Vivacta Bio and Grit Bio have secured FDA orphan drug designation for GT801 in autoimmune hemolytic anemia. Early findings from two patients showed complete responses following treatment with the investigational in vivo CAR-T therapy, which uses targeted lipid nanoparticles to generate CD19-directed immune cells inside the body.
Find out how Cartesian Therapeutics’ WestGene deal could reshape in vivo CAR-T strategy in autoimmune disease and what risks remain.
T-CURX and Pantherna are combining CAR-T, mRNA and LNP platforms. Europe’s in vivo cell therapy race just became more serious.
Find out how Scribe Therapeutics’ STX-1150 could reshape long-acting LDL-C lowering through CRISPR-based epigenetic silencing.
Macrophage cell therapy has promise but painful execution risk. United Immunity’s Carisma deal tests whether in vivo CAR-M can scale.
Eli Lilly’s Engage Bio deal is small by its standards, but DNA delivery may become a big battleground in genetic medicines.
Viral vectors built gene therapy’s first era. Safety, redosing and manufacturing pressure are pushing non-viral platforms into the spotlight.
Explore how CPTx’s targeted lipid nanoparticle platform could reshape CAR T therapy scalability and access. Read the full analysis now.
Eli Lilly and Company has entered into a definitive agreement to acquire Orna Therapeutics, Inc., gaining access to the biotechnology firm’s circular RNA platform and its clinical trial ready in vivo CAR-T candidate ORN-252 targeting CD19 for B cell driven autoimmune diseases. The deal, valued at up to 2.4 billion dollars in cash including milestone