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Dimension Therapeutics, Inc. (NASDAQ:DMTX), a leading gene therapy company advancing novel, liver-directed treatments for diverse rare diseases, today announced its plans to expand the company’s internal research and process development capabilities. Dimension has signed a lease agreement with Alexandria Real Estate Equities, Inc. (NYSE:ARE) for 17,475 square feet of state-of-the-art laboratory and office space at 19 Presidential Way in Woburn, Massachusetts, with occupancy expected in April 2016. The expansion builds upon Dimension’s existing mammalian cell platform in Cambridge, and will support continued innovation in vector optimization and development of manufacturing processes required for IND-enabling studies and the reliable production of high quality AAV vectors at commercial scale.

 

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WuXi PharmaTech (Cayman) Inc. (NYSE:  WX), a leading open-access R&D capability and technology platform company serving the pharmaceutical, biotechnology, and medical device industries, today announced that its Laboratory Testing Division (LTD) has launched preclinical medical device testing services in China. These services are being offered for Chinese and international clients for their product registration with the China Food and Drug Administration (CFDA) and for Chinese clients for product registration in global markets.

LTD’s preclinical medical device testing services in China include a complete portfolio of biocompatibility, microbiology, and chemistry testing services. Major tests being offered include in vitro cytotoxicity; acute, sub-chronic and chronic toxicity; irritation; sensitization; hemocompatibility; implantation; genotoxicity; carcinogenicity; effects on reproduction, including development effects; extractable and leachable testing; and chemical characterization. WuXi has been a leader in providing preclinical and clinical medical device testing services in the United States for many years.

 

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Dimension Therapeutics, Inc. (“Dimension” or the “Company”), a leading gene therapy company advancing novel, liver-directed treatments for diverse rare diseases, today announced the pricing of its initial public offering of 5,500,000 shares of its common stock at a public offering price of $13.00 per share. Dimension’s underwriters have been granted a 30-day option to purchase up to an additional 825,000 shares of common stock at the public offering price less underwriting discounts and commissions. All shares in the offering will be sold by Dimension and are expected to begin trading on the NASDAQ Global Select Market on October 22, 2015 under the symbol “DMTX”.

 

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WuXi PharmaTech (Cayman) Inc. (NYSE:  WX), a leading open-access R&D capability and technology platform company serving the pharmaceutical, biotechnology, and medical device industries, today announced that it has received the 2015 Asian CRO Company of the Year award from Frost & Sullivan, a leading business research and consulting firm. The award is part of the Frost & Sullivan Asia Pacific Best Practices Awards series, recognizing companies in Asia Pacific that demonstrate outstanding achievement and superior performance in areas such as leadership, technological innovation, customer service, and strategic product development.

 

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Aclaris Therapeutics, Inc. announced today that its wholly owned subsidiary, Aclaris Therapeutics International Limited (ATIL), and Rigel Pharmaceuticals, Inc. have entered into an exclusive, worldwide license agreement for the development and commercialization of Rigel’s JAK inhibitor compounds for the treatment of alopecia areata and other dermatological conditions.

Under the license agreement, ATIL has agreed to assume responsibility for the continued development of specified Rigel JAK inhibitor compounds for the treatment of alopecia areata and other dermatological conditions. ATIL has also agreed to make an upfront payment of $8 million to Rigel, as well as make various milestone payments and tiered royalties on any future sales of these compounds.

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Proteostasis Therapeutics, Inc. (PTI), a company that develops disease-modifying therapeutics for diseases involving protein processing, recently announced at a presentation during the 29th Annual North American Cystic Fibrosis Conference data on itstransmembrane conductance regulator (CFTR) amplifier program for use in combined treatments for patients with cystic fibrosis(CF).

In Proteostasis’ presentation entitled “CFTR Amplifiers Are a New Class of CFTR Modulators,” the company demonstrated an increase in CFTR modulating activity in human bronchial epithelial (hBE) cells when its composites were used as single therapies, as well as results that these composites are able to further enhance CFTR when utilized as a combined treatment with the currently available clinical-stage correctors.

 

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Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced it has successfully filed a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for aceneuramic acid prolonged release (Ace-ER; UX001) tablets intended for patients with GNE Myopathy. Ace-ER is designed to replace the deficient sialic acid substrate in patients with GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), a rare, progressive muscle-wasting disease.

“We have reached an important milestone for both Ultragenyx and patients living with GNE Myopathy,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer of Ultragenyx. “It is the company’s first filing for marketing approval, just five years after its founding, and the first marketing application of a potential treatment for patients affected by this progressive debilitating muscle disease.”

Ultragenyx is seeking to obtain conditional approval from the EMA for Ace-ER for the treatment of adult patients with GNE Myopathy. The MAA submission is based on positive data from a Phase 2 randomized, double-blind, placebo-controlled clinical study. If a positive opinion is received from the Committee for Medicinal Products for Human Use (CHMP), a decision from the European Commission would be expected in the second half of 2016.

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In a recent paper published in the Nature Reviews in Neurology journal, a team of US researchers discussed the current challenges, opportunities and emerging evaluation methods in preclinical Alzheimer disease treatments, describing a convergent initiative to improve Alzheimer disease prevention research coined as Collaboration for Alzheimer’s Prevention.

Alzheimer disease (AD) is progressive disorder affecting brain neurons, and is the most common cause of dementia. At the moment, this devastating illness has no cure and imposes a heavy burden to patients, caregivers and society. Studies have showed that specific medications, dietary supplements and lifestyle attitudes might potentially reduce the risk of developing AD. Nonetheless, the complete implications of these preventive measures have not been adequately assessed so far. Clinical studies evaluating the effects of AD prevention strategies in at-risk individuals without cognitive impairment are of enormous public benefit.

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