Avidity Biosciences, Inc. (Avidity) is a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates, or AOCs.
The company's proprietary AOC platform is designed to combine the specificity of monoclonal antibodies, or mAbs, with the precision of RNA therapeutics to target the root cause of diseases previously untreatable with such therapeutics.
The company's pipeline has three programs in potentially registrational clinical trial...
Avidity Biosciences, Inc. (Avidity) is a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates, or AOCs.
The company's proprietary AOC platform is designed to combine the specificity of monoclonal antibodies, or mAbs, with the precision of RNA therapeutics to target the root cause of diseases previously untreatable with such therapeutics.
The company's pipeline has three programs in potentially registrational clinical trials. Delpacibart etedesiran, abbreviated as del-desiran (formerly AOC 1001), is designed to treat people with myotonic dystrophy type 1 (DM1) and is in Phase 3 development with the global HARBOR trial. Delpacibart braxlosiran, or del-brax (formerly AOC 1020), is the first investigational therapy designed to directly target DUX4 in people living with facioscapulohumeral muscular dystrophy (FSHD) and is in Phase 1/2 development with the FORTITUDE trial. Delpacibart zotadirsen, or del-zota (formerly AOC 1044), is designed for people with Duchenne muscular dystrophy (DMD) and is in development with the Phase 2 EXPLORE44 Open-Label Extension (OLE) study. Del-zota is specifically designed for people with mutations amenable to exon 44 skipping (DMD44) and is the first of multiple AOCs the company is developing for DMD. Del-desiran, del-brax, and del-zota have all been granted Orphan Designation by the FDA and the European Medicines Agency (EMA), and Fast Track designation by the FDA. In addition, the FDA has granted del-desiran Breakthrough Therapy designation for the treatment of DM1 and granted del-zota Rare Pediatric Disease designation.
In February 2025, the company announced it will be reporting top-line del-zota data from its completed Phase 1/2 EXPLORE44 trial for people living with DMD44 at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March 2025. The company also announced it has now completed enrollment in the EXPLORE44-OLE study for people living with DMD44. The data from the Phase 1/2 EXPLORE44 and EXPLORE44-OLE studies will support the company's first BLA submission anticipated at year end 2025.
Additionally, the company shared its full year 2024 highlights across its three clinical development programs for del-zota, del-desiran and del-brax, and other pipeline advancements. These include:
Del-zota for DMD44
In February 2024, Avidity announced the FDA granted Rare Pediatric Disease designation for del-zota for the treatment of DMD44.
In August 2024, Avidity reported positive initial del-zota data from the 5 mg/kg cohort of the Phase 1/2 EXPLORE44 trial in people living with DMD44 which demonstrated remarkable delivery to skeletal muscle, exceptional, unadjusted increase of 25% in near full-length dystrophin production with a profound reduction in creatine kinase levels to near normal, and robust exon 44 skipping. Del-zota demonstrated favorable safety and tolerability with most treatment emergent adverse events being mild or moderate.
In addition to the participants rolling over from the Phase 1/2 EXPLORE44 trial, Avidity announced it was enrolling additional participants in the EXPLORE44 Open-label Extension (OLE) study to support a potential BLA submission at year end 2025. Enrollment in the EXPLORE44-OLE study is now complete.
Del-desiran for DM1
In March 2024, Avidity announced it achieved global regulatory alignment with FDA, EMA and other global regulatory authorities on the design of the del-desiran Phase 3 HARBOR study.
In March 2024, Avidity reported positive del-desiran long-term 4 mg/kg data from the MARINA-OLE study, which showed reversal of disease progression in people living with DM1 across multiple endpoints, including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data.
In May 2024, Avidity announced the FDA granted Breakthrough Therapy designation for del-desiran for the treatment of DM1.
Enrollment for the global Phase 3 HARBOR trial is ongoing and on track for completion in mid-2025.
Del-brax for FSHD
In June 2024, Avidity reported positive initial del-brax 2 mg/kg data at four months from the Phase 1/2 FORTITUDE trial which demonstrated remarkable and consistent reductions of greater than 50% in DUX4 regulated genes, mean reductions of 25% or greater in novel circulating biomarker and creatine kinase, trends of functional improvement, and favorable safety and tolerability in people living with FSHD.
In October 2024, Avidity announced the initiation of the biomarker cohort in the Phase 1/2 FORTITUDE trial of del-brax. 2 mg/kg of del-brax will be administered every six weeks, designed to ensure continuous suppression of DUX4.
Pipeline Advancements
In November 2024, Avidity announced the expansion of its pipeline into precision cardiology, including two wholly-owned candidates: one for PRKAG2 syndrome and one for PLN cardiomyopathy. In addition, Avidity shared details of its next-generation technology innovations with up to 30-fold improvements in delivery observed in preclinical studies.
In August 2024, Avidity announced it plans to advance additional DMD product candidates following robust del-zota data; a product candidate for exon 45 skipping is in IND-enabling studies.
In addition, the company announced its upcoming clinical and regulatory outlook for 2025 as the company accelerates the expansion of its capabilities to support potential launches of product candidates in clinical development and to potentially operate as a commercial organization. Avidity’s anticipated 2025 clinical and regulatory highlights include:
Del-zota for the treatment of DMD44:
Presentation of topline data from the EXPLORE44 trial in the first quarter of 2025.
Presentation of topline data from the ongoing EXPLORE44-OLE trial in the fourth quarter of 2025.
Potential BLA submission year at end 2025.
The FDA confirmed the accelerated approval path is available for del-zota and that the clinical data package from the EXPLORE44 program could support a BLA filing.
Del-desiran for the treatment of DM1:
Completion of enrollment of the ongoing Phase 3 HARBOR trial in mid-2025.
Update from the ongoing MARINA-OLE trial, including long-term 4mg/kg and safety data in the fourth quarter of 2025.
Publication of data analyses from the completed Phase 1/2 MARINA trial in 2025.
Planned marketing application submissions in 2026, including in the U.S. and European Union.
Del-brax for the treatment of FSHD:
Potential regulatory alignment on a global Phase 3 trial design in the second quarter of 2025.
Potential alignment on an accelerated approval path for the ongoing FORTITUDE biomarker cohort in the second quarter of 2025.
Completion of enrollment of the FORTITUDE biomarker cohort in the second quarter of 2025.
Presentation of topline data from the FORTITUDE trial in the second quarter of 2025.
Initiation of a global, potentially registrational trial in FSHD in the second quarter of 2025.
Strategy
The key elements of the company's strategy to harness the power of the company's AOC platform to develop a new class of drugs; continue advancing its three clinical stage product candidates in rare neuromuscular indications; expand its pipeline in its focus areas of neuromuscular and precision cardiology indications, as well as into additional tissue types; and build an agile and diverse company.
AOC Product Platform
The company is committed to delivering a new class of RNA therapeutics called AOCs, designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. It utilizes its proprietary AOC platform to design, engineer, and develop therapeutics that combine the specificity of mAbs with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with such therapeutics. All of the company's oligonucleotides target disease-related RNA. RNA is a polymeric molecule essential in the coding, decoding, regulation, and expression of genes. The company has accumulated deep experience regarding oligonucleotide therapeutics, modulation of RNA processes, antibody engineering and conjugation, and drug delivery techniques. It collectively refers to the know-how and proprietary technology borne out of this experience, and their systematic application in the design and development of its product candidates, as its AOC platform.
Approach
Based on the data-driven hypothesis that the delivery of oligonucleotides can be greatly enhanced by using antibodies as conjugates, the company's scientists have established a framework for screening potential cell surface protein-mAb pairs to determine which pairs are well suited to deliver active oligonucleotides to specific cell types. The company has identified multiple cell surface protein-mAb pairs that can deliver oligonucleotides into various tissue and cell types to induce pharmacologic changes. For example, it has employed AOCs built on a scaffold of a mAb or mAb fragment that binds with high selectivity and affinity to TfR1 to deliver oligonucleotides to cell types outside of the liver.
The company's deep experience with oligonucleotide therapeutics, modulation of RNA processes, antibody engineering and conjugation, and drug delivery techniques provides the foundation for its efforts to address the current limitations of oligonucleotide therapies. Its disruptive and broad AOC platform also affords the company's the option to deploy various types of oligonucleotides, including small interfering RNAs (siRNAs) and phosphorodiamidate morpholino oligomers (PMOs), whose specific mechanisms of action modify RNA function in different ways. The company has programs utilizing both siRNAs and PMOs in clinical development. This flexibility allows the company to use oligonucleotides that are tailored to modulate a given disease process. Mechanisms of these oligonucleotides can range from reducing the expression of a disease-related RNA with siRNAs to correcting aberrant processing of RNAs with splice-modifying oligonucleotides.
AOCs are designed to do the following: Combine the proven technologies of approved mAbs and oligonucleotides; Deliver to tissues previously untreatable with RNA therapeutics, starting with muscle and broadening to other tissues and cell types; and Scale with experienced manufacturers who are able to utilize well-established and scalable methods for manufacturing mAbs and oligonucleotides. The company also has the ability to use a single mAb across multiple programs, providing significant leverage around development costs and timelines associated with each incremental program.
Development Programs
The company is advancing and expanding its AOC pipeline to develop potential treatment options for patients and their families across a wide range of therapeutic areas. Its first AOC programs are from its rare neuromuscular disease franchise, where it has leveraged its deep experience with oligonucleotide therapeutics, modulation of RNA processes, antibody engineering and conjugation, and drug delivery techniques. The company has programs in its early-stage development pipeline through internal efforts and external collaborations that explore utilizing AOCs in additional indications, including cardiology and immunology. It has expanded beyond rare neuromuscular disorders and into precision cardiology, advancing two wholly-owned precision cardiology development candidates targeting rare genetic cardiomyopathies for PRKAG2 syndrome and PLN cardiomyopathy.
Clinical Programs
Del-desiran for the Treatment of DM1
Del-desiran is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary mAb that binds to the transferrin receptor 1 (TfR1) conjugated with an siRNA targeting DMPK mRNA. Del-desiran is being studied in the global Phase 3 HARBOR trial and in the ongoing MARINA-OLE trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints, including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Commission.
Solution
Del-desiran consists of a proprietary mAb that binds to TfR1 conjugated with an siRNA, siDMPK.19, targeted to DMPK RNA; and is designed to be administered to the patient as an intravenous infusion:
Addresses the underlying cause of the disease-DM1 is caused by an increase in the number of CUG triplet repeats occurring in the DMPK gene product. Del-desiran is designed to reduce the expression levels of DMPK RNA, thereby reducing the CUG burden in the nucleus and thereby releasing muscle blind-like protein to allow for normal mRNA processing;
Efficient delivery of drug substance to diseased cells-In an effort to solve for challenges identified in prior unsuccessful efforts to deliver an unconjugated oligonucleotide into muscle cells, the TfR1 antibody component of del-desiran facilitates efficient delivery of del-desiran to skeletal and cardiac muscle cells. Once inside the muscle cells, the siRNA component of del-desiran, siDMPK.19, acts to reduce levels of DMPK mRNA in both the nucleus and the cytoplasm; and
Reproducible and scalable therapeutic-As with all the company's AOCs, del-desiran is readily synthesized using well-established and scalable methods for manufacturing mAbs and oligonucleotides.
Phase 3 HARBOR Study
In June 2024, the company announced the initiation of its global Phase 3 HARBOR trial and began administration of del-desiran. It anticipates completion of enrollment of the ongoing Phase 3 HARBOR trial in mid-2025. The HARBOR trial is a randomized, placebo-controlled, double-blind pivotal study designed to evaluate del-desiran in approximately 150 people (ages 16 and older) living with DM1. The trial will be conducted at approximately 40 sites globally.
Phase 1/2 MARINA Clinical Trial and MARINA-OLE Study
The MARINA trial was a randomized, double-blind, placebo-controlled, Phase 1/2 clinical trial that enrolled 38 adults with DM1. The primary objective of this study was to evaluate the safety and tolerability of single and multiple ascending doses of del-desiran administered intravenously. The MARINA trial assessed the activity of del-desiran across key biomarkers, including spliceopathy, an important biomarker for DM1, and knockdown of DMPK mRNA. Though the Phase 1/2 trial was not powered to assess functional benefit, it explored the clinical activity of del-desiran in multiple measures of muscle function, including myotonia, muscle strength, measures of mobility, as well as patient reported outcomes and quality of life measures. Patients had the option to enroll in MARINA-OLE, an open label extension study, at the end of the treatment period. MARINA-OLE is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran in participants with DM1 who were previously enrolled in the MARINA Phase 1/2 trial. This trial continues to evaluate the safety, tolerability, PK, PD, and efficacy of del-desiran in participants enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical trial. All participants who completed the MARINA study rolled-over into the MARINA-OLE study and continue to receive del-desiran regardless of whether they received treatment or placebo in the MARINA study.
The sites participating in the MARINA-OLE study are all part of the Myotonic Dystrophy Clinical Research Network (DMCRN). The DMCRN is running a natural history study called Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1). The company has entered into an agreement supporting END-DM1, a non-interventional study designed to advance the understanding of disease progression in approximately 700 people with DM1.
Long-Term Del-desiran Data Reported in March 2024 from the Phase 2 MARINA-OLE Trial
Del-desiran data reported in March 2024 showed reversal of disease progression in people living with DM1 across multiple endpoints, including video hand opening time, or vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data.
In January 2025, the company announced that it will be presenting an update from the ongoing MARINE-OLE trial, including long-term 4mg/kg and safety data, in the fourth quarter of 2025. The company anticipates providing publication of data analyses from the completed Phase 1/2 MARINA trial in 2025. It also anticipates submitting marketing applications in 2026, including in the U.S. and European Union.
Del-brax for the Treatment of FSHD
The company is developing del-brax to treat the underlying cause of FSHD, one of the most common forms of muscular dystrophy. FSHD is a genetic muscle disorder in which the muscles of the face, shoulder blades, and upper arms are among the most affected. Symptoms usually begin before age 20, with weakness and atrophy of the muscles around the eyes and mouth, shoulders, abdominal muscles, upper arms, and lower legs, usually with asymmetric involvement. FSHD is caused by the aberrant expression, and subsequent translation, of a gene called double homeobox 4, or DUX4, which leads to cell death, immune response and oxidative stress.
The company's therapeutic strategy in FSHD employs an AOC based on its proprietary mAb that targets TfR1 to deliver an siRNA targeted to DUX4 mRNA. By directly targeting DUX4 RNA in the muscle, leading to destruction of the DUX4 transcripts, it can reduce the downstream effects, including cell death and oxidative stress.
Solution
Del-brax consists of the company's proprietary mAb that is designed to bind to TfR1 conjugated with an siRNA targeted to DUX4 mRNA to be administered as an intravenous infusion.
Addresses the underlying cause of the disease-del-brax is designed to reduce the expression of the DUX4 mRNA, thereby reducing the expression of the DUX4 protein resulting in reduced expression of the downstream genes that are believed to cause FSHD.
Efficient delivery of drug substance to diseased cells- The TfR1 antibody component of the AOC is designed to facilitate efficient delivery to skeletal and cardiac muscle cells, an advantage over other companies' previous unsuccessful efforts to deliver an unconjugated oligonucleotide into muscle cells.
Once inside the muscle cells, the siRNA component of del-brax, siDUX4, acts to reduce levels of DUX4 mRNA.
Reproducible and scalable therapeutic-As with all its AOCs, del-brax is readily synthesized using well-established and scalable methods for manufacturing mAbs and oligonucleotides.
Phase 1/2 FORTITUDE Clinical Trial
Del-brax is being studied in the Phase 1/2 FORTITUDE trial in adult and adolescent participants with FSHD. The FORTITUDE trial is a randomized, placebo-controlled, double-blind clinical trial designed to evaluate single and multiple doses of del-brax in approximately 100 participants with FSHD. The study is ongoing with dose escalation cohorts A and B fully enrolled and the biomarker cohort enrolling participants. The FORTITUDE study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of del-brax administered intravenously. Activity of del-brax will be assessed using key biomarkers, including DUX4-regulated muscle and circulating biomarkers and magnetic resonance imaging (MRI) measures of muscle volume and composition. Though the Phase 1/2 trial is not statistically powered to assess functional benefit, it will explore the clinical activity of del-brax, including measures of mobility and muscle strength, as well as patient reported outcomes and quality of life measures. Participants will have the option to enroll in FORTITUDE-OLE, an open-label extension study, once their participation in the FORTITUDE study is complete.
In October 2024, the company announced the initiation of the biomarker cohort in the FORTITUDE trial. The biomarker cohort will assess the impact of del-brax 2 mg/kg every six weeks in people living with FSHD, ages 16-70. The primary endpoints of the study are changes in DUX4 regulated gene expression and DUX4 regulated circulating biomarker. Enrollment is expected to be completed in the second quarter of 2025. In addition, enrollment of the FORTITUDE-OLE is ongoing.
The majority of sites participating in the FORTITUDE trial are part of the FSHD clinical trial research network, or FSHD CTRN. The company is partnering with the FSHD CTRN on the ongoing natural history study called Motor Outcomes to Validate Evaluations in FSHD or MOVE FSHD. The company is sponsoring the MOVE Plus, or MOVE+, sub-study which is enrolling approximately 100 participants in the U.S. The goal of MOVE+ is to enhance the community's understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can accelerate and support future clinical trial design.
In June 2024, the company reported positive initial 2 mg/kg del-brax data from the Phase 1/2 FORTITUDE trial which demonstrated remarkable and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with FSHD.
In January 2025, the company announced that it anticipates reaching regulatory alignment on a global Phase 3 trial design and alignment on a potential accelerated approval path for the ongoing FORTITUDE biomarker cohort in the second quarter of 2025. In addition, the company announced that the presentation of topline data from the FORTITUDE trial and the initiation of a global, potentially registrational trial in FSHD are also anticipated in the second quarter of 2025.
Del-zota for the Treatment of DMD44
Del-zota is being studied for the treatment of people living with DMD44 and is the first of multiple AOCs the company is developing for DMD. Del-zota is designed to deliver PMO to skeletal muscle and heart tissue to specifically skip exon 44 of dystrophin mRNA to enable production of near full-length dystrophin. Del-zota is in Phase 2 development as part of the EXPLORE44-OLE study in people living with DMD44. The FDA and European Commission granted Orphan designation for del-zota. The FDA has granted del-zota Rare Pediatric Disease designation and Fast Track designation.
Duchenne Muscular Dystrophy (DMD) Programs
The company is developing AOCs to treat the underlying cause of DMD and restore dystrophin levels. The oligonucleotides in its AOCs are designed to promote the skipping of specific exons to allow the production of near full-length dystrophin protein. The company's most advanced DMD program, del-zota, is designed to treat people with mutations amenable to Exon 44 skipping (DMD44). Del-zota is in clinical development in the Phase 2 EXPLORE44-OLE study. Additionally, its ongoing preclinical development programs target additional mutations that are amenable to exon-skipping, including Exon 45.
Solution
The company's development efforts in DMD are focused on AOCs based on PMOs that can induce exon skipping for Exon 44 and additional exons, including Exon 45, conjugated to its proprietary mAb targeting TfR1. Del-zota is the company's lead program in development for DMD and targets Exon 44. First, based on recent advances in the understanding of the splicing process and placement of skipping agents on pre-mRNA described in published literature, it has screened for and identified PMOs with optimized skipping activity. Second, the mAb targeting TfR1 allows for more efficient delivery to muscle cells, therefore allowing for better uptake of the PMO.
Phase 1/2 EXPLORE44 Clinical Trial and EXPLORE44-OLE Study
In August 2024, the company reported initial data from the 5 mg/kg cohort of its EXPLORE44 trial in people living with DMD44. These data demonstrated consistent delivery of PMO in skeletal muscle, an increase in the mean dystrophin production of 25% of normal and a mean increase of 37% in exon 44 skipping. In addition, del-zota showed greater than 80% reduction of creatine kinase compared to baseline in people living with DMD44. Additional data presented in early 2025 from the EXPLORE44 and EXPLORE44-OLE studies demonstrated significant and sustained reductions in CK in the 5 mg/kg cohort. Placebo-treated patients who continued into the EXPLORE44-OLE study showed a rapid decrease in CK after beginning treatment with del-zota.
The initial assessment from the randomized, double-blind, placebo-controlled EXPLORE44 trial assessed the safety and tolerability for 26 participants across two dose levels (5 mg/kg and 10 mg/kg). Del-zota demonstrated favorable safety and tolerability, with most treatment emergent AEs mild or moderate in participants with DMD44. Two participants discontinued from the study due to treatment emergent adverse events: one due to a serious adverse event of anaphylaxis which fully resolved, and one due to moderate infusion related reaction. For the 5 mg/kg cohort, participants received three doses of 5 mg/kg of del-zota, or placebo, every six weeks.
Enrollment is now complete for the EXPLORE44 study. Participants in the EXPLORE44 trial had the option to enroll in the EXPLORE44-OLE for del-zota. In addition, the company announced it was enrolling additional participants in the EXPLORE44-OLE study to support a BLA submission anticipated at year-end 2025.
Enrollment in the EXPLORE44-OLE study is now complete. The company also announced plans to advance additional exon-skipping candidates from its DMD franchise. Exon 45 is currently in IND-enabling studies for the treatment of people living with DMD mutations amenable to exon 45 skipping (DMD45).
In January 2025, the company announced that the FDA confirmed an accelerated approval path is available for del-zota and that the clinical data package from the EXPLORE44 program could support a BLA filing. Avidity is now planning a potential BLA submission anticipated at year-end 2025. The company also announced that it will be presenting topline data from the EXPLORE44 trial in the first quarter of 2025, and topline data from the ongoing EXPLORE44-OLE study in the fourth quarter of 2025.
Discovery Programs
Opportunities in Additional Neuromuscular Diseases, Cardiology and Immunology
The company is committed to the advancement and expansion of its pipeline with multiple research and development candidates to treat conditions in skeletal muscle, cardiology, and immunology as part of its internal discovery efforts and collaborations with Eli Lilly and Company (Lilly) and Bristol-Myers Squibb Company (BMS). In November 2024, the company announced that it had expanded beyond rare neuromuscular disorders and opened up a new therapeutic field, precision cardiology, to address the root cause of genetic diseases of the heart. It is advancing its first two wholly-owned precision cardiology development candidates targeting rare genetic cardiomyopathies: AOC 1086 targeting PLN (phospholamban) cardiomyopathy and AOC 1072 targeting PRKAG2 (Protein Kinase AMP-activated non-catalytic subunit Gamma 2) syndrome. All of the preclinical programs have been engineered using the company's AOC platform technology.
The company is collaborating with Lilly for the discovery, development, and commercialization of AOCs directed to up to six selected mRNA targets in immunology and other select indications outside of muscle. Through its research collaboration with BMS and its internal discovery efforts, the company's development activities target multiple cardiac-specific indications.
Intellectual Property
As of December 31, 2024, the company’s intellectual property portfolio consisted of 40 issued U.S. patents and 30 pending U.S. patent applications that the company owns or co-owns. Collectively, these patent rights relate to various aspects of its AOC product candidates and technology platform. In addition, the company has an exclusive license to certain patent rights from the University of Alberta and Fred Hutchinson Cancer Center. In addition to filing and prosecuting patent applications in the United States, the company often files counterpart patent applications in additional countries and jurisdictions. The company also files patent applications pursuant to the Patent Cooperation Treaty, or PCT. The company's PCT patent applications are in the first phase of the PCT process, which is the international phase, in which patent protection is pending under a single patent application filed with the United States Patent and Trademark Office, or USPTO, as a contracting state of the PCT. These PCT patent applications have not yet entered the second phase of the PCT process, which is the national and regional phase, in which rights are continued by filing necessary documents with the patent offices of separate contracting states of the PCT. The national phase of the PCT patent application process occurs 30 months after the earliest priority date of the PCT patent application.
Intellectual Property Relating to Del-Desiran (AOC 1001)
With regard to del-desiran, as of December 31, 2024, the company owned 5 issued U.S. patents, 3 pending U.S. patent applications, 7 granted foreign patents, and 23 pending patent applications in foreign jurisdictions, including Australia, Brazil, Canada, China, Europe, Hong Kong, Israel, Japan, Mexico, Singapore, Taiwan, New Zealand, and South Korea. These patent rights relate to del-desiran composition of matter, formulations containing del-desiran, methods of manufacturing, and methods of treating diseases, using del-desiran. Any patents issued from these applications are expected to expire in 2038-2041; however, a patent term extension may be available.
Intellectual Property Relating to Del-Brax (AOC 1020) and Other FSHD AOC Product Candidates
With regard to del-brax and other FSHD AOC product candidates, as of December 31, 2024, the company owned or co-owned 8 issued U.S. patents, 5 pending U.S. patent applications, 1 granted foreign patent, 24 pending patent applications in foreign jurisdictions, including Australia, Brazil, Canada, China, Europe, Hong Kong, Israel, Japan, Mexico, Singapore, Taiwan, New Zealand, and South Korea. These patent rights relate to the del-brax and other FSHD AOC composition of matter, formulations containing del-brax and other the FSHD AOC, methods of manufacturing, and methods of treating diseases, using its FSHD AOC. Any patents issued from these applications are expected to expire in 2041-2042; however, a patent term extension may be available. The company has one patent family licensed from Fred Hutchinson Cancer Center, which includes one issued U.S. patent, one pending U.S. patent application, one granted foreign patent, and one pending application in Europe.
Intellectual Property Relating to Del-Zota (AOC 1044) and Other DMD AOC Product Candidates
With regard to del-zota and other DMD AOC product candidates, as of December 31, 2024, the company owned 6 issued U.S. patents, 8 pending U.S. patent applications, 3 granted foreign patents, 39 pending patent applications in various countries and regions, including Australia, Canada, China, Europe, Hong Kong, Israel, Japan, Mexico, Singapore, Taiwan, New Zealand, and South Korea; and one pending patent application filed pursuant to the PCT. These patent rights relate to del-zota and other DMD AOCs composition of matter, formulations containing del-zota and other DMD AOCs, methods of manufacturing, and methods of treating diseases, using del-zota and other DMD AOCs. Any patents issued from these applications are expected to expire in 2038-2044; however, a patent term extension may be available.
Intellectual Property Relating to AOC 1072 and Other PRKAG2 AOC Product Candidates
With regard to AOC 1072 and other PRKAG2 AOC product candidates, as of December 31, 2024, the company owned two pending U.S. patent applications, and one pending patent application filed pursuant to the PCT. These patent rights relate to the AOC 1072 and other PRKAG2 AOC composition of matter, formulations containing AOC 1072 and other the PRKAG2 AOC, methods of manufacturing, and methods of treating diseases, using its PRKAG2 AOC. Any patents issued from these applications are expected to expire in 2044-2045; however, a patent term extension may be available.
Intellectual Property Relating to AOC 1086 and Other PLN AOC Product Candidates
With regard to AOC 1086 and other PLN AOC product candidates, as of December 31, 2024, the company owned one pending U.S. patent applications, and one pending patent application filed pursuant to the PCT. These patent rights relate to the AOC 1086 and other PLN AOC composition of matter, formulations containing AOC 1086 and other the PLN AOC, methods of manufacturing, and methods of treating diseases, using its PLN AOC. Any patents issued from these applications are expected to expire in 2044; however, a patent term extension may be available.
Intellectual Property Relating to the company's AOC Product Platform
As of December 31, 2024, the company owned 29 families of U.S. and foreign patents and patent applications generally covering its AOC product platform. These families include 37 issued U.S. patents, 26 granted foreign patents, 30 pending U.S. patent applications, 4 pending PCT patent applications, and 133 pending foreign patent applications in Europe, Australia, Brazil, Canada, China, Israel, Hong Kong, Japan, South Korea, Mexico, Singapore, New Zealand, and Taiwan, relating to key aspects and components of the company's AOC product platform systems. The company's patent applications contain claims covering (i) proprietary antibodies; (ii) proprietary oligonucleotide chemical structures; (iii) proprietary oligonucleotide sequences; (iv) proprietary AOC structures; and (v) methods for manufacturing and using its AOC technologies. Some of these AOC platform cases generically cover the company's various product candidates. The issued U.S. patents and any U.S. patents issuing from the company's pending U.S. patent applications are expected to expire between 2037 and 2045. The company has one patent family licensed from the University of Alberta, which includes 2 issued U.S. patents, one pending U.S. patent application, 4 granted foreign patents, and six pending applications in Europe, Canada, China, Japan, South Korea, and Hong Kong. Additionally, the company has one patent family licensed from GenAhead Bio Inc, which includes 2 pending U.S. applications, 4 granted foreign patents, and 3 pending applications in Europe, China, and Japan.
The company has filed trademark applications for registration of the Avidity, Avidity Bioscience logo, del-brax, del-desiran, del-zota, EXPLORE44, EXPLORE44 logo, EXPLORE44-OLE logo, FORTITUDE, FORTITUDE logo, HARBOR, HARBOR logo mark, MARINA, MARINA logo, MARINA-OLE, and MARINA-OLE logo marks with the United States Patent and Trademark Office and certain foreign trademark offices.
Research Collaboration with Bristol Myers Squibb
In November 2023, the company entered into a Research Collaboration and License Agreement with BMS, or the BMS Collaboration Agreement, to expand on the research with MyoKardia for up to five targets utilizing its proprietary AOC platform technology.
Research Collaboration with Lilly
In April 2019, the company entered into a research collaboration and license agreement, or the Lilly Agreement, with Lilly for the discovery, development and commercialization of antibody-oligonucleotide conjugate products, or Products, in immunology and other select indications on a worldwide basis.
Under the Lilly Agreement, the company granted Lilly an exclusive, worldwide, royalty-bearing license under its technology to research, develop, manufacture, and sell products directed to up to six mRNA targets. Lilly has the right to sublicense its rights under the Lilly Agreement, subject to certain conditions. Lilly granted the company a non-exclusive license under certain Lilly technology solely to conduct research under the Lilly Agreement. The company retains the right to use its technology to perform its obligations under the Lilly Agreement and for all purposes not granted to Lilly. The company agreed that it will not, itself or with a third party, research, develop, manufacture, or commercialize, or otherwise exploit any compound or product directed against targets subject to the collaboration.
Research Collaboration with MyoKardia, a wholly owned subsidiary of BMS
In December 2020, the company entered into a research collaboration, referred to as the MyoKardia Agreement, with MyoKardia, a wholly-owned subsidiary of BMS, to demonstrate the potential utility of AOCs in cardiac tissue by leveraging MyoKardia's genetic cardiomyopathy platform, including among other aspects, its novel target discovery engine and proprietary cardiac disease models. Under the terms of the MyoKardia Agreement, in July 2023, BMS, as the successor in interest to MyoKardia, exercised its option to negotiate and enter into a license agreement covering AOCs that modulate the function of cardiovascular targets. The research collaboration with MyoKardia was terminated in November 2023 upon execution of the BMS Collaboration Agreement.
History
Avidity Biosciences, Inc. was founded in 2012. The company was incorporated in 2012.
