Latest news with #DMD44
Los Angeles Times
18-05-2025
- Business
- Los Angeles Times
Bioscience Firms in Southern California Focus on Innovation & Collaboration
Southern California is one of the nation's leading life science hubs, led by the vast cluster of companies in San Diego County that focus on research and development. However, the sector is spread throughout the region with employers such as Amgen in Thousand Oaks and Edwards Lifesciences in Irvine featured among the top companies. New drug developments and delivery systems are some of the leading recent breakthroughs from local companies. Treatments have been created for a wide range of ailments, such as neuromuscular diseases, diabetes, eye disease and cancer, among others. Over the past few months, San Diego-based Avidity Biosciences Inc. reported multiple positive datasets from its three clinical-stage programs in rare muscle diseases. It is preparing for commercialization in anticipation of three potential products that could launch in close succession next year. 'Avidity's mission is to revolutionize the delivery of RNA therapeutics to make a profound difference in the lives of people living with serious rare diseases, many of whom have no or limited treatment options,' said Sarah Boyce, Avidity's chief executive. 'We plan to file our first biologics license application for our DMD44 drug candidate and accelerate commercial preparations for three potential product launches in rapid succession in all three rare neuromuscular diseases.' In Northridge, Medtronic Diabetes received approval from the Food and Drug Administration in April for its Simplera Sync sensor for use with the insulin delivery system MiniMed 780G. The new sensor is a disposable all-in-one that requires no fingerpricks. It plans a limited launch of the product this fall. 'We're committed to driving innovation that makes life easier for those living with diabetes so they can forget about their diabetes as much as possible throughout the day,' said Que Dallara, president of Medtronic Diabetes, in a statement. Aliso Viejo-based Glaukos Corp. reported positive clinical updates in January for its iDose TR sustained-release procedural pharmaceutical platform. The company focuses on treatments of chronic eye diseases such as glaucoma, cataracts and diseases that impact the cornea and retina. In February, it followed up on their previous announcement with FDA acceptance of its new drug application for Epioxa, a therapy for keratoconus, which affects the cornea, and set an October goal for the FDA to review the treatment. In January, Agoura Hills-based A2 Biotherapeutics closed an $80-million Series C funding round that will be used for three clinical development programs of its precision cell therapies. Investors include The Column Group and Samsara BioCapital. 'We are excited by the initial clinical data from our lead programs, which we believe validates our proprietary logic-gate technology approach to solid tumor cancers,' said Jim Robinson, CEO of A2 Bio, in a statement. Other companies are expanding through collaboration. San Diego-based Amprion announced a collaboration with Mayo Clinic Laboratories to expand access to Amprion's SAAmplify test across the United States. The partnership is expected to improve diagnostic accuracy for neurodegenerative diseases. 'Amprion has developed early and accurate diagnostic tools for a range of neurodegenerative diseases, including Parkinson's, Lewy Body Dementia, and Multiple System Atrophy. Through this partnership, Amprion and Mayo are able to provide patients, physicians and families with one of the most comprehensive diagnostic offerings available,' said Russ Lebovitz, Amprion chief executive.
Yahoo
12-03-2025
- Business
- Yahoo
Avidity Biosciences Announces Upcoming Presentations at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Aravindhan Veerapandiyan, MD, Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital and EXPLORE44® trial investigator, will present topline del-zota data from Phase 1/2 EXPLORE44 trial at 2025 MDA Clinical & Scientific Conference in Dallas, Texas Avidity to host investor and analyst webcast event March 17, 2025, at 8:00 a.m. ET Key opinion leaders to participate in Avidity sponsored industry forum event "Advancing RNA Therapeutics", March 17, 2025, at 7:00 a.m. CT/8:00 a.m. ET SAN DIEGO, March 12, 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 that the company will be presenting one oral and two poster presentations at the 2025 MDA Clinical & Scientific Conference (MDA) in Dallas, Texas, being held March 16-19, 2025 and will host an investor and analyst webcast event on March 17, 2025. 2025 MDA Clinical & Scientific Conference Oral & Poster Presentations March 19, 2025: 9:00 a.m. – 9:15 a.m. CT Del-zota produced statistically significant increases in exon skipping and dystrophin levels in EXPLORE44, a Phase 1/2 study in patients with DMD44 (oral presentation) Aravindhan Veerapandiyan, MD, Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital, and EXPLORE44® trial investigator, will present topline del-zota data from Avidity's Phase 1/2 EXPLORE44 trial for people living with Duchenne Muscular Dystrophy amenable to exon 44 skipping (DMD44). March 16-18, 2025: 6:00 p.m. – 8:00 p.m. CT Del-zota produced statistically significant increases in exon skipping and dystrophin levels in EXPLORE44, a Phase 1/2 study in patients with DMD44 (poster presentation) Phase 3, randomized, global study assessing efficacy and safety of del-desiran for the treatment of myotonic dystrophy type 1: HARBOR™ trial design (poster presentation) The presentation and posters will be available on the publications page of Avidity's website at following the conference. Avidity Sponsored Industry Forum Breakfast Event During MDA Conference On March 17, 2025, 7:00 a.m. CT, Mike Flanagan, PhD, Chief Scientific Officer at Avidity, will host an industry forum breakfast titled "Advancing RNA Therapeutics: Exploring Antibody Oligonucleotide Conjugates (AOCs) for Rare Neuromuscular Diseases." Dr. Flanagan will be joined by guest speaker, Nicholas E. Johnson, MD, MSCI, FAAN, Vice Chair for Research, Department of Neurology, Virginia Commonwealth University, and will also be joined by Jeffrey M. Statland, MD, Professor of Neurology, University of Kansas Medical Center, and Johanna Hamel, MD, Associate Professor of Neurology, Psychology and Laboratory Medicine, University of Rochester to take part in a Q&A session. Participation will be open to all MDA Conference registered attendees. Avidity Investor and Analyst Webcast Avidity management will be joined by Kevin Flanigan, MD, Robert F. and Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research at Nationwide Children's Hospital, and Professor of Pediatrics and Neurology at the Ohio State University College of Medicine, on March 17, 2025, at 8:00 a.m. ET for a live video webcast to discuss del-zota topline data from the EXPLORE44 trial, which will be available on the company's website at 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 muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and 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. 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
