Latest news with #AvidityBiosciences
Yahoo
8 hours ago
- Business
- Yahoo
Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial
Avidity Biosciences, Inc. (NASDAQ:RNA) revealed topline data from the dose escalation cohorts of the delpacibart braxlosiran (del-brax) Phase 1/2 FORTITUDE program in Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is a genetically acquired disease that leads to progressive muscle weakness and severely decreased functional capacity. The data will be presented at the 32nd Annual FSHD Society International Research is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the disease-causing gene, double homeobox 4 (DUX4). FSHD affects approximately 45,000 to 87,000 people in the United States and Europe. Topline data from these cohorts for del-brax treated participants, compared to placebo, demonstrated: Consistent improvement of functional mobility and muscle strength as measured by 10-meter Walk-Run test (10MWRT), Timed Up and Go (TUG), and quantitative muscle testing (QMT) as compared to placebo; Consistent improvement in multiple measures of quality of life as measured by patient-reported outcomes and compared to placebo; Rapid and significant reductions in levels of KHDC1L and creatine kinase, a biomarker of muscle damage. Favorable long-term safety and tolerability with most adverse events (AEs) mild or moderate, with no related serious or severe adverse events and no discontinuations. Topline data from the ongoing, fully enrolled del-brax Phase 1/2 FORTITUDE biomarker cohort are anticipated in Q2 2026. The primary endpoint of the FORTITUDE biomarker cohort is reduction of KHDC1L, a novel DUX4-regulated circulating biomarker. Avidity also announced that the accelerated approval regulatory pathway in the U.S. is open for del-brax and that the company has initiated the global, confirmatory Phase 3 FORWARD study in FSHD. In a recent research note, Chardan Capital analyst Keay Nakae set a 12-month price target of $75 per share for Avidity Biosciences. The valuation stems from a Net Present Value (NPV) model of the company's projected revenues from its RNA therapeutics pipeline, forecasted out to the year 2034. Chardan maintained its Buy rating and increased its price forecast from $65 to $75. According to Nakae's analysis, the valuation incorporates specific 'probability of success' rates for each of Avidity's drug candidates to account for clinical and regulatory risks. His model assigns a 60% chance of success for the company's FSHD candidate, 50% for its DM1 and DMD exon 44 candidates, and lower probabilities for other assets. The analyst applied a 14% Weighted Average Cost of Capital (WACC) as the discount rate to reflect the high-risk profile of the development-stage company. Nakae also highlighted several significant risks that could impede Avidity from reaching the $75 price target. He noted that the company, which currently has no revenue-generating products, will likely require additional financing, potentially leading to shareholder dilution. The analyst also pointed to the risks of intellectual property challenges, the critical nature of upcoming clinical trial outcomes, and the intense competition from larger, better-resourced companies in the biotechnology sector. Price Action: RNA stock is trading lower by 11.6% to $32.03 at last check Monday. Read Next:Photo by Gorodenkoff via Shutterstock UNLOCKED: 5 NEW TRADES EVERY WEEK. Click now to get top trade ideas daily, plus unlimited access to cutting-edge tools and strategies to gain an edge in the markets. Get the latest stock analysis from Benzinga? AVIDITY BIOSCIENCES (RNA): Free Stock Analysis Report This article Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
Business Insider
15-05-2025
- Business
- Business Insider
Bank of America Securities Sticks to Its Buy Rating for Avidity Biosciences (RNA)
Bank of America Securities analyst Tazeen Ahmad reiterated a Buy rating on Avidity Biosciences (RNA – Research Report) yesterday and set a price target of $48.00. The company's shares closed yesterday at $28.40. Confident Investing Starts Here: Quickly and easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks straight to you inbox with TipRanks' Smart Value Newsletter According to TipRanks, Ahmad is an analyst with an average return of -5.3% and a 43.00% success rate. Ahmad covers the Healthcare sector, focusing on stocks such as Sarepta Therapeutics, PTC Therapeutics, and Alnylam Pharma. The word on The Street in general, suggests a Strong Buy analyst consensus rating for Avidity Biosciences with a $67.50 average price target, implying a 137.68% upside from current levels. In a report released on May 12, H.C. Wainwright also maintained a Buy rating on the stock with a $68.00 price target.
Yahoo
14-05-2025
- Business
- Yahoo
Antibody Conjugate Oligonucleotide Market Analysis and Forecast, 2025-2035: CRISPR/Cas9 Integration with Oligonucleotide Conjugates Opens New Avenues in Gene Therapy
The global antibody conjugate oligonucleotide market is on track for significant growth, driven by increased demand for targeted therapies in oncology, autoimmune diseases, and genetic disorders, along with advances in genetic medicine. By 2035, the market is projected to expand exponentially due to the rise of oligonucleotide-based therapies and innovation in gene editing technologies. Key players like Avidity Biosciences and AstraZeneca are leading advancements, particularly in North America, with strategic collaborations and investments. However, high development costs remain a challenge. Dublin, May 14, 2025 (GLOBE NEWSWIRE) -- The "Antibody Conjugate Oligonucleotide Market - A Global and Regional Analysis: Focus on Type, Oligonucleotide Type, Target Disease, and Country Analysis - Analysis and Forecast, 2025-2035" has been added to offering. The global antibody conjugate oligonucleotide market is positioned for significant expansion, propelled by increasing demand for targeted therapies and advancements in genetic medicine. The market is expected to grow in 2024 as research into monoclonal antibody-oligonucleotide conjugates intensifies, especially in fields like oncology, autoimmune diseases, and genetic disorders. By 2035, the market could experience exponential growth, driven by the adoption of oligonucleotide-based therapies and advances in genomic technologies, including small interfering RNA (siRNA) and antisense oligonucleotides. The prevalence of cancer and genetic disorders remains a crucial driver. The World Health Organization (WHO) estimates 18 million new cancer cases and 9.6 million deaths annually, emphasizing the need for effective targeted therapies. Companies like Ionis Pharmaceuticals demonstrate promising results with antisense oligonucleotide therapy for spinal muscular atrophy. Similarly, there's a rise in genetic disorders such as Duchenne muscular dystrophy and cystic fibrosis, increasing demand for therapies targeting the genetic causes of these diseases. Integrating oligonucleotide conjugates with gene editing technologies like CRISPR/Cas9 is expected to propel market growth further. This fusion not only facilitates precise genetic material delivery but also targeted gene modification. Avidity Biosciences and Editas Medicine are pioneering this integration to enhance genetic therapies' delivery and efficacy, opening new possibilities for treating gene-based diseases. One key opportunity lies in the rapid growth of personalized medicine. Genetic profiling and biomarker identification are enabling companies to develop therapies tailored to individual patients, particularly in oncology and rare genetic disorders. Dyne Therapeutics and Tallac Therapeutics are at the cutting edge of this personalized approach, crafting targeted therapies for previously untreatable conditions. However, high development and production costs pose a challenge, particularly restricting accessibility in low-income regions. Substantial capital investment in research, development, and specialized infrastructure remains a barrier to broader adoption. Leading companies in the market, including Avidity Biosciences, Dyne Therapeutics, Tallac Therapeutics, and AstraZeneca, are leveraging innovation, strategic collaborations, acquisitions, and expansion into new therapeutic areas to boost competitiveness. Dyne's Dyn101, for example, focuses on improved muscle delivery for Duchenne muscular dystrophy therapy. Market Segmentation: Monoclonal antibody-oligonucleotide conjugates are leading due to their ability to provide precise therapies for complex diseases. Companies like Avidity Biosciences are advancing treatments such as AOC 1001 for Duchenne muscular dystrophy, demonstrating the technology's adoption in developing targeted therapies for rare diseases. North America to Lead the Market North America dominates the market owing to a robust biotech ecosystem and substantial R&D investments. Key players like AstraZeneca and Ionis Pharmaceuticals are advancing gene-based treatments, with Ionis achieving a milestone in gene therapy with Spinraza for spinal muscular atrophy. Additionally, AstraZeneca's collaboration with Ionis in oncology underscores the region's investment in biotechnology, supported by favorable regulatory environments and FDA's expedited approval processes, fostering the growth of oligonucleotide conjugate therapies. Key Topics Covered: 1. Global Antibody Conjugate Oligonucleotide Market Outlook2. Global Antibody Conjugate Oligonucleotide Market: Industry Analysis3. Global Antibody Conjugate Oligonucleotide Market (by Type)4. Global Antibody Conjugate Oligonucleotide Market (by Oligonucleotide Type)5. Global Antibody Conjugate Oligonucleotide Market (by Target Disease)6. Global Antibody Conjugate Oligonucleotide Market (by Region)7. Global Antibody Conjugate Oligonucleotide Market: Competitive Insights and Company Profiles AstraZeneca Avidity Biosciences Dyne Therapeutics Tallac Therapeutics For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900
Yahoo
21-04-2025
- Business
- Yahoo
Avidity Biosciences Completes Enrollment in Biomarker Cohort in Phase 1/2 FORTITUDE™ Trial for Delpacibart Braxlosiran (del-brax) in People Living with Facioscapulohumeral Muscular Dystrophy
Del-brax FORTITUDE biomarker cohort designed for potential accelerated approval; plan to share regulatory update in Q2 2025 Regulatory alignment on global Phase 3 del-brax trial design and study initiation anticipated in Q2 2025 Plan to present topline data from FORTITUDE dose escalation cohorts in Q2 2025 On track to be first globally approved drug for FSHD SAN DIEGO, March 31, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced the completion of enrollment in the biomarker cohort in the Phase 1/2 FORTITUDE™ clinical trial of delpacibart braxlosiran (del-brax) in people living with facioscapulohumeral muscular dystrophy (FSHD). A total of 51 participants were enrolled in the FORTITUDE biomarker cohort. "Completing enrollment in the FORTITUDE biomarker cohort is an important milestone as we pursue a potential accelerated approval path in the U.S. for del-brax and work to bring the first approved drug to people living with this rare, devastating neuromuscular disease who have no treatment options as quickly as possible," said Steve Hughes, M.D., chief medical officer at Avidity. "We are very encouraged by the del-brax 2 mg/kg data thus far, demonstrating unprecedented and consistent reductions in DUX4-regulated genes, significant decreases in novel circulating biomarker and creatine kinase, trends of functional improvement, and favorable safety and tolerability, and look forward to sharing additional data as well as other key milestones in the second quarter of this year." Avidity is on track to deliver multiple updates from the del-brax program in Q2 including: Regulatory alignment on a potential accelerated approval path in the U.S. for the ongoing FORTITUDE biomarker cohort; Regulatory alignment on the design of the global Phase 3 trial as well as initiation of the trial; and Topline data from the dose escalation cohorts in the FORTITUDE trial. Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the mRNA transcript of the disease-causing gene, double homeobox 4 (DUX4). Currently, there are no approved therapies for the treatment of FSHD, a rare, hereditary disorder marked by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability. FSHD affects approximately 45,000 to 87,000 people in the United States and European Union. About the Phase 1/2 FORTITUDE™ trial The FORTITUDE™ trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate single and multiple doses of delpacibart braxlosiran or del-brax in 90 participants with facioscapulohumeral muscular dystrophy (FSHD). FORTITUDE is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of del-brax administered intravenously. Activity of del-brax is being assessed using key biomarkers, including DUX4-regulated muscle and circulating biomarkers and magnetic resonance imaging (MRI) measures of muscle volume and composition. Though the Phase 1/2 trial is not statistically powered to assess functional benefit, it explores the clinical activity of del-brax including measures of mobility and muscle strength as well as patient reported outcomes and quality of life measures. The trial has a total of three dose cohorts. The first two dose escalation cohorts evaluated 2 mg/kg or 4 mg/kg of del-brax versus placebo and were designed to assess safety as well as inform the dose and dose regimen of del-brax for additional studies. Avidity has completed enrollment in the dose escalation cohorts and identified 2 mg/kg every six weeks of del-brax as the dose for future clinical trials. The third, ongoing biomarker cohort in the FORTITUDE trial is designed for a potential accelerated approval path in the U.S. It is assessing the impact of del-brax 2 mg/kg every six weeks versus placebo for 12 months in people living with FSHD, ages 16-70. The primary endpoints of the study cohort are changes in DUX4-regulated gene expression and DUX4-regulated circulating biomarker. Enrollment in the biomarker cohort is complete. Participants who complete FORTITUDE have the option to enroll in the ongoing FORTITUDE open-label extension (FORTITUDE-OLE™) study evaluating the long-term safety and tolerability of del-brax in participants living with FSHD. For more information about the FORTITUDE trial, visit the FORTITUDE study website or visit and search for NCT05747924. About the Phase 2 FORTITUDE-OLE™ trial FORTITUDE-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of delpacibart braxlosiran or del-brax in participants with facioscapulohumeral muscular dystrophy (FSHD) who were previously enrolled in the Phase 1/2 FORTITUDE™ trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-brax in participants who enrolled in the randomized, placebo-controlled, Phase 1/2 FORTITUDE clinical trial. Participants who enroll in the FORTITUDE-OLE study will receive del-brax regardless of whether they received active treatment or placebo in the FORTITUDE study. The total duration of active treatment with del-brax in the FORTITUDE-OLE is approximately 24 months. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on the FORTITUDE-OLE study click here or visit and search for NCT06547216. About Del-brax Del-brax is designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4. The abnormal expression of DUX4 protein leads to changes in gene expression in muscle cells that are associated with the life-long, progressive loss of muscle function in patients with FSHD. Del-brax aims to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in people with FSHD. Del-brax consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA. Avidity reported positive initial del-brax 2 mg/kg data at four months from the Phase 1/2 FORTITUDE™ trial demonstrating unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with FSHD. Del-brax is currently in Phase 1/2 development as part of the FORTITUDE trial in individuals with FSHD. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan designation for del-brax and the FDA has granted del-brax Fast Track designation. About Facioscapulohumeral Muscular Dystrophy (FSHD) Facioscapulohumeral muscular dystrophy (FSHD) is a rare, progressive, and variable hereditary muscle-weakening condition marked by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability. It is characterized by progressive and often asymmetric skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk and progresses to weakness in muscles in the lower body. FSHD is an autosomal dominant disease caused by the aberrant expression of the DUX4 (double homeobox 4) gene in the skeletal muscle, which activates genes that are toxic to muscle cells and leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function. Skeletal muscle weakness results in physical limitations throughout the whole body, including an inability to lift arms for more than a few seconds, loss of ability to show facial expressions and serious speech impediments. These symptoms cause many people affected by FSHD to become dependent on the use of a wheelchair for mobility. Currently, there are no approved treatments for people living with FSHD. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit and engage with us on LinkedIn and X. Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: Avidity's plans to present topline data from the dose escalation cohorts of the FORTITUDE study and the timing thereof; Avidity's plans to pursue an accelerated approval path for del-brax and the anticipation of regulatory alignment with such plans; the status of the FORTITUDE trial and cohorts therein, including but not limited to initiation, enrollment, design and goals; the ability for del-brax to achieve accelerated approval; the characterization of data associated with del-brax and the FORTITUDE trial, the conclusions drawn therefrom, the impact of such data on the advancement of del-brax and its abilities to treat FSHD; and the possibility of del-brax becoming the first globally approved drug for FSHD. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: the data and results produced in the FORTITUDE trial and FORTITUDE-OLE as of the most recent respective cutoff dates may not be indicative of final results, may not support BLA submissions or accelerated approvals, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute a successful product launch for del-brax; unexpected adverse side effects to, or inadequate efficacy of, del-brax that may delay or limit its development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of clinical trials; Avidity's dependence on third parties in connection with clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and subsequent filings with the SEC. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact:Kat Lange(619) 837-5014investors@ Media Contact:(619) 837-5016media@ View original content to download multimedia: SOURCE Avidity Biosciences, Inc. Sign in to access your portfolio
Yahoo
05-04-2025
- Business
- Yahoo
Avidity Biosciences (RNA): Among Unstoppable Stocks That Could Double Your Money
We recently published a list of . In this article, we are going to take a look at where Avidity Biosciences, Inc. (NASDAQ:RNA) stands against other unstoppable stocks that could double your money. Generating significant returns and multiplying their money in the stock market remains a primary goal for most investors. However, high excess returns (alpha) are challenging to generate, let alone doubling money. For example, if someone took a bet on the overall economy and bought the broader market index, it would have taken around five to seven years for them to double the investments, as these indices usually take that much time, depending on the economic cycle and market trends. Such gains are never easy to replicate, but certain companies and sectors are better positioned for high growth due to strong fundamentals, innovation, or macroeconomic trends. Investors who can identify these stocks through research and understanding market cycles can generate extra returns. Moreover, specific stocks' valuation and growth trajectory over the next few years must be precisely analysed to make good returns. Over the last five years, the stock market has been highly dynamic, reflecting broader economic shifts, interest rate cycles, and technological advancements. While 2023 and 2024 were volatile because of concerns over inflation, the Federal Reserve policy, and geopolitical tensions, 2025 has been equally volatile, with the S&P 500 down 3% and the Nasdaq down around 8% (as of March 27). This volatility makes higher returns riskier. Nevertheless, market analysts are still optimistic about gains in 2025. In an interview with CNBC on April 1, Chris Hyzy, Merrill and BofA Private Bank CIO, said he would use recent market weakness to increase positions and favour broad market exposure through equal-weighted S&P positions. He identifies financials and consumer discretionary stocks as particularly oversold and attractive. He also believes that certain areas, like software and cybersecurity, could lead the technology sector in share market gains in the coming months. Chris also suggested that while uncertainty may persist into the summer, markets will likely begin pricing in anticipated improvements in economic conditions and corporate earnings later in the year. According to his assessment, the job market remains stable and strong, which would mean a sharp economic downturn is unlikely. He expects the market to experience a 'sawtooth bottom' rather than a sharp V-shaped recovery, suggesting that long-term opportunities remain despite persisting volatility. Fundstrat's head of research, Tom Lee, stated to CNBC on March 31 that market conditions indicate oversold status and potential bottom formation regardless of ongoing downward trends. Investors maintain their focus on government policies and tariff situations, and their economic impact. According to his estimates, the April 2 tariff updates should also clarify the future of policies and could potentially reduce selling pressure in the market. He also believes that as and when the Federal Reserve communicates further on interest rates, inflation, and other policies, it should provide more direction to investors. In essence, opportunities could emerge in the near term, and investors should look for better entry points to create positions to generate more substantial returns. But stock selection also remains key. According to Goldman Sachs Asset Management's March 24 report, Embracing a Broader Equity Landscape, while the technology sector remains a key driver of growth in 2025, the dominance of a few large U.S. tech companies appears to be waning. The authors highlighted that capital is beginning to diversify beyond the Magnificent 7, and many of today's market leaders may not sustain their positions at the top. This evolving market dynamic presents new opportunities for active investors, particularly in smaller-cap equities, high-quality businesses outside the U.S., and differentiated long-term investment themes. As leadership broadens, they believe this shift could mark the beginning of a more favorable environment for stock selection across the global equity landscape. In recent months, many analysts and fund managers have favored diversifying towards small- and mid-cap stocks; thus, this space should remain on investors' radars. At the same time, investors should be aware of the risks involved in high-performing equities. To identify the unstoppable stocks that could double investors' money, we used online screeners to compile a list of U.S.-listed companies with a market capitalization exceeding $2 billion and with a greater than 20% return in the last one year. We then applied an additional criterion, considering only those stocks with an expected upside of around 100% or more. From the refined list, we took the top 10 stocks with the highest upside potential and ranked them in ascending order of respective upsides. Additionally, we provided insights into hedge fund sentiment surrounding these stocks, using data from Insider Monkey's Q4 2024 are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here). A scientist holding a vial of a promising product candidate for therapeutic development. 1-Year Return: 32% Avidity Biosciences, Inc. (NASDAQ:RNA) is a clinical-stage biotech company developing a new type of RNA-based medicines called Antibody Oligonucleotide Conjugates (AOCs). These medicines are designed to deliver genetic treatments directly to the tissues affected by disease, helping target rare genetic conditions that currently have no effective treatments. The company is trying to revolutionise the delivery of RNA therapeutics, and several of its therapies are under clinical trials. Leerink Partners analyst Joseph Schwartz recently reiterated a Buy rating on Avidity Biosciences, Inc. (NASDAQ:RNA) with a $60 price target, citing promising clinical data from the company's exon skipper candidate, del-zota, for Duchenne muscular dystrophy (DMD). The analyst pointed to encouraging results and believes these improvements could provide meaningful patient clinical benefits. He also highlighted that Avidity (NASDAQ:RNA) is on track to submit a Biologics License Application (BLA) by the end of 2025, without requiring additional biopsies for new patients. The analyst stated that Avidity's upcoming program represents their initial commercial entry, while internal activities like manufacturing, along with patient services and market access teams' development, are already in motion. The small commercial potential of DMD for Avidity does not deter the analyst from seeing this program as essential groundwork for expanding the company's muscular dystrophy medication development. Overall, RNA ranks 9th on our list of unstoppable stocks that could double your money. While we acknowledge the potential of RNA to grow, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. There is an AI stock that went up since the beginning of 2025, while popular AI stocks lost around 25%. If you are looking for an AI stock that is more promising than RNA but that trades at less than 5 times its earnings, check out our report about this cheapest AI stock. READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
