Thalassemia

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

Retrieved on: 
Wednesday, March 6, 2024

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: 
Thursday, February 15, 2024

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: 
Sunday, February 4, 2024

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: 
Tuesday, January 30, 2024

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: 
Wednesday, January 17, 2024

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).

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: 
Wednesday, January 17, 2024

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).

Clearway Health Launches Innovative Model to Support Health Systems in Enabling Access to Breakthrough Gene Therapy

Retrieved on: 
Tuesday, January 16, 2024

BOSTON, Jan. 16, 2024 /PRNewswire/ -- Clearway Health, a specialty pharmacy accelerator partnering with hospitals and health systems, has pioneered a model to support hospitals in enabling access to novel gene therapy treatments. The model was used to execute and accelerate payor, legal and procurement processes for Children's National Hospital in Washington, D.C., to treat their first pediatric patient with ZYNTEGLO®, a one-time, potentially curative breakthrough gene therapy treatment for transfusion-dependent beta-thalassemia, a rare blood disorder requiring regular red blood cell transfusions.

Key Points: 
  • BOSTON, Jan. 16, 2024 /PRNewswire/ -- Clearway Health , a specialty pharmacy accelerator partnering with hospitals and health systems, has pioneered a model to support hospitals in enabling access to novel gene therapy treatments.
  • Clearway Health launches innovative model to support health systems in enabling access to breakthrough gene therapy.
  • "Many health systems are not set up to coordinate the procurement of a novel gene therapy.
  • "Clearway Health's framework allowed us to quickly and efficiently address these concerns, positioning our hospital to provide the latest pharmaceutical advancement."

bluebird bio Provides Update on Commercial Launch Progress, Program Milestones, and 2024 Financial Outlook

Retrieved on: 
Monday, January 8, 2024

bluebird bio, Inc. (Nasdaq: BLUE) (the Company; bluebird) today announced updates to be presented at the 42nd Annual J.P. Morgan Healthcare conference including commercial launch progress, 2024 program milestones and financial outlook.

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) (the Company; bluebird) today announced updates to be presented at the 42nd Annual J.P. Morgan Healthcare conference including commercial launch progress, 2024 program milestones and financial outlook.
  • “In 2024 we are leveraging our validated commercial strategy to accelerate the launch of LYFGENIA and drive continued strong uptake for ZYNTEGLO.
  • Presentation at the 2024 J.P. Morgan Healthcare Conference
    Andrew Obenshain, chief executive officer, bluebird bio, will present a corporate update on Tuesday, January 9 at 10:30 a.m. PT/1:30 p.m.
  • A replay of the webcast will be available on the bluebird bio website for 30 days following the event.

Clarivate Identifies Thirteen Potential Blockbuster Drugs and Gamechangers in Annual Drugs to Watch Report

Retrieved on: 
Monday, January 8, 2024

LONDON, Jan. 8, 2024 /PRNewswire/ -- Clarivate Plc (NYSE:CLVT), a global leader in connecting people and organizations to intelligence they can trust to transform their world, today announced the release of its annual Drugs to Watch™ report. The report provides in-depth predictive analysis of drugs with the potential for standout commercial and/or clinical success. The report, a key industry resource in the evolving healthcare landscape, has identified over 85 Drugs to Watch over the 11-year course of its publication.

Key Points: 
  • The report provides in-depth predictive analysis of drugs with the potential for standout commercial and/or clinical success.
  • The report, a key industry resource in the evolving healthcare landscape, has identified over 85 Drugs to Watch over the 11-year course of its publication.
  • Leveraging deep industry expertise and comprehensive therapeutic area differentiated data, this year's Drugs to Watch report identifies innovative medicines based on recent scientific breakthroughs poised to have extraordinary impacts on patient outcomes."
  • Included in Drugs to Watch 2023, a delayed U.S. launch meant that it remains a drug to watch for 2024.

Clarivate Identifies Thirteen Potential Blockbuster Drugs and Gamechangers in Annual Drugs to Watch Report

Retrieved on: 
Monday, January 8, 2024

LONDON, Jan. 8, 2024 /PRNewswire/ -- Clarivate Plc (NYSE:CLVT), a global leader in connecting people and organizations to intelligence they can trust to transform their world, today announced the release of its annual Drugs to Watch™ report. The report provides in-depth predictive analysis of drugs with the potential for standout commercial and/or clinical success. The report, a key industry resource in the evolving healthcare landscape, has identified over 85 Drugs to Watch over the 11-year course of its publication.

Key Points: 
  • The report provides in-depth predictive analysis of drugs with the potential for standout commercial and/or clinical success.
  • The report, a key industry resource in the evolving healthcare landscape, has identified over 85 Drugs to Watch over the 11-year course of its publication.
  • Leveraging deep industry expertise and comprehensive therapeutic area differentiated data, this year's Drugs to Watch report identifies innovative medicines based on recent scientific breakthroughs poised to have extraordinary impacts on patient outcomes."
  • Included in Drugs to Watch 2023, a delayed U.S. launch meant that it remains a drug to watch for 2024.