This week in drug discovery (10-14 March)
News round-up for 10-14 March by DDW Senior Digital Content Editor Diana Spencer.
Clinical trials of all stages have dominated the DDW news section this week. All the stories represent a significant step forward for drug discovery, either due to the innovative nature of the therapies being studied, or the unique methods employed in the trial.
The top stories:
First clinical genetic correction of a disease-causing mutation reported
Biotechnology company Beam Therapeutics says initial data from its Phase I/II trial establish clinical proof-of-concept for BEAM-302 as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and for in vivo base editing.
First patient dosed in gene therapy study for Stargardt disease
SpliceBio has dosed the first patient in the Phase I/II ASTRA study of SB-007, a dual adeno-associated viral (AAV) vector, for the treatment of Stargardt disease.
Dual approach could transform treatment of rare cancer
Researchers have combined immunotherapy and chemotherapy to offer new hope for patients with thymic carcinoma, a rare and aggressive cancer.
Trial supports immunoglobulin in post-polio patients
Positive results from a Phase II/III clinical trial evaluating Grifols’ intravenous immunoglobulin (IVIG) to treat patients with post-polio syndrome (PPS) have demonstrated a significant improvement in distance walked compared to placebo.
Deep learning provides enhanced imaging analysis in IPF trial
Qureight and biopharmaceutical company Remedy Cell have agreed a partnership to validate RC-0315’s mechanism of action in idiopathic pulmonary fibrosis (IPF).