Topic: sickle cell anemia
The proportion of patients who had a greater than 1 g/dL increase in hemoglobin is down on GBT’s prior update but still superior to placebo.
A Fred Hutchinson team developed gold nanoparticles that can deliver CRISPR to mouse models of HIV and inherited blood disorders.
Sickle cell disease biotech Imara has nabbed Pfizer’s clinical rare disease lead Willem H. Scheele, M.D., as its new chief medical officer.
Scientists suggest that using more precise CRISPR techniques could contain blood stem cells' p53-mediated response to gene editing.
The series B sets Imara up to wrap up a phase 2a trial of a phosphodiesterase-9 inhibitor it picked up from Lundbeck and push deeper into the clinic.
Aruvant Sciences will focus on developing gene therapies for blood diseases, starting with sickle cell disease and beta-thalassemia.
The FDA lifted a clinical hold it placed on Vertex and CRISPR Therapeutics’ gene-edited stem cell therapy for sickle cell disease.
CRISPR Therapeutics and Vertex Pharmaceuticals will test their gene therapy in patients with beta thalassemia.
A discontinued heart disease drug will be developed for sickle cell, with Roche handing off its inclacumab antibody to Global Blood Therapeutics.
Modus Therapeutics raised over 140 million Swedish krona, or about $15.7 million, to advance clinical studies of sevuparin in sickle cell disease.