Autosome

Vertex Announces FDA Clearance of Investigational New Drug Application for VX-407 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Retrieved on: 
Thursday, March 21, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, FDA, Clinical Trials, Death, Autosome, VRTX, Growth, Disease, PC1, Patient, Kidney failure, Kidney transplantation, Polycystic kidney disease, IND, Vertex Pharmaceuticals, PKD1, Vertex, ADPKD, Dialysis, Cystic fibrosis, Food, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
  • ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease.
  • ADPKD is a life-shortening genetic kidney disease characterized by the growth of numerous kidney-enlarging cysts that impair kidney function and can ultimately lead to kidney failure, requiring dialysis or kidney transplantation, and premature death.
  • The majority of ADPKD cases are caused by variants in the PKD1 gene, which encodes the polycystin 1 (PC1) protein.

XORTX Submits a New Patent for the Treatment of Chronic Kidney Disease

Retrieved on: 
Wednesday, January 3, 2024
DN, Immunoglobulin A, IgA nephropathy, Kidney disease, Diabetic nephropathy, Uric acid, Trial of the century, Oxipurinol, Therapy, Autosome, Pharmacokinetics, Focal segmental glomerulosclerosis, Patent, Chronic kidney disease, ADPKD, Treating, Study, Polycystic kidney disease, XRX, Safety, Lupus nephritis, XOI, CKD, Pharmaceutical industry

CALGARY, Alberta, Jan. 03, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces submission of a new patent for the treatment of chronic kidney disease (“CKD”).

Key Points: 
  • CALGARY, Alberta, Jan. 03, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces submission of a new patent for the treatment of chronic kidney disease (“CKD”).
  • This patent is designed to protect new discoveries and strategies for the treatment of individuals with varied degrees of kidney function in the setting of CKD.
  • This patent application is intended to claim new opportunities to enhance how the xanthine oxidase inhibitor class of drugs may be dosed in the future.
  • Importantly, how to further improve the safe and effective administration of this class of drugs, including oxypurinol.”

AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations

Retrieved on: 
Monday, November 6, 2023
Biotechnology, Pharmaceutical, Oncology, General Health, Health, FDA, Genetics, Clinical Trials, GRN, IND, Autosome, AAV, Food, Genetic, European Commission, Fast Track, Prevalence, Investigational New Drug, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Apathy, Personality, Dementia, Civil service commission, Abnormal psychology, Frontotemporal dementia, Gene, Brain, Safety, Patient, Death, FTD, Granulin, Pharmaceutical industry

In addition, the FDA has granted Fast Track designation to AVB-101 for the treatment of FTD-GRN to slow disease progression.

Key Points: 
  • In addition, the FDA has granted Fast Track designation to AVB-101 for the treatment of FTD-GRN to slow disease progression.
  • Both the U.S. FDA and the European Commission (EC) granted orphan designation to AVB-101 for the treatment of FTD in 2022.
  • “The IND clearance and Fast Track designation of AVB-101 are important milestones for the FTD community and for our company.
  • Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with frontotemporal dementia with progranulin mutations.

Science Table by Anatomage Brings Real Learning Experiences through Virtual Experiments

Retrieved on: 
Tuesday, October 3, 2023
Lithium, Simulation, Nuclear fission, Learning, Science, Student, Accident, Life, Selection, IB, Light, AP, Eye color, Chemistry, Genetics, NGSS, Diving, Battery, Physics, STEM, Biology, PCR, Autosome, Injury, Tutoring, Nursing

SANTA CLARA, Calif., Oct. 3, 2023 /PRNewswire/ -- Anatomage Inc, an education technology company, today released Science Table by Anatomage, a digital laboratory platform that enables students to perform virtual scientific experiments on a large scale 7 foot-long touch-screen table.

Key Points: 
  • Science Table by Anatomage inspires the next generation of learners with its 60+ virtual experiments in biology, chemistry, and physics.
  • With this in mind, Science Table by Anatomage experiments feature striking visuals bringing real-world examples into the laboratory activities.
  • To enhance student learning experiences, the Science Table by Anatomage platform makes lab experiments - integral to science learning - both safe and odorless, removing risks associated with chemical and biological hazards.
  • Reimagine your science classroom and bring STEM to life with Science Table by Anatomage's virtual experiments.

Poxel Announces its Participation at Upcoming Scientific Conference

Retrieved on: 
Monday, October 2, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, NASH, Adrenoleukodystrophy, Scientific communication, ALD, AMPK, DSM-IV codes, Adenosine, Metabolism, Polycystic kidney disease, Fatty liver disease, R, ADPKD, Pharmacology, Autosome, Pharmaceutical industry, Vaccine, Euronext

POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced that it will participate at the upcoming 12th International Meeting on AMPK on October 4th, 2023.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced that it will participate at the upcoming 12th International Meeting on AMPK on October 4th, 2023.
  • Sophie Bozec, Executive Vice President, R&D Pharmacology and Scientific Communication at Poxel, will host a virtual presentation on “Direct AMPK Activation: From Preclinical to Clinical.
  • Perspectives in Rare Metabolic Diseases”.
  • The presentation will review Poxel’s molecule PXL770, which is a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, focused on rare diseases treatment, including adrenoleukodystrophy (ALD) and autosomal dominant polycystic kidney disease (ADPKD).

Peer-Reviewed Publication of Positive Preclinical Data of SiSaf’s SIS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis

Retrieved on: 
Monday, September 18, 2023
Human, AL, Osteopetrosis, University, RNA, Bone disease, Autosome, Nucleic acid, Messenger, Small RNA, University of L'Aquila, Bone, House, American Society for Bone and Mineral Research, Patient, Annual general meeting, Mouse, Silicon, LNP (newspaper), FDA, Phenotype, IND, Society, Safety, Pharmaceutical industry, Medical imaging, Vaccine

These results could have significant translational impact on bone disease therapies and open the path to human trials of SiSaf’s potentially curative treatment for Osteopetrosis ADO2.

Key Points: 
  • These results could have significant translational impact on bone disease therapies and open the path to human trials of SiSaf’s potentially curative treatment for Osteopetrosis ADO2.
  • There are currently no approved treatments for this debilitating disease and no other treatments currently in clinical trials.
  • SIS-101-ADO, SiSaf’s lead in house program, is an ADO2-specific siRNA specifically designed against the human CLCN7G215R mRNA formulated with the company’s sshLNP.
  • In addition, due to the serious manifestations of this rare skeletal disorder in children, the FDA granted SIS-101-ADO Rare Pediatric Disease Designation for the treatment of Autosomal Dominant Osteopetrosis.

With One-in-Fifty People in the US at Risk, the Brain Aneurysm Foundation Launches New Campaign to Increase Disease Awareness and Accelerate Research

Retrieved on: 
Wednesday, September 6, 2023
Survivor, Fibromuscular dysplasia, Marfan syndrome, Beth Israel Deaconess Medical Center, Education, Prevalence, Blood, Stroke, Policy, Aortic rupture, Skull, Research, Brain, Risk, Pop, Pain, Subarachnoid hemorrhage, Polycystic kidney disease, BAF, Rupture, Patient, Physician, Need, Seizure, Nerve, Nausea, Biology, Diagnosis, City, Headache, Woman, Smoking, Death, Family, Memory, Social media, Autosome, Disease, Ehlers–Danlos syndromes, Pharmaceutical industry, Nursing, Aneurysm

HANOVER, Mass., Sept. 6, 2023 /PRNewswire/ -- The Brain Aneurysm Foundation (BAF), the leading advocacy organization supporting education, research, and policy to transform the treatment of brain aneurysms, today announced the launch of "Stop the Pop," a new campaign to increase awareness of the prevalence and impact of the disease and the need for ongoing investment to fund new innovations, screening, and treatment. 

Key Points: 
  • The campaign aims to build awareness of symptoms, genetic and environmental risk factors, and to increase research funding to better understand the underlying causes of the condition and to advance new treatments.
  • "A feeling that something has 'popped' is what we hear most from patients who have experienced a rupture.
  • A brain aneurysm is a weak, bulging area in an artery in the brain that in some instances can rupture.
  • Women over the age of 55 have a higher risk of brain aneurysm rupture than men and are generally at greater risk.

XORTX Submits Orphan Drug Designation Application to the European Medicines Agency (EMA) to Treat Progressive Kidney Disease

Retrieved on: 
Tuesday, August 29, 2023
Marketing, ODD, European Medicines Agency, Acute kidney injury, COMP, Oxipurinol, Research, Science, Committee, EMA, Polycystic kidney disease, Patient, Hyperuricemia, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Kidney disease, Autosome, Pharmaceutical industry, ADPKD

CALGARY, Alberta, Aug. 29, 2023 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. (“XORTX” or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces that it has submitted an Orphan Drug Designation application for XORLO™ to the European Medicines Agency (the “EMA”). The “orphan-drug designation request is for the use of XORTX’s patented unique proprietary formulation of oxypurinol – XORLO™ – for the treatment of autosomal dominant polycystic kidney disease (ADPKD)”.

Key Points: 
  • CALGARY, Alberta, Aug. 29, 2023 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. (“XORTX” or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces that it has submitted an Orphan Drug Designation application for XORLO™ to the European Medicines Agency (the “EMA”).
  • The “orphan-drug designation request is for the use of XORTX’s patented unique proprietary formulation of oxypurinol – XORLO™ – for the treatment of autosomal dominant polycystic kidney disease (ADPKD)”.
  • The orphan drug designation process (“ODD”) initiated with the submission of this application and is made to the EMA’s COMP (Committee for Orphan Medicinal Products) office.
  • It also follows on receipt of ODD status granted by the U.S. Food and Drug Administration in April 2023.

First Tunisian study examining age of onset for biallelic mutations in Huntington's disease

Retrieved on: 
Sunday, August 27, 2023
HD, Huntington's disease, Zygosity, University, HTT, Disease, Allele, Huntingtin, Mutation, Doctor of Philosophy, CAG, Movement, Patient, University of Lisbon, Parkinson's disease, International Congress on Tuberculosis, Autosome, Pharmaceutical industry, Vaccine

This Tunisian cohort study reported that the age of disease onset for patients with a homozygous mutation in the Huntingtin gene (HTT) did not differ significantly from that of heterozygous patients.

Key Points: 
  • This Tunisian cohort study reported that the age of disease onset for patients with a homozygous mutation in the Huntingtin gene (HTT) did not differ significantly from that of heterozygous patients.
  • The age of disease onset between the homozygous and heterozygous patients did not differ significantly.
  • Patients with smaller all were associated with a later age of onset, 62 years for homozygous and 74 years for heterozygous.
  • The number of CAG repeats and age of disease onset was shown to be inversely correlated for homozygous patients.

Poxel Announces Upcoming Participation at the H.C. Wainwright 2nd Annual Kidney Conference

Retrieved on: 
Tuesday, July 18, 2023
Research, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Other Science, Science, NASH, AMPK, DSM-IV codes, Adenosine, Polycystic kidney disease, CEST, Fatty liver disease, ADPKD, Conference, Autosome, Pharmaceutical industry, Cryptocurrency, Euronext

POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its participation at the upcoming H.C. Wainwright 2nd Annual Kidney Conference , to be held virtually on July 25, 2023.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its participation at the upcoming H.C. Wainwright 2nd Annual Kidney Conference , to be held virtually on July 25, 2023.
  • Thomas Kuhn, CEO, and members of the management team of Poxel, will be available for one-on-one virtual meetings on July 25, 2023.
  • The Company’s virtual presentation is scheduled the same day at 9:30 am ET (3.30 pm CEST) and will focus on PXL770, a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, for the treatment of autosomal dominant polycystic kidney disease (ADPKD).