SAN DIEGO, CA, USA I January 31, 2023 I Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, “Arcturus Therapeutics”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced that the Clinical Trial Application (CTA) for ARCT-032, an inhaled investigational mRNA medicine to treat cystic fibrosis (CF), received approval to proceed into a Phase 1 First-in-Human study in New Zealand.
“Arcturus continues to progress ARCT-032, a potential mRNA medicine for people with cystic fibrosis regardless of their underlying mutation type, through the approval of a CTA to proceed into First-in-Human studies,” said Joseph Payne, President and CEO of Arcturus Therapeutics. “We believe the advantages driven by our proprietary LUNAR® delivery technology, advanced mRNA purification processes, and manufacturing know-how may allow ARCT-032 to restore healthy CFTR protein in the lungs of people with CF, including those that currently do not have effective treatment options. Preclinical data shared at the recent North American Cystic Fibrosis Conference demonstrated robust expression and functional restoration of CFTR in human bronchial epithelial cells from CF donors, providing additional support for the advancement of ARCT-032 into clinical development.”
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the Cystic Fibrosis Transmembrane (CFTR) gene result in a reduction or absence of CFTR quantity and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ineffective in many people with CF as a result of their underlying mutations.
About ARCT-032
ARCT-032 will utilize Arcturus’ LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease. The ARCT-032 program is supported by preclinical data in rodents, ferrets and primates, as well as demonstrating restoration of expression and function in human bronchial epithelial cells.
About Arcturus Therapeutics
Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global late-stage clinical mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR™ mRNA Technology (samRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus’ diverse pipeline of RNA therapeutic and vaccine candidates includes mRNA vaccine programs for SARS-CoV-2 (COVID-19) and Influenza, and other programs to potentially treat ornithine transcarbamylase (OTC) deficiency, and cystic fibrosis, along with partnered programs including glycogen storage disease type III, and hepatitis B virus. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (patents and patent applications issued in the U.S., Europe, Japan, China and other countries). For more information, visit www.ArcturusRx.com. In addition, please connect with us on Twitter and LinkedIn.
SOURCE: Arcturus Therapeutics
Post Views: 497
SAN DIEGO, CA, USA I January 31, 2023 I Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, “Arcturus Therapeutics”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced that the Clinical Trial Application (CTA) for ARCT-032, an inhaled investigational mRNA medicine to treat cystic fibrosis (CF), received approval to proceed into a Phase 1 First-in-Human study in New Zealand.
“Arcturus continues to progress ARCT-032, a potential mRNA medicine for people with cystic fibrosis regardless of their underlying mutation type, through the approval of a CTA to proceed into First-in-Human studies,” said Joseph Payne, President and CEO of Arcturus Therapeutics. “We believe the advantages driven by our proprietary LUNAR® delivery technology, advanced mRNA purification processes, and manufacturing know-how may allow ARCT-032 to restore healthy CFTR protein in the lungs of people with CF, including those that currently do not have effective treatment options. Preclinical data shared at the recent North American Cystic Fibrosis Conference demonstrated robust expression and functional restoration of CFTR in human bronchial epithelial cells from CF donors, providing additional support for the advancement of ARCT-032 into clinical development.”
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the Cystic Fibrosis Transmembrane (CFTR) gene result in a reduction or absence of CFTR quantity and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ineffective in many people with CF as a result of their underlying mutations.
About ARCT-032
ARCT-032 will utilize Arcturus’ LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease. The ARCT-032 program is supported by preclinical data in rodents, ferrets and primates, as well as demonstrating restoration of expression and function in human bronchial epithelial cells.
About Arcturus Therapeutics
Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global late-stage clinical mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR™ mRNA Technology (samRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus’ diverse pipeline of RNA therapeutic and vaccine candidates includes mRNA vaccine programs for SARS-CoV-2 (COVID-19) and Influenza, and other programs to potentially treat ornithine transcarbamylase (OTC) deficiency, and cystic fibrosis, along with partnered programs including glycogen storage disease type III, and hepatitis B virus. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (patents and patent applications issued in the U.S., Europe, Japan, China and other countries). For more information, visit www.ArcturusRx.com. In addition, please connect with us on Twitter and LinkedIn.
SOURCE: Arcturus Therapeutics
Post Views: 497
