BERKELEY A simple, precise and inexpensive method for cutting DNA to insert genes into human cells could transform genetic medicine, making routine what now are expensive, complicated and rare procedures for replacing defective genes in order to fix genetic disease or even cure AIDS. Discovered last year by Jennifer Doudna and Martin Jinek of the Howard Hughes Medical Institute and University of California, Berkeley, and Emmanuelle Charpentier of the Laboratory for Molecular Infection Medicine-Sweden, the technique was labeled a "tour de force" in a 2012 review in the journal Nature Biotechnology. That review was based solely on the team's June 28, 2012, Science paper, in which the researchers described a new method of precisely targeting and cutting DNA in bacteria. Two new papers published last week in the journal Science Express demonstrate that the technique also works in human cells. A paper by Doudna and her team reporting similarly successful results in human cells has been accepted for publication by the new open-access journal eLife. "The ability to modify specific elements of an organism's genes has been essential to advance our understanding of biology, including human health," said Doudna, a professor of molecular and cell biology and of chemistry and … Continue reading →

Fetal DNA circulating in a pregnant mothers blood can be used to detect a wide variety of genetic abnormalities before birth, opening the door for noninvasive testing for more conditions. By sequencing DNA that escapes into womens bloodstreams, scientists were able to detect disease-causing mutations that are now normally found by piercing a mothers womb with a needle to get amniotic fluid, according to a study in the American Journal of Human Genetics. Amniocentesis, the standard procedure for prenatally testing for genetic conditions such as Down syndrome, carries a low risk of miscarriage. Obtaining DNA from a blood sample from the mother carries virtually no risk, and may enable doctors to expand their reach and accuracy as they look for genetic disease, said Cynthia Morton, a Harvard Medical School geneticist who performs prenatal tests at Brigham & Womens Hospital in Boston. This could largely replace invasive testing, she said in a telephone interview, and, no doubt, is an exciting next step in the future of prenatal testing. The study was done by scientists at Tufts Medical Center in Boston and Verinata Health Inc. in Redwood City, California. Illumina Inc. (ILMN), the biggest maker of DNA sequencers, said this week that … Continue reading →

Geron Corporation (GERN) announced that it has signed an Asset Contribution Agreement with BioTime, Inc. (BTX) and BioTime's recently formed subsidiary, BioTime Acquisition Corporation (BAC). Per the terms of the agreement, Gerons stem cell assets will be divested to BAC on the closing of the deal. The deal is scheduled to close by September 30, 2013. Once the deal is closed, Geron will provide BAC with the intellectual property and other assets related to its discontinued human embryonic stem cell programs. BioTime will provide BAC with cash ($5 million), shares (8.9 million), warrants to purchase 8 million shares at a price of $5.00 per share, rights to use certain human embryonic stem cell lines, and minority stakes in two subsidiaries of BioTime. Meanwhile, Geron stockholders would have a 21% stake in BAC. Geron will also be entitled to receive royalties on the sale of products covered by the patents acquired by BAC from Geron. Apart from Geron, BioTime would have a 72% stake in BAC and the remaining 7% would be owned by a private investor (for an investment of $5 million). BioTime will also have warrants which would allow the company to increase its stake in BAC by 2%. … Continue reading →

Jan. 10, 2013 Type 1 and type 2 diabetes results when beta cells in the pancreas fail to produce enough insulin, the hormone that regulates blood sugar. One approach to treating diabetes is to stimulate regeneration of new beta cells. There are currently two ways of generating endocrine cells (cell types, such as beta cells, that secrete hormones) from human embryonic stem cells, or hESCs: either generating the cells in vitro in culture or transplanting immature endocrine cell precursors into mice. Researchers from the University of California, San Diego School of Medicine, collaborating with scientists from San Diego-based biotech company ViaCyte, Inc., looked at the differences and similarities between these two types of hESC-derived endocrine cell populations and primary human endocrine cells, with the longer-term goal of developing new stem cell therapies for diabetes. The results of their study will be published on line January 10, in advance of the February 7 print edition of the journal Cell Stem Cell. The scientists compared gene expression and chromatin architecture -- the structure of combined DNA and proteins that make up the nucleus of the cell, in which dynamic remodeling occurs at various stages of differentiation -- in both primary human endocrine … Continue reading →

UC San Diego-based study looks at differentiation of hESCs in endocrine cell progression Newswise Type 1 and type 2 diabetes results when beta cells in the pancreas fail to produce enough insulin, the hormone that regulates blood sugar. One approach to treating diabetes is to stimulate regeneration of new beta cells. There are currently two ways of generating endocrine cells (cell types, such as beta cells, that secrete hormones) from human embryonic stem cells, or hESCs: either generating the cells in vitro in culture or transplanting immature endocrine cell precursors into mice. Researchers from the University of California, San Diego School of Medicine, collaborating with scientists from San Diego-based biotech company ViaCyte, Inc., looked at the differences and similarities between these two types of hESC-derived endocrine cell populations and primary human endocrine cells, with the longer-term goal of developing new stem cell therapies for diabetes. The results of their study will be published on line January 10, in advance of the February 7 print edition of the journal Cell Stem Cell. The scientists compared gene expression and chromatin architecture the structure of combined DNA and proteins that make up the nucleus of the cell, in which dynamic remodeling occurs at … Continue reading →

BIND Biosciences and Amgen Sign Agreement for the Worldwide Development and Commercialization of a Kinase Inhibitor Nanomedicine Collaboration to Develop a Novel and Targeted Accurin Based on BINDs Nanomedicine Platform and Amgens Kinase Inhibitor BIND Biosciences, a clinical-stage biopharmaceutical company developing a new class of highly selective targeted and programmable therapeutics called Accurins, announced recently that it has entered into a global collaboration agreement with Amgen Inc. to develop and commercialize a kinase inhibitor nanomedicine for treating a range of solid tumors. The collaboration will develop a novel Accurin based on BINDs platform for targeted and programmable nanomedicines and Amgens undisclosed proprietary kinase inhibitor. The collaboration aims to create a kinase inhibitor nanomedicine with optimized therapeutic properties, applying for the first time tissue targeting to molecularly targeted drugs. Under the terms of the agreement, Amgen will have the exclusive right to pursue development and commercialization of the Accurin kinase inhibitor against solid tumor targets to be selected by Amgen. Both companies will work together on preclinical development and Amgen will assume responsibility for future development and commercialization. BIND could receive up-front and development milestone payments totaling $46.5M, and BIND could receive up to an additional $134M in regulatory and sales … Continue reading →