-- Collaboration to leverage Orna's differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT --

-- Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties --

WATERTOWN, Mass., Jan. 7, 2025 /PRNewswire/ -- Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.) announced a three-year strategic research collaboration with Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) to utilize Orna's novel and proprietary LNP delivery solutions to enhance Vertex's efforts in developing next generation gene editing therapies for patients with SCD and TDT.

TORONTO, Sept. 26, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has accepted for review its New Drug Submission (NDS) for vanzacaftor/tezacaftor/deutivacaftor, a once-daily triple combination therapy for people living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

– First regulatory authorization of a CRISPR-based gene-editing therapy in Canada –  

TORONTO, Sept. 25, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for PrCASGEVY® (exagamglogene autotemcel), an autologous genome edited hematopoietic stem cell-based therapy, for the treatment of patients 12 years of age and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). There are an estimated 2,000 patients eligible for CASGEVY in Canada, the majority of whom are living with SCD.