Disclosed herein are potency assays for a gene therapy treatment for β-thalassemia. Also disclosed herein are methods for measuring relative potency of a drug product.

Disclosed herein are potency assays for a gene therapy treatment for sickle cell disease. Also disclosed herein are methods for measuring relative potency of a drug product used for the treatment of sickle cell disease.

Methods for identifying patients with anemia, distinguishing thalassemia-trait anemia from iron-deficiency anemia, and identifying pre-anemic patients several weeks before anemia becomes clinically detectable. Also, methods for detecting blood doping in athletes and for optimizing therapy with erythropoiesis stimulating agents or iron supplementation. Computer-readable storage devices and systems, e.g., for use in the described methods.

The invention concerns a method for determining, by flow cytometry, the hemoglobin content of each erythroid cell of a set of erythroid cells. This method applies in particular to determining the hemoglobin content of each red blood cell of a set of red blood cells. The invention also concerns a method for determining the amount of red blood cells transfused into a patient and for monitoring the therapeutic efficacy of a treatment for sickle cell disease or β-thalassemia.

Provided are methods of treating a disease or condition characterized by a deficiency of or a defect in an iron transporter using a small molecule. For example, the method may increase transepithelial iron transport, or it may increase iron release. Additionally, the small molecule may be hinokitiol, or it may be selected from the group consisting of amphotericin B, calcimycin, nonactin, deferiprone, purpurogallin, and maltol. Also provided is a method of identifying a compound capable of treating a disease or condition characterized by a deficiency of or a defect in an iron transporter.

Provided herein are methods and kits for analyzing a biological sample obtained from a subject having, suspected of having, or being at risk for a disease associated with the contact activation system.

The present invention relates to the field of treating iron deficiency with IV iron carbohydrate complexes such ferric carboxymaltose, monitoring or identifying subjects to determine their eligibility for being administered said IV iron carbohydrate complexes, and combining said IV iron carbohydrate complexes with additional drugs in order to mitigate or reduce side effects induced by said IV iron carbohydrate complexes.

The present invention is directed to a method to detect in a blood sample whether the red blood cells (RBCs) contained in said blood sample present or not an alteration of their deformability by using molecular rotor (MR) able to penetrate RBCs cell membrane. The invention also relates to diagnostic methods of RBC related pathologies associated to the modification of the distribution of the viscosity, rigidity or deformability of RBCs by detection and measurement of the RBCs fluorescence intensity image intensity implementing optical methods. Finally, the present invention is directed to the use of MRs for testing the deformability of red blood cells (RBCs) in a blood sample and kit comprising MR and red blood cells (RBCs) control.

The present invention relates methods of treatment using BMP6 antagonists.

The present invention relates to the field of tumor treatment and molecular immunology, and particularly, to an anti-CTLA4/anti-PD-1 bispecific antibody and use thereof. Specifically, the anti-CTLA4/anti-PD-1 bifunctional antibody comprises a first protein functional region targeting PD-1 and a second protein functional region targeting CTLA4, wherein, according to the EU numbering system, the heavy chain constant region of the immunoglobulin comprised in the bispecific antibody has mutations at any 2 or 3 of positions 234, 235 and 237, and the affinity constant of the bispecific antibody to FcγRIIIa and/or C1q is reduced after the mutation as compared to that before the mutation. The bifunctional antibody of the present invention can well and specifically bind to CTLA4 and PD-1, specifically relieve immunosuppression of CTLA4 and PD-1 in an organism, and activate T lymphocytes, thus having good application prospect.