Scribe Therapeutics Inc. has announced an expanded collaboration with Sanofi SA, under which Sanofi receives an exclusive license to use Scribe’s CRISPR X-Editing (XE) genome editing technologies for the development of in vivo therapies, including for sickle cell disease.
Shanghai Institute of Materia Medica of the Chinese Academy of Sciences have developed nitrogenous heterocyclic compounds described as as receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors.
Recent studies suggest that neutrophils are not only involved in the innate immune response but also may play extended roles in chronic inflammatory disorders and adaptive immune responses as well.
Arrowhead Pharmaceuticals Inc. has filed an application in New Zealand for clearance to initiate a phase I/II trial of ARO-DUX4, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD).
Nysnobio Inc. has received a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to assist the advancement of the company’s lead parkin gene replacement therapy candidate, NB-001. The funding will support product manufacturing to enable IND safety studies in preparation for clinical trials evaluating NB-001 in Parkinson’s disease patients with biallelic loss-of-function mutations in the parkin gene, or parkin-PD.
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