Urea cycle

iECURE Receives FDA Orphan Drug Designation for GTP-506, an Investigational Gene Editing Product Candidate for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency

Retrieved on: 
Tuesday, September 6, 2022

The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.

Key Points: 
  • The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.
  • Orphan Drug Designation provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs.
  • Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products.
  • 1 iECURE has licensed the ARCUS nuclease from Precision BioSciences for four gene insertion programs including OTC, CTLN1 and PKU.

Arcturus Announces Regulatory Approval to Proceed with Phase 2 Study of ARCT-810 mRNA Therapeutic Candidate for Ornithine Transcarbamylase (OTC) Deficiency

Retrieved on: 
Wednesday, July 28, 2021

The ARCT-810 Phase 2 study is a randomized, double-blind, placebo-controlled, nested single and multiple ascending dose design for adolescents and adults with OTC deficiency.

Key Points: 
  • The ARCT-810 Phase 2 study is a randomized, double-blind, placebo-controlled, nested single and multiple ascending dose design for adolescents and adults with OTC deficiency.
  • We are pleased to initiate this multiple dose Phase 2 clinical trial of ARCT-810 in OTC deficiency patients.
  • Advancement to a Phase 2 study is supported by the safety results from our ARCT-810 Phase 1 trial in Healthy Volunteers, said Steve Hughes, M.D., Chief Medical Officer of Arcturus.
  • Ornithine Transcarbamylase (OTC) deficiency is a serious urea cycle disorder with a prevalence of approximately 10,000 people worldwide.

Metropolitan Wins Top Honors for Best Tasting Tap Water in the U.S.

Retrieved on: 
Tuesday, June 8, 2021

Metropolitan garnered top honors in the municipal water category, with its member agency, the City of Santa Ana, coming in at second place.

Key Points: 
  • Metropolitan garnered top honors in the municipal water category, with its member agency, the City of Santa Ana, coming in at second place.
  • While water quality and safety are always our utmost priorities, its also important to us that consumers like the taste of our water.
  • Good taste is an important confirmation for consumers of the quality of the water coming from their taps, Metropolitan General Manager Jeffrey Kightlinger said.
  • Our panel of tasters take months-long training to become certified sensory analysts to ensure that our customers are receiving the best tasting drinking water.

Aeglea BioTherapeutics Launches THINK ARGININE™, a Disease Education Initiative to Improve Awareness and Diagnosis of Arginase 1 Deficiency

Retrieved on: 
Thursday, May 6, 2021

In order to support healthcare providers in making diagnoses, THINK ARGININE also includes no-charge diagnostic testing for adults and children with suspected ARG1-D.

Key Points: 
  • In order to support healthcare providers in making diagnoses, THINK ARGININE also includes no-charge diagnostic testing for adults and children with suspected ARG1-D.
  • "Our THINK ARGININE initiative is the result of input from those in the ARG1-D community, including patients, caregivers and clinicians.
  • Together, greater awareness and earlier diagnosis can lead to more effective disease management strategies earlier in life.
  • "Because Arginase 1 Deficiency is a progressive disease, delays in diagnosis and treatment can lead to worse clinical outcomes.

Global Fumaric Acid Market Report 2021: Industry Trends, Share, Size, Growth, Opportunity and Forecasts, 2015-2020 & 2021-2026 - ResearchAndMarkets.com

Retrieved on: 
Monday, April 26, 2021

b'The "Fumaric Acid Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2021-2026" report has been added to ResearchAndMarkets.com\'s offering.\nThe global fumaric acid market reached a value of US$ 660.9 Million in 2020.

Key Points: 
  • b'The "Fumaric Acid Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2021-2026" report has been added to ResearchAndMarkets.com\'s offering.\nThe global fumaric acid market reached a value of US$ 660.9 Million in 2020.
  • Looking forward, the global fumaric acid market to exhibit moderate growth during the next five years.\nFumaric acid is a white or colorless crystalline chemical compound with a fruit-like taste.
  • Similar to malic acid, fumaric acid helps in the production of energy from food in the form of ATP.
  • Commercially available in the form of dry powder, liquid or large pellet crystals, fumaric acid is also used in the manufacturing of medicines, animal feed, unsaturated polyester, cleaning agents and printing inks.\nGlobal Fumaric Acid Market Drivers:\nFumaric acid is used as an acidulant, pH controller and flavor enhancer in the food and beverage industry.

Aeglea BioTherapeutics and Immedica Announce Commercialization Agreement for Pegzilarginase for the Treatment of Arginase 1 Deficiency in Europe and Middle East

Retrieved on: 
Monday, March 22, 2021

"There is an urgent need to deliver new therapies for Arginase 1 Deficiency due to its progressive and devastating nature.

Key Points: 
  • "There is an urgent need to deliver new therapies for Arginase 1 Deficiency due to its progressive and devastating nature.
  • Under the terms of the agreement, Immedica will make an upfront payment of $21.5 million to Aeglea.
  • Immedica receives commercialization rights in Europe and several Middle East countries.
  • Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine.

Jubilant Therapeutics Announces Research Collaboration with Boston Children's Hospital, Harvard Medical School, to Evaluate PAD4 Inhibitors in Autoimmune/Inflammation Disease Models

Retrieved on: 
Thursday, February 25, 2021

PAD4 is an enzyme that converts arginine to citrulline in histones and is highly expressed in neutrophils.

Key Points: 
  • PAD4 is an enzyme that converts arginine to citrulline in histones and is highly expressed in neutrophils.
  • Specific PAD4 inhibitors, that are well tolerated by animals, would be great for pre-clinical and, later, human studies.
  • Jubilant Therapeutics Inc. is a patient-centric biopharmaceutical company advancing potent and selective small molecule modulators to address unmet medical needs in oncology and autoimmune diseases.
  • Jubilant Therapeutics is headquartered in the U.S. and guided by globally renowned key opinion leaders and scientific advisory board members.

Jubilant Therapeutics Announces Research Collaboration with Boston Children's Hospital, Harvard Medical School, to Evaluate PAD4 Inhibitors in Autoimmune/Inflammation Disease Models

Retrieved on: 
Thursday, February 25, 2021

PAD4 is an enzyme that converts arginine to citrulline in histones and is highly expressed in neutrophils.

Key Points: 
  • PAD4 is an enzyme that converts arginine to citrulline in histones and is highly expressed in neutrophils.
  • Specific PAD4 inhibitors, that are well tolerated by animals, would be great for pre-clinical and, later, human studies.
  • Jubilant Therapeutics Inc. is a patient-centric biopharmaceutical company advancing potent and selective small molecule modulators to address unmet medical needs in oncology and autoimmune diseases.
  • Jubilant Therapeutics is headquartered in the U.S. and guided by globally renowned key opinion leaders and scientific advisory board members.

Jubilant Therapeutics Announces Research Collaboration with Boston Children's Hospital, Harvard Medical School, to Evaluate PAD4 Inhibitors in Autoimmune/Inflammation Disease Models

Retrieved on: 
Thursday, February 25, 2021

PAD4 is an enzyme that converts arginine to citrulline in histones and is highly expressed in neutrophils.

Key Points: 
  • PAD4 is an enzyme that converts arginine to citrulline in histones and is highly expressed in neutrophils.
  • Specific PAD4 inhibitors, that are well tolerated by animals, would be great for pre-clinical and, later, human studies.
  • Jubilant Therapeutics Inc. is a patient-centric biopharmaceutical company advancing potent and selective small molecule modulators to address unmet medical needs in oncology and autoimmune diseases.
  • Jubilant Therapeutics is headquartered in the U.S. and guided by globally renowned key opinion leaders and scientific advisory board members.

Arcturus Therapeutics Announces Initiation of Dosing ARCT-810 in Patients with Ornithine Transcarbamylase (OTC) Deficiency

Retrieved on: 
Monday, December 7, 2020

The ongoing Phase 1b study will evaluate approximately twelve patients with OTC deficiency.

Key Points: 
  • The ongoing Phase 1b study will evaluate approximately twelve patients with OTC deficiency.
  • Arcturus previously announced that it had completed the ARCT-810 Phase 1, dose escalation study in healthy subjects, at doses up to 0.4 mg/kg.
  • We are pleased to have now initiated dosing of ARCT-810 in patients with OTC deficiency.
  • Ornithine Transcarbamylase (OTC) deficiency is a serious urea cycle disorder with a prevalence of approximately 10,000 worldwide.