Boston Children’s Hospital

Children’s Hospital Los Angeles to study “Bubble Boy” syndrome

Tuesday, October 10, 2017

Michael Pulsipher, M.D., of the Children’s Center for Cancer and Blood Diseases at Children’s Hospital Los Angeles, along with co-principal investigator, Sung-Yun Pai, M.D., of Boston Children’s Hospital, has been awarded nearly $9 million from the National Institute of Allergy and Infectious Diseases of the NIH to study a new treatment approach for babies born with severe combined immunodeficiency (SCID), which prevents the immune system from functioning normally. In a multi-site study, investigators plan to determine the lowest dose of chemotherapy needed for babies with SCID undergoing bone marrow transplant—the standard treatment for SCID. The goal is to restore the immune system safely and effectively with less toxicity than the higher dose regimens currently in use.

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The Institute for Protein Innovation launches

Friday, May 12, 2017

A team of scientists from the Massachusetts biomedical community launched a new research institute to expand protein science and enhance drug discovery. Backed by a unique and collaborative combination of public investment and academic philanthropy, The Institute for Protein Innovation (IPI) brings together leaders in academic research, biotechnology, pharmaceuticals and biomedical investing to seek new therapies for currently intractable diseases. 

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Cambridge Epigenetix licenses DNA Methylation IP from Boston Children’s Hospital

Tuesday, May 2, 2017

Cambridge Epigenetix, a pioneer in the development and application of epigenetic technologies, has entered into an exclusive license agreement with Boston Children’s Hospital giving access to DNA profiling technologies, which may hold the key to improved diagnoses and treatment of many diseases, including cancer. The licensed intellectual property (IP) is based upon the early findings of Professor Anjana Rao; an expert in immunology and cancer epigenetics, formerly of Boston Children’s Hospital and now with the La Jolla Institute, and a member of the Cambridge Epigenetix Scientific Advisory Board (SAB). Professor Rao’s pioneering research group was the first to identify 5-hydroxymethylcytosine (5hmC) as an important biomarker for cancer diagnosis that cannot be measured by traditional epigenetic sequencing methodologies. She also determined that the ten-eleven translocation (TET) family of enzymes generate 5hmC from 5-methylcytosine (5mC) and are key regulators of cellular differentiation that are frequently mutated in cancer.

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Study shows that almost half of pediatric trials are not finished or published

Tuesday, November 1, 2016

As Research Practitioner highlighted earlier this year, clinical research results have been disclosed late or not at all at least 95% of the time since reporting of results from human studies to the federal government’s database became mandatory in 2008.1  Most of these trials, however, use adult subjects. Researchers at Boston’s Children Hospital in Massachusetts decided to examine the reporting of pediatric study results and found that almost half of the studies had been discontinued or not published.

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PureTech, Siddhartha Mukherjee launch Vor BioPharma

Wednesday, May 11, 2016

PureTech Health has launched Vor BioPharma, an immuno-oncology company dedicated to developing a new class of targeted cell therapies. The company, which is advancing a novel approach to chimeric antigen receptor (CAR) T-cell therapy, has licensed its core technology from the lab of Vor scientific co-founder, Siddhartha Mukherjee, M.D., Ph.D., assistant professor of Medicine at Columbia University and Pulitzer Prize-winning author of The Emperor of All Maladies: A Biography of Cancer.

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