Arcturus Therapeutics


Ultragenyx Pharmaceutical, Inc.: LUNAR-RARE1 and LUNAR-RARE2

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™) nanoparticle delivery platform to design and optimize mRNA medicines for rare disease targets. These medicines will be developed and commercialized by Ultragenyx.

Arbutus: 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.

Arrowhead: ARC-AAT

Arcturus has licensed our unlocked nucleomonomer (UNA) Oligomer technology to Arrowhead Pharmaceuticals, Inc. to develop RNA medicines that treat liver disease caused by Alpha-1 antitrypsin deficiency (AATD).

The alpha-1 antitrypsin protein is produced in the liver and released into the blood. It circulates around the body and helps protect different organs, including the liver, lung and skin, from proteins called proteases that break down tissue. Patients with AATD do not have enough functioning alpha-1 antitrypsin protein and so can develop severe lung and liver disease including infections, emphysema, chronic obstructive pulmonary disease, hepatitis and liver cancer.

This disease is caused by a genetic mutation. There are no therapeutics available to treat the cause of this disease. Patients are given supportive treatments to reduce their symptoms. Patients with severe symptoms may be treated with IV alpha-1 antitrypsin infusions and liver and lung transplants.

Arrowhead is developing an RNA medicine, called ARC-AAT, that stops the liver from producing mutant alpha-1 antitrypsin protein. The mutant protein accumulates in the liver of AATD patients and causes inflammation and disease. ARC-AAT can prevent this from happening and allow the liver to repair itself. This medicine has Orphan Drug Designation in the USA and Europe.

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