Autoinflammatory diseases

EQS-News: Secarna Pharmaceuticals publishes new preclinical data in The Journal of Immunology demonstrating potent therapeutic activity of LNAplus™ ASO targeting NLRP3 in debilitating autoinflammatory diseases

Retrieved on: 
Thursday, July 6, 2023

Using its proprietary Oligofyer™ bioinformatics system, Secarna designed LNAplus™ ASOs to specifically suppress the expression of the NLR family pyrin domain containing 3 (NLRP3), the central component of the inflammasome pathway.

Key Points: 
  • Using its proprietary Oligofyer™ bioinformatics system, Secarna designed LNAplus™ ASOs to specifically suppress the expression of the NLR family pyrin domain containing 3 (NLRP3), the central component of the inflammasome pathway.
  • CAPS are a spectrum of autoinflammatory diseases caused by activating mutations in the NLRP3 gene.
  • The activity of NLRP3-ASO was tested in an in vivo NOMID disease model; NOMID is the most severe form of CAPS.
  • After systemic ASO treatment, life expectancy was significantly increased, with weight gains and reductions in the severity of skin lesions, showing an overall reduction in systemic inflammation.

ZyVersa Therapeutics Announces a Publication in Frontiers in Medicine Showing Direct Inflammasome Activation in Children with Juvenile Idiopathic Arthritis

Retrieved on: 
Thursday, May 25, 2023

In the paper titled, “Inflammasome activation and formation of ASC specks in patients with juvenile idiopathic arthritis,” the authors reported the following:

Key Points: 
  • In the paper titled, “Inflammasome activation and formation of ASC specks in patients with juvenile idiopathic arthritis,” the authors reported the following:
    Il-1β production from macrophages exposed to extracellular ASC specks was enhanced in serum of JIA patients with a high titer of ANA, suggesting that autoantibodies favor a secondary inflammatory response to ASC specks by macrophages
    The authors stated, “We clearly discovered direct inflammasome activity ex vivo in CD14+ CD16− monocytes of oligo- and poly-articular JIA patients.” To read the article, Click Here .
  • “The research published in Frontiers in Medicine demonstrates the role of inflammasome activation in two of the most common types of JIA, oligo- and poly-articular, which were previously thought to be classical antigen-driven autoimmune conditions.
  • This research provides support for the broad range of conditions that are impacted by activation of the innate immune response,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO and President.
  • “Additionally, the data demonstrating enhanced IL-1β production from macrophages exposed to extracellular ASC specks in JIA patients with a high titer of ANA supports the potential of ASC speck inhibition in helping to control inflammation in this population.”

BioAegis Therapeutics to Sponsor a Webinar, Below the Eyes of the Hippopotamus: The Expanding Spectrum of Auto- and Hyper- Inflammatory Diseases

Retrieved on: 
Wednesday, May 10, 2023

The webinar, Below the Eyes of the Hippopotamus: The Expanding Spectrum of Auto- and Hyper- Inflammatory Diseases will delve beneath the surface of this rapidly growing field of auto-inflammatory and hyper-inflammatory diseases.

Key Points: 
  • The webinar, Below the Eyes of the Hippopotamus: The Expanding Spectrum of Auto- and Hyper- Inflammatory Diseases will delve beneath the surface of this rapidly growing field of auto-inflammatory and hyper-inflammatory diseases.
  • The major scientific breakthrough in identifying genes responsible for autoinflammatory diseases and related genotypes.
  • Key biomarkers for autoinflammatory diseases and new targets for treatments, like the NLRP3 inflammasome.
  • A live Q&A discussion with key opinion leader, David Fajgenbaum, MD, will follow the presentation.

AB2 Bio Completes Enrolment in Pivotal Phase 3 Trial of Tadekinig alfa to Treat Primary Monogenic IL-18 Driven HLH

Retrieved on: 
Tuesday, March 7, 2023

The study is designed to show the efficacy and safety of Tadekinig alfa (r-hIL-18BP) for the treatment of primary monogenic IL-18 driven HLH, an ultra-rare and life-threatening condition with no approved therapies, mainly affecting children.

Key Points: 
  • The study is designed to show the efficacy and safety of Tadekinig alfa (r-hIL-18BP) for the treatment of primary monogenic IL-18 driven HLH, an ultra-rare and life-threatening condition with no approved therapies, mainly affecting children.
  • “This is a truly exciting time as we pioneer development of the first targeted treatment option for IL18 driven diseases and conditions,” said Dr. Djordje Filipovic, CEO of AB2 Bio.
  • “We are pleased to have completed the targeted enrollment in this pivotal study which represents a significant milestone for AB2 Bio and the patient community.
  • “Tadekinig alfa is an important potential new treatment option for patients suffering from IL-18 driven autoinflammatory diseases with no current standard of care.