treatment News

Study highlights: Ex vivo treatment of AML and MDS bone marrow samples with bexmarilimab led to increased antigen-presenting human leukocyte antigen DR isotype (HLA-DR) expression, indicating improved antigen presentation capacity. Bexmarilimab , when combined with azacitidine or venetoclax, enhanced HLA-DR expression and in many samples, reduced the viability of leukemic blasts, particularly in...
Houston, Texas – Rice University researchers have developed a rapid, accurate test for diagnosing malaria that is significantly faster and easier to use than traditional tests. The advancement has the potential to improve patient outcomes, especially in rural regions with limited health care resources. Malaria remains a significant global health...
SAN DIEGO – Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer, today announced encouraging interim Phase 1 data from the Company’s off-the-shelf, iPSC-derived natural killer (NK) cell programs in relapsed / refractory acute myeloid leukemia (AML). The ongoing Phase...
5 of 6 Patients Achieve Objective Response, including 4 Patients with Complete Response, with Single Dose of FT596 at 900 Million Cells in Combination with Rituximab 13 of 19 Patients Achieve Objective Response with Single Dose of FT596 at 90 Million and 300 Million Cell Dose; 10 of 11 Patients...
Nashville, Tennessee – Among adult patients with severe sickle cell disease (SCD), favorable outcomes were reported with use of a modified transplantation procedure, according to results from a multicenter study presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition in December 2023. Although the use of...
EMERYVILLE, Calif. — XOMA Corporation the biotech royalty aggregator, announced today that based upon the U.S. Food and Drug Administration’s (FDA) acceptance of Zevra Therapeutics’ New Drug Application (NDA) for arimoclomol, an orally-delivered, first-in-class therapy for Niemann-Pick disease type C (NPC), XOMA will make a $1 million milestone payment to...
Stockholm, Sweden – Sobi® (STO: SOBI), a global biopharmaceutical company dedicated to delivering innovative treatments for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) seeking approval for Nanoecapsulated Sirolimus plus Pegadricase (NASP), formerly SEL-212, for the treatment...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the company’s PALYNZIQ® (pegvaliase-pqpz) supplemental Biologics License Application (sBLA) to expand treatment to include adolescents aged 12-17 with phenylketonuria (PKU). The FDA has set a Prescription Drug User...
DEER PARK, Ill. — Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the Company’s New Drug Application (NDA) for ET-600, a proprietary patented formulation of desmopressin oral solution, has been accepted for review by...
PRINCETON, N.J. — Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage, biopharma company advancing GTx-104, a clinical-stage, novel, injectable formulation of nimodipine being developed for IV infusion to address significant unmet medical needs in aSAH patients, today announced that on August 22, 2025 the U.S. Food...