Taysha Gene Therapies has encouraging data from an open-label, Phase 1/2 study in the rare disease giant axonal neuropathy. It might not be enough. The FDA recommended the biotech conduct a randomized and placebo-controlled study a challenge in any ultra-rare disease.
In partnering with clinical-stage Arcellx, Gilead Sciences gains the opportunity to develop a cell therapy for multiple myeloma that could offer advantages over currently available therapies for the blood cancer. The deal announced Friday came as Arcellx reported additional data during the annual meeting of the American Society of Hematology that showed continued durability of the cell therapy.
CSL Behring's hemophilia B gene therapy, Hemgenix, carries a $3.5 million list price. But the company contends that by reducing economic burdens associated with bleeding and prophylactic infusions, the one-time treatment will save money over time.
Breast cancer drugs and cell therapies were prominent in this year’s annual meeting of the American Society of Clinical Oncology. Here’s a recap of some of the news highlights from the biggest cancer conference of the year.
Two natural killer (NK)cell therapy candidates from Nkarta now have preliminary Phase 1 clinical data suggesting these therapies are helping patients in two types of blood cancers. Nkarta therapies based on engineered NK cells could offer both efficacy and safety advantages over the first generations of cell therapies, which are made from a patient’s own T cells.