Avidity Biosciences, Inc. engages in the development of a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOC). It utilizes its AOC platform to design, engineer, and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. The company was founded by Troy E. Wilson, Kent Hawryluk, Mark E. Davis, and Francis Patrick McCormick on November 13, 2012 and is headquartered in San Diego, CA.
相关临床试验
12
3 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2013
进行中（未招募）
2
16.7%
Available
1
8.3%
已完成
5
41.7%
Enrolling By Invitation
1
8.3%
招募中
3
25.0%
暂无批准数据
- Novartis discontinued development of VHB937 (lifonebart) after the Phase 2 ASTRALS trial in early-stage ALS missed both primary and secondary endpoints. - The 251-patient trial tested the TREM2-stabilizing monoclonal antibody or placebo for 40 weeks, with no numerical data released by the company. - The setback adds to a difficult pipeline stretch for Novartis, following Phase 3 misses for pelacarsen and del-desiran and halted rap-cel studies. - Detailed ASTRALS findings are scheduled for presentation at the 37th International Symposium on ALS/MND in Amsterdam on December 9-11, 2026.
- Novartis' Phase 3 HARBOR study of del-desiran failed to show statistically significant improvement over placebo on video hand opening time in myotonic dystrophy type 1. - The antibody oligonucleotide conjugate targets DMPK messenger RNA, and Novartis reported clinical activity on secondary endpoints and exploratory analyses including muscle strength. - Novartis shares fell about 9-10% while Dyne Therapeutics and Sarepta Therapeutics dropped sharply on read-through risk to their related DMPK programs. - Novartis is evaluating the full HARBOR dataset and will engage health authorities on a development path; no approved treatment exists for the disease itself.
- Immunic, Inc. has appointed Chitrang Davé as its newly created Chief Data and Digital Officer, effective September 1, 2026, reporting directly to CEO Erik Lundgren. - The role will establish enterprise-wide data, analytics, AI, and digital capabilities to support the potential NDA submission and commercialization of vidofludimus calcium (IMU-838) in multiple sclerosis. - Top-line data from the Phase 3 ENSURE trials in relapsing MS are expected by year-end 2026, with a confirmatory Phase 3 program in progressive MS advancing. - Davé brings more than 25 years of data and analytics leadership experience from Avidity Biosciences, Edwards Lifesciences, and Medtronic.
- RNA interference (RNAi) therapies using small interfering RNA (siRNA) are already approved by the FDA, with at least seven treatments available for genetic diseases and more in clinical trials. - The global RNAi therapeutics market is projected to grow from about $1.5 billion in 2024 to roughly $5.1 billion in 2034, reflecting rapid commercial expansion. - Delivery beyond the liver is a key frontier, with researchers developing siRNAs targeting muscles, nerves, the placenta, and other organs. - Korean firms OliX and rznomics signed technology transfer deals with Eli Lilly worth 900 billion won and 1.9 trillion won, respectively, underscoring global interest in RNA-based platforms.
- Atrium Therapeutics earned a $15 million milestone payment from Bristol Myers Squibb for successfully delivering a second lead compound targeting an undisclosed cardiology indication. - The payment stems from a global licensing and research collaboration focused on RNA-based therapies for multiple cardiovascular indications. - Atrium is eligible for up to approximately $1.35 billion in R&D milestones, $825 million in commercial milestones, and tiered royalties up to low double-digits on net sales. - The milestone reflects the strength of Atrium's RNA delivery platform, which uses monoclonal antibodies and siRNA to target the underlying drivers of genetically driven cardiac diseases.
- The FORTITUDE Phase I/II biomarker cohort met its primary endpoint of reducing KHDC1L levels and key secondary endpoint of lowering creatine kinase in FSHD patients. - Del-brax, an investigational antibody oligonucleotide conjugate (AOC), is designed to suppress aberrant DUX4 expression, the root cause of FSHD. - The validated 2 mg/kg every-6-weeks dosing regimen is now being evaluated in the ongoing Phase III FORTITUDE-3 trial enrolling 200 patients. - FSHD affects approximately 45,000 to 87,000 people in the US and EU, with no currently approved disease-modifying therapies available.
- The FSHD market across the 7MM is projected to grow substantially by 2036, driven by the anticipated launch of disease-modifying therapies and increasing diagnosis rates. - In 2025, the United States accounted for approximately 45% of total diagnosed FSHD cases across the 7MM, with nearly 16,000 FSHD1 and 800 FSHD2 cases. - Novartis completed its acquisition of Avidity Biosciences in January 2026, gaining access to Delpacibart braxlosiran, with global regulatory submissions anticipated in 2028. - Promising pipeline candidates include del-brax, GYM329/RO7204239/RG6237, EPI-321, MDL-103, and Restem-L, targeting DUX4 suppression and myostatin inhibition.
- Novartis AG successfully raised $11 billion through investment-grade bonds across seven tranches to repay a bridge loan for its $12 billion acquisition of Avidity Biosciences, which closed last month. - The acquisition strengthens Novartis's innovative medicines portfolio with Avidity's Antibody Oligonucleotide Conjugates (AOCs™) platform that delivers RNA therapeutics directly to muscle tissue for genetic neuromuscular diseases. - The deal is projected to increase Novartis's expected annual sales growth rate from 5% to 6% between 2024 and 2029, with potential product launches anticipated before 2030. - Bond pricing came at favorable terms with the longest-dated 2056 bond priced at 0.9 percentage point over Treasuries, approximately 0.3 percentage point tighter than initial projections despite rising market borrowing costs.
