Platforms

RNA/mRNA Medicine Platforms in Rare Diseases and Vaccines

Partnership Platform

Program
Partner
Indication
Status
Partner
Indication
Glycogen Storage Disease Type III
Status
Phase 1/2 *
Program
LUNAR®-RARE
Partner
Indication
Undisclosed Rare Disease
Status
Preclinical
Partner
Indication
Hepatitus B Virus (HBV)
Status
Preclinical
Partner
Indication
Nonalcholic Steatohepatitis (NASH)
Status
Preclinical
$1B+ in Potential Milestones & Royalties
We have partnered with a number of industry leaders and have a robust and diverse preclinical and clinical drug development pipeline.

Our external development partners include Janssen Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc., Takeda Pharmaceutical Company Limited, CureVac AG and Synthetic Genomics, Inc.

* https://clinicaltrials.gov/ct2/show/NCT04574830

LUNAR®-GSDIII

Arcturus has partnered with Ultragenyx to develop messenger RNA (mRNA) therapeutic candidates for certain rare disease targets. The first disclosed indication is Glycogen Storage Disease Type III.
Glycogen Storage Disease Type III
Glycogen Storage Disease Type III is a genetic disease affecting 1 in 100,000 Americans. GSDIII is caused by mutations in the AGL gene that allow glycogen to accumulate in cells. This can cause low blood sugar levels, muscle weakness and liver damage. There are no specific treatments for GSDIII.
LUNAR®-GSDIII
Arcturus and Ultragenyx are developing mRNA medicines to replace the defective AGL gene product and allow cells to breakdown glycogen using normal pathways.
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LUNAR®-HBV

Arcturus has partnered with Janssen Pharmaceuticals, Inc., a Johnson & Johnson company, to develop nucleic acid medicines for the treatment of hepatitis B virus infection and other diseases.
Hepatitis B
Hepatitis B is caused by the Hepatitis B Virus (HBV) and affects more than 350 million people worldwide. It is a major cause of liver disease including cirrhosis and liver cancer. There is no cure for chronic HBV infection. In most cases of chronic hepatitis B, antiviral medication suppresses the virus, but does not clear it completely. Therefore, these medications must be taken for life. Unfortunately, many of these medications have common side effects, including fever, headaches, hair loss, and mental problems. As soon as a patient comes off the medication the virus can return.
LUNAR®-HBV
Arcturus and Janssen are developing a nucleic acid medicine to fight HBV without the same side effect issues that plague existing therapies.
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LUNAR®-NASH

Arcturus, in partnership with Takeda Pharmaceutical Company Ltd., is developing RNA medicines for the treatment of nonalcoholic steatohepatitis (NASH) and other gastrointestinal disorders, utilizing our wholly owned therapeutic delivery platform, LUNAR®.
Nonalcoholic steatohepatitis (NASH)
Nonalcoholic steatohepatitis (NASH) is the most serious form of nonalcoholic fatty liver disease (NAFLD) and affects 3.5-5% of Americans. NASH is predicted to become the leading cause of end-stage liver disease, liver transplant, and hepatocellular carcinoma in the next two decades. There are no FDA-approved drugs to treat NASH. Doctors currently recommend weight loss and increased exercise to control fat deposition, fibrosis and inflammation in the liver.
LUNAR®-NASH
Takeda and Arcturus are collaborating to develop RNA-based therapeutic candidates to treat NASH. Current program activities include lead candidate identification and duration of action studies in various models of disease.
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