Avidity Biosciences, Inc. (NASDAQ:RNA) is one of the Best Performing Long Term Stocks According to Analysts. On September 10, Avidity Biosciences, Inc. (NASDAQ:RNA) announced positive one-year data for its, del-zota, in the EXPLORE44 and EXPLORE44-OLE trials for Duchenne muscular dystrophy patients amenable to exon 44 skipping. Management noted that the data show a reversal of disease progression and significant functional improvements compared to baseline and natural history. Del-zota works by delivering ...

Core Insights - Avidity Biosciences, Inc. (NASDAQ:RNA) is highlighted as a top stock to buy for the next three months according to hedge funds, with a reiterated Buy rating and a price target of $96 from Cantor Fitzgerald [1][2] Business Updates - The company reported significant progress in three late-stage neuromuscular programs targeting Duchenne muscular dystrophy, myotonic dystrophy type 1, and facioscapulohumeral muscular dystrophy [2][3] - The FDA granted Breakthrough Therapy designation for Delpacibart Zotadirsen, with plans to submit a Biologics License Application for del-zota by the end of 2025 [3] Financial Performance - Avidity Biosciences, Inc. achieved an 88.12% increase in collaboration revenue, reaching $3.85 billion, surpassing consensus estimates by $2.24 million [4]

Company Insights - Moderna's stock (MRNA) has surged approximately 37% in the last month but has dipped around 15% year to date, with a recent trading increase of about 7% [1] - The FDA approved Moderna's supplemental Biologics License Application for Spikevax, allowing its COVID-19 vaccine for children aged six months to 11 years at increased risk for COVID-19 [1] - Moderna's mRNA-1010, a seasonal influenza vaccine candidate, achieved a relative vaccine efficacy (rVE) of 26.6% in a Phase 3 study, with specific strain efficacies of 29.6% for A/H1N1, 22.2% for A/H3N2, and 29.1% for B/Victoria lineages [2][3] - The FDA also expanded the approval of Moderna's respiratory syncytial virus vaccine to adults aged 18-59 at increased risk for the disease [3] Industry Trends - Global healthcare stocks are currently cheaper than they have been in decades, with a slow return of investment into the sector [4] - Despite low prices, investor interest remains weak due to uncertainty around U.S. drug pricing policies, including potential revival of "most-favored-nation" rules and 200% tariffs on pharmaceutical imports [5] - Healthcare funds have seen net inflows of $7.2 billion since early 2024, although this is still 41% lower than the previous year [7] - U.S. healthcare has underperformed the S&P 500 by over 60 percentage points in the past three years, with its valuation at a near-record 27% discount compared to the S&P [8] - Investors are awaiting a clear catalyst for the sector, with ongoing policy uncertainty complicating future earnings predictions [9]

Company Overview - ProQR Therapeutics NV is focused on developing transformative RNA therapies using its proprietary Axiomer RNA editing technology platform [1][4] - The company aims to create a new class of medicines targeting both rare and prevalent diseases with unmet medical needs [4] Axiomer Technology - Axiomer is a next-generation RNA base editing technology that allows for specific single nucleotide changes in RNA [3][4] - The technology utilizes the human cell's own ADAR (Adenosine Deaminase Acting on RNA) machinery to convert Adenosine (A) to Inosine (I), which is then translated as Guanosine (G), effectively correcting disease-causing mutations [3] Upcoming Events - ProQR management is scheduled to present at the Citizens (JMP) Life Sciences Conference in New York City on May 8, 2025, at 11:30 AM EDT [1] - A live webcast of the presentation will be available on ProQR's website, with a replay accessible for approximately 30 days [2]