Posted on 28/06/202128/06/2021Intellia and Regeneron Announce Landmark Clinical Data Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis
Posted on 18/05/202108/07/2024Biogen Announces Topline Results From Phase 2/3 Gene Therapy Study for XLRP
Posted on 11/05/202108/07/2024Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
Posted on 29/04/202108/07/2024Adverum Biotechnologies announced a Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony (clinically-relevant decrease in ocular pressure) in its INFINITY clinical trial evaluating ADVM-022 gene therapy for the treatment of diabetic macular edema
Posted on 29/03/202108/07/2024Independent investigation shows that etranacogene dezaparvovec is highly unlikely to be the cause of HCC in HOPE-B pivotal trial
