, <a href="https://www.businessinsider.com/lyft-uber-compared-ahead-of-ipos-2019-3">Uber</a>, <a href="https://www.businessinsider.com/slack-confidentially-filed-ipo-2019-2">Slack</a>, and <a href="https://www.businessinsider.com/pinterest-ipo-2019-2">Pinterest</a> are all set to debut on the public markets in the coming months after a <a href="https://www.businessinsider.com/2019-was-supposed-to-be-a-banner-year-for-ipos-but-now-its-turning-into-a-shitshow-2019-1">sleepy first-quarter</a>. A host of others, like <a href="https://www.businessinsider.com/exclusive-airbnb-may-defer-its-ipo-plan-2019-3">Airbnb</a> and <a href="https://markets.businessinsider.com/news/stocks/peloton-has-reportedly-picked-banks-for-an-ipo-which-could-value-it-at-more-than-8-billion-2019-2-1027977907">Peleton</a>, have also reportedly explored going public.</p><p>And even after a slow start start to IPOs this year - due in part to the <a href="https://www.businessinsider.com/government-shutdown-bill-reopen-congress-senate-federal-courts-2019-1">partial government shutdown</a> and one of the worst fourth-quarters on record - an overshadowed corner of the market is enjoying a strong run: healthcare.</p><p>The sector is the most active to debut in the US public market so far in 2019, followed by financials, according to data from Renaissance Capital. Healthcare was also the most active over the past year.</p><p>Investors' excitement around newly public biotechnology companies suggests a strong year ahead after outperforming the broader US IPO market in 2018, said Oppenheimer analysts Jay Olson and Silvan Türkcan.</p><p>While it's still too early to compare, initial IPO returns in the biotech space are positive so far this year. They've averaged 2% in about one month compared to 2018 IPOs returning 4% for the whole year, according to their analysis.</p><p>So far this year, 11 biotech IPOs have raised a total of $1.2 billion. Notably, those IPOs in 2019 have been predominantly in the cancer immunotherapy space, according to Oppenheimer's analysis. The largest so far is <a href="https://markets.businessinsider.com/stocks/goss-stock">Gossamer Bio</a>, which raised $317 million.</p><p>"In discussions with investors, we have received favorable feedback and continued interest in 2019 Biotech IPOs," they wrote.</p><p><em><strong>Read more: </strong></em><em><a href="https://www.businessinsider.com/top-biotech-ipos-of-2018-2018-9">The market for biotech IPOs is red hot - here are the top 10 of 2018</a></em></p><p>The relatively high number of healthcare companies coming onto the market isn't just confined to the US. Globally, healthcare and technology were the most active sectors in the first-quarter, according to Ernst &amp; Young data.</p><p>Between January and March, there were 45 IPOs in the technology space - the most of any sector - followed by the 32 in the healthcare sector.</p><p>So where is all this excitement coming from, and is it a sign of a group that's overheating?</p><p>Put simply, and while trying not to sound "cliché," Stifel biotechnology analyst Paul <span>Matteis said there's a widespread perception that science around drug development is improving and that investors want to get in on advances in different areas like gene therapy, neuroscience, and oncology.</span></p><p><span>"It's still early days for leveraging some of the newer tools, like gene therapy and <a href="https://www.businessinsider.com/r-alnylams-gene-silencing-drug-wins-fda-approval-2018-8">RNAi</a>, that have emerged as vehicles to leverage science that's improving across an array of disease areas," Matteis told Markets Insider on Thursday, adding that companies feel like they have better access to capital.</span><span></span></p><p><span>"There's been this expansion of tools to tackle and target specific genes," he said. </span></p><p><span>Still, Matteis doesn't think investor sentiment is near the uber-bullish heights of 2015. Between 2014 and 2015, investors saw a relatively high 50 to 60 biotech IPOs per year, according to Oppenheimer's analysis.</span><span></span></p><p><span>The <a href="https://markets.businessinsider.com/index/nasdaq_biotechnology">NASDAQ Biotechnology Index</a> is up just over 13% so far this year, barely outperforming the <a href="https://markets.businessinsider.com/index/s&amp;p_500">S&amp;P 500</a>'s 12.8% gain. Meanwhile, the <a href="https://markets.businessinsider.com/etfs/health-care-select-sector-spdr-fund">Health Care Select Sector SPDR Fund</a> has gained 6% this year.</span></p><p><em><strong>Now read more markets coverage from Markets Insider and Business Insider: </strong></em></p><ul><li><em><a href="https://markets.businessinsider.com/news/stocks/levi-strauss-and-co-initial-public-offering-starts-trading-2019-3-1028049207">Levi's spikes 31% as it returns to the public market</a></em></li><li><a href="https://www.businessinsider.com/next-recession-cause-overlooked-signal-could-send-warning-months-in-advance-2019-3"><em>A chief market strategist at a $1.4 trillion investing giant reveals the hidden red flag that can signal a recession months in advance</em></a></li><li><a href="https://www.businessinsider.com/inside-lyft-ipo-roadshow-stop-in-nyc-2019-3"><em>Inside the Lyft roadshow in NYC where investors packed the penthouse of a $1,000 a night hotel</em></a></li></ul><p class="tagline"><hr><a href="http://www.businessinsider.com/intelligence/ai-in-us-healthcare?IR=T&amp;utm_source=businessinsider&amp;utm_medium=ctgr&amp;utm_term=ctgr_leadgen&amp;utm_content=dhealth_ctgr_leadgen&amp;utm_campaign=dhealth_ctgr_leadgen_ai_in_us_healthcare"><img src="https://static.businessinsider.com/image/5b1988cc1ae66235008b54c1" alt="Business Insider Intelligence Exclusive FREE Report: The 5 Ways AI Will Change U.S. Healthcare" border="0" style="display: block; width: 100% !important; max-width: 640px !important; margin: 0 auto !important" /></a><hr /></hr></p>

Lyft, Uber, and Slack are the IPOs getting all the attention this year - but a completely different corner of the market is heating up

<p><div class="KonaFilter image-container display-table float_right"><div><div class="image on-image"><img src="https://static-ssl.businessinsider.com/image/5412b86d6bb3f7b27b9d1186-1200-924/ebola-14.jpg" alt="ebola" border="0"><p class="source"><span><a href="https://www.flickr.com/photos/niaid/14674486019/">Flickr/NIAID</a></span></p><p class="caption">Colorized scanning electron micrograph of filamentous Ebola virus particles (green) attached and budding from a chronically infected VERO E6 cell (orange) (25,000x magnification). </p></img></div><p></p></div></div>Shares of Tekmira Pharmaceuticals were up more than 14% on Monday after the company announced that it reached a "regulatory framework" with the FDA for using its TKM-Ebola treatment to treat Ebola.</p>
<p>In a <a href="http://investor.tekmirapharm.com/releasedetail.cfm?ReleaseID=871918">press release</a>, Tekmira said:</p>
<p style="padding-left: 30px;"><span style="font-size: 15px; line-height: 1.5em;">Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, today announced that the FDA has authorized Tekmira to provide TKM-Ebola for treatment under expanded access protocols to subjects with confirmed or suspected Ebola virus infections.</span></p>
<p style="padding-left: 30px;">"Tekmira is reporting that an appropriate regulatory and clinical framework is now in place to allow the use of TKM-Ebola in patients. We have worked with the FDA and Health Canada to establish this framework and a treatment protocol allowing us to do what we can to help these patients," said Dr. Mark J. Murray Tekmira's President and CEO.</p>
<p style="padding-left: 30px;">"We have insisted on acting responsibly in the interest of patients and our stakeholders," added Dr. Murray. "Today we are reporting that, working closely with regulators in the United States and Canada, we have established a framework for TKM-Ebola use in multiple patients. In the US, the FDA has granted expanded access use of TKM-Ebola under our Investigational New Drug application (IND) and Health Canada has established a similar framework, both of which allow the use of our investigational therapeutic in more patients."</p>
<p>This summer, <a href="http://www.businessinsider.com/s?q=tekmira">shares of Tekmira were all over the place</a> on hopes that the company's Ebola treatment would get expanded approval from the FDA.</p>
<p>The latest update from the company, which came on August 13, said it was <a href="http://www.businessinsider.com/ebola-company-tekmira-news-2014-8">"evaluating" options</a> with regard to expanded use of its Ebola treatment.</p>
<p>This chart shows the rally in shares of Tekmira over the last three months, with the stock gaining more than 65% over that period. </p>
<p><div class="KonaFilter image-container display-table"><div><div class="image"><img src="https://static-ssl.businessinsider.com/image/542048a7eab8ea9e454b6b6b-1024-389/tkmr chart.png" alt="TKMR chart" border="0"><p class="source"><span>Google Finance</span></p></img></div><p></p></div></div></p>

You Searched For "RNAi" and got 4 results

Pioneering RNA vaccines could tackle all future virus mutations, eliminate need for booster doses

Ankush Banerjee

A biotech company lost $3 billion in value overnight after one of its drugs failed a key trial

Lydia Ramsey

Lyft, Uber, and Slack are the IPOs getting all the attention this year - but a completely different corner of the market is heating up

Rebecca Ungarino

Ebola Drug Firm Tekmira Is Surging Again

Myles Udland