Glycolipids

Lysogene Announces FDA Fast Track Designation for LYS-GM101 Gene Therapy for the Treatment of GM1 Gangliosidosis

Retrieved on: 
Thursday, July 8, 2021

GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration.

Key Points: 
  • GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration.
  • A product that receives Fast Track designation is eligible for more frequent interactions with FDA, potential eligibility for accelerated approval, priority review, and rolling Biologics License Application (BLA) review.
  • This Fast Track designation demonstrates the regulators sustained interest in Lysogenes cutting edge gene therapy program.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).

Azafaros Appoints Stefano Portolano, M.D., as Chief Executive Officer

Retrieved on: 
Wednesday, June 30, 2021

Azafaros B.V. today announced that Stefano Portolano, M.D., has joined Azafaros as Chief Executive Officer (CEO) and has concurrently been appointed to the Board of Directors of the company.

Key Points: 
  • Azafaros B.V. today announced that Stefano Portolano, M.D., has joined Azafaros as Chief Executive Officer (CEO) and has concurrently been appointed to the Board of Directors of the company.
  • We are very happy to welcome Stefano to Azafaros as the company progresses further into clinical development.
  • "Stefano joins an outstanding team that is rapidly advancing lead candidate AZ-3102, the companys proprietary orally-available azasugar molecule for the treatment of GM1 and GM2 gangliosidoses.
  • Since the companys inception in 2018, Azafaros has made remarkable progress by building a strong and committed organization that has reached the clinic in less than three years, said Stefano Portolano, M.D., Azafaros' Chief Executive Officer.

New, biological, and safer soaps

Retrieved on: 
Wednesday, May 19, 2021

Biosurfactants, produced by bacteria, are safer and can replace synthetic surfactants.\nRhamnolipid molecules are some of the safest surfactants known and are particularly attractive, thanks to their biodegradability, minimal toxicity, and amenability to be produced from industrial waste.

Key Points: 
  • Biosurfactants, produced by bacteria, are safer and can replace synthetic surfactants.\nRhamnolipid molecules are some of the safest surfactants known and are particularly attractive, thanks to their biodegradability, minimal toxicity, and amenability to be produced from industrial waste.
  • They are made using Pseudomonas aeruginosa, a pathogenic bacterium harmful to humans.\n"If we want to fully gain the benefits of rhamnolipids, we need to grow these pathogenic bacteria on a huge scale.
  • And because that\'s a health risk, the industry is looking for alternatives," explained Professor Gauthier.
  • The INRS community includes more than 1,500 students, postdoctoral fellows, faculty members, and staff.\n'

Sigyn Therapeutics™ Discloses In-Vitro Viral Pathogen Study

Retrieved on: 
Wednesday, April 21, 2021

Incorporated within Sigyn Therapy is a cocktail of adsorbent components with unique binding and capture characteristics to optimize the broad-spectrum depletion of inflammatory targets from the bloodstream.

Key Points: 
  • Incorporated within Sigyn Therapy is a cocktail of adsorbent components with unique binding and capture characteristics to optimize the broad-spectrum depletion of inflammatory targets from the bloodstream.
  • An objective of this study was to rebalance elevated cytokine levels and optimize the elimination of endotoxin from human blood plasma.
  • The study was conducted in triplicate over four-hour time periods with a pediatric version of Sigyn Therapy.\xc2\xa0 Average reduction of endotoxin load peaked at 83% during the studies.
  • Sigyn anticipates that subsequent events and developments may cause its views to change.

Associates of Cape Cod, Inc. announces launch of PyroSmart NextGen™ Recombinant LAL Reagent

Retrieved on: 
Friday, April 23, 2021

b'EAST FALMOUTH, Mass., April 23, 2021 /PRNewswire/ -- Associates of Cape Cod, Inc. (ACC), a Seikagaku Group Company, announces the launch of a new, innovative, sustainable recombinant LALreagent, PyroSmart NextGen for Bacterial Endotoxin Testing (BET).

Key Points: 
  • b'EAST FALMOUTH, Mass., April 23, 2021 /PRNewswire/ -- Associates of Cape Cod, Inc. (ACC), a Seikagaku Group Company, announces the launch of a new, innovative, sustainable recombinant LALreagent, PyroSmart NextGen for Bacterial Endotoxin Testing (BET).
  • The new LAL reagent is sustainably produced using recombinant technology; it is not based on raw materials from animals, unlike traditional LAL reagents which are manufactured using the blood harvested from horseshoe crabs.
  • "We were the first FDA-licensed LAL reagent manufacturer, and we continue our pioneering efforts to be the first to make a full recombinant cascade reagent available to the market.
  • "\nTransitioning to the use of a new recombinant LAL reagent requires some level of validation, irrespective of the article being tested.

Associates of Cape Cod, Inc. announces launch of PyroSmart NextGen™ Recombinant LAL Reagent

Retrieved on: 
Friday, April 23, 2021

b'EAST FALMOUTH, Mass., April 23, 2021 /PRNewswire/ -- Associates of Cape Cod, Inc. (ACC), a Seikagaku Group Company, announces the launch of a new, innovative, sustainable recombinant LALreagent, PyroSmart NextGen for Bacterial Endotoxin Testing (BET).

Key Points: 
  • b'EAST FALMOUTH, Mass., April 23, 2021 /PRNewswire/ -- Associates of Cape Cod, Inc. (ACC), a Seikagaku Group Company, announces the launch of a new, innovative, sustainable recombinant LALreagent, PyroSmart NextGen for Bacterial Endotoxin Testing (BET).
  • The new LAL reagent is sustainably produced using recombinant technology; it is not based on raw materials from animals, unlike traditional LAL reagents which are manufactured using the blood harvested from horseshoe crabs.
  • "We were the first FDA-licensed LAL reagent manufacturer, and we continue our pioneering efforts to be the first to make a full recombinant cascade reagent available to the market.
  • "\nTransitioning to the use of a new recombinant LAL reagent requires some level of validation, irrespective of the article being tested.

Safer medical devices with Picosun's antimicrobial ALD coatings

Retrieved on: 
Friday, February 12, 2021

Numerous ALD oxide coatings deposited with Picosun's processes showed remarkable reduction of microbial growth and had low values of bacterial endotoxin contamination(*).

Key Points: 
  • Numerous ALD oxide coatings deposited with Picosun's processes showed remarkable reduction of microbial growth and had low values of bacterial endotoxin contamination(*).
  • The coatings were characterized by an independent third party laboratory according to ISO 22196:2011 antimicrobial standard and ANSI/AAMI ST72:2019 bacterial endotoxin standards.
  • These results, along with the earlier tests validating the non-cytotoxicity of Picosun's ALD films, prove the safety and aseptic benefits of these materials in medical devices, both implanted and external ones.
  • "The aseptic properties of our ALD films are so excellent that they surpass even the strictest requirements of the medical implant industry.

Safer medical devices with Picosun's antimicrobial ALD coatings

Retrieved on: 
Friday, February 12, 2021

Numerous ALD oxide coatings deposited with Picosun's processes showed remarkable reduction of microbial growth and had low values of bacterial endotoxin contamination(*).

Key Points: 
  • Numerous ALD oxide coatings deposited with Picosun's processes showed remarkable reduction of microbial growth and had low values of bacterial endotoxin contamination(*).
  • The coatings were characterized by an independent third party laboratory according to ISO 22196:2011 antimicrobial standard and ANSI/AAMI ST72:2019 bacterial endotoxin standards.
  • These results, along with the earlier tests validating the non-cytotoxicity of Picosun's ALD films, prove the safety and aseptic benefits of these materials in medical devices, both implanted and external ones.
  • "The aseptic properties of our ALD films are so excellent that they surpass even the strictest requirements of the medical implant industry.

Global VENGLUSTAT Emerging Insight and Market Forecast 2030: Also Known as Ibiglustat - An Investigational Oral Inhibitor of an Enzyme Called Glucosylceramide Synthase (GCS) - ResearchAndMarkets.com

Retrieved on: 
Tuesday, February 9, 2021

The "VENGLUSTAT - Emerging Insight and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "VENGLUSTAT - Emerging Insight and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.
  • Further, it also consists of future market assessments inclusive of the market forecast, SWOT analysis, market competitors, and other emerging therapies.
  • VENGLUSTAT (also known as Ibiglustat) is an investigational oral inhibitor of an enzyme called glucosylceramide synthase (GCS), which leads to the reduction in the formation of glucosylceramide-based glycosphingolipids.
  • VENGLUSTAT is in clinical development for the treatment of conditions caused by lysosomal dysfunction such as Fabry, Gaucher, and Parkinson's diseases.

Passage Bio Receives MHRA Clinical Trial Authorization for PBGM01 for Treatment of GM1 Gangliosidosis

Retrieved on: 
Thursday, December 10, 2020

This is the first regulatory authorization for the global PBGM01 clinical trial program, the Imagine-1 study, for the treatment of infantile GM1, a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available.

Key Points: 
  • This is the first regulatory authorization for the global PBGM01 clinical trial program, the Imagine-1 study, for the treatment of infantile GM1, a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available.
  • Patients with GM1 urgently need treatment options, and we are pleased to have the support of the MHRA as we continue plans to initiate our global clinical trial program for PBGM01, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.
  • It is gratifying to see Passage Bio advancing PBGM01 into clinical trials, said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania (Penn) and chief scientific advisor of Passage Bio.
  • GM1 is a devastating disease for patients and their families, said Gary Romano, M.D., Ph.D., chief medical officer of Passage Bio.