Cytoplasm

Sana Biotechnology to Present Data from Multiple Preclinical Studies at the American Society of Gene and Cell Therapy 25th Annual Meeting

Retrieved on: 
Monday, May 2, 2022
Social disruption, Gene, CAR, COVID-19, Drug development, Immune system, Sana, Company, Cell, Biotechnology, Patient, Immunosuppression, Risk, Annual report, Cytoplasm, CD19, Chronic lymphocytic leukemia, Acute leukemia, Potency (pharmacology), Society, Terminology, Non-Hodgkin lymphoma, Technology, Research, Cancer, Vaccine

Preclinical data demonstrates across a variety of cell types that these transplanted allogeneic cells are able to evade both the innate and adaptive arms of the immune system while retaining their activity.

Key Points: 
  • Preclinical data demonstrates across a variety of cell types that these transplanted allogeneic cells are able to evade both the innate and adaptive arms of the immune system while retaining their activity.
  • Sana is developing re-targetable fusogens as a platform technology to enable the in vivo delivery of genetic payloads to specific cell types.
  • We have shown in preclinical studies that we can engineer fusogens to specifically target diverse cell surface receptors that allow cell-specific delivery across multiple different cell types.
  • Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements for any reason.

Windtree Therapeutics Announces Late-Breaker Presentation on Phase 2 SEISMiC Study Results of Istaroxime in Early Cardiogenic Shock at the European Society of Cardiology (ESC) Heart Failure Conference

Retrieved on: 
Wednesday, April 20, 2022
Cardiogenic shock, European Society of Cardiology, Acute respiratory distress syndrome, U.S. Securities and Exchange Commission, COVID-19, Security (finance), GLOBE, Patient, Sarcoplasmic reticulum, Blood, Technology, Efficacy, Hypertension, Blood pressure, Study, FDA, University, Therapy, Annual report, Arrhythmia, AHF, Private Securities Litigation Reform Act, Cardiology, WINT, Conference, Science, Mortality, SBP, Company, Radiology, Marketing, Kidney, SCAI, Department, Surgery, Growth, Heart, Principal, Safety, Prevalence, HK, Medicine, Heart rate, Brain, Form 10-K, Cytoplasm, Oxygen, Risk, ADS, Institute, Seismology, Public health, Instability, Pharmaceutical industry, Medical device, Istaroxime

At the late-breaker presentation, Dr. Metra will present additional data and details from the Study.

Key Points: 
  • At the late-breaker presentation, Dr. Metra will present additional data and details from the Study.
  • Cardiogenic shock is a serious condition that occurs when the heart is failing significantly and cannot pump enough blood and oxygen to the brain, kidneys, and other vital organs.
  • Windtree Therapeutics, Inc.is advancing multiple late-stage interventions for acute cardiovascular and acute pulmonary disorders to treat patients in moments of crisis.
  • Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release.

Windtree Announces Positive Topline Results from Its Phase 2 SEISMiC Study of Istaroxime in Early Cardiogenic Shock

Retrieved on: 
Wednesday, April 20, 2022
HK, Cardiogenic shock, Annual report, CEO, Arrhythmia, Acute respiratory distress syndrome, Marketing, Â, U.S. Securities and Exchange Commission, COVID-19, Heart failure, Security (finance), Istaroxime, Heart rate, AHF, Kidney, Private Securities Litigation Reform Act, Brain, SCAI, GLOBE, Program, WINT, Patient, Form 10-K, Sarcoplasmic reticulum, Growth, Blood, Mortality, SBP, Technology, Company, Cytoplasm, Mercury (element), Seismology, European Society of Cardiology, Safety, Oxygen, Blood pressure, Hypertension, FDA, Prevalence, Risk, ADS, Senior, Therapy, Instability, Pharmaceutical industry, Medical imaging, Medical device, Heart

The SEISMiC Phase 2 study in early cardiogenic shock is an international, randomized, double blind, placebo- controlled study enrolling 60 patients with Society for Cardiovascular Angiography & Interventions (SCAI) stage B early cardiogenic shock due to severe heart failure with systolic blood pressures (SBP) between 75-90 mmHg.

Key Points: 
  • The SEISMiC Phase 2 study in early cardiogenic shock is an international, randomized, double blind, placebo- controlled study enrolling 60 patients with Society for Cardiovascular Angiography & Interventions (SCAI) stage B early cardiogenic shock due to severe heart failure with systolic blood pressures (SBP) between 75-90 mmHg.
  • Study drug was infused for 24 hours in a 1:1 randomization to placebo or istaroxime.
  • Two istaroxime doses were evaluated, 1.5 g/kg/min in the first group and 1.0 g/kg/min in the next group.
  • Craig Fraser, President and CEO of Windtree stated, It is worth noting the area of cardiogenic shock is complementary to our AHF program.

Vesigen Therapeutics Appoints Paulash Mohsen as Chief Executive Officer

Retrieved on: 
Monday, April 11, 2022
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Infectious Diseases, Therapy, Amyotrophic lateral sclerosis, Cell, Merck & Co., Virus, Cubist Pharmaceuticals, Cytoplasm, Horizontal Environmental Genetic Alteration Agents, Chemical engineering, Intrinsic termination, ARMM Regional Legislative Assembly, Board, RNA, Brown University, Biotechnology, Doctor of Philosophy, Yuman–Cochimí languages, Harvard University, Harvard T.H. Chan School of Public Health, Tissue, Public health, Exosome, Seminal vesicles, Technology, Pfizer, Massachusetts Institute of Technology, Protein, Cubism, Physiology, Business operations, RNA transfection, MBA, Harvard Business School, HIV, Enzyme, Genetics, Optimer Pharmaceuticals, Acquisition, Patient, Pharmaceutical industry, Vaccine, Merck

Vesigen Therapeutics, Inc., a biotechnology company pioneering a novel extracellular vesicle delivery technology, ARMMs, today announced the appointment of Paulash Mohsen as Chief Executive Officer.

Key Points: 
  • Vesigen Therapeutics, Inc., a biotechnology company pioneering a novel extracellular vesicle delivery technology, ARMMs, today announced the appointment of Paulash Mohsen as Chief Executive Officer.
  • On behalf of our Board and management team, we welcome Paulash and look forward to his executive leadership.
  • Mr. Mohsen is a seasoned biopharmaceutical executive who has served in a variety of roles across multiple disciplines.
  • Prior to his appointment at Vesigen, he served as Chief Business Officer at Yumanity Therapeutics, and helped the company grow from a seed stage, research-based startup to a public company, with a lead clinical program in Parkinsons disease.

Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease

Retrieved on: 
Thursday, March 24, 2022
FDA, Health, Research, Pharmaceutical, Science, Biotechnology, Disease, Cell, Cholesterol, NPC1, Niemann–Pick disease, Therapy, Metabolic disorder, Cytoplasm, GCS, Trial of the century, Clinical trial, Tay–Sachs disease, ODD, Liver, Lists of diseases, Spleen, GM2 gangliosidoses, GBA2, Food, Glycogen storage disease, Glycosphingolipid, NPC, NP-C, Tremor, NPC2, US Food Sovereignty Alliance, Brain, Leiden University, NP, GM2, Courage, Enzyme, FDA, Protein, Patient, GM1, Pharmaceutical industry

Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in Niemann-Pick disease type C (NP-C).

Key Points: 
  • Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in Niemann-Pick disease type C (NP-C).
  • The compound already received ODD from the FDA for GM2 Gangliosidosis including both Sandhoff and Tay-Sachs diseases.
  • Azafaros completed a first-in-human clinical trial with AZ-3102 in healthy volunteers in 2021 and received Orphan Drug Designation in GM2 Gangliosidosis from the FDA in February 2022.
  • Azafaros is supported by a syndicate of leading Dutch and Swiss investors including Forbion, BioGeneration Ventures, BioMedPartners and Schroder Adveq.

Windtree Completes Enrollment of Phase 2 Study of Istaroxime in Early Cardiogenic Shock

Retrieved on: 
Tuesday, March 15, 2022
Therapy, Annual report, Heart, Mortality, MD, CMO, Program, Technology, Blood pressure, GLOBE, WINT, Patient, Heart failure, Istaroxime, Oxygen, Private Securities Litigation Reform Act, FDA, Kidney, Risk, COVID-19, Heart rate, ADS, Hypertension, Cardiogenic shock, Safety, Security (finance), Cytoplasm, Brain, Sarcoplasmic reticulum, Instability, Form 10-K, HK, Cardiology, Mercury (element), Acute respiratory distress syndrome, Company, Research, CEO, SBP, SCAI, Blood, Arrhythmia, Growth, AHF, U.S. Securities and Exchange Commission, Marketing, Pharmaceutical industry, Medical device

The study is an international, randomized double-blind, placebo-controlled study designed to assess the efficacy and safety of istaroxime and to support an intended pathway for the development in early cardiogenic shock.

Key Points: 
  • The study is an international, randomized double-blind, placebo-controlled study designed to assess the efficacy and safety of istaroxime and to support an intended pathway for the development in early cardiogenic shock.
  • The study has enrolled 60 Society for Cardiovascular Angiography & Interventions (SCAI) class B early cardiogenic shock patients with severe heart failure (30 assigned to istaroxime and 30 assigned to placebo) and systolic blood pressures (SBP) between 75-90 mmHg.
  • We are very pleased to have completed enrollment in the study of istaroxime in early cardiogenic shock due to heart failure.
  • Market research revealed 99% of 100 U.S.-based clinical cardiologists interviewed who treat cardiogenic shock patients responded that new drug innovation to treat SCAI class B cardiogenic shock patients is highly needed.

Key Opinion Leaders in mRNA Therapeutics and Vaccines - ResearchAndMarkets.com

Retrieved on: 
Friday, February 18, 2022
Health, Pharmaceutical, History, Patient, Lists of diseases, RNA transfection, Genome, Pfizer–BioNTech COVID-19 vaccine, RNA, Safety, Therapy, Technology, Vaccine, Paramount, Research, COVID-19, Cytoplasm

The "mRNA Therapeutics and Vaccines: Key Opinion Leaders" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "mRNA Therapeutics and Vaccines: Key Opinion Leaders" report has been added to ResearchAndMarkets.com's offering.
  • In fact, currently, there are more than 65 mRNA-based therapeutics and over 85 mRNA vaccines under development.
  • In vitro transcribed mRNA is now being evaluated as a treatment option for several oncological and genetic disorders.
  • Synthetic mRNA can also be engineered to facilitate the in vitro development of several types of cell and gene therapies.

Curve Therapeutics Announces Collaboration with MSD for Next Generation Drug Discovery Platform

Retrieved on: 
Wednesday, February 16, 2022
MSD, Merck & Co., Merck, Department, Doctor of Philosophy, Therapy, Technology, NJ, Protein, University, Princeton University Department of Chemistry, Research, Drug discovery, MBA, Cytoplasm, Pharmaceutical industry, Generic drug, Fine chemical, Curve

Simon Kerry, PhD, MBA, Chief Executive Officer of Curve, said: "This collaboration is a major milestone for Curve and an important endorsement of our ground-breaking drug discovery platform.

Key Points: 
  • Simon Kerry, PhD, MBA, Chief Executive Officer of Curve, said: "This collaboration is a major milestone for Curve and an important endorsement of our ground-breaking drug discovery platform.
  • Curve's novel, proprietary platform enables the direct discovery of biologically active molecules against targets that have been difficult to address using conventional drug discovery methods.
  • Curve is pioneering a game-changing, functional, drug discovery engine to generate higher quality hits and leads with the aim of discovering first-in-class therapeutics.
  • Curve's platform enables the discovery of biologically active molecules against targets that have evaded conventional drug discovery techniques.

Curve Therapeutics Announces Collaboration with MSD for Next Generation Drug Discovery Platform

Retrieved on: 
Wednesday, February 16, 2022
MSD, Merck & Co., Merck, Department, Doctor of Philosophy, Therapy, Technology, NJ, Protein, University, Princeton University Department of Chemistry, Research, Drug discovery, MBA, Cytoplasm, Pharmaceutical industry, Generic drug, Fine chemical, Curve

SOUTHAMPTON, England, Feb. 16, 2022 /PRNewswire/ -- Curve Therapeutics (Curve), a private biotechnology company pioneering a potentially game-changing, functional drug discovery platform, today announces a global research collaboration with MSD, the trade name of Merck & Co., Inc., Kenilworth, NJ USA, to discover and validate modulators of up to five therapeutic targets using its Microcycle® technology, initially for oncology and neurology indications.

Key Points: 
  • Simon Kerry, PhD, MBA, Chief Executive Officer of Curve, said: "This collaboration is a major milestone for Curve and an important endorsement of our ground-breaking drug discovery platform.
  • Curve's novel, proprietary platform enables the direct discovery of biologically active molecules against targets that have been difficult to address using conventional drug discovery methods.
  • Curve is pioneering a game-changing, functional, drug discovery engine to generate higher quality hits and leads with the aim of discovering first-in-class therapeutics.
  • Curve's platform enables the discovery of biologically active molecules against targets that have evaded conventional drug discovery techniques.

DGAP-News: Atriva receives FDA Orphan Drug Designation for Zapnometinib to treat Hantavirus Infections

Retrieved on: 
Monday, January 10, 2022
Safety, CDC, Viral hemorrhagic fever, RSV, RNP, Risk, Macrophage inflammatory protein, Severe acute respiratory syndrome coronavirus 2, Viral load, Death, Orthohantavirus, CSO, Patient, Orthornavirae, ODD, Chemokine, Gene expression, Category, Orphan drug, Hantavirus pulmonary syndrome, RNA, III, Food, Patent, ECDC, Therapy, Degenerative disease, Cell, Company, Cytoplasm, Cytokine, Human, Vaccine, CEO, COVID-19, FDA, Disease, Gesellschaft mit beschränkter Haftung, Orphan, Fatality, Hantavirus hemorrhagic fever with renal syndrome, HPS, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, MEK, Pharmaceutical industry, HFRS, zapnometinib's mode of action with dual effect, Atriva Therapeutics GmbH, ZAPNOMETINIB'S MODE OF ACTION WITH DUAL EFFECT, ATRIVA THERAPEUTICS GMBH

"Antiviral efficacy of zapnometinib against hantavirus has been previously proven in vitro in an academic collaboration.

Key Points: 
  • "Antiviral efficacy of zapnometinib against hantavirus has been previously proven in vitro in an academic collaboration.
  • Dr. Rainer Lichtenberger, CEO of Atriva Therapeutics, commented: "The Orphan Drug Designation we received for our lead drug candidate zapnometinib is an important milestone towards addressing the urgent needs of patients infected with hantavirus.
  • [1]
    The FDA may grant Orphan Drug Designation (ODD) to support the development and evaluation of new treatments for rare diseases.
  • [11]
    The Atriva lead product zapnometinib (ATR-002) is developed specifically to treat diseases such as influenza and COVID-19, caused by RNA viruses.