Organ dysfunction

Ayala Pharmaceuticals Announces AL102 Receives Orphan Drug Designation for Desmoid Tumors

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Monday, November 6, 2023
Aggressive fibromatosis, FDA, Organ dysfunction, Incidence, Food, Chronic pain, OTCQX, DT, Head, Plantar fibromatosis, Quality of life, Abdominal wall, Medicine, Patient, Food and Drug Administration, Diagnosis, GSI, Plasma protein binding, Pharmaceutical industry

REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AL102, a Gamma Secretase Inhibitor (GSI), for the treatment of desmoid tumors (DT).

Key Points: 
  • REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AL102, a Gamma Secretase Inhibitor (GSI), for the treatment of desmoid tumors (DT).
  • Orphan drug designation is granted by the FDA to drugs and biologics intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation.
  • Under the FDA’s Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval.
  • “Receiving FDA orphan drug status for AL102 underscores the significant unmet need for novel treatment options for people living with desmoid tumors.

Prothena Reports Third Quarter 2023 Financial Results and Business Highlights

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Thursday, November 2, 2023
Biotechnology, Pharmaceutical, Health, Amyloid, Survival, FDA, Bristol Myers Squibb, Research, Organ dysfunction, AD, IND, PD, Food, VITAL, ATTR, Therapy, Fast track (FDA), AL, Mortality, G&A, Novo Nordisk, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Acquisition, European Medicines Agency, American Society of Hematology, MAD, SAD, Orphan, R, TTR, ASH, Clinical trial, Biology, Risk, Alpha-synuclein, Epitope, Amyloidosis, Society, SPA, Patient, Failure, Pharmaceutical industry, Vaccine, Fine chemical, Security (finance), Blood

Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the third quarter and first nine months of 2023 and provided business highlights.

Key Points: 
  • Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the third quarter and first nine months of 2023 and provided business highlights.
  • Robust reduction of brain amyloid plaque has been demonstrated to likely predict clinical benefit for people with early AD.
  • The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for PRX012 for the treatment of AD.
  • As of September 30, 2023, Prothena had $673.1 million in cash, cash equivalents and restricted cash, and no debt.

eGenesis Presents Preclinical Data Demonstrating Recipient Survival and Compatibility with Genetically Engineered Porcine Kidneys at 2023 IPITA-IXA-CTRMS Joint Conference

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Thursday, October 26, 2023
Massachusetts General Hospital, Survival, RNA, Antigen, CRISPR, Cell Transplantation, Organ dysfunction, Kidney, Cell, Pelvic floor dysfunction, Transgene, System, Organ transplantation, Risk, Congress, Complement, Gene, Endothelium, Transplant, Patient, NHP, Ischemia, Pharmaceutical industry, Dentistry, Vaccine, Nature

Two presentations will highlight data from the company’s landmark preclinical proof of concept study for kidney transplant recently published in Nature.

Key Points: 
  • Two presentations will highlight data from the company’s landmark preclinical proof of concept study for kidney transplant recently published in Nature.
  • A third presentation will share the results of a study evaluating pancreatic islets from genetically engineered porcine donors transplanted into a mouse model.
  • “We’re thrilled to share data highlighting our lead candidate for kidney transplant, EGEN-2784, as well as additional data supporting our genome engineering platform.
  • Humanized porcine donors were genetically engineered using the Yucatan miniature pig breed to carry 69 genomic edits through CRISPR/Cas 9 technology.

Fapon, LASCCO, Abionic Formed Strategic Collaboration for PSP Sepsis Diagnosis in China

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Thursday, October 26, 2023
PSP, Survival, Patient, FDA, Research, C-reactive protein, Organ dysfunction, Fatigue, IVD, Biomarker, Ophthalmology, Septic shock, Immunoassay, Physician, Mortality, Septic, Obstetrics, IVDR, CE, Infection, Incidence, POC, Nausea, Metabolism, CLIA, Sepsis, Fever, Acute inhalation injury, Disease, Peritonitis, Medicine, Partnership, Burn, Neurology, Blood, Chills, CRP, Pain, Diagnosis, Death, Trauma, ICU, Medical imaging, Pharmaceutical industry, Silviculture, Hospital

This strategic partnership will usher in a significant breakthrough in the medical field, removing barriers to early sepsis diagnosis.

Key Points: 
  • This strategic partnership will usher in a significant breakthrough in the medical field, removing barriers to early sepsis diagnosis.
  • Performing PSP testing on high-risk individuals can flag potential sepsis before the onset of clinical symptoms.
  • However, these methods cannot meet the clinical needs for early diagnosis of sepsis as they have major limitations.
  • Obtaining the PSP license for the diagnosis of sepsis will further enhance Fapon's potential for innovative applications in the diagnosis of infectious diseases.

eGenesis Announces Publication in Nature of Landmark Preclinical Data Demonstrating Long-Term Survival with Genetically Engineered Porcine Kidneys

Retrieved on: 
Wednesday, October 11, 2023
Xenotransplantation, Engineering, Biomarker, Hope, Inflammation, Genome, Endothelium, Risk, Data, Massachusetts General Hospital, Survival, Coagulation, Transplant, Hospital, Patient, Organ dysfunction, Pelvic floor dysfunction, Gene, Harvard Medical School, Transgene, NHP, Human, Filtration, Vaccine, Dentistry, Nature

CAMBRIDGE, Mass., Oct. 11, 2023 (GLOBE NEWSWIRE) -- eGenesis, a biotechnology company developing human-compatible (HuCo™) organs for the treatment of organ failure, today announced publication in the journal Nature of long-term survival data from a proof-of-concept study evaluating engineered porcine donor kidneys transplanted into a cynomolgus macaque model. This dataset will support advancement of the company’s lead candidate for kidney transplant, EGEN-2784, toward clinical development.

Key Points: 
  • These results represent the largest and most comprehensive preclinical dataset published in the field to date.
  • Recipient survival in the preclinical setting has historically been measured in weeks or months.
  • The publication titled: “ Design and Testing of a Humanized Porcine Donor for Xenotransplantation ,” reported long-term survival of NHP recipients of the company’s genetically engineered porcine kidneys.
  • “At eGenesis, we are focused on transformational progress for the field – improving long-term survival for transplant recipients from months to years,” said Michael Curtis, Ph.D., Chief Executive Officer of eGenesis.

Novel classification of allergic disorders published by the European Academy of Allergy and Clinical Immunology: SIAF

Retrieved on: 
Tuesday, October 10, 2023
Medicine, European Academy of Allergy and Clinical Immunology, EAACI, Research, Health, Classification, Patient, Education, Nanotechnology, Wrocław Medical University, Tissue, Swiss Institute of Allergy and Asthma Research, Skin, Human, Allergy, Infection, Organ dysfunction, Medical device, Vaccine

DAVOS, Switzerland, Oct. 10, 2023 /PRNewswire/ -- The revision of the current allergic disease nomenclature based on symptoms and organ dysfunction has been long-awaited at the time of modern patient-tailored treatments and precision medicine. The new classification is based on disease mechanisms, thus facilitating targeted and personalised disease management.

Key Points: 
  • The new classification is based on disease mechanisms, thus facilitating targeted and personalised disease management.
  • The world's key opinion leaders gathered around the initiative of the European Academy of Allergy and Clinical Immunology (EAACI) to present a new classification of allergic disorders based on the mechanisms of diseases.
  • Published online today in Allergy, the position paper reveals the new way allergic diseases are perceived.
  • We expect the new classification to  profoundly change the healthcare professionals' approach to managing allergic diseases since it provides key solutions to a personalised approach.

Novel classification of allergic disorders published by the European Academy of Allergy and Clinical Immunology: SIAF

Retrieved on: 
Tuesday, October 10, 2023
Medicine, European Academy of Allergy and Clinical Immunology, EAACI, Research, Health, Classification, Patient, Education, Nanotechnology, Wrocław Medical University, Tissue, Swiss Institute of Allergy and Asthma Research, Skin, Human, Allergy, Infection, Organ dysfunction, Medical device, Vaccine

DAVOS, Switzerland, Oct. 10, 2023 /PRNewswire/ -- The revision of the current allergic disease nomenclature based on symptoms and organ dysfunction has been long-awaited at the time of modern patient-tailored treatments and precision medicine. The new classification is based on disease mechanisms, thus facilitating targeted and personalised disease management.

Key Points: 
  • The new classification is based on disease mechanisms, thus facilitating targeted and personalised disease management.
  • The world's key opinion leaders gathered around the initiative of the European Academy of Allergy and Clinical Immunology (EAACI) to present a new classification of allergic disorders based on the mechanisms of diseases.
  • Published online today in Allergy, the position paper reveals the new way allergic diseases are perceived.
  • We expect the new classification to  profoundly change the healthcare professionals' approach to managing allergic diseases since it provides key solutions to a personalised approach.

EQS-News: Adrenomed presents new findings on its precision medicine treatment for septic shock with enibarcimab during Weimar Sepsis Update

Retrieved on: 
Saturday, September 16, 2023
ADM, DPP3, Research and development, Sepsis, Radboud University Medical Center, Septic, Organ dysfunction, Biomarker, Septic shock, Intensive care medicine, Plasma, Patient, Risk, Sir Ewan Forbes, 11th Baronet, Medicine, Mortality, Nursing care plan, Congress, Conference, Pharmaceutical industry

Adrenomed is preparing a confirmatory trial

Key Points: 
  • Adrenomed is preparing a confirmatory trial
    Enibarcimab is targeting loss of vascular integrity, a previously unaddressed pathophysiological mechanism [1]
    Adrenomed presenting on individualized precision medicine treatment at the World Sepsis Day Event, Berlin, September 12 [2]
    Hennigsdorf/Berlin (Germany), September 8, 2023 – Adrenomed AG, the vascular integrity company, today announced new findings on the biomarker-guided treatment of septic shock with enibarcimab.
  • New data analyses of the phase II clinical trial AdrenOSS-2 validate the precision medicine approach applied by Adrenomed in the development of its non-neutralizing antibody enibarcimab for the treatment of septic shock.
  • They underpin Adrenomed’s hypothesis that there is an interplay between baseline DPP3 levels and treatment effects of enibarcimab in patients with septic shock.
  • The AdrenOSS-2 trial has marked an important step forward in bringing our precision medicine approach to patients.

Adrenomed presents new findings on its precision medicine treatment for septic shock with enibarcimab during Weimar Sepsis Update

Retrieved on: 
Friday, September 8, 2023
ADM, DPP3, Research and development, Sepsis, Radboud University Medical Center, Septic, Organ dysfunction, Biomarker, Septic shock, Intensive care medicine, Plasma, Patient, Risk, Medicine, Mortality, Poster, Nursing care plan, Congress, Conference, Medical device, Pharmaceutical industry

Adrenomed is preparing a confirmatory trial

Key Points: 
  • Adrenomed is preparing a confirmatory trial
    Enibarcimab is targeting loss of vascular integrity, a previously unaddressed pathophysiological mechanism1
    Adrenomed presenting on individualized precision medicine treatment at the World Sepsis Day Event, Berlin, September 122
    HENNIGSDORF, Germany and BERLIN, Sept. 08, 2023 (GLOBE NEWSWIRE) -- Adrenomed AG, the vascular integrity company, today announced new findings on the biomarker-guided treatment of septic shock with enibarcimab.
  • New data analyses of the phase II clinical trial AdrenOSS-2 validate the precision medicine approach applied by Adrenomed in the development of its non-neutralizing antibody enibarcimab for the treatment of septic shock.
  • They underpin Adrenomed’s hypothesis that there is an interplay between baseline DPP3 levels and treatment effects of enibarcimab in patients with septic shock.
  • The AdrenOSS-2 trial has marked an important step forward in bringing our precision medicine approach to patients.

Preeclampsia Foundation-funded Genetic Research Study Finds Two Distinct Molecular "Faces" of Preeclampsia

Retrieved on: 
Tuesday, August 29, 2023
HELLP, Pregnancy, Placenta, Rambam Health Care Campus, Fetus, Organ dysfunction, Research, MD Anderson Cancer Center, Inflammation, RNA, Doctor of Philosophy, Late-onset hypogonadism, Pre-eclampsia, HELLP syndrome, MD, Patient, Hypertension, Place of birth, Infant, Growth, Death, Gene, Disease, Pharmaceutical industry, Vaccine, Technion – Israel Institute of Technology

For this study, single-cell transcriptomic research measured tens of thousands of placental cells, one cell at a time, using single-cell RNA sequencing.

Key Points: 
  • For this study, single-cell transcriptomic research measured tens of thousands of placental cells, one cell at a time, using single-cell RNA sequencing.
  • "This is important because it means there is a real molecular basis for two forms of preeclampsia, and that treatments will need to be tailored to these distinct subtypes."
  • "For example, the role of inflammation has been well described in preeclampsia research, but not fully understood," said Zeisel.
  • "We are looking forward to the continued growth of research stemming from this important finding related to preeclampsia," said Clement Pappas.