SAN DIEGO , April 22, 2024 /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 that on April 20, 2024, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 37,500 shares of its common... Read More

SAN DIEGO , March 27, 2024 /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 that Avidity management will be participating in the following upcoming conferences: Cantor Fitzgerald Virtual Muscular Dystrophy Symposium on April 3 at 9:40 a.m. PT / 12:40 p.m. ET 23 rd... Read More

SAN DIEGO , March 21, 2024 /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 that on March 20, 2024, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 30,800 shares of its common... Read More

Avidity accelerates global Phase 3 HARBOR™ study initiation to Q2 2024 following regulatory agreement on study design; primary endpoint is video hand opening time (vHOT) and secondary endpoints include muscle strength and activities of daily living Delpacibart etedesiran (AOC 1001) demonstrated consistent and durable improvements in myotonia, muscle strength and activities of daily living in people with DM1 in long-term data... Read More

SAN DIEGO , Feb. 29, 2024 /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 its support for Rare Disease Day ® , highlighting the importance of bringing awareness to the devastating impact rare diseases have on patients, families and caregivers worldwide. "Today, on... Read More

Funding from new and existing investors, including Adage Capital Partners LP, Boxer Capital, Casdin Capital, LLC, Farallon, Janus Henderson Investors, RA Capital Management, RTW Investments, Wellington Management, as well as multiple large investment management firms Pro forma cash, cash equivalents and investments expected to provide runway into late 2026 Avidity to host Volume 8 of investor and analyst event series via... Read More

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. 28, 2024 /PRNewswire/ --... Read More

SAN DIEGO , Feb. 27, 2024 /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 that Avidity management will be participating at the following upcoming conferences: TD Cowen 44 th Annual Health Care Conference on March 5 at 10:30 a.m. PT / 1:30 p.m. ET Leerink Partners... Read More

SAN DIEGO , Feb. 21, 2024 /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 that on February 20, 2024, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 75,500 shares of its... Read More

SAN DIEGO , Feb. 20, 2024 /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 that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to AOC 1044, the company's investigational therapy for the treatment of Duchenne muscular... Read More

John W. Day, MD, PhD, Professor of Neurology and Pediatrics, and Director, Division of Neuromuscular Medicine, Stanford University School of Medicine and a principal investigator in END-DM1, ongoing natural history study will join Avidity management to present data from MARINA-OLE™ Avidity to host Volume 8 of investor and analyst event series via webcast March 4, 2024 , at 8:00 a.m. ET SAN DIEGO , Feb. 15, 2024 /PRNewswire/... Read More

SAN DIEGO , Jan. 22, 2024 /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 that on January 20 , 2024, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 37,650 shares of its... Read More

Avidity plans to initiate global Phase 3 HARBOR™ trial of AOC 1001 for DM1 mid-2024 Company to report data from all three ongoing clinical programs in 2024 AOC 1001 data for DM1 in Q1, AOC 1020 data for FSHD in Q2, and AOC 1044 data for DMD44 in 2H Avidity to advance wholly-owned and partnered cardiology programs toward clinical development Avidity appoints Eric B. Mosbrooker as Chief Strategy Officer SAN DIEGO , Jan. 5,... Read More

SAN DIEGO , Dec. 21, 2023 /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 that on December 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 7,100 shares of its... Read More

AOC 1044 delivered up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg and increased exon skipping in all participants Avidity plans to provide first look at AOC... Read More

Avidity to receive $100 million up front with the potential to receive up to $2.2 billion in milestone payments and up to low double-digit royalties Global licensing and research collaboration to focus on discovery, development and commercialization of up to five cardiovascular targets leveraging Avidity's proprietary AOC platform technology SAN DIEGO , Nov. 28, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a... Read More

SAN DIEGO , Nov. 21, 2023 /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 that on November 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 27,000 shares of its... Read More

SAN DIEGO , Nov. 21, 2023 /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 that Avidity management will be participating at the following upcoming conference: 6 th Annual Evercore ISI HealthCONx Conference on November 28 at 9:30 a.m. PT/12:30 p.m. ET A live webcast of... Read More

Avidity reported new positive AOC 1001 data at World Muscle Society (WMS) Congress in October demonstrating consistent improvement in multiple additional functional endpoints and favorable long-term safety and tolerability in people living with myotonic dystrophy type 1 (DM1) AOC 1044 receives Orphan Designation for the treatment of DMD in US and EU Avidity remains on track to report data readouts from three clinical... Read More

SAN DIEGO , Oct. 23, 2023 /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 that on October 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 96,200 shares of its... Read More

New AOC 1001 data demonstrate improvement in additional functional measures including hand grip, muscle strength and patient reported outcomes, augmenting previously reported positive data showing improvements in myotonia, muscle strength and mobility New long-term safety data of AOC 1001 continue to demonstrate favorable safety and tolerability with over 200 infusions totaling 46.2 patient-years of exposure Data from 12... Read More

SAN DIEGO , Sept. 26, 2023 /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 that the company will be presenting one oral and six poster presentations at the 28th Annual Congress of the World Muscle Society (WMS), being held October 3-7, 2023 , in Charleston, South... Read More

SAN DIEGO , Sept. 25, 2023 /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 that Avidity management will be participating at the following upcoming conferences: Chardan 7 th Annual Genetic Medicines Conference on October 3 at 6:00 a.m. PT/9:00 a.m. ET BMO Biopharma... Read More

SAN DIEGO , Sept. 21, 2023 /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 that on September 20, 2023 , the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 90,300 shares of its... Read More

Avidity supports World Duchenne Awareness Day, International Myotonic Dystrophy Awareness Day, FSHD Society Walk & Roll to Cure FSHD and Global Genes Week in RARE Avidity joins with MDF community leaders, patients, caregivers, and legislators in educational briefing to highlight the latest scientific advancements in myotonic dystrophy research and development Company advancing clinical development programs for three types of... Read More

SAN DIEGO , Aug. 31, 2023 /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 that the company will be presenting two poster presentations at the 2023 Myotonic Dystrophy Foundation Annual Conference, being held September 7-9, 2023 , in Washington, D.C. POSTER... Read More

SAN DIEGO , Aug. 30, 2023 /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 that Avidity management will be participating at the following upcoming conferences: 2023 Wells Fargo Healthcare Conference on September 6 at 5:45 a.m. PT / 8:45 a.m. ET 2023 Cantor Global... Read More

SAN DIEGO , Aug. 21, 2023 /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 that on August 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 42,100 shares of its common... Read More

SAN DIEGO , Aug. 15, 2023 /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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1044, the company's investigational therapy in development for the treatment of Duchenne muscular... Read More

Advancing three clinical development programs – DM1, DMD, FSHD - with data anticipated from each program over the next 12 months SAN DIEGO , Aug. 8, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today reported financial results and recent highlights for the second quarter... Read More

SAN DIEGO , July 21, 2023 /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 that on July 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 33,150 shares of its common... Read More

SAN DIEGO , June 21, 2023 /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 that on June 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 23,650 shares of its common... Read More

Avidity is currently enrolling participants in the Phase 1/2 FORTITUDE™ study of AOC 1020 for the treatment of FSHD Company anticipates data from a preliminary assessment in half of the participants in the FORTITUDE study in 1H 2024 SAN DIEGO , June 20, 2023 /PRNewswire/ - - Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody... Read More

SAN DIEGO , June 8, 2023 /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 that the company will be presenting two oral presentations and two poster presentations at the 30th Annual FSHD Society International Research Congress, being held June 15-16, 2023 , in Milan,... Read More

SAN DIEGO , May 22, 2023 /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 that on May 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 36,900 shares of its common... Read More

Avidity plans to double the number of participants receiving 4 mg/kg of AOC 1001 in MARINA Open-Label Extension (MARINA-OLE™) study by dose escalating participants currently on 2 mg/kg of AOC 1001 FDA allows new participant enrollment for 2 mg/kg of AOC 1001 First look at data from the MARINA-OLE study planned for the end of 2023 SAN DIEGO , May 17, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a... Read More

Positive topline data from AOC 1001 Phase 1/2 MARINA™ trial demonstrated functional improvement, disease modification and favorable safety and tolerability profile in people living with myotonic dystrophy type 1 Advancing three programs in clinical trials with data expected later this year - first look at data from open-label extension study, MARINA-OLE™; healthy volunteer data from EXPLORE44™ Expands research and... Read More

Topline data presented at AAN annual meeting showed AOC 1001 achieved directional improvements in multiple functional endpoint assessments - myotonia (vHOT), measures of strength (total QMT) and mobility (10mWRT, Timed Up and Go) AOC 1001 data demonstrate favorable safety/tolerability profile with most adverse events mild or moderate Topline AOC 1001 data identify dose range for pivotal study; Avidity continues to work to... Read More

SAN DIEGO , April 24, 2023 /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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon... Read More

SAN DIEGO , April 21, 2023 /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 that on April 20, 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 58,300 shares of its common... Read More

SAN DIEGO , April 17, 2023 /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 presentations at the 75 th American Academy of Neurology (AAN) Annual Meeting, including an oral presentation on the topline safety, biomarker and functional data from the AOC 1001 Phase 1/2... Read More

Nicholas E. Johnson , M.D., M.Sci., FAAN, MARINA lead investigator will present AOC 1001 topline data Volume 7 of investor and analyst series will be an in-person event in Boston and available via webcast SAN DIEGO , April 13, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™),... Read More

Discussions with the FDA ongoing as Avidity submits emerging AOC 1001 data from the MARINA trial AOC 1001 continues to be generally well tolerated; Avidity provides more information on the rare serious adverse event in a single participant that led to the partial clinical hold AOC 1001 top-line safety and functional data to be presented at the American Academy of Neurology (AAN) Annual Meeting on April 27, 2023 Avidity to... Read More

SAN DIEGO , March 21, 2023 /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 that on March 20 , 2023, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 73,700 shares of its... Read More

SAN DIEGO , March 13, 2023 /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 that the company will be presenting two oral presentations and multiple poster presentations at the 2023 MDA Clinical & Scientific Conference in Dallas, Texas , being held March 19-22, 2023... Read More

SAN DIEGO , March 1, 2023 /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 that Avidity management will be participating at the following conference: Cowen 43 rd Annual Healthcare Conference on March 7 at 8:10 a.m. PT/ 11:10 a.m. ET A live webcast of the event,... Read More

SAN DIEGO , Feb. 28, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today reported financial results for the fourth quarter and year ended December 31, 2022 and highlighted recent corporate progress. "In 2022, we executed on our goal of advancing three rare disease programs... Read More

SAN DIEGO , Feb. 28, 2023 /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 its support for Rare Disease Day®, highlighting the importance of bringing awareness to the devastating impact rare diseases have on patients, families and caregivers. "This Rare Disease Day,... Read More

Avidity adds new research and development programs in skeletal muscle and cardiology Avidity executing on three distinct rare disease programs in Phase 1/2 clinical development - myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne muscular dystrophy (DMD) SAN DIEGO , Feb. 27, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to... Read More

W. Michael Flanagan , Ph.D. named chief scientific and technical officer SAN DIEGO , Feb. 23, 2023 /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 that Arthur A. Levin , Ph.D. has been appointed to the board of directors of the company and is transitioning from chief... Read More

SAN DIEGO , Feb. 21, 2023 /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 that on February 20, 2023 , the Compensation Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 27,400 shares of its common stock... Read More

SAN DIEGO , Feb. 14, 2023 /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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD is a serious, rare,... Read More

SAN DIEGO , Feb. 7, 2023 /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 that Avidity management will be participating at the following conference: SVB Securities Global Biopharma Conference on February 14 at 1:20 p.m. PT/ 4:20 p.m. ET A live webcast of the event,... Read More

SAN DIEGO , Jan. 20, 2023 /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 that on January 20, 2023, the Compensation Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 12,350 shares of its common stock and... Read More

SAN DIEGO , Jan. 18, 2023 /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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD is a serious, rare,... Read More

SAN DIEGO , Dec. 15, 2022 /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 pricing of an underwritten public offering of 12,000,000 shares of its common stock at a price to the public of $17.25 per share. All of the shares to be sold in the offering are to be sold... Read More

SAN DIEGO , Dec. 15, 2022 /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 that it intends to offer and sell, subject to market and other conditions, $150 million of shares of its common stock in an underwritten public offering. In addition, Avidity intends to grant... Read More

AOC 1001 delivered siRNA to skeletal muscle and produced meaningful DMPK reduction in 100% of participants with a 45% mean reduction in DMPK after a single dose of 1 mg/kg or two doses of 2 mg/kg AOC 1001 produced splicing improvement of 31% in a key set of muscle-specific genes in people with DM1, with a splicing improvement of 16% across a broad 22-gene panel Early signs of clinical activity, with improvement in myotonia... Read More