Thalassemia

EQS-News: BB Biotech presents solid first-quarter results; biotech stock market impacted by shifting views on interest rates

Retrieved on: 
금요일, 5월 3, 2024
USD, Annual, Alnylam Pharmaceuticals, TTR, Federal Reserve, Agios, BB Biotech, Mitapivat, NAV, Esperies, Accounting, Federal, Cardiomyopathy, NBI, Public expenditure, Prostate cancer, Obesity, Environment, DSM-IV codes, ADC, CRISPR, Patient, Thalassemia, Biotechnology, CRISPR Therapeutics, Initial public offering, Beam Therapeutics, Inflation, CHF, IRA, Drug development, Clinical trial, Significance, Therapy, Pharmaceutical industry

BB Biotech presents solid first-quarter results; biotech stock market impacted by shifting views on interest rates

Key Points: 
  • BB Biotech presents solid first-quarter results; biotech stock market impacted by shifting views on interest rates
    The issuer is solely responsible for the content of this announcement.
  • Media release as at April 26, 2024
    Portfolio of BB Biotech AG as at March 31, 2024
    BB Biotech presents solid first-quarter results; biotech stock market impacted by shifting views on interest rates
    Investor sentiment in the biotech space continues to be shaped by the general interest rate environment.
  • Net profit for the first quarter of 2024 amounted to CHF 260 mn (net loss of CHF 254 mn in prior-year quarter).
  • While activity in the secondary market has already picked up, there have been few biotech IPOs so far this year.

Orphan designation: mitapivat sulfate Treatment of thalassaemia intermedia and major, 12/01/2024 Positive

Retrieved on: 
목요일, 4월 18, 2024
Patient, Committee, Thalassemia, EMA, IRIS, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of thalassaemia alpha intermedia and major on 12 January 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

bluebird bio Reports Fourth Quarter and 2023 Annual Results and Highlights Operational Progress and 2024 Guidance

Retrieved on: 
화요일, 3월 26, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, PIN, Sickle cell disease, MSA, Bluebird bio, Telephone, Webcast, Patient, Gene, CMMI, QTC, Bank statement, Thalassemia, Annual report, Medicaid, Cell, Medicare, Bluebird, FDA, Pharmaceutical industry

bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today reported fourth quarter and annual financial results and business highlights for the year ended December 31, 2023, including recent commercial and operational progress.

Key Points: 
  • bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today reported fourth quarter and annual financial results and business highlights for the year ended December 31, 2023, including recent commercial and operational progress.
  • “In 2023, bluebird established a validated, commercial gene therapy strategy that brought ZYNTEGLO and SKYSONA to individuals living with beta-thalassemia and cerebral adrenoleukodystrophy.
  • Completed 2 patient starts for SKYSONA since the beginning of 2024, in addition to 6 patient starts completed for SKYSONA in 2023.
  • In the first quarter of 2024, bluebird signed its first Medicaid outcomes-based agreement for LYFGENIA with the state of Michigan.

bluebird bio Secures up to $175 Million Debt Financing with Hercules Capital

Retrieved on: 
월요일, 3월 18, 2024
Health, FDA, Stem Cells, Genetics, General Health, Pharmaceutical, Biotechnology, Sickle cell disease, Patient, DSM-IV codes, Security (finance), Piper, Thalassemia, DLA Piper, Latham & Watkins, Bluebird, Pharmaceutical industry

bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today announced that it has entered into a $175 million five-year, term loan facility with Hercules Capital, Inc. (NYSE: HTGC) (“Hercules”).

Key Points: 
  • bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today announced that it has entered into a $175 million five-year, term loan facility with Hercules Capital, Inc. (NYSE: HTGC) (“Hercules”).
  • The term loan facility provides for up to $175 million of term loans in aggregate, available in four tranches.
  • This financing should help support the availability of their novel gene therapies for patients,” added John Miotti, Principal at Hercules Capital.
  • Latham & Watkins LLP served as legal counsel to bluebird and DLA Piper served as legal counsel to Hercules.

International Harrington Prize Awarded to Dr. Arlene Sharpe

Retrieved on: 
화요일, 3월 19, 2024
LDL, Human papillomavirus infection, Patient, DSM-IV codes, Lecture, National Cancer Institute, Harvard University, Creativity, AAP, PD-1, Massachusetts General Hospital, Science, Diabetes, Marfan syndrome, All India Mahila Congress, Immunology, AUC, NIH, Cancer, Nivolumab, Immune system, Biochemistry, Harvard Medical School, Cervical cancer, Research, University, Antibody, ASCI, FDA, Mount Sinai Hospital, Sickle cell disease, University of Pennsylvania, PCSK9, COVID-19, Doctor of Philosophy, Biology, Kidney, Thalassemia, Johns Hopkins University, Society, Division, Leptin, Pharmaceutical industry

CLEVELAND, March 19, 2024 /PRNewswire/ -- The eleventh annual Harrington Prize for Innovation in Medicine has been awarded to Arlene H. Sharpe, MD, PhD, Kolokotrones University Professor and Chair of the Department of Immunology at Harvard Medical School. The award recognizes her breakthrough discoveries in immune regulation, which have established foundational principles in immunology and led to new cancer therapies that act by boosting the immune response to cancer.

Key Points: 
  • CLEVELAND, March 19, 2024 /PRNewswire/ -- The eleventh annual Harrington Prize for Innovation in Medicine has been awarded to Arlene H. Sharpe, MD, PhD, Kolokotrones University Professor and Chair of the Department of Immunology at Harvard Medical School.
  • Her work helped identify key pathways that restrain the activity of T lymphocytes (immune cells) to fight cancer.
  • A committee composed of members of the ASCI Council and the Harrington Discovery Institute Scientific Advisory Board reviewed nominations from leading academic medical centers globally before selecting the 2024 Harrington Prize recipient.
  • 2023: Jean Bennett, MD, PhD, and Albert M. Maguire, MD, for their groundbreaking translational research to restore sight in inherited genetic diseases.

Generation Bio Reports Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
수요일, 3월 6, 2024
Therapy, Research, Haemophilia, DSM-IV codes, HSC, Health, Thalassemia, Sickle cell disease, G&A, Hepatocyte, DNA, Mouse, Vaccine

CAMBRIDGE, Mass., March 06, 2024 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and fourth quarter and full year 2023 financial results.

Key Points: 
  • (Nasdaq:GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and fourth quarter and full year 2023 financial results.
  • “Late last year we announced important advances in our cell-targeted lipid nanoparticle and immune-quiet DNA platforms that we are now translating to our portfolio strategy,” said Geoff McDonough, M.D., chief executive officer of Generation Bio.
  • We are also continuing to optimize iqDNA to progress our hemophilia A program and to explore therapeutic applications in cell types beyond hepatocytes.
  • In 2024, the company intends to optimize iqDNA for application in hepatocytes and T cells with a focus on improving potency.

Agios Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
목요일, 2월 15, 2024
Agios Pharmaceuticals, Security, AGIO, Total, Research, New Drug Application, ASH, FDA, Research and development, PKU, Growth, Sale, Exposition, Thalassemia, Society, MDS, Phenylketonuria, Administration, Sickle cell disease, Anti-transglutaminase antibodies, Patient, Public expenditure, Net, Small RNA, IND, Pharmaceutical industry

CAMBRIDGE, Mass., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Launch: Generated $7.1 million in U.S. net revenue for the fourth quarter of 2023, a 4 percent decrease from the third quarter of 2023, primarily driven by lower customer inventory levels at the end of the fourth quarter of 2023, partially offset by favorable gross-to-net adjustments.
  • A total of 109 patients are on PYRUKYND® therapy, a 9 percent increase from the third quarter of 2023.
  • Cost of Sales: Cost of sales was $0.6 million for the fourth quarter of 2023 and $2.9 million for the full year ended Dec. 31, 2023.
  • ET to discuss fourth quarter and full year 2023 financial results and recent business highlights.

Orphan designation: mitapivat sulfate Treatment of thalassaemia intermedia and major, 13/10/2023 Positive

Retrieved on: 
일요일, 2월 4, 2024
COMP, IRIS, European Commission, Thalassemia, Committee, Patient, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - mitapivat sulfate
    - Intended use
    - Treatment of thalassaemia intermedia and major
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2827
    - Date of designation
    - Sponsor
    Agios Netherlands B.V.
  • Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

EQS-News: BB Biotech AG: End of year biotech equity market rally – M&A and end of rate hike cycle

Retrieved on: 
화요일, 1월 30, 2024
Neuroscience, Patient, Annual general meeting, United States dollar, Research, ADC, Health care, Policy, Biotechnology, NAV, Dax, Intelligence, Annual, SGA, BB Biotech, AGM, Euro, Investment, Hereditary angioedema, EQT, Consolidated, Lifting, Hunziker, CHF, Affibody molecule, Thalassemia, Annual report, Clinical trial, MSCI, Cardiomyopathy, Revolution, Pharmaceutical industry

Media release as at January 19, 2024

Key Points: 
  • Media release as at January 19, 2024
    Portfolio of BB Biotech AG as at December 31, 2023
    In 2023, global equity performed better than anticipated by many.
  • Consequently, BB Biotech ended the year with a net loss of CHF 207 mn.
  • This represents a 5% return based on the volume-weighted average closing price of BB Biotech shares in December 2023.
  • The US central bank held rates steady in the fall, lifting market sentiment in the last two months of 2023.

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
수요일, 1월 17, 2024
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).