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BioWorld - Wednesday, July 15, 2026
Home » Boston Children’s Hospital

Articles Tagged with ''Boston Children’s Hospital''

Concept art for "unlocking the secrets of the mind"
Drug design, drug delivery & technologies

New assay for drug discovery against chemo-induced peripheral neuropathy

May 8, 2026
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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.
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Gold wireframe handshake

Harbour Biomed newco HBM Alpha pens $395M hyperplasia drug deal

March 4, 2025
By Marian (YoonJee) Chu
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.
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Gold wireframe handshake

Harbour Biomed newco HBM Alpha pens $395M hyperplasia drug deal

Feb. 26, 2025
By Marian (YoonJee) Chu
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.
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Real fluorescence microscopic view of human neuroblastoma cells
Cancer

Less is more: ALK inhibitors increase exposed ALK expression

Dec. 15, 2023
By Coia Dulsat
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.
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3D illustration of a ribosome constructing messenger RNA molecules
Drug Design, Drug Delivery & Technologies

Even personalized drugs could benefit multiple patients

July 18, 2023
By Mar de Miguel
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.
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Neurology/Psychiatric

Two mutations acquired during development could cause schizophrenia in adulthood

July 7, 2023
By Mar de Miguel
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.
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Gene editing illustration

Base editing rescues spinal muscular atrophy in vivo

April 10, 2023
By Mar de Miguel
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.
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Genetic/Congenital

Base editing rescues spinal muscular atrophy in vivo

April 6, 2023
By Mar de Miguel
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.
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Biomarkers

RELMβ as novel biomarker and pathogenic player in children with food allergy

March 2, 2023
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.
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Endocrine/Metabolic

HBM Alpha completes seed financing to advance programs for congenital adrenal hyperplasia and PCOS

Jan. 27, 2023
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).
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