Business Development
Arcturus Business Development Opportunities
Arcturus Therapeutics is actively engaged in partnering discussions. Please contact[email protected] regarding any business development opportunities.
Partnered RNA Medicines
Partnered RNA Medicines
Infectious Disease Vaccines
In December 2022, Arcturus partnered with CSL Seqirus for the research, development, manufacture, and global commercialization of infectious disease vaccines. The collaboration combines CSL Seqirus’ established global vaccine commercial and manufacturing infrastructure with Arcturus’ manufacturing expertise and innovative STARR™ self-amplifying mRNA vaccine and LUNAR® delivery platform technologies. Arcturus will bring its mRNA design and modification expertise, LUNAR® lipid nanoparticle (LNP) technology and manufacturing know-how, which has enabled the Company’s low dose, lyophilized and durable self-amplifying mRNA vaccines against COVID-19. Under the terms of the agreement, Arcturus will provide CSL Seqirus with a license to its STARR™ self-amplifying mRNA technology, LUNAR® lipid-mediated delivery, along with mRNA drug substance and drug product manufacturing expertise. CSL Seqirus will lead development and commercialization of vaccines under the collaboration. The collaboration plans to advance vaccines against SARS-CoV-2 (COVID-19), influenza, pandemic preparedness as well as three other globally prevalent respiratory infectious diseases.
LUNAR®-GSDIII and LUNAR®RARE
In October 2015, Arcturus has partnered with Ultragenyx Pharmaceutical, Inc. to develop RNA medicines that treat rare diseases. This collaboration combines Arcturus’ expertise in RNA medicines with Ultragenyx’ proven ability in rare disease drug development. In this partnership, Arcturus is exploiting the versatility of our proprietary Lipid-enabled and Unlocked Nucleic Acid modified RNA (LUNAR®) lipid-mediated delivery platform to design and optimize mRNA medicines for rare disease targets. These medicines will be developed and commercialized by Ultragenyx. Ultragenyx has advanced LUNAR®-GSDIII into clinical development.
Internal Programs
Internal Programs
LUNAR®-OTC
The LUNAR®-OTC (ARCT-810) development program addresses ornithine transcarbamylase (“OTC”) deficiency, a rare, genetic disease caused by mutations in the OTC gene that lead to dysfunctional or deficient OTC levels. Our LUNAR®-OTC development candidate, ARCT-810, uses our LUNAR® platform to deliver normal OTC mRNA into liver cells, where OTC is produced and functions, to produce normal functioning OTC with possible disease-modifying effects. Our LUNAR®-OTC approach has the potential to treat the underlying defect that causes the debilitating symptoms of OTC deficiency, rather than mitigating symptoms by sequestering ammonia. LUNAR®-OTC has received orphan drug designation from the FDA for treatment of OTC deficiency. LUNAR®-OTC has received approval from the UK Health Research Authority to initiate a Phase 2 clinical study. Worldwide development and commercialization rights to ARCT-810 are entirely held by Arcturus.
LUNAR®-CF (ARCT-032 Grant by CFF)
ARCT-032 (LUNAR®-CF) LUNAR®- program addresses cystic fibrosis lung disease, a progressive lung disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (“CFTR”) gene. In December 2020, we announced the selection and advancement of ARCT-032 as a development candidate for treatment of cystic fibrosis. ARCT-032 uses our LUNAR® platform to deliver codon-optimized CFTR mRNA into airway epithelial cells. This allows airway cells to produce functional CFTR protein using their native translational machinery and protein trafficking pathways. This approach has the potential to treat the underlying defect that causes CF lung disease, regardless of what mutation type a patient has. The Cystic Fibrosis Foundation (“CFF”) has recognized the potential of the LUNAR®-CF program and has partnered with us to develop this important therapy. ARCT-032 represents the first LUNAR®-based mRNA therapeutic that will be delivered by the inhaled route, offering direct delivery to the affected airways with the potential to restore functional CFTR. We have announced selection of a development candidate and plan to initiate a CTA in 2022.
Technology Platforms
Technology Platforms
Arcturus’ versatile RNA therapeutics and LNP Delivery Systems platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (patents and patent applications issued in the U.S., Europe, Japan, China and other countries). These technologies are potentially available for licensing as part of a collaboration. Please contact [email protected] regarding any business development opportunities.
- LUNAR® LNP Lipid-mediated delivery
- STARR™ mRNA Technology
- RNA Manufacturing Expertise
Partnered RNA Medicines
Infectious Disease Vaccines
In December 2022, Arcturus partnered with CSL Seqirus for the research, development, manufacture, and global commercialization of infectious disease vaccines. The collaboration combines CSL Seqirus’ established global vaccine commercial and manufacturing infrastructure with Arcturus’ manufacturing expertise and innovative STARR™ self-amplifying mRNA vaccine and LUNAR® delivery platform technologies. Arcturus will bring its mRNA design and modification expertise, LUNAR® lipid nanoparticle (LNP) technology and manufacturing know-how, which has enabled the Company’s low dose, lyophilized and durable self-amplifying mRNA vaccines against COVID-19. Under the terms of the agreement, Arcturus will provide CSL Seqirus with a license to its STARR™ self-amplifying mRNA technology, LUNAR® lipid-mediated delivery, along with mRNA drug substance and drug product manufacturing expertise. CSL Seqirus will lead development and commercialization of vaccines under the collaboration. The collaboration plans to advance vaccines against SARS-CoV-2 (COVID-19), influenza, pandemic preparedness as well as three other globally prevalent respiratory infectious diseases.
LUNAR®-GSDIII and LUNAR®RARE
In October 2015, Arcturus has partnered with Ultragenyx Pharmaceutical, Inc. to develop RNA medicines that treat rare diseases. This collaboration combines Arcturus’ expertise in RNA medicines with Ultragenyx’ proven ability in rare disease drug development. In this partnership, Arcturus is exploiting the versatility of our proprietary Lipid-enabled and Unlocked Nucleic Acid modified RNA (LUNAR®) lipid-mediated delivery platform to design and optimize mRNA medicines for rare disease targets. These medicines will be developed and commercialized by Ultragenyx. Ultragenyx has advanced LUNAR®-GSDIII into clinical development.
News & Events
March 28, 2023
Arcturus Therapeutics Announces Fourth Quarter 2022 Financial Update and Pipeline Progress
January 31, 2023
Arcturus Therapeutics Announces Clinical Trial Application for ARCT-032 Received Approval to Proceed into First-in-Human Studies to Treat Cystic Fibrosis
December 19, 2022
Arcturus Therapeutics to Present at the 41st Annual J.P. Morgan Healthcare Conference
December 13, 2022
Arcturus Appoints John Markels, Ph.D. to its Board of Directors
December 12, 2022
CSL Announces Closing of Global Collaboration and Licensing Agreement with Arcturus Therapeutics
November 9, 2022
Arcturus Therapeutics Announces Third Quarter 2022 Financial Update and Pipeline Progress
November 8, 2022
Arcturus Therapeutics to Present at the Following Investor Conferences in November
November 1, 2022
Arcturus Announces Collaboration with CSL to Develop and Commercialize Self-amplifying mRNA Vaccines
October 26, 2022
Arcturus Therapeutics to Report Third Quarter 2022 Financial Results and Provide Corporate Update on November 9, 2022
September 27, 2022
Arcturus Therapeutics to Attend the Following Investor Conferences
September 1, 2022
Arcturus Therapeutics to Attend the Following Investor Conferences
August 31, 2022
Arcturus Announces $63.2 Million Award from the U.S. Government to Support Development of Self-amplifying mRNA Vaccine for Rapid Pandemic Influenza Response
August 18, 2022
Arcturus Reports Durability Data Against Omicron Variants, Including BA.5, at Six-Month Timepoint Following Booster with ARCT-154 COVID-19 Vaccine
August 9, 2022
Arcturus Therapeutics Announces Second Quarter 2022 Financial Update and Pipeline Progress
August 1, 2022
Arcturus Therapeutics to Report Second Quarter 2022 Financial Results and Provide Corporate Update on August 9, 2022
May 9, 2022
Arcturus Therapeutics Announces First Quarter 2022 Financial Update and Pipeline Progress
May 5, 2022
Arcturus Reports Strong Three-Month Durability Results from ARCT-154 Booster Trial
April 27, 2022
Arcturus Therapeutics to Report First Quarter 2022 Financial Results and Provide Corporate Update on May 9, 2022
April 20, 2022
Arcturus Announces Self-amplifying COVID-19 mRNA Vaccine Candidate ARCT-154 Meets Primary Efficacy Endpoint in Phase 3 Study
March 25, 2022
Arcturus Therapeutics to Participate in the Cantor Fitzgerald Virtual Rare Orphan Disease Summit