Ultragenyx Pharmaceutical Inc., of Novato, Calif., reported interim results from the first dose cohort of a phase I/II trial testing its gene therapy DTX301, as a treatment of ornithine transcarbamylase deficiency. Read More
Bluebird Bio Inc., of Cambridge, Mass., closed its previously announced public offering, issuing an additional 277,109 shares at $185 per share, raising approximately $51.3 million additional capital as part of the underwriters' over-allotment option. Read More
Resverlogix Corp., of Calgary, Alberta, entered a licensing agreement with Medison Pharma Ltd., of Petach Tikva, Israel, for apabetalone (RVX-208) in Israel and the Palestine Authority. Read More
NEW DELHI – The World Health Organization (WHO) has pre-qualified Indian company Bharat Biotech International Ltd.'s Typbar TCV, its conjugate vaccine against typhoid fever. The pre-qualification paves the way for possible use in childhood immunization programs against typhoid in high-risk, endemic countries. Read More
SHANGHAI – Jiangsu Hengrui Medicine Co., of Shanghai and Lianyungang, continues to find success licensing-out the global rights to U.S. biopharmas from its pipeline of 21 new molecular entities. Read More
SHANGHAI – The CFDA recently released "Guiding Principles for the Research and Evaluation of Cell Therapy Products" for trial implementation. This hotly anticipated move now adds cell therapies such as stem cells and chimeric antigen receptor (CAR T) therapies under the umbrella of CFDA reforms that are aligning China's drug regulations with global drug markets. Read More
SAN FRANCISCO – From angel investors to big pharma partners to corporate VCs, life sciences entrepreneurs got an earful about what to do – and what to avoid – when seeking funding and building relationships during the Redefining Early Stage Investments (RESI) conference and partnering forum in San Francisco, held in conjunction with the 36th Annual J.P. Morgan Healthcare Conference. Read More
SAN FRANCISCO – If 2017 marked a year of big wins for scientific innovation with the FDA approvals of the first three gene therapies – Novartis AG's Kymriah (tisagenlecleucel) CAR T immunotherapy for B-cell acute lymphoblastic leukemia, Kite Pharma Inc.-developed CAR T Yescarta (axicabtagene ciloleucel) for certain types of non-Hodgkin lymphoma and more "traditional" gene therapy Luxturna (voretigene neparvovec-rzyl) from Spark Therapeutics Inc. to treat RPE65 mutation-associated retinal dystrophy – then 2018 might be the year when the innovative capabilities on the payer side are put to the test. Read More
"All drug prices are too high in this country," Alex Azar acknowledged Tuesday as he sat in the hot seat at a Senate Finance Committee hearing convened to consider his nomination as the next secretary of the U.S. Department of Health and Human Services (HHS). Read More
Advice from the independent data monitoring committee (IDMC) that Axsome Therapeutics Inc. quit the phase III trial called CREATE-1 with AXS-02 (disodium zoledronate tetrahydrate) put a dent in the company's shares and reshuffled the landscape for complex regional pain syndrome (CRPS) candidates. Read More
