Additional programs for neurometabolic disorders, primary immune deficiencies and
hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline.
An orally administered, highly potent and selective phosphodiesterase 9 inhibitor, IMR-687 has the potential to be a disease-modifying therapeutic for sickle cell disease as well as other
hemoglobinopathies.
M2 PRESSWIRE-June 19, 2019-: Global $12.6Bn
Hemoglobinopathies Market by 2026 - Pre-Implant Genetic Diagnosis is Estimated to Witness a Lucrative CAGR of Over 8%
Hemophilia, Dengue and patient with
Hemoglobinopathies (Thalassemia).
CRISPR Therapeutics (CRSP) and Vertex (VRTX) reviewed recent progress in the clinical development programs for CTX001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe
hemoglobinopathies. The companies announced that the first patient has been treated with CTX001 in a Phase 1/2 clinical study of patients with transfusion-dependent beta thalassemia, or TDT, marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial.
Finally, the hospital tested for
hemoglobinopathies using high-performance liquid chromatography.
The circular showed the expanded newborn screening test will be able to detect endocrine, amino and fatty acid disorders organic acid, urea cycle defect, cystic fibrosis,
hemoglobinopathies, biotinidase deficiency, and other disorders.
The premarital screening program is a comprehensive program that aims to limit the spread of genetic
hemoglobinopathies such as sickle cell anemia (SCA) and thalassemia, in addition to infectious diseases such as hepatitis B and C, and human immunodeficiency virus/acquired immune deficiency syndrome (HIV/ AIDS).
However, in patients with
hemoglobinopathies, such as sickle cell disease, an aplastic crisis can be triggered.
Thalassemic syndromes Heterozygous beta thalassemia Other
hemoglobinopathies Artifact in the presence of Hb S Some Hb variants with thalassemic phenotype Acquired conditions Megaloblastic anemia Hyperthyroidism Pseudoxanthoma elasticum Hypertrophic osteoarthropathy Treatment-related conditions Antiretroviral therapy in patients with HIV