Acute myeloid leukemia (AML) is a hematological cancer with limited treatment options and characterized by frequent relapse and poor prognosis. The only approved antibody-drug conjugate for AML is gemtuzumab ozogamicin, which targets CD33.
Enveda has obtained IND clearance from the FDA and initiated a phase I trial of ENV-6946, a first-in-class oral small molecule for the treatment of inflammatory bowel disease, including ulcerative colitis and Crohn’s disease.
Vyriad Inc. has announced the closing of a $25 million final tranche to its series B financing, bringing the total series B round to $85 million. This additional funding will support first-in-human testing of VV-169, Vyriad’s in vivo CAR T candidate, in patients with relapsed or treatment-refractory multiple myeloma.
Mutations in the NF1 gene lead to neurofibromatosis type 1, which often leads to bone abnormalities and spine deformity. Efforts to treat or even prevent the disease have been stymied by lack of understanding about how the disease occurs and progresses.
In October, the Nobel Committee awarded the 2025 Nobel Prize in Physiology or Medicine to Shimon Sakaguchi, Mary Brunkow and Fred Ramsdell for their discoveries in the field of autoimmunity. As has become typical for the scientific Nobel Prizes, the award-winning research is by now several decades old. But the discoveries were the basis for ongoing research into how to prevent autoimmunity that notched significant wins in 2025, in both basic research and in the clinic.
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Laekna Pharmaceutical Ningbo Co. Ltd. has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.
Shanghai Xianxiang Medical Technology Co. Ltd. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN)-binding moiety coupled to an EGFR (HER1; erbB1) mutant-targeting moiety through a linker reported to be useful for the treatment of cancer.
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