Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal electrical signaling. These alterations give rise to a severe, early-onset developmental and epileptic encephalopathy that is typically associated with a high seizure burden and resistance to standard antiseizure medications.
ENL-YEATS is an epigenetic reader that sustains transcriptional programs essential for AML, whereas FLT3 mutations, present in approximately 30% of patients, drive malignant proliferation. Dual inhibition of ENL-YEATS and FLT3 may therefore more effectively disrupt complementary drivers of leukemogenesis than FLT3 targeting alone.
Exicure Inc.’s buyout early this year of GPCR Therapeutics Inc. is paying off in a big way with data from the finished phase II trial testing burixafor (GPC-100). The agent is used with propranolol and granulocyte colony-stimulating factor to mobilize hematopoietic progenitor cells in patients with multiple myeloma (MM) undergoing autologous hematopoietic cell transplant.
Impressive results of a potential second-line combination treatment for multiple myeloma from the Majestec-3 trial of teclistamab plus daratumumab raised eyebrows at the American Society of Hematology’s 67th annual meeting, with the combination showing an 83.4% rate of progression-free survival at three years vs. 29.7% for standard of care.
Investigators from Secarna Pharmaceuticals GmbH & Co. KG recently presented data for their antisense oligonucleotide (ASO) SECN-15 that targets and downregulates the expression of neuropilin-1 (NRP1), a transmembrane co-receptor that promotes tumor progression in several tumor types, including breast and gastric cancers.
Evidence suggests the composition of the gut microbiome modulates a tumor’s response to therapy. Maat Pharma SA has thus developed Microbiome Ecosystem Therapies (METs) that replicate, at a large scale, the effects of the gut microbiome in people who respond to immune checkpoint inhibitor (ICI) therapy.
Targeted therapies and immunotherapies continue to show better results than chemotherapy in investigator-initiated and company-sponsored cancer trials, and newer options demonstrate improvements over older ones, supporting potential shifts in how patients are treated.
While several targeted therapies are approved for acute myeloid leukemia, a 2023 U.K. study found that median survival following diagnosis was only about seven months, highlighting the need not only for new therapies, but for a faster regulatory strategy. At the American Society of Hematology’s 67th annual meeting in Orlando Dec. 6, researcher Jesse Tettero presented data supporting the use of a measurable residual disease (MRD) surrogate endpoint in acute myeloid leukemia (AML) research.
Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster.
The overexpression of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) is tied to poor prognosis in several cancer types, including osteosarcoma and Ewing sarcoma, and is therefore a potential therapeutic target in these cancers. In this regard, Avammune Therapeutics Inc. is developing an ENPP1 inhibitor – AVA-NP-695.
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