Arcturus Therapeutics


LUNAR-OTC (Co-Development 50/50)

In Jan 2018, Arcturus Therapeutics and CureVac announced that they entered into a broad strategic collaboration to jointly discover, develop and commercialize novel messenger RNA (mRNA) therapeutics. Under the agreement, the companies will collaborate to develop up to four molecular therapy products for rare diseases using Curevac’s optimized natural mRNA sequence (RNAoptimizer®) and Arcturus’ lipid-mediated nucleic acid delivery system (LUNAR®). The agreement focuses on developing mRNA therapeutics for enzyme replacement and antibody generation. Development costs will be shared between the companies, with plans to co-commercialize products in the future under a profit sharing arrangement. The first mRNA therapy to be jointly developed and potentially commercialized by the companies will target ornithine transcarbamylase (OTC) deficiency, a genetic disease characterized by the accumulation of ammonia in the blood. The collaboration also grants CureVac access to the full suite of Arcturus’ lipid-mediated delivery intellectual property to enable the development of additional mRNA product candidates.

Johnson & Johnson


In October 2017, Arcturus entered into a partnership with Janssen, a Johnson & Johnson company, to develop and commercialize nucleic acid-based drug products for the treatment of specified diseases, including HBV, using Arcturus’ LUNAR lipid-mediated delivery platform and UNA oligomer chemistry. Under the agreement, facilitated by J&J Innovation, Arcturus received an upfront cash payment and will receive R&D support, as well as pre-clinical, development, and sales milestone payments, and royalty payments on any future licensed product sales. Janssen will assume responsibility for development costs and all commercialization costs associated with the program.



In December 2016, Arcturus entered into a partnership with Takeda to develop RNA-based therapeutic candidates for the treatment of NASH and other GI-related disorders, utilizing Arcturus’ wholly-owned therapeutic delivery platform, LUNAR, and UNA oligomer chemistry.


LUNAR-RPL (Replicon RNA)

In October 2017, Arcturus entered into a research collaboration and worldwide license agreement with Synthetic Genomics Inc., to develop self-amplifying RNA-based vaccines and therapeutics in both human and animal health. The collaboration will bring together Arcturus’ LUNAR® lipid-mediated delivery platform with Synthetic Genomics’ RNA replicon platform to potentially enable more efficacious and lower cost vaccines and therapeutics.



In October 2015, Arcturus has partnered with Ultragenyx Pharmaceutical, Inc. to develop RNA medicines that treat rare and ultra-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 unlocked nucleomonomer agent (UNA) Oligomer chemistry and 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.


HBV program (ARB-1467)

Arbutus Biopharma is using Arcturus’ innovative unlocked nucleomonomer agent (UNA) Oligomer technology to develop RNA medicines to treat hepatitis B virus (HBV) infections. Approximately 240 million people worldwide are chronically infected with HBV and over 700,000 people die each year from liver disease caused by HBV. There is no effective cure for HBV infection. Patients may be treated with supportive treatments or liver transplant to ease symptoms and antiviral medications to stop the virus from replicating. The hepatitis B virus can develop resistance to currently available antiviral drugs. Arbutus has developed an RNA medicine, called ARB-1467, containing three RNAi molecules that work to stop all HBV mRNA from being turned into protein. Therefore, there is less chance of the virus developing resistance to this drug than to conventional antivirals. A Phase I clinical trial showed this drug is safe and well tolerated in healthy adults. A Phase II clinical trial testing ARB-1467 in HBV infected patients is now underway.

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