1) With the help of patient groups, biotechs hone in on rare diseases – BBJ, February 26, 2015
This Saturday is worldwide Rare Disease Day, the eighth such day coordinated by the European Organization for Rare Diseases to draw attention to the some 7,000 diseases which are shares by a small number of patients each — many just a few thousand — but collectively affect 350 million people globally. Fueled both by scientific advances in genetics and the support of patient organizations, the biotech industry is showing more interest than ever in developing drugs for such diseases. Besides Genzyme Corp. and Shire, scores of smaller biotechs are focusing on genetically defined conditions with small patient populations.
2) Cost concerns at center of debate over rare disease treatment – Nashoba Publishing, February 26, 2015
As drug companies pioneer new methods of treating an array of diseases, some lawmakers want to limit the out-of-pocket cost to health plan members purchasing medications while others worry that tack would cause costs to soar.
3) What 23andMe’s FDA Approval Means For The Future Of Genomics – Forbes, February 20, 2015
Last night, the Food and Drug Administration approved a test made by 23andMe, the Mountain View, Calif.-based personal genetics company, for a gene that can cause a rare disorder called Bloom Syndrome, which causes short stature and a heightened risk of cancer.
4) Cambridge firms will use gene database to make new drugs – Boston Globe, February 23, 2015
Two Cambridge life sciences companies have struck a deal to collaborate on making new cancer drugs.Foundation Medicine, which tests tumors for genetic mutations and suggests treatment plans to doctors, said it would make its genetic database available to H3 Biomedicine as part of a multi-year agreement to create and commercialize new drugs. The database, called FoundationCORE, contains genetic information on more than 35,000 samples.
5) Dicerna doubles in size and moves to West Cambridge – BBJ, February 26, 2015
With two clinical trials underway and a third set to begin later this year, Dicerna Pharmaceuticals is today moving into a new lab and office space that’s twice the size of its Watertown headquarters.
Together, as partners with our customers, we are committed to provide product development services with new and innovative ways to reduce costs and lead times, while continually maintaining the highest quality standards. Quality is a fundamental and integral part of our business. Through our People and Systems, we provide high quality products and professional services to the pharmaceutical industry. We always strive to exceed all the expectations of our customers. CuriRx Incorporation’s goal is to create new standards of care in therapeutic areas with high unmet medical needs by partnering and providing innovative solutions for drug development of pharmaceutics, biopharmaceutics, vaccines, generics and biosimilars products. Website.
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Rubius Therapeutics is focused on developing circulating cell therapies for autoimmune and rare metabolic diseases.
MassCONNECT, the only entrepreneur mentorship program in Massachusetts that dives deep into the life sciences industry, kicked off its first cycle of 2015 with a Technology Showcase at LabCentral on February 24th. At the Showcase, the chosen entrepreneurs presented their ideas to a room of industry executives and potential mentors for early feedback.
First up to present was Yoav Golan, MD, MS of Tufts NewCo. This new company started by Golan and his colleague Abraham (Linc) Sonenshein, PhD is developing a new non-antibiotic molecular entity to prevent and treat C. Difficile, a leading cause of healthcare associated bacterial infections.
Next up was Cinzia Metallo, PhD of Biomille Technologies which has developed the first device specifically designed for facial muscle rehabilitation. Biomille’s electrodes are shaped to match the muscle anatomy of the face and enable patients affected by a vast range of facial impairments to regain their facial muscle functionality.
On Monday, February 23rd, more than 100 patient advocates, biotechnology industry stakeholders and legislators gathered at the Massachusetts State House to mark the 8th annual Rare Disease Day.
The complex nature of rare diseases, coupled with limited access to treatment and services, means that family members are often the primary source of solidarity, support and care for their loved ones. The Rare Disease Day 2015 theme Living with a Rare Disease pays tribute to the millions and millions of parents, siblings, grandparents, spouses, aunts, uncles, cousins, and friends whose daily lives are impacted and who are living day-by-day, hand-in-hand with rare disease patients.
In 2011, Governor Deval Patrick designated the last day of February as Rare Disease Day in order to call attention to the public health issues associated with rare diseases, which affect nearly 30 million Americans and countless others around the world.
Our Rare Disease Day at the State House began with a panel discussion in the morning. The Forum, “State of Rare: Taking Stock of Rare Disease Research Today & Tomorrow” featured speakers: Norman Barton, M.D., Ph.D., Vice President, Clinical Development, Shire, Ed Kaye, M.D., Senior Vice President, Chief Medical Officer, Sarepta Therapeutics, Karen Peluso, Executive Director, Neurofibromatosis Northeast, Steve Uden M.D., Senior Vice President, Research, Alexion Pharmaceuticals and moderator Ken Dhimitri, Vice President, Operations, Boston Biomedical Associates.
1) New research sheds light on cancer, Alzheimer’s origins – Boston Globe, February 18, 2015
Boston scientists have developed a technique that can trace a cancer cell back to the tissue where it started, raising hope for one day improving treatment for mysterious cancers of unknown origin.Using the same data set, a team in Cambridge discovered, to their surprise, that immune cells — and not brain cells involved in learning and memory — appear to be at the genetic root of Alzheimer’s disease.
2) F.D.A. Eases Access to DNA Tests of Rare Disorders – New York Times, February 19, 2015
On Thursday, the F.D.A. approved a test from 23andMe that would be administered to prospective parents to see if they carry mutations that could cause a rare disorder called Bloom syndrome in their children. The agency also said that, in general, such so-called carrier tests would no longer need to be approved in advance before being marketed. It also voiced support for allowing consumers in some situations to have direct access to genetic testing, without a doctor being involved.
3) Our View: Welcome, MassBiologics – The Herald News, February 17, 2015
There have been many twists and turns — and even a couple different names — as the construction of the anchor facility of the SouthCoast Life Sciences and Technology Park at Fall River has progressed. The facility that partially opened last week is now known as MassBiologics SouthCoast.
4) FDA Outlines Plan for Expected $10M Under President’s Precision Medicine Plan – Genome Web, February 18, 2015
The US Food and Drug Administration is planning to put the $10 million the agency expects to receive under the President’s Precision Medicine Initiative to set up databases that can help inform its oversight of next-generation sequencing tests, and to establish the bioinformatics infrastructure to facilitate the curation and sharing of the information in those repositories, an agency official said today.
5) How Pfizer Is Using Big Data To Power Patient Care – Forbes, February 17, 2015
Through data, we are identifying the most important aspects of a disease to study in our trials. In developing a medicine for lupus, combing data for patterns can help us identify characteristics of patients that are more likely to experience life-threatening disease flares. These analyses help us more precisely find which patients to study, and which endpoints will provide the most relevant findings for both quality of care and cost. Ultimately, understanding which patients to include will help us complete our clinical trials in a shorter timeframe and deliver a medicine in an area where there hasn’t been a new treatment option in 50 years.