Fetal hemoglobin

MiNA Therapeutics Presents New Data for its HbF Program at the Keystone Symposia’s Delivery of Nucleic Acid Therapeutics Conference

Retrieved on: 
Wednesday, January 24, 2024
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Patient, Liposome, Therapy, RNAa, Keystone, MST, RNA, Mina, Fetal hemoglobin, Conference, Bone marrow, Sickle cell disease, Safety, HBF, Vivo, HBG, Pharmaceutical industry

MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, announced today that it will present new pre-clinical data for its lead RNA activation program targeting fetal hemoglobin (HbF) at the Keystone Symposia’s Delivery of Nucleic Acid Therapeutics Conference in Banff, Alberta, Canada.

Key Points: 
  • MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, announced today that it will present new pre-clinical data for its lead RNA activation program targeting fetal hemoglobin (HbF) at the Keystone Symposia’s Delivery of Nucleic Acid Therapeutics Conference in Banff, Alberta, Canada.
  • The data will be presented on January 24, 2024, at 7:30 pm MST during a poster presentation.
  • MiNA’s HbF program is designed to increase transcription of the gamma globin (HBG) gene, enabling patients with beta-hemoglobinopathies to produce enhanced levels of HbF.
  • MiNA anticipates advancing its HbF program, the first program to emerge from its genetic medicine portfolio, into pre-clinical development in 2024.

Qvin™ Introduces Q-Pad™: Transforming Women's Health with FDA-Cleared Lab Testing using Menstrual Blood

Retrieved on: 
Monday, January 8, 2024
Menopause, Biotechnology, Woman, Research, FDA, Anemia, Health, Diabetes, Fetal hemoglobin, HPV, SAN, Fertility, Stanford University, Obstetrics, Endometriosis, Biomarker, A1C, Patient, Safety, Prediabetes, OTC, Gynaecology, Physician, Human papillomavirus infection

SAN FRANCISCO, Jan. 8, 2024 /PRNewswire/ -- Qvin™, the biotechnology research company that developed the first and only healthcare service that collects menstrual blood samples as an alternative to traditionally collected venous blood draws, today announced FDA clearance of its Q-Pad™ and A1c Test. The clearance makes it possible for the millions of women in America who live with diabetes to receive monitoring of A1c, using laboratory tests performed on the Q-Pad. More broadly, this marks an opportunity for testing important biomarkers for the more than 80 million people who menstruate in the U.S.

Key Points: 
  • The traditional methods of blood testing require invasive procedures administered by medical professionals, and those can be time-consuming and expensive.
  • And yet, menstrual samples had never previously been explored as a diagnostic source for health information.
  • "Utilizing menstrual samples, the Q-Pad can address critical women's health issues that have historically been neglected," stated Søren Therkelsen, Co-founder of Qvin.
  • "For instance, women seeking to understand their fertility status can soon monitor various reproductive hormones via menstrual blood using the Q-Pad.

Editas Medicine Announces New EDIT-301 Safety and Efficacy Data in 17 Patients, Presented Today at the American Society of Hematology (ASH) Annual Meeting and in a Company-sponsored Webinar

Retrieved on: 
Monday, December 11, 2023
SCD, Cleveland Clinic, Sickle cell disease, Șaeș, Division, EHA, Editas Medicine, Anemia, Data, Fetal hemoglobin, MD, Society, Clinical trial, Busulfan, Blood, Event, ASH, European Hematology Association, Patient, TDT, Safety, Senior, Pharmaceutical industry

The total dataset of 17 treated patients includes 12 additional patients since the data presentation at the European Hematology Association (EHA) Annual Congress and in a Company-sponsored webinar this past June.

Key Points: 
  • The total dataset of 17 treated patients includes 12 additional patients since the data presentation at the European Hematology Association (EHA) Annual Congress and in a Company-sponsored webinar this past June.
  • Editas Medicine will present the RUBY and EdiTHAL trial data today at 1 p.m.
  • The data will also be presented in a poster presentation at the American Society of Hematology (ASH) Annual Meeting in San Diego, CA, at 6:00 p.m. PT (9:00 p.m.
  • Presenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s, Cleveland, OH, United States

FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

Retrieved on: 
Friday, December 8, 2023
Malignancy, VOC, DNA, Sickle cell disease, Abdominal pain, Headache, Patient, Research, Hemoglobin A, Cas9, CRISPR, Oxygen, Volatile organic compound, Recurrence, Fever, Mutation, Priority review, DSM-IV codes, Vomiting, Bone marrow, Hematology, China Biologic Products, Safety, Food, Multimedia, Rejection, HBF, Ronas Voe, Health, Person, Nausea, Throat, Mouth, Hispanic and Latino Americans, Public, Adolescence, Stomatitis, Risk, Tissue, Febrile neutropenia, History, End organ damage, Fetal hemoglobin, Animal, Pain, SCD, B cell, Regenerative medicine, Heated tobacco product, Disease, FDA, African Americans, Mouth ulcer, Vaccine, Cosmetics, Blood, Human services, Dietary supplement, Freedom, Pharmaceutical industry

Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S.

Key Points: 
  • Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S.
  • The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body's tissues.
  • Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises.
  • In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.

Editas Medicine Announces Third Quarter 2023 Results and Business Updates

Retrieved on: 
Friday, November 3, 2023
FDA, Research, Regenerative Medicine Advanced Therapy, Food, Spinal muscular atrophy, Biogen, SCD, Life, Public expenditure, TDT, Cas9, Sickle cell disease, Regulation of tobacco by the U.S. Food and Drug Administration, Poster, Society, ASH, Commercialization, HSC, File, Fetal hemoglobin, Collaboration, Lead, Beta thalassemia, Anemia, Multiple sclerosis, Bone marrow, Therapy, Patient, BLA, MS, Pharmaceutical industry, Cryptocurrency, Edith A. Perez, Editas Medicine, EU

CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the third quarter 2023 and provided business updates.

Key Points: 
  • CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the third quarter 2023 and provided business updates.
  • Cash, cash equivalents, and marketable securities as of September 30, 2023, were $446.4 million compared to $480.0 million as of June 30, 2023.
  • The increase is primarily related to an upfront payment for the non-exclusive Cas9 license to Vor Bio in the third quarter of 2023.
  • Editas Medicine plans to participate in the following scientific and medical conference:
    Editas Medicine plans to participate in the following investor events:

Editas Medicine to Present Clinical Data from the RUBY and EdiTHAL Trials of EDIT-301 at the ASH 2023 Annual Meeting and in a Company-sponsored Webinar

Retrieved on: 
Thursday, November 2, 2023
Trial of the century, OH, Event, Safety, Sickle cell disease, Society, ASH, Senior, American Society of Pediatric Hematology/Oncology, Caregiver, Fetal hemoglobin, Editas Medicine, Patient, Blood, Pharmaceutical industry, Nursing, Blood product, Edith A. Perez

CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that a scientific abstract detailing safety and efficacy clinical data from the RUBY trial of EDIT-301 in patients with severe sickle cell disease and from the EdiTHAL trial of EDIT-301 in patients with transfusion-dependent beta thalassemia has been accepted for a poster presentation at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition being held December 9-12, 2023, in San Diego, CA, and online.

Key Points: 
  • Company to host a webinar to discuss EDIT-301 clinical data on Monday, December 11, at 1:00 p.m.
  • Editas Medicine will also host a Company-sponsored webinar on Monday, December 11, at 1:00 p.m.
  • ET to discuss the RUBY and EdiTHAL clinical data.
  • Clinical data on six patients, including at least five months data from the first two patients treated.

Beam Therapeutics Reports Pipeline Updates and Second Quarter 2023 Financial Results

Retrieved on: 
Tuesday, August 8, 2023
Well, Tissue, ESCAPE, Research and development, HBF, Biotechnology, Allele, Gene editing, SCD, Clinical trial, Cancer, SMART Process Acceleration Development Environment, G&A, CD34, BCL11A, AATD, Fetal hemoglobin, CGMP, Patient, Investment, Security (finance), Administration, Cas9, Hematology, Sickle cell disease, Liver, Growth, Hospital, Trial of the century, LNP, LNP Media Group, R, Services General Administration and Coordination Department, Government of Sindh, Treatment, Safety, Pharmaceutical industry, Vaccine, Medical imaging, Television, Beam

CAMBRIDGE, Mass., Aug. 08, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported second quarter 2023 financial results and provided an update on its clinical and pipeline progress.

Key Points: 
  • Beam continues to expect initiation of current good manufacturing practice compliant operations at its North Carolina manufacturing facility in late 2023.
  • Research & Development (R&D) Expenses: R&D expenses were $97.6 million for the second quarter of 2023, compared to $74.6 million for the second quarter of 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $24.7 million for the second quarter of 2023, compared to $24.1 million for the second quarter of 2022.
  • Net Loss: Net loss was $82.8 million for the second quarter of 2023, or $1.08 per share, compared to $72.0 million for the second quarter of 2022, or $1.02 per share.

Trinity Biotech Announces That It Has Received 510(k) Clearance From The U.S. Food and Drug Administration for the Premier Resolution System

Retrieved on: 
Monday, August 7, 2023
Hemoglobin A2, IEX, Trinity, Sickle cell disease, Food and Drug Administration, Sale, Incidence, Fetal hemoglobin, Laboratory, Thalassemia, FDA, Food, HPLC, Software, Medical device, A2

DUBLIN, Ireland, Aug. 07, 2023 (GLOBE NEWSWIRE) -- Trinity Biotech has received U.S. Food and Drug Administration (FDA) 510(k) clearance for the Premier Resolution System, an automated analyzer for the accurate & precise quantification of hemoglobins F and A2, and the detection of >200 hemoglobin variants.

Key Points: 
  • DUBLIN, Ireland, Aug. 07, 2023 (GLOBE NEWSWIRE) -- Trinity Biotech has received U.S. Food and Drug Administration (FDA) 510(k) clearance for the Premier Resolution System, an automated analyzer for the accurate & precise quantification of hemoglobins F and A2, and the detection of >200 hemoglobin variants.
  • The Premier Resolution System is now cleared for sale in the United States.
  • The Premier Resolution System is a modern successor to Trinity Biotech’s Ultra System that held a leading position in the US hemoglobin variant diagnostic market.
  • The Premier Resolution gives laboratories the tools to easily identify clinically important variants.

Editas Medicine Announces Second Quarter 2023 Results and Business Updates

Retrieved on: 
Wednesday, August 2, 2023
Linda, File, Life, European Hematology Association, Appointment, Biogen, Biotechnology, SCD, Research, DSM-IV codes, Public expenditure, Acquisition, Fetal hemoglobin, Patient, Anemia, EHA, Drug discovery, Sickle cell disease, IND World's Fair Line, FDA, Collaboration, LG Chem, BLA, TDT, Management, Pharmaceutical industry, Plumbing, Cryptocurrency, Editas Medicine

CAMBRIDGE, Mass., Aug. 02, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the second quarter 2023 and business updates.

Key Points: 
  • Strengthened Executive Team with Appointments of Erick Lucera as Chief Financial Officer and Linda C. Burkly, Ph.D., as Chief Scientific Officer
    CAMBRIDGE, Mass., Aug. 02, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the second quarter 2023 and business updates.
  • The Editas team is excited and continues driving execution towards our goals,” commented Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine.
  • ET to provide and discuss a corporate update and financial results for the second quarter of 2023.
  • To access the call, please dial 1-877-407-0989 (domestic) or 1-201-389-0921 (international) and ask for the Editas Medicine earnings call.

Editas Medicine Announces Positive Initial EDIT-301 Safety and Efficacy Data from the First Four Patients Treated in the RUBY Trial and the First Patient Treated in the EdiTHAL Trial

Retrieved on: 
Friday, June 9, 2023
Male, HBF, Blood, Senior, European Hematology Association, Life, Event, OH, Gene editing, SCD, Clinical trial, Aes, RAF Sullom Voe, Female, EDT, Editas Medicine, Șaeș, American Society of Pediatric Hematology/Oncology, Fetal hemoglobin, MD, Patient, Anemia, Busulfan, EHA, Sickle cell disease, Safety, Pharmaceutical industry, Nursing, Edith A. Perez

ET

Key Points: 
  • ET
    Company to host a virtual event on the RUBY and EdiTHAL data on Monday, June 12 at 8:00 a.m.
  • Editas Medicine will present the RUBY trial data and the EdiTHAL trial data on Monday, June 12, at 8 a.m.
  • All four treated patients in the RUBY trial are free from vaso-occlusive events since infusion with EDIT-301, with 2-10 months follow-up.
  • EDIT-301 is currently being investigated in clinical studies in patients with severe sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta thalassemia (EDITHAL trial, NCT05444894).