Bristol Myers Squibb Co. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN) E3 ubiquitin ligase-binding moiety covalently bonded to a B-cell lymphoma 6 protein (BCL-6)-targeting moiety through a linker. They are reported to be useful for the treatment of cancer and autoimmune diseases.
It is far easier and safer to inject drugs into veins than directly into the brain, yet it is extremely difficult for systemically delivered drugs to cross the blood-brain barrier and achieve therapeutic concentrations there.
Oncolytic viruses trigger immunogenic cell death in tumors, releasing signals that activate innate immune cells and promote tumor-specific T-cell responses. While some oncolytic viruses, including FDA-approved T-VEC, have shown safety and some clinical benefit, their limited effectiveness as standalone treatments underscores the need for combination therapies.
Age-related macular degeneration (AMD) is the primary cause of blindness in people aged 65 and older in developed nations. Recent estimates suggest that, globally, the number of individuals affected by AMD could rise to approximately 288 million by 2040.
Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is an important factor from a complex downstream of Bruton tyrosine kinase (BTK) in the B-cell receptor (BCR) signaling pathway.
CAR T-cell therapy for B-cell malignancies has still to be improved regarding durability, manufacturing complexity or toxicity, among others. Precigen Inc. has presented data regarding the preclinical development and efficacy of PRGN-3008, a PD-1-expression inhibitor cell therapy targeting CD19 that was built in a next-generation ultra CAR T platform.
Suzhou Sanegene Bio Inc. has gained clinical trial approval in China for SGB-3383 for the treatment of complement-mediated kidney diseases, including IgA nephropathy, C3 glomerulopathy, immune complex-mediated membranoproliferative glomerulonephritis and atypical hemolytic uremic syndrome.
Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
Breast cancer is the most frequent malignancy among women worldwide, and all subtypes of breast cancer involve upregulation of the c-Myc gene, making it a compelling therapeutic target. G-rich regions of the c-Myc promoter can form G-quadruplex structures, which can be targeted using small molecules containing a styrylquinolinium core, which then downregulate oncogenic c-Myc. The challenge, however, is specificity.
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