Etavopivat is designed to activate PKR and thereby modulate RBC metabolism by impacting two critical pathways in RBCs. The etavopivat clinical development program will investigate whether decreasing 2,3-DPG may help oxygen bind to hemoglobin (i.e. increasing oxygen affinity), and thereby increase ATP and impact RBC function. This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients age 12 to 65 years (inclusive), with sickle cell disease. There are two

FT-4202 is a potent, selective, orally bioavailable, small-molecule activator of pyruvate kinase-red blood cell (PKR) being developed by Forma Therapeutics, Inc and is intended for use as a treatment for patients with sickle cell disease (SCD). The clinical hypothesis is that PKR activation will reduce the rate of sickle cell polymerization and improve red blood cell (RBC) membrane function, thereby reducing RBC sickling and RBC hemolysis that lead to vascular obstruction and anemia, two hallmar

This is an interventional, multi-national, multi-centre, randomised (2:1), double-blind, placebo-controlled Phase 3 study in participants aged 12 and older with SCD, who had 1−15 VOCs in the year prior to screening and who have moderate to severe anaemia. Participants will be randomised in a blinded fashion (2:1) to the etavopivat or placebo dosing groups. A total of 408 participants are expected to be enrolled in the study and receive treatment for 52 weeks in the double-blind treatment period

Sickle Cell Disease (SCD) is a serious life-long illness that causes low red blood cell counts (anaemia), fatigue, and damage to blood vessels leading to problems such as acute painful episodes, strokes and leg ulcers. It can also result in organ damage and lead to premature death. The main purpose of this research study is to determine the effect of the study drug GBT440, compared to placebo in increasing red blood cell counts and reducing the severity of symptoms of SCD. A placebo is a “dummy

A open label, non-randomized, first-in-patient, phase I/II, proof-of-concept study following subjects for two years after transplantation of either genome-edited autologous HSPC investigational drug product. The study consist of 3 parts - Part A include treatment of adults with OTQ923; Part B include treatment of adults with HIX763; Part C include treatment of kids 2-17 years old with either OTQ923 or HIX763

The ReSPECT trial is a global, randomized, double-blind, controlled, pivotal Phase 3 trial of rezafungin versus the standard antimicrobial regimen to prevent invasive fungal disease due to Candida, Aspergillus and Pneumocystis in subjects undergoing allogeneic BMT. Rezafungin, dosed once-weekly, will be compared to a daily regimen containing multiple drugs including fluconazole or posaconazole, and trimethoprim-sulfamethoxazole, also known as Bactrim, for 90 days, at which time fungal-free survi

A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients with Sickle Cell Disease

The study drug CSL889 is being developed for the treatment of vaso-occlusive crisis (also known as pain crisis) that occurs in patients with sickle cell disease. All humans have hemopexin. However, patients with sickle cell disease may have very little hemopexin. The main job of hemopexin is to combine with a substance in blood called haem and to clear haem from the blood. The haem comes from red blood cells when red blood cells die. Research suggests that haem may play a role in pain crisis. CS

This observational study will look at how patients progress over a five year period. Detailed medical history retrospective data will be collected on study enrollment and then at routine annual review appointments. Data from investigations will be collected from the electronic patient record (including laboratory results and imaging reports). Patients will also complete a health-related quality of life questionnaire every year.