Using whole genome sequencing, scientists at Boston Children’s Hospital have studied the genes and mutations of ataxia-telangiectasia (A-T) that would respond to treatments with splice-switching antisense oligonucleotides (ASOs). Their work, published on July 12, 2023, in Nature, determined the appropriate individualized genetic therapy for these patients and identified a new drug. Read More
An oral drug has shown promise for treating metabolic syndrome in mouse models by selectively blocking the production of free radicals in the mitochondria, a research group has found. The study, carried out by a group led by Martin Brand, Professor Emeritus at the Buck Institute for Research on Aging, could lead to treatments that tackle the underlying causes of metabolic syndrome and multiple other conditions linked to aging. Read More
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. Read More
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). Read More
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. Read More
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. Read More
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. Read More
Sangamo Therapeutics Inc. has signed an evaluation and option agreement with Prevail Therapeutics Inc., a wholly owned subsidiary of Eli Lilly & Co., through which Prevail has been granted rights to evaluate certain proprietary adeno-associated virus (AAV) capsids developed by Sangamo and may exercise certain options to license these capsids for multiple undisclosed neurological targets. Read More
Adcentrx Therapeutics Inc. has received FDA clearance of its IND application for ADRX-0706 for the treatment of select advanced solid tumors. Read More
Bivictrix Therapeutics plc has released promising final data from a second in vivo efficacy study of its lead clinical candidate BVX-001, a first-in-class Bi-Cygni antibody-drug conjugate (ADC) for the treatment of acute myeloid leukemia (AML). Read More
