Arcturus Therapeutics

We’re proud to stand with the #CF community—and especially with the final 10% still waiting for effective treatments.    The 2025 EE Scientific Symposium on Gene Delivery brought forward bold science, fresh thinking, and renewed hope. We're energized by the progress and committed to being part of the solution.    Learn more about our clinical trial investigating ARCT-032 as a potential therapy for individuals with nonsense and rare CF mutations that do not respond to CFTR modulators here: https://lnkd.in/gDWuJUqb #RareDiseases #CysticFibrosis #CF #CFTR #mRNA #mRNATherapies #mRNATherapeutics 

Throwback Thursday to six months ago when Arcturus Therapeutics attended the North American Cystic Fibrosis Conference #NACFC in Boston! Arcturus had a poster: Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults with Cystic Fibrosis. #RareDiseases #CysticFibrosis #CF #CFTR #mRNA #mRNATherapies #mRNATherapeutics

Swift identification of elevated ammonia levels in the Emergency Department is needed to save lives.     Zoey was a beautiful, spirited 19-year-old college student who lost her life to undiagnosed OTC deficiency. In Zoey's memory, her family has partnered with the National Urea Cycle Disorders Foundation to help raise awareness and educate medical professionals on the need to obtain blood ammonia levels. Watch Zoey’s video now: https://lnkd.in/g8v-sx8X     To learn more about our investigational mRNA treatment, ARCT-810 for OTCD, visit: https://lnkd.in/g3fWb6Kc or ClinicalTrials.gov (NCT06488313). #mRNATherapy #OrnithineTranscarbamylaseDeficiency #OTCD #UreaCycleDisorders #Arcturus

LunairCF is a clinical trial investigating the potential of using mRNA therapy (also known as ARCT-032) as a potential treatment therapy for #CysticFibrosis.     While ARCT-032 is a potential therapy for all those living with CF, it may be especially helpful for those individuals with nonsense and rare CF mutations that do not respond to CFTR modulators.    Learn more about the study on the clinicaltrials.gov website: https://lnkd.in/gM_V9NXa #RareDiseases #CysticFibrosis #CF #CFTR #mRNA #mRNATherapies #mRNATherapeutics

Our friends at CONNECTING FAMILIES UREA CYCLE DISORDERS FOUNDATION are hosting their annual Family Camp Weekend from May 1–4, 2025, in Seattle, WA. This special event is designed to bring families affected by #UreaCycleDisorders (UCD) together from across the country. It is a chance to connect with others who understand your journey, learn more about UCD through helpful education sessions, enjoy fun activities for all ages, and access resources that can make a difference for you and your family.   If you or a loved one living with a UCD and interested in attending Family Camp, please contact Robin@ucdfamily.org or visit: www.ucdfamily.org.   Arcturus is proud to support this meaningful event, and we encourage you to watch the video to learn more about the foundation and the camp experience: https://lnkd.in/gtuaiMhd

Did you know that the Cystic Fibrosis Foundation has its own Clinical Trial Finder? You can use their website to find clinical trial opportunities that might be right for you or a loved one living with #CysticFibrosis. The Clinical Trial Finder allows you to search by location and distance to a trial site and allows you to sign up for clinical trial alerts. Learn more on the clinical trial finder website: https://lnkd.in/dBTcVJus #RareDisease #CF #CFTR #mRNA #mRNATherapeutics #ClinicalTrials #Arcturus 

collectively coexisting is a podcast hosted by our friends at Remember The Girls that explores the experiences of women affected by #Xlinked conditions. This podcast highlights the challenges women face in healthcare, and the experiences of women with genetic testing, family planning, and mental health. Listen to Dr. Andrea Gropman, an expert in OTC deficiency discuss the latest research on females with OTC and their risk of symptoms: https://lnkd.in/eAwX_ESP    To learn more about our investigational mRNA treatment, ARCT-810 for OTCD, visit: https://lnkd.in/g3fWb6Kc or ClinicalTrials.gov (NCT06488313). #mRNA #OrnithineTranscarbamylaseDeficiency #OTCD #UreaCycleDisorders #Arcturus

Did you know that Arcturus has its own YouTube channel? It’s a great resource to learn more about our work and how we’re developing potential #mRNA therapies for those with #CysticFibrosis and #OTCDeficiency. Check out our videos to hear inspiring stories from community guest speakers, learn how mRNA works and its potential benefits, and meet the dedicated team members at Arcturus working to make a meaningful difference. Access our YouTube channel here: https://lnkd.in/gW_sb8NT #mRNATherapeutics #LUNAR #CF #OTCD #Arcturus

Have questions about genetic therapies? Join us March 20 at 7 p.m. ET as we discuss how genetic therapies, including RNA therapy and gene therapy, could potentially work for any person with cystic fibrosis, at our research webinar.   Led by Kara Foshay, Phd, and Heather Hathorne, PhD, RRT, CCRC, you’ll learn how genetic therapies work in the body, what makes them different from existing therapies, and much more. At the end, we’ll be taking your questions during a Q&A.   Register now, and you’ll receive the recording post-event: https://lnkd.in/gSJNTUEQ

#Hyperammonemia is a state in which blood ammonia levels get dangerously high. This can cause seizures, coma, brain damage and even death.     Kate Vinokurov tells her son's story. Etan lives with #OTCdeficiency which means his body can't get rid of the ammonia that builds up as a natural part of the metabolic process.     The "Think Ammonia!" campaign from Metabolic Support UK aims to improve outcomes for individuals experiencing hyperammonemia.      Watch Kate and Etan’s story here: https://lnkd.in/gRBSbA8x     To learn more about our LUNAR-OTC program for OTCD, visit: https://lnkd.in/g3fWb6Kc #ThinkAmmonia #mRNA #OTCD #UreaCycleDisorders #Arcturus