About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.
About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.
Vertex Pharmaceuticals Inc.’s chief operating officer Stuart Arbuckle said during the Stifel Healthcare Conference in November that his firm’s launch last year of Casgevy (exagamglogene autotemcel) for sickle cell disease and transfusion-dependent beta-thalassemia “was the beginning of diversifying on top of our cystic fibrosis [CF] base” – a base that itself continues to grow.
Clinical data reported by a handful of biopharmas on Dec. 19 sent stocks soaring or plunging, as company leaders laid out next-step plans. Investors showed enthusiasm for Omeros Corp.’s narsoplimab to treat hematopoietic stem cell-transplant-associated thrombotic microangiopathy (TA-TMA), and Roche AG’s Parkinson’s prospect prasinezumab, which is partnered with Prothena Corp. plc.
The U.S. CMS has negotiated outcomes-based agreements with Bluebird Bio Inc. and Vertex Pharmaceuticals Inc. to make their costly sickle cell gene therapies the first treatments to become available through the voluntary Medicaid Cell and Gene Therapy Access Model.
Visterra Inc., a subsidiary of Otsuka Pharmaceutical Co. Ltd., reported positive top-line data from the ongoing Visionary phase III study of sibeprenlimab, an anti-APRIL monoclonal antibody for immunoglobulin A nephropathy (IgAN).
Although more and more gene therapies are getting the FDA stamp of approval, concerns persist about their potential long-term risks. U.S. lawmakers have proposed several pieces of legislation over the past few years to address some of the uncertainties. Now the Congressional Research Service (CRS) is suggesting other requirements Congress may want to consider to improve the regulatory landscape for gene therapies, especially those intended to treat blood disorders.
Visterra Inc., a subsidiary of Otsuka Pharmaceutical Co. Ltd., reported positive top-line data from the ongoing Visionary phase III study of sibeprenlimab, an anti-APRIL monoclonal antibody for immunoglobulin A nephropathy (IgAN).
Vertex Pharmaceuticals Inc. has described orexin OX2 receptor agonists reported to be useful for the treatment of amyotrophic lateral sclerosis, obesity, hypertension, retinopathy, multiple sclerosis, narcolepsy, hypersomnia and Parkinson’s disease.
In denying Medicaid patients with sickle cell disease or transfusion-dependent beta-thalassemia access to Vertex Pharmaceuticals Inc.’s fertility preservation program, which is intended to counteract a side effect of the company’s gene-editing therapy, Casgevy, “the federal government now stands as the barrier between thousands of predominantly Black Americans and the necessary medical care that would protect their basic right to have biological children,” Vertex said in a lawsuit filed July 15.
