Division of Hematology News

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FDA Approves Two Gene Therapies for Sickle Cell Disease

Dec 8, 2023

The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

CHOP Hematologist Honored by American Society of Hematology for Leadership in Promoting Diversity

Nov 20, 2023

Alexis Thompson, MD, MPH, received the award at the society’s annual meeting.

Gene Splicing Reduces Effectiveness of CD20-Targeting Monoclonal Antibodies Designed to Treat Variety of Blood Cancers and Disorders

Nov 16, 2023

Researchers found CAR T-cell therapy may serve as effective alternative for patients with these disorders.

CHOP Researchers Improve Fitness of Cells Used in Cell Transplants

Sep 26, 2023

CHOP researchers find exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.

Experts in the Hemostasis and Thrombosis Center at CHOP Provide Expert Commentary on Hemophilia

Aug 18, 2023

Leslie Raffini, MD, and Regina Butler, RN, were quoted in the Philadelphia Inquirer on a story featuring a CHOP patient family living with hemophilia, an inherited bleeding disorder that prevents blood from clotting properly.

Researchers Develop “In Vivo” Gene Editing Model for Blood Disorders

Jul 27, 2023

CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.

CHOP’s First In-person Cure Sickle Cell Walk & Family Fun Day Raises More Than $173,000 for Sickle Cell Research and Care

Jun 20, 2023

On a beautiful Saturday in June, Children’s Hospital of Philadelphia (CHOP) hosted the first in-person Cure Sickle Cell Walk & Family Fun Day at Citizens Bank Park raising more than $173,000 to support CHOP’s large and comprehensive sickle cell program.

CHOP Researchers Identify Potential Target in Myeloid Malignancies

Jun 15, 2023

CHOP researchers have identified a molecule that plays a role in post-translational modification and activation in myeloid cancers, making it a potential therapeutic target.

CHOP Expert Breaks Down Latest Hemophilia A Gene Therapy Data

Feb 23, 2023

CHOP gene therapy expert Dr. Lindsey George reacts to a phase 3 hemophilia gene therapy study in an editorial in the New England Journal of Medicine.

Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program

Feb 9, 2023

The Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program is a Children’s Hospital of Philadelphia (CHOP) Frontier Program within the Division of Hematology that aims to provide advanced clinical care of inherited bleeding disorders and develop breakthrough treatments through research.

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