RNA

NiceGuy0606

Your observation about the June 4th price action is right. If new shorts piled in at the $14 level, a major gap-up on Monday is going to put them under immediate pressure, even if the overall squeeze parameters look calm on paper.The competition from existing mAbs and RNA therapies is real, and market penetration will be a steady climb. However, the cost economics are what most shorts completely miss. HAE is one of the most expensive chronic conditions in the world. takzyhro/ Orladeyo cost insurers roughly 500 k per Patient every year. Over 10 years, a single well-controlled patient costs health insurance $5M+. Intellia is likely to cost less than this. But the true macro comed from Platform validation. Proving that the in-vivo delivery mechanism is safe and durable in a Phase 3 trial completely changes the risk profile for NTLA's entire pipeline (like ATTR).

Equivalent-Aside-404

I’ve seen that NTLA has 2.7 million shares to borrow. The short interest was 34% last reporting(Schwab). There was a bump in price 6/4/26 to over 14. I suspect at that time there have been new shorts with the price rise, in addition I suspect some shorts were churned with the pressure of positive news on FDA clearance of one of NTLA’s other trials and institutional investing. How the shorts play this with the current news I do not have the sophisticated knowledge to even make a guess. There are two other companies to compete with one using monoclonal antibodies and is in stage three the other is an RNA-targeted prophylactic therapy that is already commercially available(Gemini AI). I don’t have any biotech or pharmaceutical experience to know how competitive this will be for NTLA. It does appear that this is an effective and safe treatment. The disease HEA itself can be life threatening. The prevalence in the US is estimated to be up to 10,000 people those many of those may be unreported. So there is not a huge market, but it is great news because it validates NTLA as a serious player in the genetic engineering biotech field. I believe this will squeeze due to price momentum from the news, not necessarily due to great squeeze parameters, and am hoping for it to rise to at least 15 and maybe even 18 based on previous charts. I have been increasing my shares, now 700, with some calls. I’ll probably dump some shares above 15 and see where it consolidates before getting in again, or maybe not, because the company is expecting approval this year for this drug and the FDA has cleared their other drug trial to resume.

WorldFamousAstronaut

It’s a long way from being on par because of cost. If it were cheaper there would never be a reason to use short reads. So if you think PacBio will take market share it mostly comes down to the Revio 2 outperforming the next Illumina instrument on cost, which is possible but highly unlikely based on past improvement rates and the fact that Illumina can drop consumable costs until PacBio goes bankrupt if it has to. Comparing BioNano is entirely fair as an example of a company in the biotech instruments space that investors had high hopes in, with a proven and unique technology related to genomics, that essentially failed without getting bought up. It’s irrelevant to my point that it’s not DNA sequencing. It’s more likely that long reads won’t take market share at this point. Look at the revenue trends in the past 5 years. Also consider how sequencing is being used increasingly for single cell RNA-seq which is almost fully dominated by Illumina because long reads give almost no advantage there. I work in this field and I’m invested in PacBio and ONT so I’m not talking out of my ass. At the moment I think it’s hard to deny that the future is not bright for long reads, even if they stay around as a niche product for genome assembly.

Expensive-Wash5622

Go Go RNA 🤑🤑🤑🤑

illtakeoneplease

Co-Diagnostics, Inc. The Utah corporation, is a molecular diagnostics company that develops, manufactures and markets state-of-the-art diagnostics technologies. The Company's technologies are utilized for tests that are designed using the detection and/or analysis of nucleic acid molecules (DNA or RNA). Co-Diagnostics Develops Ebola Assay Strategy Following Recent Global Outbreak Alert

XunclericoX

Illumina was the shit at RNA sequencing. Not the market leader anymore

Nelsons93

it has definitely been a brutal ride. But you are factually wrong about it being the exact same info. Last year at ASGCT they presented data on Non Human Primates. The data dropping today is specifically focused on Type 2 Diabetes using high fat diet mouse models. It is a much deeper dive that includes new single cell RNA sequencing and electron microscopy showing exactly how the beta cells are maturing and increasing insulin granules. The study achieved complete rescue of hyperglycemia at four weeks.  It is definitely not the same press release. It is new proof of concept for the massive Type 2 market. The stock price is a different story, but the science is definitely advancing.

tendytinglings

This and also, we are not gonna hit AGI in our lifetime. There’s just no chance. Biology is incomprehensibly complex and while our analytical tools have come a long way, we still don’t understand shit. Hell, just two years ago, we found viroid RNA (obelisks) doing God knows what in both prokaryotic and eukaryotic life forms… AI will continue to be invested in without question. But with life science funding in a dire position in the US, I’d say we’re slowing down our progress towards biological AGI at present.

DoctorNezuko

Allbirds makes no sense to me. Tempus has always been an "AI" company. And by that I mean they develop pipelines for analyzying cancer biopsy samples collected from RNA and proteomic analytical sequencing. If anything, this is just an example of how the term AI is meaningless because all AI means is that the analysis is done using a weighted average of mathmatical forumulations, and we don't understand why the weights are what they are. At least in biological analytic techiques, we understand why we don't understand the weights, and that is because (1) our techniques are not perfect, (2) random impurities always exists that affect the results, and (3) we don't fully understand biology, and thus there will be factors we didn't know to consider. Does AI know what these factors are? No, but it psuedo accounts for it.

bigboyvapesinc

PMOs and RNA therapeutic treatments are the future of modern medicine. To be able to influence expression of diseased genes and even make the protein functional (Exondys 51 is one of their best examples) is going to change everything. We are still in the very early stages of the applications of this technology. Sarepta at least deserves a very close eye. I like this DD, thanks OP

DiarrheaFartLover

[I aw a guy at Walmart the other day with this shirt:](https://m.media-amazon.com/images/I/B1pppR4gVKL._CLa%7C2140%2C2000%7CA14Y9RNA5fL.png%7C0%2C0%2C2140%2C2000%2B0.0%2C0.0%2C2140.0%2C2000.0_AC_UY1000_.png) Boy, he sure showed them!

JernauG

I can give you a shot of RNA

kibo_blaster

Just putting this on the radar for people. Circio Holding ASA is a small Norwegian biotech working on circRNA technology (circVec). Unlike many early-stage biotech companies that focus on a single drug candidate, Circio is building a platform technology designed to improve gene expression in viral gene therapy vectors. Recent in-vivo data showed up to 50x enhanced gene expression in the eye and strong reproducibility across different tissues, which strengthens the case for circRNA-based approaches in gene therapy. The company has also mentioned an ongoing R&D collaboration with a large pharma company, and management has stated they expect multiple partnerships during 2026 as the platform matures. The coming months are quite event-heavy for the company, with participation in several industry conferences including: • LSX RNA Leaders Europe – March 18–19 • BioEquity – May 4–6 • ASGCT – May 11–15 • Circular & Self-Amplifying RNA Therapeutics Summit – May 26–28 • BIO International Convention – June 22–25 For companies developing platform technologies, these events are often where pharma partnerships and collaborations begin through meetings with potential partners and investors. There are also many rumours about Eli Lilly, buying Circio’s technology, which would make the stock price go very high. Not financial advice – just an interesting small biotech to keep an eye on :) Right now Circio’s stocks are priced 0.54 USD, if Circio’s plan works out and they get big partnerships this year, it could go as high as 10 USD if not more. Here are links to articles and interviews with the CEO if you want to do your own research: https://medwatch.no/nyheter/legemidler_biotek/article19055802.ece https://biostock.se/en/2026/01/circios-vd-om-emission-big-pharma-avtal-och-vagen-framat/ https://www.circio.com/en/event/bioeurope-spring-2026/

silverfutes

Damnit Kennedy, I don’t care about RNA testing, just tell us if it’s safe to eat bats again.

Lunar_Excursion

patents: INHBE RNA INTERFERENCE AGENTS: [https://patentscope.wipo.int/search/en/WO2025264948](https://patentscope.wipo.int/search/en/WO2025264948)

Lunar_Excursion

Biotech deals: Ventyx Biosciences - acquired for $1.2B - NLRP3 inhibitors Adverum Biotechnologies - acquired - vision gene therapy Rznomics - acquired for $1.3B - hearing loss RNA therapy

Astronaut100

Of course, China is playing 42D chess. No wonder they are the first to release new technology such as smartphones, AI, advanced semiconductors, RNA vaccines… oh wait.

Magnet_Pull

Just go to r/biotechnology and r/labrats to witness how we suffer. I feel these Issues are holding back Biotech atm: \- The AI bubble sucked up alot of VC which otherwise may have ended up in Biotech \- Religious zealotism hinders Biotech research in the US (see transgenic mice debate) and ecological zealotism hinders it in Europe (See GMO crops) \- It shows more and more that the promises of several processes could not be held, especiallyin a world which no longer prioriteses environmental protection However there are chances: \- EU is changing it's GMO legislature --> look at the BAYER stock \- Food industry is adapting precise fermentation for meat replacement products \- Health sector has a chance, esp. RNA vaccines etc (again in case the FDA does not go full MAHA-BS) \- Germany needs to transfer large sectors of its chemical industry, industrial biotechnology might be a chance I think investing in peptine industry can be a good idea, yet I have studied it and not even I want to invest the time to do my DD with all those companies business cases (nothing here is financial advice)

Brilliant_Voice1126

This is my whole operating principle right now. Everyone thinks the US primacy will last forever. Buffet said “never bet against America”. But this is based on the assumption we act as we had for almost 70 years as a source of stability in policy, treaties, foreign policy etc. Now that we’re as predictable as a toddler who missed nap time that assumption no longer holds. We are causing long lasting reputational damage and at the same time sabotaging our formerly world class research infrastructure, university system, our relationships with allies, including threatening the most important treaty for stability in the last century. This is unprecedented, and deeply stupid. When the idiot got elected last Jan I began to position out of USD. I have gone into gold (AAAU 25%) and a mixture of international ETFs and funds (SGOVX, IEUR, SCHF, ILF and EWY 60%) and a small mixture of US and European equities in fields that I largely have some expertise (10-15% and shrinking) - those have still done the worst. EWY went up 100% last year, ILF 50% , SCHF 40%) so this pivot has been great, just wish I’d done it all at once and taken some early losses. Overall 2025 was ~40% with my US equities dragging me down - even though they’ve largely gained only RNA, and CNC have popped enough to be competitive with my intl positions since I bought those at ATLs. Still thinking of shedding them for more non USD positions. Not to gloat but this is working and not gonna stop until the US rights its policy.

Yung_Ceejay

Thanks for the kind words! Just connecting dots with the PPD partnership and Thermo-Fisher providing the diagnostic tools for the Myelomatch framework. They carry a wide range of WT1 antibodies for immunohistochemistry as well as WT1 RNA sequencing panels, they also routinely sequence for ASXL1 mutation as part of their Myelomatch routine panel.

GarfieldLeZanya-

There is a huge amount of posters here who genuinely seem to think the only AI out there right now are LLM chat bots. Like that all of this gargantuan investment is just to build a machine to give you brownie recipes lol.  I have colleagues from grad school who are biostatisticians using AI for RNA sequence modeling and drug development for big pharma. Multimodal Foundation models (synthesizing audio + video + text + sensor data) are everywhere in robotics/production, medical imagining tech, and surveillance state shit. 3D perception and object tracking models are basically creating a new era for the military industrial complex and autonomous driving. At least from personal experience, banks and finance are shoving graph neural nets into KYC and AML systems as much as we can because it is so damn good at fraud detection. Hell, our entire online feed from TikTok to Reddit to Youtube and streaming suggestions are now all powered by deep learning recommender systems.  As you say, this tech is real. It is genuinely value generating. And it isnt going anywhere. It may certainly be way overvalued and due for a correction, but anyone who thinks we are gonna have some pop and be done with this is clueless.

GarfieldLeZanya-

I can not believe how many times this had to be repeated: LLM chatbots are not the only, let alone the primary, form of ML/AI behind this boom. I have no clue why so many people seem to sincerely think all of this investment are just models for asking chatGPT to make you grocery lists or whatever. I have a colleague from grad school, who is a Biostatistician, who is using a huge amount of compute for deep learning models to power RNA sequence modeling for a pharma company. You have multimodal foundation models, ML/AI models designed to parse image/video/audio/sensor data for things like robotics and manufacturing, security and surveillance tasks, medical imaging tech, etc. Those also feed into deep learning models for 3D perception, object tracking, and planning/prediction transformers for things like self-driving cars. Your entire social media algorithm, from Tiktok/Youtube feeds and ads optimization and what posts show up on what sites and what ads get surfaced, are largely being moved to transformer architecture and new deep learning models. I can tell you from personal experience, deep learning models are being integrated all over the finance world. Graph neural nets are being used everywhere for doing AML (anti-money laundering) and real-time fraud checks on financial transactions and to capture fraud rings. I agree with many that it is \*very\* overhyped right now and will have some deflation, eventually. However you're absolutely clueless if you sincerely think all of this is for some fucking brownie recipes and roleplay chats on OpenAI.

Jergle24

Crispr-cas9 is the hot thing in biotech right now. It’s used for gene editing and in the future gene editing will be able to solve basically any disease ever. CRBU is co-founded by the woman who co-invented the tech and also co-founded NTLA. She is working with CRBU to develope a crispr hybrid RNA-DNA (chRDNA). This chRDNA will make the existing crispr tech work better be more accurate and have less unintended gene edits which have unintended consequences. ChRDNA will make crispr safer and more effective, it is also a platform that can be patented and then CRBU can license it to all the other biotech crispr companies and make billions. Trial data for their two proof of concept therapies CB-010 and CB-011 will be released by end of year. Stock has moved from 0.66 to 2.60 in anticipation but mcap is still around 300m which means this has upside potential to easily 10-20x if the results are good. I’m betting on the competence of Jennifer Dounda who invented crispr, won a Nobel prize, and cofounded NTLA. Bad results kills the thesis but she has proven to be a good bet and the upside far outward the downside here.

Mother___Night

Pharma, full index. Old people gonna pay bigly for custom made DNA/RNA therapies--millions per treatment.

Kooky-You6511

They sell Nanopore based DNA/RNA sequencing devices

Better-Sea7632

This Reddit post is one of the most accurate and well-balanced warnings I’ve seen about small-cap pharma investing — and it aligns with my firsthand experience. That said, it’s also worth noting how fascinating this sector has been over the past six months. The **biotech indices have more than doubled the S&P 500’s last 6 months returns** and even outperformed tech ETFs at times — and the sector just had its **best week in years.** I'm tracking and benefiting from real momentum in areas like **gene therapy**, **RNA therapeutics**, and even some **psychedelic-related neuroscience** plays. Plus many exciting one-product in-development companies are there to be found - if you know the business (few people do). A lot of companies that were absolutely crushed last year and very undervalued at the start of this year have been quietly recovering from the floor — some example names like *Ionis, Arrowhead, Nektar, ATAI, UniQure* have all had solid runs recently, some of course due to new Clinical Trial results (this is NOT a recommendation to buy in now given the price rises these saw - just examples) Of course, there are always the “one great drug” hopefuls and takeover dreams floating around — that’s part of the ecosystem. But what makes biotech so different from “normal” investing is just how unpredictable it is. Even when you know the science, the market reaction can appear irrational if you do not know the bigger picture/dynamics that will not obvious to most retail investors. For me, the safest (and most enjoyable) approach is a **portfolio mindset** — not betting too much on single miracles, but following themes, a bit like Big Pharma does with its pipelines. It’s risky, but fascinating — part science, part psychology. PS I'm ex-Pharma/Biotech and I invest entirely in Biotech - as it's the sector I know, even still - I keep most my initial biotech penny stocks to 1-2% of my portfollio

Doodl3s

You can argue they're both forms of gene therapy... however, Injecting an RNA via viral vector is MRNAs technology. Its exactly how the COVID shot worked. Actually cutting DNA and splicing the gene itself is Very different with much worse risks that have occurred which is why CRSP and EDIT got knocked for years after killing people. You may be right that CRSP will go up, and i also do believe in their potential, but your DD about QURE is irrelevant and actually applies to MRNA QURE was a huge deal, I'm actually ecstatic. QURE worked by injection of RNA via viral vector. For that reason, MRNA is what was validated by QURE, NOT CRSP. - science degree in some bullshit nobody cares about

Doodl3s

CRSP has very little to do with QURE. QUREs technology it's RNA... you should buy MRNA, not CRSP

DanneEng90

There’s a growing rumor circulating that CAMP4 Therapeutics (CAMP) could announce three major developments as early as Monday. While nothing is confirmed yet, the buzz suggests: 1️⃣ Progress on their clinical pipeline – potential updates on early trial results. 2️⃣ A possible new partnership/collaboration with a larger biotech. 3️⃣ Strategic expansion news that could strengthen their position in the RNA therapeutics space. If even part of this turns out to be true, Monday could mark a turning point for CAMP. Volume has been heating up, sentiment is shifting bullish, and a breakout might be around the corner. Not financial advice, just sharing what I’m seeing – but definitely keeping my eyes on this one 👀📈

DanneEng90

There’s a growing rumor circulating that CAMP4 Therapeutics (CAMP) could announce three major developments as early as Monday. While nothing is confirmed yet, the buzz suggests: 1️⃣ Progress on their clinical pipeline – potential updates on early trial results. 2️⃣ A possible new partnership/collaboration with a larger biotech. 3️⃣ Strategic expansion news that could strengthen their position in the RNA therapeutics space. If even part of this turns out to be true, Monday could mark a turning point for CAMP. Volume has been heating up, sentiment is shifting bullish, and a breakout might be around the corner. Not financial advice, just sharing what I’m seeing – but definitely keeping my eyes on this one 👀📈

DanneEng90

There’s a growing rumor circulating that CAMP4 Therapeutics (CAMP) could announce three major developments as early as Monday. While nothing is confirmed yet, the buzz suggests: 1️⃣ Progress on their clinical pipeline – potential updates on early trial results. 2️⃣ A possible new partnership/collaboration with a larger biotech. 3️⃣ Strategic expansion news that could strengthen their position in the RNA therapeutics space. If even part of this turns out to be true, Monday could mark a turning point for CAMP. Volume has been heating up, sentiment is shifting bullish, and a breakout might be around the corner. Not financial advice, just sharing what I’m seeing – but definitely keeping my eyes on this one 👀📈

DanneEng90

There’s a growing rumor circulating that CAMP4 Therapeutics (CAMP) could announce three major developments as early as Monday. While nothing is confirmed yet, the buzz suggests: 1️⃣ Progress on their clinical pipeline – potential updates on early trial results. 2️⃣ A possible new partnership/collaboration with a larger biotech. 3️⃣ Strategic expansion news that could strengthen their position in the RNA therapeutics space. If even part of this turns out to be true, Monday could mark a turning point for CAMP. Volume has been heating up, sentiment is shifting bullish, and a breakout might be around the corner. Not financial advice, just sharing what I’m seeing – but definitely keeping my eyes on this one 👀📈

DanneEng90

There’s a growing rumor circulating that CAMP4 Therapeutics (CAMP) could announce three major developments as early as Monday. While nothing is confirmed yet, the buzz suggests: 1️⃣ Progress on their clinical pipeline – potential updates on early trial results. 2️⃣ A possible new partnership/collaboration with a larger biotech. 3️⃣ Strategic expansion news that could strengthen their position in the RNA therapeutics space. If even part of this turns out to be true, Monday could mark a turning point for CAMP. Volume has been heating up, sentiment is shifting bullish, and a breakout might be around the corner. Not financial advice, just sharing what I’m seeing – but definitely keeping my eyes on this one 👀📈

BioTrends_USA

CYDY. Key results & Findings Highlight “for the NCT03902522” posted today; 1) Most patients achieved plasma HIV-1 RNA levels less than 50 copies/mL after 24 weeks, indicating viral suppression in a treatment-experienced population. 2) Leronlimab was generally well tolerated, and no drug-related serious adverse events were reported. 3) Adverse events were predominantly mild, with no significant safety concerns apparent After 1 week, 64% of leronlimab recipients achieved ≥0.5 log₁₀ reduction in HIV-1 RNA (versus 23% for placebo; intent-to-treat analysis) After 24 weeks of extension with leronlimab plus optimized background therapy, most participants maintained plasma HIV-1 RNA levels below 50 copies/mL, suggesting robust long-term viral control Leronlimab was well-tolerated, with the majority of adverse events being mild and no drug-related serious adverse events Compared to current HIV drugs, CYDY’s leronlimab shows several notable efficacy and safety advantages, especially in heavily treatment-experienced patients with multi-drug resistant HIV. Key points of comparison include; Efficacy: In a phase 3 pivotal trial, leronlimab combined with standard antiretroviral therapy achieved viral suppression (<50 copies/mL) in about 81% of heavily treatment-experienced patients, significantly higher than the approximately 43-45% viral suppression rates seen with some recently approved drugs in this difficult-to-treat population. In a phase 2b/3 trial, leronlimab monotherapy maintained viral suppression for several years in some patients, a rare outcome for monotherapy in HIV treatment. Mechanism: Leronlimab is a monoclonal antibody blocking the CCR5 receptor, a co-receptor HIV uses to enter immune cells. This contrasts with most current antiretroviral drugs, which target viral enzymes. This unique mechanism allows leronlimab to remain effective even when resistance has developed against other drug classes. Resistance and Safety: Leronlimab retains full activity despite extensive resistance to the four main antiviral drug classes, and its efficacy is not compromised by exposure to maraviroc, another CCR5 antagonist. It also has a favorable safety profile with lower toxicity, less drug-drug interactions, and fewer serious adverse events reported. It is administered once weekly via subcutaneous injection compared to daily oral dosing for most ART drugs. Convenience and Long-Term Benefits: The less frequent dosing of leronlimab offers greater convenience and potentially better adherence. It also shows promise in protecting healthy cells from HIV entry and preventing transmission. In summary, leronlimab demonstrates superior efficacy in multi-drug resistant HIV cases, a novel entry-inhibition mechanism, improved safety, and more convenient dosing compared to many current HIV drugs, positioning it as a valuable option for patients with limited treatments. However, leronlimab is still investigational and undergoing regulatory review for broader use

AppTB

Non RNA vaccines

wholovesshortshorts

RNA-targeted biotech company at a 5 year low? What could go wrong!

Horsemeatburger

>ie see operation warp speed with the covid vaccine Not sure that's a great example of government efficiency. >the government selected and pushed these companies to innovate on its behalf using government dollars Well, the "Pfizer" vaccine, one of the early COVID RNA vaccines and amongst the most powerful, was actually developed by the German company BioNTech with development funded by the German tax payer and the EU. Pfizer then supported the trials and manufacturing. Moderna developed their vaccine using US government dollars, though, and I believe Johnon & Johnson did so as well. Still, more COVID vaccines were developed without US govt funding than with it.

ActiveCelebration843

Amazingly, I told everyone in here to buy RNA, but nobody listened and downvoted. Now we are rich. I tried to tell everyone!

StinkyNameRightHere

As far as I understand it, they don't need a big workforce at this point. They are paying Arrowhead to develop their next gen RNA drug. So they just need to be able to keep making those payments. Which is what the layoffs are supposed to help enable them to do. They have some cash still and some untapped credit and with shipments resuming so quickly on elevidys, they should be fine for now. Once the FDA puts somebody who is "right to try" in Vinay's spot and once Roche resumes shipments in the EU, I think we will see the stock increase modestly.

zeezooguy1

I told everyone RNA and nobody listened! Could’ve been rich.

ActiveCelebration843

Take a look at RNA, they are doing amazing things. Trust me

Prestigious-Ant-7210

Not sure if it qualifies but it’s market cap is still under $500 million so I’m going to say CADL. They have an awesome drug pipeline. If it has to truly be under $1, then GBIO. A company with a similar thesis was bought out for $2.1 billion a couple years ago and other big pharma companies almost certainly want to compete in the space. Highly specific delivery of RNA to T cells is going to be huge if they can show it works in humans

ActiveCelebration843

Take a look at Avidity Biosciences RNA, interesting things happening there.

Academic-Forever1492

They seem to be a well run company with an innovative product and a clear vision. The future for their products could very interesting.By providing RNA and DNA sequencing on a small low cost device, it could become a lot easier to detect exact variants of viruses or genetic predisposition to diseases. I think the virus angle could be interesting, GPs would have the ability to finally diagnose viruses in real time. They also are leveraging from AI to improve the speed, ease and accuracy of their product. It seems to be quite niche and in its infancy at the moment, but the potential market could be large.

StinkyNameRightHere

Just curious, what's your position? I'm full port in SRPT for better or worse. I think you are right on a few points. For one thing, AAV which they are using to deliver their gene therapy is known to be higher risk for patients whose muscles have already significantly wasted as the muscle wasting primes the immune system to overreact. The deaths occurred in this high-risk group of non-ambulatory patients whose muscle tone had already significantly decreased. There is a whole population of people who can still be safely helped by elevidys if they just limit the scope to only ambulatory patients. Of course there is also a chance they will get the immune suppression figured out better and expand back out into non-ambulatory patients as well, but that seems like it is probably a much longer road at this point. They also have an entirely separate project involving RNA rather than AAV that could eventually bear fruit. So I'm cautiously optimistic. But... Leadership, finances, and relationship with the FDA are all very questionable. The decision to initially refuse the FDA request to stop shipping elevidys and then immediately flip-flop accomplished nothing except significantly pissing off the FDA, which is basically a death sentence for a pharma company. The initial refusal is really hard to understand unless they are so strapped for cash that they did not think they could survive stopping shipments. I do think there is a real risk at this point that the FDA actively makes an example of them. Even if the FDA does not have a bone to pick with them, there is also a risk that the FDA will just move ponderously slowly and SRPT will simply run out of money before they can start making money again. I don't think they are in a good position to survive a slow war of attrition, even with the layoffs. Best case is that the FDA lets them start shipping to ambulatory patients again soon and that allows them to remain financially solvent long enough to pick up the pieces on some other things like non-ambulatory patients or future RNA treatments. No guarantee that will happen though. So basically I think you are right that people are overreacting to the deaths and acting like they came out of nowhere when in reality the risks were well known, and the solutions are probably within reach. However, that being true does not necessarily mean that SRPT can survive the sticky situation they are in. But forget all this, Cramer said today that SRPT should be prosecuted, so in that case, I'm all in.

Prestigious-Ant-7210

No idea why it’s going up. But if they show that their product works in humans and is safe, even if whatever RNA they put in it doesn’t work I think there is a high chance of them being acquired at the least. T cell specific delivery with lipid nanoparticles is a huge deal and they have strong data in non-human primates, but it may not pan out in humans. If it does though, it would be huge.

G0x209C

Because of the dollar per token not being viable with current computation methods. "Everyone is being hyped that this will solve world hunger, it will solve everything!!!" Yet, people are getting laid off, artists are forced to become factory workers even more instead of getting room to be creatives, it's using a significant portion of our drinking water, it's using a lot of electricity, it largely produces mediocrity unless you provide it with just the right context. (which means anyone working on systems and programs is becoming a somewhat meta-cognitive engineer, aka \[cognitive\] context engineer. Tell the lesser but faster simulated consciousness what to do) I see it being very viable in the fields of research and development. Finding new ways of discovering DNA and RNA sequences, unfolding proteins etc.. But otherwise? It's a potential helpful learning tool, or a tool that will make you stop using your brain. People are already losing competency in writing emails. Software developers already noticed a drop in the sharpness of their technical skills. We ought not to become reliant on these tools for thinking and producing. We ought to use these tools as they are best used for the human psyche: As a learning aid. As a perception expander. As a better translation tool than google translate. As a summarized note maker of meetings (its really good at that). And as the concept these "AI" LLMs came from: Machine Learning. The only thing we should stay aware of with Machine Learning is its limitations. It's only as good as the data you feed it. It can make mistakes, we need to check. If we only used it for the purposes it would be humanly best for: it is not a commercially viable operation.

No_Culture_5921

AMBR is quietly building differentiated technology: custom enzymes designed specifically for RNA correction. Not off-the-shelf CRISPR. That kind of proprietary control over the editing system could create real IP and value.

Odd_Watercress_7409

AMBR is very early-stage, so revenue is zero and risk is high. But their platform, if validated, could serve as a base for multiple therapeutic programs. Like an early Editas or Beam, but focused on RNA.

Odd_Watercress_7409

I’ve been watching the patent activity around RNA editing, and AMBR seems to be carving out a niche in programmable enzymes. Still a high-risk bet, but the IP side looks strong.

Odd_Watercress_7409

Amber Bio (AMBR) is developing a programmable RNA editing platform, aiming to correct disease-causing RNA transcripts without touching DNA. Still in early stages, but the science could be disruptive if it pans out.

Ready-Baseball-4300

If you’re comparing AMBR to something like Beam Therapeutics or Intellia, remember that AMBR isn’t doing CRISPR or base editing in the traditional sense. RNA editing is a different category, and that distinction matters for both risks and opportunities.

Ready-Baseball-4300

AMBR’s recent presentations highlighted their ability to target single-base RNA mutations, which could open the door to treatments for diseases like ALS and certain liver disorders. Still early, but the precision here is what makes it interesting.

Ready-Baseball-4300

The RNA editing space is heating up, and AMBR is one of the few public companies focused exclusively on it. Their approach could eventually allow in-body "repairs" of genetic conditions without permanent DNA edits.

Ready-Baseball-4300

AMBR is building a synthetic biology platform centered around RNA editing. Unlike traditional gene editing, this allows for reversible, precise changes. Still speculative, but the science has real long-term promise.

jefbe80

In SLXN NASDAQ:SLXN) ("Silexion" or the "Company"), a clinical-stage biotechnology company pioneering RNA interference (RNAi) therapies for KRAS-driven cancers, today announced compelling preclinical data demonstrating the efficacy of its next-generation RNAi therapeutic candidate, SIL204, against human pancreatic, colorectal and lung (NSCLC) cancer cell lines. These results significantly expand SIL204's therapeutic potential beyond pancreatic cancer, allowing it to potentially address major KRAS-driven cancers with substantial unmet medical needs. Following the Company's recent announcement of completion of preclinical studies, a comprehensive analysis of the data has revealed that SIL204 effectively inhibited the proliferation and metabolic activity of human cancer cell lines harboring KRAS G12D mutations across multiple cancer types, resulting in the following obvervations: The data reveals SIL204 successfully inhibited the proliferation and metabolic activity of human cancer cell lines harboring a specific KRAS mutation (G12D): GP2D (colorectal), A427 (lung) and Panc-1 (pancreatic), in a statistically significant manner.

Formal-Plate-8242

maybe this IDEXX offers testing for bird flu, also known as avian influenza. IDEXX provides various tests for detecting avian influenza, including antibody tests and real-time PCR assays. IDEXX's Avian Influenza Testing Options: * Avian Influenza (AI) Ab Test: This test is an enzyme-linked immunosorbent assay (ELISA) that detects antibodies to avian influenza viruses in chicken serum. * AI MultiS-Screen Ab Test: This test is an ELISA designed to detect avian influenza antibodies in serum from multiple avian species, including chickens, turkeys, ducks, and geese. * RealPCR Influenza RNA Mix: This test identifies Influenza A RNA using the IDEXX RealPCR platform. * Avian Pneumovirus (APV) Ab Test: This test detects APV-specific antibodies in serum samples. * Influenza A Ab Test: This blocking ELISA detects serum antibodies to any influenza A subtype.

ThomasMarcZC

Its price hit $2+ twice in March. Although I don't know if it'll hit $2 again today but I am optimistic. Here's the news summary (by ChatGPT): The sentiment of this news is **strongly positive**, driven by promising clinical data, high clinical benefit rates, and the absence of significant safety concerns in early trial phases for REYOBIQ™. The tone is optimistic, with credible scientific and regulatory backing (NCI, CPRIT) * New **Phase 1 trial data** for REYOBIQ in Leptomeningeal Metastases (LM) shows **dose-dependent efficacy** and **high clinical benefit**: * Immune response insights from RNA sequencing suggest **apoptosis and immune activation** by Day 28. **Implications:** The data reinforces REYOBIQ’s potential as a **safe, high-dose radiotherapeutic** for a **highly fatal and underserved condition (LM)**. The lack of FDA-approved therapies for LM and the grim prognosis for these patients highlight the **market need** and **clinical urgency**. The tolerability of higher doses without significant toxicity is particularly promising.

Smurf_Crime_Scene

The RNA didn't do it on purpose!

RIP_Michael_Hotdogs

Lowest intelligence being to cause market crash 2020: literal strands of RNA in a protein coat, dubiously alive 2025: The elected representatives of a county of 350 million

workonlyreddit

That was the Fed rescuing the market and we were saved by the success of RNA covid vaccines, which is a miracle on its own.

Muted-Watercress9599

Liking LON:ONT - Oxford Nanopore - Oxford Nanopore Technologies (ONT) is a leading innovator in real-time, portable, and scalable DNA/RNA sequencing. It is the only producer of portable, handheld gene-sequencing devices worldwide. Its technology can read more of the genome, faster and more accurately than any competitor.  My two cents on the bull & Bear case Bullish about Expanding Clinical & Biopharma AdoptionONT is rapidly growing in clinical genomics, with large-scale contracts supporting long-term revenue expansion. Technology Leadership Oxford Nanopore is the only company globally producing handheld, portable sequencing devices for real-time, in-field DNA/RNA analysis, —that sets it apart in next-generation genomic analysis. Broad Global Applicability ONT devices are deployed across multiple sectors spanning healthcare, agriculture, environmental science, and academic research, enhancing ONT’s market resilience and expanding its TAM. Bearish Competition from Established Players Illumina and PacBio remain formidable competitors, leveraging existing market share and technology advancements. Profitability Concerns ONT has yet to achieve consistent profitability, with high R&D and expansion costs potentially delaying EBITDA breakeven. Execution Risk in Clinical Expansion The transition from research to clinical applications presents regulatory and adoption challenges that may slow market penetration. Couple interesting catalysts in this story too Cutting-Edge Technology & Innovation Pipeline - ONT is advancing into protein sequencing, potentially unlocking a new multi-billion-dollar market in the coming years. Leadership in Multiomic Sequencing and the Dark Genome - Oxford Nanopore is already a leader in multiomic sequencing, with its ability to read native DNA and RNA in their natural forms providing richer insights into complex biological systems. The exploration of the "dark genome," which contains 25% of human disease-related variants, will further solidify Oxford Nanopore’s leadership in precision medicine. As the company continues to innovate in genomics, proteomics, and metabolomics, it will expand its applications in clinical diagnostics and therapeutic development, driving long-term growth.

justUseAnSvm

MRNA. Incredibly strong drug pipeline. Not everything in there will work out, but we know RNA vaccines work, and the technology is superior to other approaches. The COVID hangover is almost over, the price has been knocked down, and the promise is still there.

birdflustocks

H5N1 has been known for almost 30 years, there was a lot of concern almost 20 years ago, and in the last few years there was the Covid pandemic and also increased concern regarding H5N1. But none of this is related, the H5N1 panzootic is unprecedented and could cause a pandemic at any time. The virus doesn't care about human issues. Our past perceptions of the virus or the Covid pandemic have nothing to do with the H5N1 pandemic risk. "An international survey, to be published next weekend, will reveal that 57% of senior disease experts now think that a strain of flu virus will be the cause of the next global outbreak of deadly infectious illness." [Source: Next pandemic likely to be caused by flu virus, scientists warn](https://www.theguardian.com/world/2024/apr/20/next-pandemic-likely-to-be-caused-by-flu-virus-scientists-warn) "In recent years, an H5N1 problem that was once mainly confined to Asia and poultry has now spread globally, and into new species of mammals, endangering wildlife, agricultural production, and human health. The problem began in 2020, when a new genotype of H5N1 viruses belonging to clade 2.3.4.4b emerged that spread rapidly in wild birds from Europe to Africa, North America, South America, and the Antarctic. At first, H5N1’s arrival in North America seemed manageable. Back in 2014, when an earlier H5 virus was introduced to North America from Asia, US poultry farmers successfully eliminated the virus through intensive monitoring and culling of 50 million chickens and turkeys, ending the largest foreign animal disease outbreak in US history. This time, despite culling \~90 million US domestic birds since 2022, poultry outbreaks continue to be reseeded from wild birds. Wild birds also introduced H5N1 to dairy cattle and marine mammals. Images of seal carcasses decaying on Argentine beaches and yellow, curdled milk on H5N1-affected dairy farms show how the 2.3.4.4b H5N1 panzootic is different and previous control strategies are not working." [Source: The global H5N1 influenza panzootic in mammals](https://www.nature.com/articles/s41586-024-08054-z_reference.pdf) "Influenza viruses do not follow our human rules. Instead, they follow the biology defined by a negative-sense, single-stranded, segmented RNA genome and interactions with their environment and hosts. They will not wait for us to work through our politics, our man-made rules, or even our science to exert their damage. They will merely follow their biology. This is a point we humans often fail to recognize, one that allows a virus, such as H5 influenza, to wreak havoc, leaving us in the dust. This is where we went wrong and where we need to do better. (…) Influenza is not going to wait for politics or publications. We must do better now.” Kay Russo Veterinarian [Source](https://www.jdscommun.org/action/showPdf?pii=S2666-9102%2824%2900151-0)

Series_Grand

The Stock that would profit the most is Novavax $NVAX Why? Because RFK doesn‘t like RNA vaccines and Novavax ran the most during the covid pandemic. Additionally they have partnered with Sanofi so they have an experienced distributor. https://preview.redd.it/f02f6y31vuke1.jpeg?width=1179&format=pjpg&auto=webp&s=9d03c02a5dbbd3ff3fcc929f0b9b565fc3414884

mewalkyne

They're an RNA company and Republicans just announced they want to ban mRNA specifically.

pancakesvt

i need to resist selling my WOLFSPEED but its so friggin hard. i sold my RNA when it looked like this and i need to learn to"push and believe"

Firm_Swing

I have a list of companies I wish I had more money to invest in. What is your area of expertise? Always good to focus on industries you’re familiar with. Personally, the bulk of my account is in index funds, but I have positions in multiple sectors I find exciting. Nuclear energy and uranium mining, domestic lithium mining (more precarious with Trump, but long term I think for national security reasons we will expand production), and biotech. There’s multiple early stage companies developing RNA and gene editing technologies that have the potential to revolutionize medicine. Depends on your risk tolerance, but there are definitely companies with massive growth potential.

J2VVei

$1000 on $CAN $10 ON $DNA (Crypto) $1 on $RNA (Crypto) And $1 on $PURPE (Crypto)

terdferguson

Is your put strategy for the short term, then long term it go up with calls? Sorry, just trying to understand. Not sure about any disease x, I think you are referencing the Congo issue here. https://www.scientificamerican.com/article/mysterious-disease-x-outbreak-might-be-malaria-what-we-know/ There is also a growing concern about a potential for a quad-demic outside of that. https://news.sky.com/story/what-is-the-quad-demic-and-how-can-you-protect-yourself-13262850 Not sure what either of these have to do with company fundamentals or mRNA (messenger RNA not the ticker) vaccines in the short term for a squeeze? Vaccine development takes years. Again, just trying to learn more about how you view the investment value.

terdferguson

This post should be in another certain subreddit...puts on a company that specialized in RNA vaccines with a "planned pandemic" for "disease x" is some short bus level of analysis. I'm not fluent in options but puts are a bet the price will go down no?

anycept

I don't see how that is going to be different from any other seasonal shots sold by other pharmas. RNA vaccine isn't a cutting-edge technology anymore, there aren't capacity issues, pandemic logistics quirks were ironed out, there's no room for price gouging. What is there to make a big impact? Big pharma can't turn the stock around even with a revolutionary obesity treatment. I'm holding NVO and MRK like a regard, losing nearly 15% on each so far 🤷‍♂️

phatbob198

Happy cake day. Here's some additional text from the link under more information: >...By using such RNA molecules designed to be highly specific to its target—in this case, the male sterility genes in cannabis—the sprayer can control male flower formation using natural, endogenous mechanisms to the plant. This technology's high specificity also prevents off-target effects on beneficial organisms or humans. >Male sterility, a condition in which plants' male reproductive organs do not produce viable pollen, is also a crucial trait in crop breeding programs because it enables the development of hybrid seeds with superior yield, quality and uniformity compared to conventionally bred cultivars (e.g., F1 hybrid seed). Akin to the industry’s use of STS (silver thiosulfate) to induce male flowering, LeafWorks’ spray technology can be applied to plants to make 100% male-sterile seed lines for hybrid-line production... >How does it work? >This is a plant spray that you apply to the leaves, or anywhere on the plant. The highly specific RNA molecules that match target genetic sequences you want to silence are taken up by the plant and tell the plant’s natural silencing mechanisms to degrade mRNA transcripts so proteins are never formed... https://leafworks.com/blogs/news/rna-spray-cannabis-hermaphroditism-solution

Orennji

RNAi suppression is transient, so you'd need to spray throughout the entire flower phase to ensure no viable pollen is produced. And how systemic is the RNA delivery vehicle? If the spray must absolutely contact a male flower to be effective, even a few flowers that are not exposed can still ruin a grow.

phatbob198

>LeafWorks... has introduced a cutting-edge non-GMO classified RNA spray designed to control male sterility genes. This technology offers wide-ranging benefits, including mitigating the risk of cross-pollination and hermaphroditism and producing male-sterile plants for efficient hybrid seed production... https://www.reddit.com/r/weedstocks/s/FiU1wp8bLP

goodreadKB

In an unexpected twist, the scientists observed that the RNA from the SARS-CoV-2 virus – responsible for COVID-19 – triggered the development of a unique type of immune cell with anti-cancer properties. These cells, dubbed “inducible nonclassical monocytes (I-NCMs),” were found to attack cancer cells and could potentially be harnessed to treat cancers that are resistant to current therapies. These findings possibly explain the mechanism behind the reported regression of certain cancers following COVID-19 infection.

bdavid21wnec

$RNA

Ol_Maxxie_Solt_DB

I think it's more likely that GLP-1 agonists spur research into new mechanisms of action that work better with fewer side effects. For example, did you know over 50% of patients that start a GLP-1 drug discontinue treatment by Day 365? That's not very durable. Instead of mimicking hormones that make people want to eat less (which also causes them to lose muscle mass = bad for long-term immune health), we're likely to explore other ways to tackle obesity. In the last five years, researchers have discovered half a dozen genes linked to obesity, courtesy hundreds of thousands of genomes in the UK Biobank. Humans with loss of function mutations in these genes can be protected from obesity, type 2 diabetes, and cardiovascular disease. Because these are natural genotypes, recreating the effect with a genetic medicine shouldn't have very many side effects or cause loss of muscle mass. Almost all of these genes are expressed in multiple tissue types in the body, including adipose tissue (fat cells). There aren't any therapeutic modalities that can deliver into adipose tissues in clinical trials right now, although RNA interference will start in 2025. However, one of the genes with the most profound effect on obesity is INHBE -- and ~98% of expression occurs in the liver. If you could design the perfect obesity target for existing RNAi platforms, then it might be INHBE. Arrowhead Pharma is about to begin a phase 1 study in obesity targeting INHBE this month (November 2024). Novo Nordisk (via its 2021 acquisition of Dicerna) will be close behind. Eli Lilly is also heavily invested in RNAi. Short-term weight loss won't be as significant with INHBE inhibition, so comparisons to GLP-1 should be made with caution. But imagine an obesity drug that only needed to be take 2-4x per year, didn't make you lose muscle mass, and had potentially no side effects. That's where the industry is likely to focus.

Ol_Maxxie_Solt_DB

Independent biotech analyst here. Biogen is suffering from some self-inflicted wounds that dropped its positioning within its core competitive landscapes, but primarily neuro. While competition is inherent to drug development and investors often overestimate the risks, Biogen doesn't appear to have a best in class asset in any of its chosen domains. That's impressive. Despite having a late-stage neuro pipeline, the company didn't develop assets aimed at emerging targets or disease indications that led to a recent renaissance for the field. Companies like Karuna Therapeutics and Cerevel Therapeutics represent two of the largest acquisitions in recent years. Biogen chased some targets with good, but not great, science. It's now paying the price with relatively underwhelming results in Alzheimer's and depression. Similarly, the commercial potential of the antisense oligo (ASO) asset looks increasingly limited. That has more to do with how quickly RNA medicines have evolved, but Biogen could've moved more quickly or spread its bets better. The immunology pipeline isn't terrible and could provide momentum, but once again Biogen has relatively little exposure to newer modalities, like bispecific antibodies. It still has solid commercial potential, but it's easy to not get excited. Within the neuro pipeline, the LRRK2 asset (an antibody) could be competitive and have broad potential. But there will soon be competition from emerging assets in protein degraders (potential advantage of being taken orally) and RNAi (potential advantage of once quarterly or twice annual dosing). On top of all that, the business is relatively stagnant. Revenue decreased 18% from 2021 to 2023, while operating cash flow fell by more than half in that span. Both metrics have improved through the first half of 2024 though. TLDR The biggest knock on Biogen is that it hasn't been successful at reinvesting cash flows into productive or competitive R&D.

bdavid21wnec

Thoughts on $RNA

Objective_Ad3539

$RNA prairie dogging above that trendline. breakout imminent!

birdflustocks

2/3 Pfizer is developing a mRNA vaccine, but seems to be a bit behind Moderna and maybe CureVac/GSK: [https://www.pfizer.com/news/announcements/pfizer-reiterates-commitment-pandemic-preparedness](https://www.pfizer.com/news/announcements/pfizer-reiterates-commitment-pandemic-preparedness) That's probably why Moderna was awarded phase 3 US government funding and not Pfizer in a direct competition: [https://www.reuters.com/business/healthcare-pharmaceuticals/us-awards-moderna-176-million-produce-bird-flu-vaccine-2024-07-02/](https://www.reuters.com/business/healthcare-pharmaceuticals/us-awards-moderna-176-million-produce-bird-flu-vaccine-2024-07-02/) According to the licensing agreement, BioNTech would receive royalties from Pfizer: "In 2018, Pfizer entered into a worldwide collaboration and license agreement with BioNTech under which Pfizer has the exclusive right to carry out the clinical development and commercialization of mRNA‐based influenza vaccines. Upon potential approval and commercialization, BioNTech would receive a royalty on Pfizer’s sales." [https://www.pfizer.com/news/press-release/press-release-detail/pfizer-initiates-phase-3-study-mrna-based-influenza-vaccine](https://www.pfizer.com/news/press-release/press-release-detail/pfizer-initiates-phase-3-study-mrna-based-influenza-vaccine) The downside with BioNTech in contrast to CureVac and Arcturus is that they have way too much cash, which limits the upside. They also work on a Mpox mRNA vaccine. Seasonal influenza consists of influenza A and influenza B strains, while "bird flu" is influenza A. mRNA vaccines are less effective against influenza B, and Sanofi was publicly proven wrong by Moderna: "The biggest names in messenger RNA like Moderna, Pfizer and CureVac are all working on their own influenza candidates, but Sanofi used a vaccines investor event yesterday to spell out bluntly why those initial attempts to target the technology on flu just won’t work." [https://www.fiercebiotech.com/biotech/first-gen-mrna-flu-vaccines-wont-win-sanofi-execs-admit-they-retool-strategy](https://www.fiercebiotech.com/biotech/first-gen-mrna-flu-vaccines-wont-win-sanofi-execs-admit-they-retool-strategy) "Moderna (MRNA.O) on Wednesday said its flu vaccine had generated a stronger immune response against all four A and B strains of the influenza virus compared to traditional flu shots in a late-stage trial." [https://www.reuters.com/business/healthcare-pharmaceuticals/moderna-scale-down-manufacturing-covid-vaccine-2023-09-13/](https://www.reuters.com/business/healthcare-pharmaceuticals/moderna-scale-down-manufacturing-covid-vaccine-2023-09-13/) Now they had so much egg on their face that they had to do something, so now they improve their mRNA technology, but also cooperate with Novavax to improve their traditional vaccines with the Matrix-M adjuvant, which is actually great, just leapfrogged by mRNA technology. [https://www.novavax.com/what-we-do/matrix-m-adjuvant-technology](https://www.novavax.com/what-we-do/matrix-m-adjuvant-technology) [https://pubmed.ncbi.nlm.nih.gov/37113023/](https://pubmed.ncbi.nlm.nih.gov/37113023/)

XYHopGuy

ironically the opportunity with CRISPR is mostly in AI. Use CRISPR for massive knockout screens + barcode the guide, take measurements (imaging, RNA, whatever), repeat. Suddenly you have a massive dataset of knockout x phenotype that's fucking impossible to use with traditional statistics. Guess what's goes good with that? About 10k H100s.

BrilliantClarity

Machine learning has many applications that you might not be thinking of. An example is DNA/RNA where a large amount of data needs to be processed using ‘AI’ on NVDA compute. This sector will grow massively in the next 10-20 years

Richard_AIGuy

There’s not much to say here, it’s all about having the cash to finance the ongoing trials. SIL-204 has indication of a Phase 2/3 trial in 2026, and that will require several hundred million in funding. The LODER trial is ongoing and anticipates moving to Phase 3 soon. The difference is the details of the delivery mechanisms and level of optimization of SIL-204. PLGA is a promising delivery system, they have shown excellent biocompatibility. However, there are questions regarding the initial burst of drugs from PLGA microparticles. https://pubs.acs.org/doi/10.1021/acsbiomaterials.0c01228 However, I’m going to assume they have some sort of innovation for this. Or at least I would hope so. As it changes delivery timelines. As for siRNA technology, it’s promising. It’s more focused than something like miRNA, which is being trialed in other cancer drugs. It’s specifically focused on a given gene. The issue is that, or the *possible* issue is that it can have unintended consequences. Meaning, it can turn off genes that are close, but not targeted. Then you have all the other stuff, stability, negative charge making cellular membrane penetration difficult, and they’re large. But if they figure out that, or some of that, it is a promising technology. But it’s a brutally expensive technology, which means that possible clinical uptake could be somewhat difficult. And it requires ultrasound guided endoscopy, to be locally and directly administered. Not the only drug of its type, of course, but definitely a challenge versus IV or port introduction. I have some faith in various RNA technologies, if I didn’t, I wouldn’t have invested in Moderna pre-pandemic, and for cancer therapeutics over vaccines. The concern is cash. It’s an incredibly expensive endeavor. But that’s the nature of biotech, 95% of startups fail.

Tigeranium

RNA

Reasonable_Yard9906

Summary: Their lead candidate is a siRNA (small piece of RNA that suppresses mRNA, the recipe cells use to make protein) that suppresses expression of KRAS oncogene in pancreatic cancer. Phase 2 looks like 1-2 patients out of 20 that got their drug lived longer than all the \~20 patients that did not get their drug. Statistically underpowered. Preclinical data they injected the drug into tumors and it did not shrink tumors but altered growth. Analysis: Their drug does something, but not shocking. Also it's pre clinical biotech, i.e. they hemorrhage money. Wouldn't hold long term. Then again you have companies like SAVA with trials based on fraudulent pre-clinical data and it's still pumping. GLTA 

bdavid21wnec

$RNA - therapy's for tons of muscle dystrophy and successful. Possible BO target as well. Just released positive data for Duchenes. Will be fast tracked cause nothing exists. Money on the bank too

overroadkill

They are both [rna](https://en.wikipedia.org/wiki/RNA_virus) viruses. Oops. There goes your credibility. Apparently an uneducated boob can google better than you. Thats embarrassing.

Auer-rod

Holy shit bro, you just love to talk in circles. The strains it tested for were effective for 98% of cases. New strains came out which were less effective. Has there been less idiots like you around and actually got vaccinated, the pandemic wouldn't have dragged on as long as it did. I never compared it to the flu vaccine. Influenza and COVID are fundamentally different on a genetic scale. One is a DNA virus, the other is a RNA virus. The way they mutate is different. They never lied. Effectiveness changed as the viruses biology changed. That's not lying, that's transparency.

Ok_Group_7873

Of course short read will remain relevant for many applications. I am not sure if Pacbio's short-read technology will become a winner, a lot of competition in that part of the market. Appearantly the quality delived with their Onse system is very-high which can be good for some applications (e.g. liquid biopsy). I have yet to have a look to the data myself, and a lot will depend on price competitiveness. My expectations are higher for the long-read segment of the company. I disagree with you statement that long-read sequencing is only useful to build a reference. I have heard clinical labs thinking about completely replacing all their other pipelines with Pacbio WGS because of the ability to detect structural variants, repeat expansions, etc. Another great advantage is the ability to directly measure DNA-methylation, which is not possible with short reads. And then there are applications in the RNA-sequencing realm where full-length mRNA sequencing can find different iso-forms of genes. Long-read will not fully replace short-read sequencing anytime soon, but there is still a lot of market share to gain by use cases where it can provide much added value. As the instruments become more readily available at large facilities I definitely predict growth. The technology was selected as the Nature method of the year 2022 for good reasons.

bdavid21wnec

$GLW - fiber play with AI, just popped today cause they are gonna print money $GXO - logistics mostly in UK, Brad Jacob company $CRK - nat gas AI play, Jerry Jones owns like 70% RNA - biotech with huge potential $VFC - turnaround play $CPS - automotive rebound play Most of these need rates to drop before they 🚀

gwelfguy

CRISPR has been around for a while and I don't know what rabbit they're suddenly going to pull out of their hat. I'm betting on Moderna and the wider applicability of their RNA-based therapies for things like cancer. That's completely separate from the recent hit they took on the effacity of their RSV vaccine.

Tiiinygecko

Ps: I had money in LEVI, RCL, RNA as well, but sold each for over 60% gain each to take profits at the top 😉.

roastshadow

I agree in general that pharma is going to do well. Today, "AI" mostly Large Language Model. What is going to help with drug companies is the large scale modeling being done, the big simulations, super-fast DNA/RNA sequencing, and some of the other techs like mrna vaccines. But at the same time, pharma has an issue that most drugs don't have to be great at something, just better than nothing, and might have terrible side effects. Large database and modeling will help with being able to find medicines and treatments that are more likely to work with less side effects. It can help by taking a DNA sample from the patient as some DNA are more likely to have certain issues, immunities, or reactions than others. AI will help with assisting doctors and nurses. It can talk to a person to help figure out something before they talk to a doctor. It can be recording the doctors' notes in real time. It can help a doctor to diagnose issues and recommend treatments. Doctors cannot know everything. An AI won't either, but it is very likely capable of putting together more variables in a new way to give suggestions. So... in the future, you go to a doctor. You do some blood tests, a DNA test, tell them how you feel, they do other tests and stuff, a urine and feces test, and other tests. Then they feed all that into the LLM and it says, "there is a 39% chance that the patient has or is developing \_\_\_\_\_\_\_", test for that. No? Ok, there is a 31% chance it is \_\_\_\_\_\_. Test for that. And so on. There are a LOT of things that can be prevented but testing and scanning can be very expensive. But, if your DNA says that there is an 80% chance of X and a 10% chance of Y, then maybe an expensive test for X is warranted.

BrisketWhisperer

PFE is my play for slow growth, due to their pipeline of promising drugs, especially RNA cancer targets, which was recently bolstered by acquisition of Seagen. I believe there is potential for faster growth if something pops in clinical trials, etc. In the meantime, the stock is priced attractively, it's an old, successful company, and you can't beat the dividend for the present time. I'm adding shares as I'm able.

b0rtb0rtb0rtb0rt

Having a great product pipeline and research and development teams is an entirely different game than profitably operating fill and distribution networks and a management team that can finesse cost control, BioNTech may have the former, but its management is struggling to meet its financial obligations and is currently being sued as a consequence. (PhD in RNA Biochemistry with an MFA, worked in biotech and pharma for a few decades at a bench and a desk)

SPAC_Time

[ANEW MEDICAL, INC. Secures Key Patent in Europe for Human Klotho Gene RNA Slice Variant and Protein](https://www.globenewswire.com/news-release/2024/06/26/2904315/0/en/ANEW-MEDICAL-INC-Secures-Key-Patent-in-Europe-for-Human-Klotho-Gene-RNA-Slice-Variant-and-Protein.html) \- WENA WENAW WENA ( was RWOD ) just changed ticker Monday. Up 120% today to $3.60, 42 million traded.

Tigeranium

Buy RNA guys!

Early-Somewhere-2198

More biotech. RNA sept alny. Does this entire thread just don’t know biotech. Not surprised. Most are idiots to get biotechnology