Ornithine transcarbamylase deficiency

Arcturus Therapeutics Announces Fourth Quarter and Fiscal Year 2023 Financial Update and Pipeline Progress

Retrieved on: 
Thursday, March 7, 2024
COVID-19, Research, Infectious Diseases, FDA, Biotechnology, Health, Pharmaceutical, General Health, Science, Pfizer–BioNTech COVID-19 vaccine, Liver, European Commission, Ministry, Arcturus Therapeutics, Messenger, Ornithine transcarbamylase deficiency, Investment, Omicron, OTC, Lyme disease, CSL, MHLW, The Lancet, Vaccine, Gonorrhea, Observation, RNA, Civil service commission, Collaboration, Therapy, COVID, Ministry of Health, Patient, Valence (chemistry), Janssen, Immunity, Orphan, ARCT, Travel, Infection

“I am excited about the continued pipeline progress and efforts toward commercialization achieved by Arcturus in 2023,” said Joseph Payne, President & CEO of Arcturus Therapeutics.

Key Points: 
  • “I am excited about the continued pipeline progress and efforts toward commercialization achieved by Arcturus in 2023,” said Joseph Payne, President & CEO of Arcturus Therapeutics.
  • This marks the first marketing approval milestone for CSL and Arcturus since signing the Collaboration and License agreement in November 2022.
  • In November 2023, Arcturus received Orphan Drug Designation from the U.S. FDA for ARCT-032, for the treatment of Cystic Fibrosis.
  • We have achieved a total of approximately $396.0 million in upfront payments and milestones from CSL as of December 31, 2023.

Precision BioSciences Announces MHRA Approval for Partner iECURE to Expand Phase 1/2 Clinical Trial of ARCUS Gene Editing Program in OTC Deficiency

Retrieved on: 
Wednesday, March 6, 2024
Other Health, Research, General Health, Pharmaceutical, Technology, Genetics, Clinical Trials, Science, Nanotechnology, Biotechnology, Other Science, Health, Liver, Nerve, Metabolic disorder, Precision BioSciences, Gene editing, Ammonia, Coma, TGA, Ornithine transcarbamylase deficiency, Blood, OTC, Precision, Risk, Health care, Death, Patient, MHRA, Disease, IND, CTA

Medicines & Healthcare products Regulatory Agency (MHRA) for the company’s Clinical Trial Authorization (CTA) application to expand the Phase 1/2 OTC-HOPE study evaluating ECUR-506 into the U.K.

Key Points: 
  • Medicines & Healthcare products Regulatory Agency (MHRA) for the company’s Clinical Trial Authorization (CTA) application to expand the Phase 1/2 OTC-HOPE study evaluating ECUR-506 into the U.K.
  • The OTC-HOPE study is investigating ECUR-506, incorporating an ARCUS nuclease, for the treatment of Ornithine Transcarbamylase (OTC) deficiency in infants.
  • In 2021, Precision licensed to iECURE an ARCUS nuclease that inserts a functional copy of the OTC gene for treatment of OTC deficiency.
  • “Congratulations to iECURE for bringing the first gene editing approach into clinical investigation for patients with OTC deficiency.

iECURE Secures Clearance from Australian Therapeutic Goods Administration for its Clinical Trial Application for the OTC-HOPE Phase 1/2 Study of ECUR-506

Retrieved on: 
Wednesday, December 13, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Research, Safety, Nerve, CTN, Gene editing, Ornithine transcarbamylase deficiency, Pediatrics, Gene, University, Coma, Mouse, Liver, Clinical trial, Disease, Risk, TGA, Patient, GTP, Nervous system, Pharmacokinetics, Child, Blood, Primate, Trial of the century, Ammonia, Death, Metabolic disorder, Neurology, Quality of life, OTC, Mount Sinai Hospital (Manhattan), Pharmaceutical industry, Dietary supplement, Medicine

“The TGA’s clearance to begin clinical trials for the OTC-HOPE study represents major milestones for the OTC deficiency community and for the iECURE team,” said Joe Truitt, Chief Executive Officer of iECURE.

Key Points: 
  • “The TGA’s clearance to begin clinical trials for the OTC-HOPE study represents major milestones for the OTC deficiency community and for the iECURE team,” said Joe Truitt, Chief Executive Officer of iECURE.
  • “ECUR-506 has the potential to restore the function of the OTC enzyme in infants who lack this critical component of the urea cycle.
  • We are hopeful that our approach will be a better option for these children.”
    The OTC-HOPE study is a Phase 1/2 first-in-human study in baby boys with genetically confirmed neonatal-onset OTC deficiency.
  • Currently available medical therapies do not correct the disease and do not eliminate the risk of life-threatening symptoms or crises.

Precision BioSciences Announces Approval of First Clinical Trial Application of ARCUS Gene Insertion Program by Partner iECURE

Retrieved on: 
Wednesday, December 13, 2023
Research, Infectious Diseases, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Precision BioSciences, Nerve, Gene editing, Ornithine transcarbamylase deficiency, HDR, Gene, Clinical trial, Coma, IND, Liver, Disease, Risk, Patient, Cell, ESGCT, Blood, Hepatocyte, Primate, CRISPR, Ammonia, Death, Neurology, Congress, CTA, OTC, Precision, Metabolic disorder, Vaccine

“The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.

Key Points: 
  • “The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.
  • This is the first ARCUS in vivo gene editing program to progress into the clinic,” said Michael Amoroso, Chief Executive Officer of Precision Biosciences.
  • A 5% threshold of enzymatic activity has the potential for clinical benefit as previously identified in Annals of Clinical and Translational Neurology .
  • “ECUR-506 is the first of several partnered and wholly owned gene editing programs leveraging ARCUS in differentiated indications and we look forward to initial clinical data from this program.”

Arcturus Therapeutics Announces Third Quarter 2023 Financial Update and Pipeline Progress

Retrieved on: 
Tuesday, November 14, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CSL, Safety, Research, DNA, Ornithine transcarbamylase deficiency, Therapy, Cystic fibrosis, Technology transfer, Liver, Clinical trial, Database, COVID-19, Patient, FDA, PMDA, Vaccine, ARCT, Messenger, Valencia CF Foundation, Arcturus Therapeutics, Conference, RNA, Ferret, Infection, Ivacaftor, CFTR, OTC, Government, HIV disease progression rates, Transfection, Travel, COVID, Pharmaceutical industry, Cryptocurrency, Arcturus

“We had considerable progress this quarter expanding our next generation STARR® vaccine platform,” said Joseph Payne, President & CEO of Arcturus Therapeutics.

Key Points: 
  • “We had considerable progress this quarter expanding our next generation STARR® vaccine platform,” said Joseph Payne, President & CEO of Arcturus Therapeutics.
  • ARCALIS Inc., the Company’s manufacturing joint venture in Japan to support the production of mRNA vaccines and therapeutics, continues to make operational progress while also obtaining financial support from the Japanese government.
  • Arcturus achieved a milestone for $35 million and anticipates receipt from CSL in November 2023.
  • Arcturus expects the final database lock to occur in the fourth quarter of 2023.

Arcturus Therapeutics Announces Second Quarter 2023 Financial Update and Pipeline Progress

Retrieved on: 
Monday, August 7, 2023
COVID-19, Research, Infectious Diseases, FDA, Genetics, Clinical Trials, Biotechnology, Pharmaceutical, Health, Science, Vaccine, Diversified Specialty Institute Holdings, Inc., Travel, Omicron, Arcturus Therapeutics, Vaccination, Marketing, SARS-CoV-2, Ornithine transcarbamylase deficiency, Infection, NDA, Pfizer–BioNTech COVID-19 vaccine, ARCT, Meiji, CSL, Multimedia, RNA, Messenger, Government, Ministry, CGMP, Patient, MedRxiv, OTC, Cystic fibrosis, Geometry, Liver, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BARDA, Food, MNT, Pharmaceutical industry, Cryptocurrency, Arcturus

Superiority of ARCT-154 in neutralizing antibody response against SARS-CoV-2 Omicron BA.4/5 variant was also demonstrated as a key secondary endpoint.

Key Points: 
  • Superiority of ARCT-154 in neutralizing antibody response against SARS-CoV-2 Omicron BA.4/5 variant was also demonstrated as a key secondary endpoint.
  • Revenue decreased by $16.6 million during the three months ended June 30, 2023 as compared to the prior year period.
  • We have collected approximately $300.0 million in upfront payments and milestones from CSL Seqirus as of June 30, 2023.
  • Additionally, we received $23.6 million under the manufacturing and supply of ARCT-154 from CSL Seqirus during the quarter ended June 30, 2023.

Arcturus Therapeutics Announces First Quarter 2023 Financial Update and Pipeline Progress

Retrieved on: 
Tuesday, May 9, 2023
Biotechnology, Infectious Diseases, Health, Genetics, Pharmaceutical, Vaccine, Diversified Specialty Institute Holdings, Inc., Travel, Therapy, Arcturus Therapeutics, Ornithine transcarbamylase deficiency, Medicines and Healthcare products Regulatory Agency, COVID-19, Infection, NDA, ARCT, Research, Pfizer–BioNTech COVID-19 vaccine, RNA, Messenger, Patient, PMDA, OTC, Cystic fibrosis, Liver, CF, Safety, Pharmaceutical industry, Cryptocurrency, Airline, Arcturus

We believe this meaningful milestone is indicative of the broader platform opportunity for our mRNA medicine technologies to result in novel vaccines and therapeutics over the coming years,” said Joseph Payne, President & CEO of Arcturus Therapeutics.

Key Points: 
  • We believe this meaningful milestone is indicative of the broader platform opportunity for our mRNA medicine technologies to result in novel vaccines and therapeutics over the coming years,” said Joseph Payne, President & CEO of Arcturus Therapeutics.
  • Arcturus is amending the protocol to allow the dosing of patients with CF and expects to initiate the enrollment in Q3 2023.
  • Additionally, we expect to collect $90.0 million in the second quarter of 2023, associated with the CSL Seqirus milestones which are in accounts receivable on March 31, 2023.
  • The cash runway remains extended through the beginning of 2026 based on the current pipeline and programs.

Novel Genetic Screening Tool Offers Hope for Babies Born With Life-Threatening Metabolic Disorder

Retrieved on: 
Thursday, May 4, 2023
Seizure, Journal of Inherited Metabolic Disease, UCDC, Pediatrics, Ornithine transcarbamylase deficiency, American Journal of Human Genetics, Hope, Metabolic disorder, Brain, Child, George Washington University, Bangladesh Technical Education Board, Associate, DSM-IV codes, Science, Human genetics, Children's National Hospital, Disorder, Genetics, American Journal, Data, Metabolism, Patient, Physician, Ammonia, Blood, Risk, OTC, Diagnosis, Gene, Disease, Dietary supplement

SEATTLE, May 4, 2023 /PRNewswire-PRWeb/ -- Every expectant parent hopes to welcome a healthy baby into the world. Unfortunately, infants with some genetic diseases appear healthy at birth, but then rapidly deteriorate—they become lethargic, stop eating, develop seizures, and may progress to coma—symptoms common to a variety of metabolic disorders. For the physicians charged with their care, it is a race against time to determine which disorder is causing their fragile patient to decline.

Key Points: 
  • For the physicians charged with their care, it is a race against time to determine which disorder is causing their fragile patient to decline.
  • A study published today in the American Journal of Human Genetics describes the tools that the Dudley Lab developed to assess the changes in a genetic sequence (variant) associated with ornithine transcarbamylase deficiency ( OTC deficiency ), a rare, inherited metabolic disorder.
  • "We hope that our data will also accelerate the development of new treatments and cures for OTC deficiency."
  • The technology built by the Dudley Lab at PNRI offers hope for a brighter future for infants with life-threatening metabolic diseases.

Arcturus Therapeutics Announces Fourth Quarter 2022 Financial Update and Pipeline Progress

Retrieved on: 
Tuesday, March 28, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Vaccine, Diversified Specialty Institute Holdings, Inc., Arcturus Therapeutics, Hepatitis B virus, Ornithine transcarbamylase deficiency, COVID-19, Infection, ARCT, CSL, Research, Pfizer–BioNTech COVID-19 vaccine, RNA, Messenger, COVID-19 vaccine, Patient, Influenza, OTC, Cystic fibrosis, CFO, Liver, Therapy, Conference, Safety, Pharmaceutical industry, Cryptocurrency, Arcturus

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, today announced its financial results for the fourth quarter ended December 31, 2022, and provided corporate updates.

Key Points: 
  • 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, today announced its financial results for the fourth quarter ended December 31, 2022, and provided corporate updates.
  • “Arcturus has continued to make remarkable operational and pipeline progress.
  • Arcturus achieved development milestones in March 2023, including milestones associated with nominating next generation vaccine candidates, resulting in $90 million invoiced to CSL Seqirus.
  • The increase in fourth quarter and year-over-year revenues primarily relates to the $200 million upfront payment we received from the CSL transaction.

European Commission Grants Orphan Designation for iECURE’s Lead Product Candidate GTP-506 for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency

Retrieved on: 
Wednesday, March 22, 2023
Biotechnology, Health Technology, Genetics, Health, Civil service commission, Liver, Marketing, Ornithine transcarbamylase deficiency, European Medicines Agency, Gene editing, European Commission, Disorder, Committee, EMA, Orphan drug, Patient, OTC, European, Diagnosis, Rare, FDA, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, EU

iECURE , a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, announced today that the European Commission granted orphan designation for the company’s lead product candidate GTP-506 for the treatment of Ornithine Transcarbamylase (OTC) deficiency.

Key Points: 
  • iECURE , a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, announced today that the European Commission granted orphan designation for the company’s lead product candidate GTP-506 for the treatment of Ornithine Transcarbamylase (OTC) deficiency.
  • GTP-506 previously received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for the treatment of OTC deficiency.
  • “Without programs like Orphan designation, thousands of patients with a myriad of rare diseases would have no medicines to treat their disorders, leading to uncontrolled symptoms and possible death.
  • Orphan designation provides companies with certain benefits and incentives in the EU, including clinical protocol assistance, possible waivers or reductions in regulatory fees and ten years of market exclusivity after approval.