CF Foundation Increases Financial Commitment to ~$25 Million
SAN DIEGO, CA, USA I September 26, 2023 I Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, announced today that Arcturus and the Cystic Fibrosis Foundation (CF Foundation) have extended their ongoing agreement. The CF Foundation agreed to increase its financial commitment to ~$25 million to advance ARCT-032, a novel messenger RNA (mRNA) therapeutic candidate formulated with Arcturus’ LUNAR® delivery technology. This agreement extends a prior research program, initiated in 2017, which has resulted in the advancement of ARCT-032 into ongoing clinical studies.
“We are thrilled to extend our productive relationship with the Cystic Fibrosis Foundation, and we are grateful for their meaningful financial support,” said Pad Chivukula, Ph.D., Chief Scientific Officer of Arcturus. “ARCT-032 has the potential to express fully functional CFTR protein in the lung, thereby addressing the root cause of cystic fibrosis. The resources and valuable expertise provided by the CF Foundation will support the continued clinical development of ARCT-032, including the completion of a Phase 1b study that we plan to initiate imminently in adults living with cystic fibrosis.”
ARCT-032 is made up of mRNA that codes for CFTR, encapsulated by lipid nanoparticles utilizing LUNAR delivery technology that has been optimized for inhaled lung delivery. ARCT-032 is agnostic to the underlying mutations associated with the disease and has the potential to provide clinical benefits across a wide range of those living with CF, including those not served by currently approved CFTR modulators. The program has completed dosing in a Phase 1 study in healthy volunteers.
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein 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 some people with CF because 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 (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. The Company has ongoing collaborations with CSL Seqirus and Meiji Seika Pharma, and a joint venture with ARCALIS. Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase deficiency and cystic fibrosis, along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. 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
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CF Foundation Increases Financial Commitment to ~$25 Million
SAN DIEGO, CA, USA I September 26, 2023 I Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, announced today that Arcturus and the Cystic Fibrosis Foundation (CF Foundation) have extended their ongoing agreement. The CF Foundation agreed to increase its financial commitment to ~$25 million to advance ARCT-032, a novel messenger RNA (mRNA) therapeutic candidate formulated with Arcturus’ LUNAR® delivery technology. This agreement extends a prior research program, initiated in 2017, which has resulted in the advancement of ARCT-032 into ongoing clinical studies.
“We are thrilled to extend our productive relationship with the Cystic Fibrosis Foundation, and we are grateful for their meaningful financial support,” said Pad Chivukula, Ph.D., Chief Scientific Officer of Arcturus. “ARCT-032 has the potential to express fully functional CFTR protein in the lung, thereby addressing the root cause of cystic fibrosis. The resources and valuable expertise provided by the CF Foundation will support the continued clinical development of ARCT-032, including the completion of a Phase 1b study that we plan to initiate imminently in adults living with cystic fibrosis.”
ARCT-032 is made up of mRNA that codes for CFTR, encapsulated by lipid nanoparticles utilizing LUNAR delivery technology that has been optimized for inhaled lung delivery. ARCT-032 is agnostic to the underlying mutations associated with the disease and has the potential to provide clinical benefits across a wide range of those living with CF, including those not served by currently approved CFTR modulators. The program has completed dosing in a Phase 1 study in healthy volunteers.
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein 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 some people with CF because 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 (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. The Company has ongoing collaborations with CSL Seqirus and Meiji Seika Pharma, and a joint venture with ARCALIS. Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase deficiency and cystic fibrosis, along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. 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: 560
