Data from the study showed that treatment with 2mg/kg dose of del-brax achieved greater than 50% mean reductions in DUX4 regulated genes for 12 patients at the four-month timepoint. The early data also showed trends of improving muscle strength and function in patients treated with this dosage. Shares of RNA were up 32.6% on Wednesday as investors cheered the results. The results show the potential of del-brax to address the underlying cause of FHSD, which is the abnormal expression of the DUX4 gene. Curren ...

Core Viewpoint - Avidity Biosciences, Inc. plans to offer $300 million of its common stock in an underwritten public offering, with an additional option for underwriters to purchase up to $45 million more [1][2]. Group 1: Offering Details - The public offering is subject to market conditions and other factors, and all shares will be sold by Avidity [1]. - The offering is being conducted under a shelf registration statement that is effective with the SEC [3]. Group 2: Use of Proceeds - The net proceeds from the offering will be used to fund clinical program development, advance research and development of the AOC platform, and for working capital and general corporate purposes [2]. Group 3: Company Overview - Avidity Biosciences focuses on RNA therapeutics, specifically Antibody Oligonucleotide Conjugates (AOCs™), which aim to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies [5]. - The company is developing clinical programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD) [5]. - Avidity is expanding its pipeline to include programs in cardiology and immunology through internal discovery and partnerships [5].

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 demonst ...

BlackJack3D/iStock via Getty Images Avidity Biosciences, Inc. (NASDAQ:RNA) specializes in targeted RNA therapeutics using its Antibody Oligonucleotide Conjugates (AOC) platform. RNA can create therapies for previously unreachable tissues and cells with this approach. RNA's platform provides monoclonal antibodies (mAbs) that accurately supply oligonucleotide treatments to interfere with or modify pathological gene expression and address the root causes of genetic diseases. RNA's pipeline includes programs in ...

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: the ability to advance into pivotal studies and expand the AOC platform into additional therapeutic areas; and the potential to develop treatments to help improve the lives of patients and revol ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q _____________________________________________________ x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED MARCH 31, 2024 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number 001-39321 _____________________________________________________ Avidity Biosciences, Inc. (Exact name of ...

Exhibit 99.1 Avidity Biosciences Reports First Quarter 2024 Financial Results and Recent Highlights Presented positive long-term del-desiran data from MARINA-OLE™ showing reversal of disease progression in people living with myotonic dystrophy type 1 across multiple endpoints; same key endpoints, with vHOT as primary, agreed for phase 3 HARBOR trial Cash position of $915 million at the end of Q124 following successful private placement SAN DIEGO, May 9, 2024 /PRNewswire/-- Avidity Biosciences, Inc. (Nasdaq: ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 _________________________ FORM 10-K _________________________ (Mark One) x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE FISCAL YEAR ENDED DECEMBER 31, 2023 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 10578 Science Center Drive, Suite 125 San Diego, CA 92121 (Address of Principal Executive Offices) (Zip Code) (858) 401-7900 ( ...

Exhibit 99.1 Avidity Biosciences Reports Fourth Quarter and Year-End 2023 Financial Results and Recent Highlights Avidity is on-track to initiate global Phase 3 HARBOR™ trial of AOC 1001 for DM1 in mid-2024 Company to share first-look at long-term efficacy and safety data from MARINA-OLE™ trial in people living with DM1 at MDA Clinical & Scientific Conference and via webcast on March 4, 2024 Avidity to report data from clinical programs for people living with FSHD and DMD44 later this year SAN DIEGO, Feb. 2 ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q _____________________________________________________ x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED SEPTEMBER 30, 2023 or Securities registered pursuant to Section 12(b) of the Act: o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number 001-39321 _______________________________ ...