Arcturus Therapeutics Holdings Inc
Rare disease drugmaker · based in San Diego, United States · founded 2013 · ticker ARCT · arcturusrx.com
Arcturus Therapeutics Holdings Inc works on Cystic Fibrosis and Urea Cycle Disorders, 2 of the rare diseases we track. It has 2 active clinical trials, and all of them are looking for volunteers now.
Arcturus Therapeutics is a commercial mRNA medicines and vaccines company developing therapies for liver and respiratory rare diseases. The company's platform technologies include STARR, a self-amplifying mRNA technology, and LUNAR, a lipid-mediated delivery system for therapeutic RNA and DNA to target cells.
Arcturus Therapeutics Holdings Inc pipeline
Each row lists the diseases Arcturus Therapeutics Holdings Inc is studying at that stage, from the ones closest to approval down to the earliest work.
Cystic Fibrosis recruiting · Urea Cycle Disorders recruiting
Arcturus Therapeutics Holdings Inc clinical trials
These are Arcturus Therapeutics Holdings Inc trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials looking for volunteers now
Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
Tests ARCT-032
For Cystic Fibrosis and CFTR Gene Mutation
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Tests ARCT-810
For OTC Deficiency, Ornithine Transcarbamylase Deficiency and OTCD
Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Arcturus Therapeutics Holdings Inc. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
More about Arcturus Therapeutics Holdings Inc
As a rare disease specialist, Arcturus Therapeutics Holdings Inc has active clinical trial programs and drug development efforts across 2 rare diseases, including Cystic Fibrosis, Urea Cycle Disorders. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows active and recruiting rare disease studies sponsored by Arcturus Therapeutics Holdings Inc, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites.
The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Arcturus Therapeutics Holdings Inc is headquartered in San Diego, United States, founded in 2013, publicly traded under the ticker symbol ARCT. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Where Arcturus Therapeutics Holdings Inc trials take place
The trials above run at 14 study sites in 1 country. Tap a country to see its states, cities and hospitals.
Rare diseases Arcturus Therapeutics Holdings Inc works on
Each disease page lists every open trial, not only Arcturus Therapeutics Holdings Inc's, plus approved treatments.
Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport.
How common: Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians
Urea Cycle Disorders (UCDs) are a group of genetic conditions affecting the enzymes that convert toxic ammonia to urea for excretion.
How common: About 1 in 35,000 births in the United States, based on newborn screening and registry data (Summar et al., 2013); Ornithine Transcarbamylase (OTC) deficiency is most common form
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Arcturus Therapeutics Holdings Inc's own site is arcturusrx.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- Cystic Fibrosis FoundationCystic Fibrosis
- NIH GARD - Cystic FibrosisCystic Fibrosis
- National Urea Cycle Disorders FoundationUrea Cycle Disorders
- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders, first revision. J Inherit Metab Dis, 2019Urea Cycle Disorders