Vaso-occlusive crisis

bluebird bio Announces FDA Approval of LYFGENIA™ (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

Retrieved on: 
Friday, December 8, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Red blood cell, Sickle cell disease, Patient, Anemia, Quality of life, Bangladesh Technical Education Board, Qualification, Webcast, Priority review, Lung, Vasculitis, Food, Incidence, Pain, Priapism, Acute myeloid leukemia, Bluebird, PIN, Genetic disorder, Regenerative medicine, Division, Ronas Voe, DSM-IV codes, Hematology, Vaso-occlusive crisis, Courage, Adolescence, Hemolytic anemia, Stomatitis, Cellular, Febrile neutropenia, History, Leukopenia, Thrombocytopenia, Neutropenia, University, QTC, Life, NHLBI, Bone marrow, Research, HBA, Bluebird bio, Telephone, Disease, Acute chest syndrome, Caregiver, NIH, MD, Blood transfusion, Pharmaceutical industry, Vaccine, Online shopping

LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Key Points: 
  • LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.
  • “LYFGENIA has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events.
  • The burden associated with VOEs is pervasive and can affect every aspect of life for patients and their families and caregivers.
  • A replay of the webcast will be available on the bluebird website for 90 days following the event.

Global Vaso Occlusive Crisis in Sickle Cell Disease Pipeline Drugs and Companies 2021 Report - ResearchAndMarkets.com

Retrieved on: 
Monday, February 21, 2022
Health, Pharmaceutical, Partnership, Vaso-occlusive crisis, Clinical trial, Company, Anti-transglutaminase antibodies, IV, Industry, Phase, News, Marketing, Sickle cell disease, Mall of America, Pharmaceutical industry, Vaso

The Vaso Occlusive Crisis in Sickle Cell Disease pipeline study analyzes and monitors the development landscape with the current status of each of the pipeline drugs under development for Vaso Occlusive Crisis in Sickle Cell Disease.

Key Points: 
  • The Vaso Occlusive Crisis in Sickle Cell Disease pipeline study analyzes and monitors the development landscape with the current status of each of the pipeline drugs under development for Vaso Occlusive Crisis in Sickle Cell Disease.
  • The drug development stage for all Vaso Occlusive Crisis in Sickle Cell Disease pipeline candidates is provided in the report enables you to understand timetable developments in Vaso Occlusive Crisis in Sickle Cell Disease therapeutic area.
  • This chapter in Vaso Occlusive Crisis in Sickle Cell Disease preclinical and clinical trials assists in gaining clear understanding of the timetable developments in Vaso Occlusive Crisis in Sickle Cell Disease product area.
  • Gain clear understanding of the Vaso Occlusive Crisis in Sickle Cell Disease drugs through our expert coverage and in-depth analysis of drugs, companies and market trends
    Gain real-time insights into the global Vaso Occlusive Crisis in Sickle Cell Disease pipeline news, developments and insights

Forma Therapeutics Reports Second Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Friday, August 13, 2021
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Thrombin, Prostate, AR, Biomarker, Research, RBC, Sickle cell disease, Transfusion-dependent anemia, Growth, Safety, Therapy, ASCO, Administration, CR, Man, Thalassemia, Research and development, CPB, Food, LinkedIn, Private Securities Litigation Reform Act, MAD, Cancer, IND, SEC, FDA, NCI, Thrombophilia, EDT, Congress, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Glioma, Population, Hemolysis, Degenerative disease, Goal, OLE, COVID-19, U.S. Securities and Exchange Commission, Tumor necrosis factor, NDA, Insurance, Conference, SCD, Prostate cancer, LDH, CRH, Hematology, Trial of the century, Inflammation, CMC, OS, Risk, Publication, Survival, Vaso-occlusive crisis, Webcast, Health, Patient, PSA, EHA, POS, Nasdaq, AML, R, Pharmaceutical industry, Medical imaging

Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today reported financial results for the second quarter ended June 30, 2021.

Key Points: 
  • Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today reported financial results for the second quarter ended June 30, 2021.
  • In January 2021, Forma announced the first patient dosed in the ongoing Phase 1 clinical trial evaluating FT-7051 for the treatment of mCRPC.
  • Net Loss: Net loss was $43.6 million for the quarter ended June 2021, compared to net loss of $25.4 million for the quarter ended June 30, 2020.
  • Forma will conduct a conference call and webcast Aug.13 at 8:00 a.m. Eastern Daylight Time (EDT) to discuss second quarter 2021 results and business updates.

GlycoMimetics to Present at Upcoming H.C. Wainwright & Co Annual Global Investment Virtual Conference

Retrieved on: 
Tuesday, September 8, 2020
Biotechnology, Pharmaceutical, Health, Proteins, Branches of biology, Integral membrane proteins, Selectins, Sickle cell disease, Vaso-occlusive crisis, E-selectin, CXCR4, Lluís Companys

GlycoMimetics, Inc. (Nasdaq: GLYC) today announced that Chief Executive Officer Rachel King will present at the upcoming H.C. Wainwright & Co 22nd Annual Global Investment Virtual Conference taking place September 14-16, 2020.

Key Points: 
  • GlycoMimetics, Inc. (Nasdaq: GLYC) today announced that Chief Executive Officer Rachel King will present at the upcoming H.C. Wainwright & Co 22nd Annual Global Investment Virtual Conference taking place September 14-16, 2020.
  • The presentation will be available on the companys website at the Investors tab for 30 days, beginning Monday, September 14 at 2:30 p.m. EDT.
  • Rivipansel, a pan-selectin antagonist, is being explored as a potential treatment for acute vaso-occlusive crisis in sickle cell disease.
  • GlycoMimetics has also completed a Phase 1 clinical trial with another wholly-owned drug candidate, GMI-1359, a combined CXCR4 and E-selectin antagonist.

Sickle Cell Disease Association of America, Inc. and Hemanext® Inc. Form New Strategic Partnership

Retrieved on: 
Wednesday, May 6, 2020
Medical specialties, Branches of biology, Medicine, Health in Africa, Hematopathology, RTT, Sickle cell disease, Crizanlizumab, Vaso-occlusive crisis

Sickle cell disease (SCD) affects 100,000 individuals in the United States, disproportionately affecting African Americans with one in 500 African Americans suffering from the disease.

Key Points: 
  • Sickle cell disease (SCD) affects 100,000 individuals in the United States, disproportionately affecting African Americans with one in 500 African Americans suffering from the disease.
  • Acute pain and vaso-occlusive crises (VOCs) are the top causes for hospitalizations and Emergency Department presentations, respectively, for sickle cell disease patients.
  • "While much has been accomplished on behalf of sickle cell disease patients, we understand that more work remains in order to fully serve people living with SCD.
  • It is a privilege to partner with the association on behalf of people living with sickle cell disease."

Nicox’s Partner Fera Pharmaceuticals Files Application for Orphan Drug Designation for Naproxcinod in Sickle-Cell Disease

Retrieved on: 
Thursday, April 2, 2020
Nonsteroidal anti-inflammatory drugs, Medical specialties, Organ systems, Naproxcinod, Organic compounds, NicOx, COX-inhibiting nitric oxide donator, Sickle cell disease, Naproxen, Vaso-occlusive crisis, Anti-inflammatory

Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, and Fera Pharmaceuticals, a privately-held, U.S. specialty pharmaceutical company, announced today that Fera has filed an application with the U.S. Food and Drug Administration (FDA) for an Orphan Drug Designation (ODD) for naproxcinod in sickle-cell disease.

Key Points: 
  • Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, and Fera Pharmaceuticals, a privately-held, U.S. specialty pharmaceutical company, announced today that Fera has filed an application with the U.S. Food and Drug Administration (FDA) for an Orphan Drug Designation (ODD) for naproxcinod in sickle-cell disease.
  • Following results from in vivo primary pharmacodynamics studies of naproxcinod in models of sickle-cell disease, Fera has decided to focus its development on the treatment of painful vaso-occlusive crisis in sickle-cell disease.
  • Naproxcinod, a Cyclooxygenase-Inhibiting Nitric Oxide (NO)-Donating (CINOD) naproxen, is a non-steroidal anti-inflammatory product candidate engineered to release NO, originally discovered and developed by Nicox.
  • Nicox and Fera entered into an agreement in December 2015, amended in September 2018, which granted Fera exclusive rights to develop and commercialize naproxcinod for the U.S. market.

FDA approves first targeted therapy to treat patients with painful complication of sickle cell disease

Retrieved on: 
Friday, November 15, 2019
Medical specialties, Medicine, Branches of biology, Health in Africa, Hematopathology, RTT, Sickle cell disease, Vaso-occlusive crisis, Hemoglobins, Draft:Rivipansel, Crizanlizumab

According to the Centers for Disease Control and Prevetion, sickle cell disease affects approximately 100,000 Americans.

Key Points: 
  • According to the Centers for Disease Control and Prevetion, sickle cell disease affects approximately 100,000 Americans.
  • The disease occurs most often in African-Americans, where 1 out of every 365 babies born have the disease.
  • "Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease."
  • The Adakveo approval was based on the results of a randomized clinical trial enrolling 198 patients with sickle cell disease with a history of vaso-occlusive crisis.

Vaso-Occlusive Crisis Pipeline Insight Report 2019 - ResearchAndMarkets.com

Retrieved on: 
Monday, October 21, 2019
Health, Infectious diseases, Vaso-occlusive crisis, Draft:Rivipansel, Medical specialties, Crizanlizumab

The "Vaso-Occlusive Crisis - Pipeline Insight, 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Vaso-Occlusive Crisis - Pipeline Insight, 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • A detailed picture of the Vaso-Occlusive Crisis pipeline landscape is provided which includes the disease overview and Vaso-Occlusive Crisis treatment guidelines.
  • The assessment part of the report embraces, in depth Vaso-Occlusive Crisis commercial assessment and clinical assessment of the pipeline products under development.
  • Vaso-Occlusive Crisis pipeline report covers 9+ companies.

Pfizer Announces Phase 3 Top-Line Results for Rivipansel in Patients with Sickle Cell Disease Experiencing a Vaso-Occlusive Crisis

Retrieved on: 
Friday, August 2, 2019
Biotechnology, Health, Pharmaceutical, Clinical trials, Health, Clinical research, Medicine, Articles, Clinical trial, Sickle cell disease, Vaso-occlusive crisis, Pfizer, Placebo, RESET, Sickle Cell Disease and Vaso-occlusive Crisis

We express our sincere gratitude to everyone who made this study possible, including the study investigators, and in particular, the patients and their families."

Key Points: 
  • We express our sincere gratitude to everyone who made this study possible, including the study investigators, and in particular, the patients and their families."
  • The RESET trial included 345 patients who were randomized1:1 to receive rivipansel or placebo, administered intravenously every 12 hours to a maximum of 15 doses.
  • Since completion of the Phase 2 clinical trial, Pfizer has been responsible for clinical development of rivipansel, including the RESET clinical trial.
  • 1 Yale SH1, Nagib N, Guthrie T. Approach to the vaso-occlusive crisis in adults with sickle cell disease.

Vaso-Occlusive Crisis Associated with Sickle Cell Disease - Pipeline Insight, 2019 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, May 8, 2019
Health, Pharmaceutical, Medical specialties, Medicine, Branches of biology, Health in Africa, Hematopathology, RTT, Sickle cell disease, Vaso-occlusive crisis, Sickle-cell disease, Drug pipeline, Draft:Rivipansel, Crizanlizumab

The "Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline Insight, 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Vaso-Occlusive Crisis Associated With Sickle Cell Disease - Pipeline Insight, 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The report offers comprehensive insights of the pipeline (under development) therapeutics scenario and growth prospects across Vaso-Occlusive Crisis Associated With Sickle Cell Disease development.
  • The report provides detailed coverage of the pipeline landscape for this mechanism of action, equipped with data from multiple sources with complete pipeline analysis by developmental stage, associated indications, route of administration and molecule type.
  • clinical, pre-clinical and discovery stages for Vaso-Occlusive Crisis Associated With Sickle Cell Disease
    The report provides pipeline product profiles which includes product description, developmental activities, licensors & collaborators and chemical information
    Provides pipeline assessment by monotherapy and combination therapy products, stage of development, route of administration, and molecule type for Vaso-Occlusive Crisis Associated With Sickle Cell Disease
    Identify the relationship between the drugs and use it for target finding, drug repurposing, and precision medicine