United States

FDA approves Brineura for CLN2 disease

Wednesday, May 3, 2017

BioMarin Pharmaceutical announced that the FDA approved Brineura (cerliponase alfa) to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is the first treatment approved to treat children with CLN2 disease, a form of Batten disease.

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Medeor receives FDA IND clearance for MDR-101 study

Wednesday, May 3, 2017

Medeor Therapeutics, a private, clinical-stage biotechnology company dedicated to the discovery, development and commercialization of personalized cellular immunotherapies, has received clearance from the FDA for an Investigational New Drug (IND) application for the company’s lead product candidate MDR-101. In addition, the FDA has agreed to a Special Protocol Assessment (SPA) for the design of a pivotal phase III clinical study of MDR-101 in.

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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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