A group led by researchers at Boston Children’s Hospital established a scalable and reproducible model of paclitaxel-induced axon degeneration and neurotoxicity in human induced pluripotent stem cell (iPSC)-derived sensory neurons.
HBM Alpha Therapeutics Inc. signed a potential $395 million licensing deal Feb. 26 with an unnamed “business partner” for its endocrine asset, HAT-001, adding another contender to the congenital adrenal hyperplasia space.
HBM Alpha Therapeutics Inc. signed a potential $395 million licensing deal Feb. 26 with an unnamed “business partner” for its endocrine asset, HAT-001, adding another contender to the congenital adrenal hyperplasia space.
Researchers at Boston Children's Hospital and Harvard Medical School have explored the possibility of using anaplastic lymphoma kinase (ALK) as a target for the application of ALK.CAR T cells to treat neuroblastoma, the most common and deadliest tumor of infancy.
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.
Schizophrenia (SCZ) could be associated with genetic alterations that can appear at the beginning of life. Such somatic variants in the NRXN1 and ABCB11 genes could lead to SCZ, according to researchers at Boston Children’s Hospital.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy. Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
Resistin-like molecule beta (RELMβ) is a gut-derived cytokine involved in both allergic responses and protection from pathogens, and it has been previously found to be dysregulated in mouse models of food allergy (FA). Researchers from Boston Children’s Hospital aimed assess the potential of RELMβ as a novel biomarker in children with FA.
HBM Alpha Therapeutics Inc. has completed seed financing to advance its lead programs, novel antibody therapies to treat congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS).