Sickle cell disease

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.

Quince Therapeutics Appoints Former Reata Pharmaceuticals Chief R&D Officer Dr. Rajiv Patni to its Board of Directors

Retrieved on: 
Thursday, February 15, 2024

“We are pleased to welcome a proven clinical development leader of Dr. Rajiv Patni’s caliber to our Board of Directors,” said David Lamond, chairperson of Quince’s Board of Directors.

Key Points: 
  • “We are pleased to welcome a proven clinical development leader of Dr. Rajiv Patni’s caliber to our Board of Directors,” said David Lamond, chairperson of Quince’s Board of Directors.
  • Most recently, he served as Chief Research and Development Officer at Reata Pharmaceuticals, a commercial-stage company recently acquired by Biogen.
  • Previously, Dr. Patni also served as Chief Medical Officer at several successful public, small-cap, and commercial-stage biopharmaceutical companies – Global Blood Therapeutics, Portola Pharmaceuticals, and Adamas Pharmaceuticals.
  • Earlier in his career, Dr. Patni held roles of increasing responsibility at Pfizer, Roche, and Actelion.

Aflac Foundation Inc. extends commitment to children and families with $1.5 million donation to Aflac Cancer and Blood Disorders Center

Retrieved on: 
Wednesday, February 14, 2024

COLUMBUS, Ga., Feb. 14, 2024 /PRNewswire/ -- Crucial for the development of new treatments and promising therapies for children with cancer and blood disorders such as sickle cell, pediatric oncology and hematology research has been a focus of support for Aflac for nearly 30 years.

Key Points: 
  • In keeping with that commitment, The Aflac Foundation Inc. recently made a $1.5 million donation to The Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, $1 million of which is earmarked for the hospital's clinical research office.
  • "Discovery leading to innovation, hope and better outcomes for patients takes place every day at the Aflac Cancer and Blood Disorders Center," said Kathelen Amos, president of The Aflac Foundation.
  • "Providing care to more than 2,600 children and young adults with sickle cell each year, the Aflac Cancer and Blood Disorders Center is home to the largest pediatric sickle cell disease program in the country," Amos added.
  • One of the most active research institutions in the country for pediatric clinical trial enrollment, the Aflac Cancer and Blood Disorders Center offers patients access to more than 380 clinical studies.

Hundreds of sickle cell patients to benefit from £1.5M technology investment

Retrieved on: 
Wednesday, February 14, 2024

Investment in the rollout of this technology could provide an additional 10,000 apheresis procedures annually.

Key Points: 
  • Investment in the rollout of this technology could provide an additional 10,000 apheresis procedures annually.
  • Sickle cell patients often find themselves in and out of hospitals, enduring severe pain and requiring regular "top-up" blood transfusions.
  • I'm very proud of the role the MedTech Funding Mandate has played in the rollout of this technology to ensure we meet the automated red blood cell exchange needs of sickle cell patients."
  • Antoinette Gawin, President and CEO, Terumo Blood and Cell Technologies: "This investment helps more patients get treatment with a proven therapy to manage sickle cell disease.

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)

Retrieved on: 
Tuesday, February 13, 2024

ZUG, Switzerland and BOSTON, Feb. 13, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

Key Points: 
  • “The approval by the European Commission is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for patients with severe sickle cell disease and transfusion-dependent beta thalassemia,” said Samarth Kulkarni, Chairman and Chief Executive Officer of CRISPR Therapeutics.
  • Vertex is working closely with national health authorities in the European Union (EU) to secure access for eligible patients as quickly as possible.
  • Through this work, they have secured early access for eligible TDT patients in France ahead of the national reimbursement process.
  • There are currently three activated ATCs in the EU with plans to activate a total of approximately 25 centers across Europe.

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, February 13, 2024

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.
  • CASGEVY is the only genetic therapy approved for SCD and TDT patients in the European Union (EU) and with this approval, there are now more than 8,000 patients potentially eligible for treatment.
  • Through this work, Vertex has secured early access for eligible TDT patients in France ahead of the national reimbursement process.
  • Vertex continues to engage with hospitals experienced in stem cell transplantation to establish a network of independently operated authorized treatment centers (ATCs) for the administration of CASGEVY.

Lexeo Therapeutics Strengthens Clinical Development Leadership with New Executive Appointments

Retrieved on: 
Monday, February 5, 2024

“The accumulating talent bench at Lexeo has overseen some of the most transformative clinical development programs, drug approvals and product launches in rare disease and precision cardiovascular medicine,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.

Key Points: 
  • “The accumulating talent bench at Lexeo has overseen some of the most transformative clinical development programs, drug approvals and product launches in rare disease and precision cardiovascular medicine,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.
  • During her tenure, Dr. See Tai led the late-stage clinical development program of tafamidis for ATTR cardiomyopathy (ATTR-ACT), which achieved global regulatory approvals, and the Phase 3 LMNA dilated cardiomyopathy clinical program.
  • In addition to late-stage clinical development, Dr. See Tai was responsible for strategic clinical planning for early-stage development candidates such as those for other genetic cardiomyopathies and Duchenne Muscular Dystrophy.
  • Dr. Adler’s work has led to the development of a novel cardiovascular gene therapy candidate entering late-stage clinical development.

Ted Love, MD, Joins Gilead Sciences’ Board of Directors

Retrieved on: 
Thursday, February 1, 2024

Gilead Sciences, Inc. (Nasdaq: GILD) today announced that Ted Love, MD, has been appointed to the company’s Board of Directors.

Key Points: 
  • Gilead Sciences, Inc. (Nasdaq: GILD) today announced that Ted Love, MD, has been appointed to the company’s Board of Directors.
  • Dr. Love is currently chair of the Board of Directors of the Biotechnology Innovation Organization and serves on the Boards of Directors of Royalty Pharma and Structure Therapeutics.
  • “We are delighted to welcome Ted Love to the Gilead Board of Directors,” said Daniel O’Day, Chairman and Chief Executive Officer of Gilead Sciences.
  • Ted will be a valuable addition to the Gilead Board as we continue to expand our impact on patients and communities worldwide.”

Emmaus Life Sciences Receives Marketing Authorization for Puerto Rico

Retrieved on: 
Friday, February 2, 2024

TORRANCE, Calif., Feb. 2, 2024 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTCQX: EMMA), a leader in sickle cell disease (SCD) treatment, today announced that it has received marketing authorization from the Puerto Rico Department of Health for Endari® (L-glutamine oral powder).

Key Points: 
  • TORRANCE, Calif., Feb. 2, 2024 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTCQX: EMMA), a leader in sickle cell disease (SCD) treatment, today announced that it has received marketing authorization from the Puerto Rico Department of Health for Endari® (L-glutamine oral powder).
  • This approval marks a significant milestone in Emmaus' mission to improve the lives of people with SCD around the world and provides access to this important therapy for the patients living with SCD in Puerto Rico.
  • "We are thrilled to bring Endari® to Puerto Rico, where the SCD community has faced significant challenges in accessing innovative treatments," said George Sekulich, Co-President of Emmaus Life Sciences.
  • Emmaus is committed to working with healthcare providers and payers in Puerto Rico to ensure that Endari® is accessible to all eligible patients.

Orphan designation: nicotinamide mononucleotide Treatment of sickle cell disease, 20/06/2023 Positive

Retrieved on: 
Sunday, February 4, 2024

EU/3/23/2786 - orphan designation for treatment of sickle cell disease

Key Points: 
  • EU/3/23/2786 - orphan designation for treatment of sickle cell disease
    nicotinamide mononucleotide
    OrphanHuman
    Lgd
    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.
  • 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: