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(Reuters) -The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel ...
Student Zoe Davis, 20, was just weeks into her junior year when she landed back in the hospital with severe sickle cell pain earlier this month. She knows new gene therapies may provide long-term ...
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
Ingelheim, Germany, 20 February 2025 – Boehringer Ingelheim, IP Group, the UK Respiratory Gene Therapy Consortium (GTC) 1 and OXB, 1 today announce the start of LENTICLAIR TM 1, a Phase I/II trial of BI 3720931, a novel, first-in-class, gene therapy aiming to improve disease outcomes in people with cystic fibrosis (CF), regardless of gene mutations that are causing the disease.
In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell. Any such modifications affect the individual patient only, and are not inherited by offspring. Somatic gene therapy represents mainstream basic and clinical research, in which ...
DALLAS, Feb. 26, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today reported financial results for the full year ended ...
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...
The Center for Cell and Gene Therapy has the largest academic GMP facility in the world, with 8,600 square feet of Class 10,000 (ISO7) cleanroom space. The GMP Gene Vector Lab produces clinical grade vectors for use in Phase I/II trials, while the GMP Clinical Research Lab prepares patient components for clinical trials.