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Avidity Biosciences, Inc. (Nasdaq: RNA) is a pioneering biopharmaceutical company dedicated to revolutionizing RNA therapeutics with its proprietary Antibody Oligonucleotide Conjugates (AOCs™). Their innovative approach merges the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, enabling targeted treatment of diseases that were previously unreachable with existing RNA therapies. They are at the forefront of developing therapies for serious genetic conditions, predominantly focusing on rare muscle diseases.
Avidity's leading clinical programs include:
- AOC 1001 (Del-desiran): Targeting Myotonic Dystrophy Type 1 (DM1), a disease with no approved therapies. Del-desiran has shown promising long-term data from the MARINA-OLE™ trial, demonstrating reversal of disease progression in patients. The global Phase 3 HARBOR™ trial is set to begin, with video hand opening time (vHOT) as the primary endpoint.
- AOC 1044: Aimed at Duchenne Muscular Dystrophy (DMD) with mutations amenable to exon 44 skipping. Recently, it achieved Rare Pediatric Disease and Orphan Drug designations from the FDA and EMA. Positive preliminary data has been reported from the Phase 1/2 EXPLORE44™ clinical trial.
- AOC 1020: Focused on Facioscapulohumeral Muscular Dystrophy (FSHD). Clinical data from the Phase 1/2 FORTITUDE™ trial is expected soon.
Avidity is also advancing its pipeline to include cardiology and immunology programs, aiming to target a broader range of diseases. The company has garnered support and collaborations with top-tier investors and strategic partners, enhancing its capability to deliver groundbreaking treatments.
With headquarters in San Diego, CA, Avidity Biosciences is committed to transforming lives through innovative RNA therapeutics. For more information, visit www.aviditybiosciences.com.
Avidity Biosciences (RNA) reported its fourth quarter and full year 2021 financial results on March 1, 2022, highlighting significant advances in RNA therapeutics. The company ended 2021 with $406 million in cash, bolstered by $175 million raised during the year. Avidity's lead candidate, AOC 1001, is undergoing clinical trials for myotonic dystrophy type 1, with additional programs (AOC 1020 and AOC 1044) expected to enter clinical development in 2022. Collaboration revenue increased to $9.3 million for 2021, up from $6.8 million in 2020.
Avidity Biosciences, Inc. (Nasdaq: RNA) announced the appointment of Steve Hughes, M.D., as chief medical officer. With over 20 years of experience in clinical development and drug approval, Dr. Hughes aims to advance Avidity's Antibody Oligonucleotide Conjugates (AOCs) platform.
He has contributed to more than 50 clinical trials and played key roles in the approval of rare disease products. Avidity is on track to initiate three clinical programs by the end of 2022, including AOC 1001 for myotonic dystrophy and programs for DMD and FSHD.
Avidity Biosciences (Nasdaq: RNA) announced participation in several major healthcare conferences, showcasing their innovative RNA therapeutic approach through Antibody Oligonucleotide Conjugates (AOCs™). Key events include a fireside chat at the SVB Leerink 11th Annual Global Healthcare Conference on February 18, a corporate panel at the Cowen 42nd Annual Health Care Conference on March 7, and another fireside chat at the Needham 21st Annual Healthcare Conference on April 12. Avidity aims to advance their clinical pipeline with AOC 1001 for myotonic dystrophy and additional therapies targeting muscular diseases.
Avidity Biosciences, Inc. (Nasdaq: RNA) announced a live webcast for investors titled "Volume 3: Delivering on AOCs - FSHD" on Dec. 9, 2021, at 11:00 am ET. The company highlighted AOC 1020 as its clinical development candidate for Facioscapulohumeral Muscular Dystrophy (FSHD), which is entering IND-enabling studies. Avidity plans to have three programs in clinical development by the end of 2022, including AOC 1001 for myotonic dystrophy and AOC 1044 for Duchenne Muscular Dystrophy. The MARINA study for DM1 continues with preliminary assessments expected in Q4 2022.
Avidity Biosciences (RNA) reported Q3 2021 financial results, highlighting a significant cash position of $413 million and positive advancements in its AOC pipeline. The initiation of the Phase 1/2 MARINA trial for AOC 1001 marks a transition to a clinical stage company, providing hope for myotonic dystrophy patients. Collaboration revenue increased to $2.2 million from $1.7 million in Q3 2020. However, R&D expenses surged to $24.8 million, up from $9.5 million in the prior year, indicating increased operational costs amid expanding clinical programs.
Avidity Biosciences (Nasdaq: RNA) announced the dosing of the first participants in the Phase 1/2 MARINA trial for its lead product candidate, AOC 1001, marking a significant milestone in RNA therapeutics. The trial targets myotonic dystrophy type 1 (DM1) and is designed to evaluate the safety and tolerability of AOC 1001, which aims to reduce levels of disease-related mRNA DMPK. AOC 1001 has received Orphan Designation from the FDA and EMA, with Fast Track Designation also granted by the FDA for expedited development.
Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical firm specializing in RNA therapeutics, will participate in two upcoming conferences. The Credit Suisse 30th Annual Healthcare Conference is scheduled for November 10, 2021, at 2:40 PM ET. Following that, the 4th Annual Evercore ISI HealthCONx Conference will take place on November 30, 2021, at 10:05 AM ET. Live webcasts and archived replays of both events will be available on the company’s website.
Avidity Biosciences, Inc. (Nasdaq: RNA) announced that the FDA granted Fast Track Designation to its lead program, AOC 1001, for treating myotonic dystrophy type 1 (DM1). This designation facilitates more frequent interactions with the FDA, expediting the development process for serious conditions. AOC 1001 aims to reduce DMPK mRNA levels, addressing the disease's root cause. The Phase 1/2 MARINA trial will evaluate the safety and tolerability of AOC 1001, enrolling around 44 adults. Currently, DM1 lacks therapeutic options, highlighting the significance of AOC 1001's advancement.
Avidity Biosciences, Inc. (NASDAQ: RNA) has announced its participation in two upcoming investor conferences. The Wells Fargo Virtual Healthcare Conference will take place on September 9, 2021, featuring a fireside chat at 11:20am ET. The Chardan Virtual 5th Annual Genetic Medicines Conference is scheduled for October 5, 2021, with a panel discussion at 12noon ET and a fireside chat at 1:30pm ET. Interested parties can access live webcasts and replays on Avidity's website.
Avidity aims to revolutionize RNA therapeutics through its Antibody Oligonucleotide Conjugates (AOCs™), with promising therapies in development for diseases like myotonic dystrophy and Duchenne Muscular Dystrophy.
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