aviscumine (CY-503) / MELEMA Pharma - LARVOL DELTA

Design, optimization, production and activity testing of recombinant immunotoxins expressed in plants and plant cells for the treatment of monocytic leukemia. (PubMed, Bioengineered) - "Plants are capable of the post-translational modifications needed for functional antibodies and can also express active protein toxins such as the recombinant mistletoe lectin viscumin, which is not possible in prokaryotes and mammalian cells respectively...One anti-CD64 targeted viscumin-based drug candidate was characterized in terms of storage stability and cytotoxicity test in vitro using human myelomonocytic leukemia cell lines. We identified bottlenecks in the plant-based expression platform that require further improvement and assessed critical process parameters that should be considered during process development for plant-made RITs."

Journal • Hematological Malignancies • Leukemia • Oncology

Rare deleterious BUB1B variants induce premature ovarian insufficiency and early menopause. (PubMed, Hum Mol Genet) - "Furthermore, our replication study using targeted sequencing revealed a novel stop-gain variant of BUB1B c.1509 T > A (p.Cys503*) in one of 200 sporadic POI cases...Notably, Bub1b+/- female mice presented late-onset subfertility, and they were more sensitive to oxidative stress than wild-type female controls, mimicking the clinical phenotypes of POI cases affected by deleterious BUB1B variants. Our findings in human cases and mouse models consistently suggest, for the first time, that heterozygous deleterious variants of BUB1B are involved in late-onset POI and related disorders."

Journal • Gynecology • Sexual Disorders