Avidity Biosciences
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2013-01-01
- Employees
- 253
- Market Cap
- $5.1B
- Introduction
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.
Avidity Biosciences Advances RNA Therapeutics with Clinical Trials for Rare Diseases
Avidity Biosciences, Inc., a biopharmaceutical company, is making strides in the development of RNA therapeutics aimed at treating rare diseases. The company's lead product candidate, AOC 1001, is currently in phase 1/2 clinical trials for myotonic dystrophy type 1, with other products targeting duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy also in clinical development.
Eli Lilly Partners with Rznomics in $1.3B Deal to Develop RNA Editing Therapy for Hereditary Hearing Loss
• Rznomics Inc. has secured a potential $1.35 billion global license option agreement with Eli Lilly to codevelop an RNA editing-based gene therapy targeting hereditary hearing loss.
• Under the partnership structure, Rznomics will lead initial R&D efforts to discover and develop the therapy, while Lilly will handle subsequent development and commercialization phases.
• The deal represents a significant expansion of Lilly's gene therapy pipeline for hearing disorders, though specific details regarding up-front payments and option terms remain undisclosed.
AbbVie and ADARx Form $335 Million Collaboration to Develop Next-Generation siRNA Therapies
• AbbVie has invested $335 million upfront in a strategic collaboration with ADARx Pharmaceuticals to develop small interfering RNA therapeutics across neuroscience, immunology, and oncology.
• The partnership combines ADARx's proprietary RNA technology for precise mRNA silencing with AbbVie's expertise in antibody engineering, ADCs, and tissue delivery approaches to overcome siRNA targeting challenges.
• Beyond the initial payment, ADARx is eligible to receive billions in additional contingent payments including milestone payments and tiered royalties as the collaboration advances.
Bulevirtide Shows Durable Response in Chronic Hepatitis Delta Virus After Treatment Cessation
• Final data from Gilead's Phase 3 MYR301 study reveals 36% of chronic HDV patients maintained virologic suppression for nearly two years after stopping bulevirtide treatment.
• Notably, 90% of patients who achieved undetectable HDV RNA for ≥96 weeks during treatment remained virus-free after discontinuation, with no relapses occurring in the second year of follow-up.
• The findings, presented at EASL Congress 2025, demonstrate bulevirtide's potential as the first effective monotherapy for chronic HDV that maintains benefits even after treatment ends.
Lilly Partners with Creyon Bio in $1B+ Deal to Develop AI-Designed RNA Therapies
• Eli Lilly and Creyon Bio have entered a global licensing and multi-target research collaboration worth potentially over $1 billion to develop RNA-targeted oligonucleotide therapies for various diseases.
• Creyon Bio will receive $13 million upfront, including cash and equity purchase, and will apply its AI-Powered Oligo Engineering Engine to design drug candidates for Lilly's targets on accelerated timelines.
• The partnership aims to create safer and more effective therapies by using quantum chemistry principles rather than traditional trial-and-error screening, representing a significant advancement in nucleic acid drug development.
GeoVax Advances Gedeptin Gene Therapy for Solid Tumors with Expanded Patent Portfolio and Promising Clinical Data
• GeoVax has strengthened its Gedeptin therapy platform with newly allowed patent claims covering the synergistic combination of gene therapy with radiation for treating various solid tumors.
• Clinical data presented at AACR 2025 demonstrated Gedeptin's safety profile and efficacy in heavily pretreated head and neck cancer patients, with median progression-free and overall survival of 7.0 months.
• The company is advancing to a Phase 2 trial combining Gedeptin with pembrolizumab (Keytruda) as a neoadjuvant therapy for relapsed head and neck cancer, leveraging potential synergy with immune checkpoint inhibitors.
Shape Therapeutics to Present Breakthrough RNA Editing Data for CNS Therapies at ASGCT Annual Meeting
• Shape Therapeutics will showcase multiple presentations at ASGCT 2025, including data demonstrating up to 88% targeted RNA editing in primate brain using systemically delivered AAV5-based capsid variants.
• The company's innovative approach combines programmable RNA editing technology (RNAfix®) with targeted AAV.ai delivery systems and TruStable™ manufacturing platforms to advance gene therapy development.
• Encoded Therapeutics will also present at ASGCT, highlighting progress in their CNS gene therapy programs for Angelman syndrome, chronic pain, and Alzheimer's disease using engineered AAV vectors.
Ugandan Scientist Secures US Patent for Revolutionary Cancer Treatment Using CRISPR Technology
• Dr. Mathias Magoola of Dei BioPharma has secured a US patent for a groundbreaking cancer treatment that uses guided RNA-Cas9 complex to target and destroy mutated genes responsible for cancer.
• The innovative therapy targets all types and stages of cancer without harming healthy cells, eliminating severe side effects commonly associated with conventional treatments like chemotherapy and radiation.
• President Museveni has pledged full government support for Dei BioPharma's mission, emphasizing the need to expedite the National Drug and Health Products Act to enable global distribution of Ugandan-made pharmaceuticals.
Isomorphic Labs Secures $600 Million to Advance AI-Powered Drug Discovery Platform
• Alphabet subsidiary Isomorphic Labs has raised $600 million in its first external funding round led by Thrive Capital, with participation from GV and Alphabet to develop its AI drug design engine.
• The company, founded by Google DeepMind CEO Sir Demis Hassabis, leverages breakthrough AI models including AlphaFold 3 to predict molecular structures and interactions for drug discovery across multiple therapeutic areas.
• Isomorphic Labs has established partnerships with pharmaceutical giants Eli Lilly and Novartis, while developing internal programs focused primarily on oncology and immunology treatments.
iOncologi Acquires TargImmune Therapeutics to Advance Novel Immunotherapies for Solid Tumors
• iOncologi, Inc. has acquired at least 99% of TargImmune Therapeutics' shares, creating a strengthened platform for developing cutting-edge immunotherapies targeting solid tumors.
• The strategic acquisition combines two experienced drug development teams while allowing TargImmune to continue operating in Basel, Switzerland as a wholly owned subsidiary of Florida-based iOncologi.
• This business move aligns with iOncologi's broader strategy to expand its therapeutic pipeline and accelerate the development of next-generation RNA-based immunotherapies for patients with limited treatment options.
Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas
• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics.
• February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million.
• Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.
UC Santa Cruz Clinical Lab Receives CLIA Certification for Revolutionary Pediatric Cancer RNA Testing
• The UC Santa Cruz Colligan Clinical Diagnostic Laboratory has received CLIA certification, enabling them to begin accepting patient samples for specialized RNA analysis in pediatric cancer cases.
• The lab's innovative Comparative Analysis of RNA Expression (CARE) test has shown superior effectiveness, identifying potential treatment options in 68% of tumor samples compared to 46% with traditional DNA testing.
• The facility plans to initially process six patient samples weekly through UCSF, with future expansion to other medical facilities and development of additional novel RNA-based cancer diagnostic tests.
Gensaic and Novo Nordisk Partner to Develop Novel Precision Therapeutics Platform
• Gensaic and Novo Nordisk have formed a strategic collaboration to develop next-generation precision therapeutics, combining Gensaic's innovative delivery platform with Novo's therapeutic expertise.
• The partnership aims to advance novel treatment approaches across multiple therapeutic areas, leveraging advanced drug delivery technologies to enhance therapeutic efficacy and patient outcomes.
• This collaboration represents a significant step forward in precision medicine development, with potential applications in metabolic disorders and other chronic diseases.
RNA Editing Breakthroughs Show Clinical Promise with Wave Life Sciences Leading First Human Results
• Wave Life Sciences achieves first-ever RNA editing results in humans for alpha-1 antitrypsin deficiency, demonstrating therapeutic protein level increases with a single dose treatment.
• RNA editing emerges as a potentially safer alternative to DNA editing, with companies like Ascidian Therapeutics and Korro Bio advancing clinical trials for rare genetic disorders.
• The field has evolved significantly over the past decade, with new editing capabilities and delivery methods opening potential treatment opportunities for over 10 million patients.
U.S. Gene Therapy Market Projected to Reach $22.23 Billion by 2034, Growing at 19.8% CAGR
• The U.S. gene therapy market is expected to grow from $4.37 billion in 2025 to $22.23 billion by 2034, driven by increasing investments, rising prevalence of chronic diseases, and integration of advanced technologies.
• Gene silencing has emerged as the dominant segment in the market, while oncology applications currently lead but neurology treatments are anticipated to gain significant market share in the coming decade.
• Artificial intelligence is revolutionizing gene therapy development by analyzing genomic data, identifying therapeutic targets, enabling personalized treatment strategies, and optimizing manufacturing processes to reduce costs.
FDA Expands Evrysdi Approval to Include Infants Under Two Months with Spinal Muscular Atrophy
• Roche and PTC Therapeutics' oral SMA drug Evrysdi (risdiplam) receives FDA approval for use in infants younger than two months, enabling treatment across all age groups from newborns to adults.
• The RAINBOWFISH study demonstrated significant efficacy, with treated pre-symptomatic babies achieving key developmental milestones including sitting, standing, and walking within 12 months.
• The expanded approval positions Evrysdi to compete directly with Biogen's Spinraza and Novartis' Zolgensma, while offering the advantage of at-home oral administration.
Isomorphic Labs to Launch Clinical Trials for AI-Designed Drugs in 2025
• Isomorphic Labs, a Google DeepMind spinoff, anticipates initiating clinical trials for AI-designed drugs by the end of 2025, potentially revolutionizing drug discovery.
• CEO Demis Hassabis aims to drastically reduce the drug discovery timeline from a decade to mere weeks or months using AI-driven approaches.
• Isomorphic Labs has established strategic research collaborations with Eli Lilly and Novartis, signaling growing confidence in AI's role in pharmaceutical R&D.
• The company's platform leverages DeepMind's AlphaFold AI to model molecular structures and predict interactions, accelerating the identification of promising drug candidates.
Arcturus Therapeutics Poised for Growth with Cystic Fibrosis and OTCD Therapies
• Arcturus Therapeutics anticipates Phase 2 LunairCF study data for ARCT-032 in cystic fibrosis patients unresponsive to existing modulators, expected in the first half of 2025.
• The company plans to release data from ARCT-810 trials for ornithine transcarbamylase deficiency (OTCD), focusing on biomarkers to potentially accelerate approval pathways.
• Regulatory developments in Japan for Arcturus' COVID-19 treatment, Kostaive, coupled with existing orders, indicate a robust revenue stream for the company.
Ionis Pharmaceuticals Advances Neurological and Cardiovascular Therapies with New Clinical Trials and FDA Approvals
Ionis Pharmaceuticals is set to enter a new chapter in 2025, focusing on advancing treatments for serious neurological diseases and cardiovascular conditions. The company has outlined plans for Phase 3 trials for ION582 for Angelman syndrome, zilganersen for Alexander disease, and ION464 for multiple system atrophy. Additionally, Ionis has received FDA approval for TRYNGOLZA™ (olezarsen) to reduce triglycerides in adults with familial chylomicronemia syndrome and for WAINUA™ (eplontersen) for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis.
Dyne Therapeutics' DYNE-101 Shows Promise in DM1 Trial, FDA Grants Fast Track Designation
• Dyne Therapeutics' DYNE-101 demonstrates compelling results in Phase 1/2 ACHIEVE trial for myotonic dystrophy type 1 (DM1).
• The FDA grants Fast Track designation to DYNE-101, expediting its development and regulatory review process.
• Dyne plans to initiate a global Registrational Expansion Cohort, aiming for U.S. Accelerated Approval submission in H1 2026.
• DYNE-251 for Duchenne muscular dystrophy (DMD) is also advancing, with potential regulatory submissions expected in early 2026.
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