Vertex inks pact with Enlaza to improve conditioning for Casgevy

Vertex has announced a multi-target drug discovery collaboration with covalent biologics platform company Enlaza Therapeutics to develop small format drug conjugates and T-cell engagers for certain autoimmune diseases and improved conditioning in sickle cell disease and beta thalassemia.

Enlaza’s War-Lock platform creates highly specific warheads that covalently bind to drug targets of interest. The platform utilizes proprietary non-natural amino acids to precision-engineer biologic medicines that expand the therapeutic window to enable safer, more effective therapies across numerous therapeutic modalities — one of which could be Vertex’s CRISPR/Cas9 gene-edited therapy, Casgevy.

Casgevy was approved by the FDA in December 2023, as the first gene-edited cell therapy for patients with sickle cell disease. In early 2024, its approval was expanded to include the treatment of transfusion-dependent beta thalassemia in patients 12 years and older. But treatment with Casgevy involves conditioning with chemotherapy — and an extended hospital stay — to remove existing blood stem cells from the bone marrow to make room for the newly edited cells.

Per the deal, Vertex will hand Enlaza $45 million inclusive of an upfront payment and equity investment. Enlaza is further eligible to receive more than $2 billion in future milestone payments.