A new RNA partnership hunts rare epilepsies; AbbVie and I-Mab tackle CD47
Zogenix is getting a new partner as it looks to tackle rare childhood seizures.
The California-based biotech, which developed and produces the seizure drug Fintepla, is teaming with Trevard Biosciences to develop RNA-based gene therapies for Dravet syndrome and other genetic epilepsies.
Spun out of MIT and advised by David Liu and Bob Langer, Trevard is trying to use transcriptional RNA to increase the expression of healthy copies of the gene behind Dravet’s syndrome in patients and decrease the expression of the faulty copy.
Zogenix will pay Trevard $10 million upfront and $5 million in stock, with milestones between $70 million and $100 million available per program. — Jason Mast
China’s I-Mab pushes ahead with combo trials of AbbVie-partnered CD47 drug
AbbVie’s CD47 partner I-Mab says the drug, lemzoparlimab, is cruising nicely along with clinical development, moving into dose expansion in a combo study.
Topline results from the study, which tests the pairing of lemzoparlimab with Rituxan and Keytruda, respectively, are expected next year. Both will enroll patients with non-Hodgkin’s lymphoma; the Rituxan portion will take place in both the US and China.
I-Mab, which has offices in Shanghai and Gaithersburg, MD, said it’s also pushing into late-stage trials in relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndrome in China after wrapping monotherapy dose escalation.
It scored a hefty partner in AbbVie in September, scoring $180 million upfront for a deal that can add up to $3 billion. — Amber Tong
Magenta and bluebird team up to look for sickle cell clues
Bluebird bio is enlisting Magenta in its efforts to clear one of the hurdles to sickle cell gene therapy.
The Cambridge biotech, which has long been one of the leaders in the sickle cell gene therapy field, is trying to find a better way of removing patients’ stem cells, so they can be edited and returned.
In many bone marrow transplants, you can get the cells in the right spot to be removed with a GCS-F antibody. But in sickle cell patients, the GCS-F blockers can lead to the painful vaso-occlusive crises that are a hallmark of the disease. An alternative, Plerixafor, exists but it’s not a perfect replacement, often requiring patients to come in for multiple collections.
Magenta’s MGTA-145 is designed to work better, when combined with Plerixafor. — Jason Mast