Company Overview and News
As gene therapy gains traction, it is expected to create a number of job opportunities. For example, the UK government projects gene and cell therapy in Britain alone will create 18,000 new jobs by 2030. The U.S. Bureau of Labor Statistics predicts a 7 percent increase in jobs for biomedical engineers and a 13 percent increase in medical scientists.
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Researchers at Johns Hopkins University, the University of California, San Diego (UCSD), and the National Institute of Mental Health grew retinas in Petri dishes, and discovered fundamental factors involved in the development of vision and color vision. Their research was published in the journal Science.
PHILADELPHIA, Oct. 08, 2018 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced new data for SPK-3006, an investigational liver-directed adeno-associated viral (AAV) gene therapy for Pompe disease. The data from the Investigational New Drug (IND)-enabling studies were presented Saturday, Oct.
Swiss drugmaker Roche is breaking into hemophilia A treatment, a US$10 billion global market dominated by rivals who have cultivated close ties to sufferers of the genetic bleeding disorder.
2018-09-26 biospace - 1
Recently, BioSpace posted a question on its website, under the heading, “We Want to Hear from You! Top Life Sciences Trends.” And don’t stop! We still want to hear from you!
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LUXTURNA wäre die erste Gentherapie für eine genetische Erkrankung, die sowohl in den USA als auch in der EU zugelassen ist
LUXTURNA diventerebbe la prima terapia genica per una malattia genetica approvata sia negli USA sia nella UE LUXTURNA sarebbe la prima e unica terapia genica approvata nell’UE per il trattamento di quei pazienti che presentano un numero sufficiente di cellule retiniche vitali e che hanno perso la vista a causa di distrofia retinica ereditaria associata a mutazioni bialleliche comprovate del gene RPE65, una condizione che porta alla cecità totale nella maggioranza dei pazienti
Novartis AG (NVS - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Gilenya for the treatment of children and adolescents aged 10 to 17 years with relapsing remitting forms of multiple sclerosis (RRMS).
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ZURICH, Sept 21 (Reuters) - A European Medicines Agency panel recommended approval of Spark Therapeutics’ gene therapy for blindness, a move that also boosts Swiss drugmaker Novartis that bought the rights to one of the world’s costliest treatments outside the United States.
Basel, September 21, 2018 - Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the approval of voretigene neparvovec, a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. Luxturna was developed and is marketed in the US by Spark Therapeutics. If approved, voretigene neparvovec will be commercialized by Novartis in markets outside the U.
LUXTURNA would be first gene therapy for a genetic disease approved in both U.S. and EU
2018-09-11 seekingalpha - 2
Cicero learned man that he was, believed in self-improvement so long as his breath lasts. He commends Socrates for learning to play the fiddle late in life and another Roman for mastering Greek when old. - Charlie Munger
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2018-10-01 - Asif
Overview The following discussion and analysis should be read in conjunction with its unaudited interim condensed consolidated financial statements and the related notes that appear elsewhere in this quarterly report on Form 10-Q. This discussion contains forward-looking statements reflecting its current expectations that involve risks and uncertainties. Actual results may differ materially from those discussed in these forward-looking statements due to a number of factors, including those set forth in the section entitled “Risk Factors” in its most recent annual report on Form 10-K. For further information regarding forward-looking statements, please refer to the “Special Note Regarding Forward-Looking Statements and Projections” immediately after the index to this quarterly report on Form 10-Q. Alimera Sciences, Inc., and its subsidiaries (we or Alimera), is a pharmaceutical company that specializes in the commercialization and development of prescription ophthalmic pharma...
2018-09-25 - Asif
Background DelMar Pharmaceuticals, Inc. is a clinical stage drug development company with a focus on the treatment of cancer. The company's mission is to benefit patients and create shareholder value by developing and commercializing anti-cancer therapies for patients whose tumors exhibit features that make them resistant to, or unlikely to respond to, currently available therapies, particularly for orphan cancer indications where patients have failed, or are unlikely to respond to, currently available therapy. DelMar Pharmaceuticals is developing VAL-083, a novel, DNA-targeting agent, for the treatment of glioblastoma multiforme (“GBM”) and potentially other solid tumors, including ovarian cancer. VAL-083 is a first-in-class, DNA-targeting chemotherapeutic that demonstrated activity against a range of tumor types in prior Phase 1 and Phase 2 clinical studies sponsored by the US National Cancer Institute (“NCI”). The company's recent research has highlighted the opportunit...
2018-09-18 - Asif
Overview Rigel Pharmaceuticals is a biotechnology company dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of patients with immune and hematologic disorders, cancer and rare diseases. The company's pioneering research focuses on signaling pathways that are critical to disease mechanisms. The company's first FDA-approved product is TAVALISSE™ (fostamatinib disodium hexahydrate), an oral spleen tyrosine kinase (SYK) inhibitor, for the treatment of adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment. The company's current clinical programs include Phase 2 studies of fostamatinib in autoimmune hemolytic anemia and IgA nephropathy, and a Phase 1 study for its IRAK program. In addition, Rigel Pharmaceuticals has product candidates in development with partners BerGenBio AS, Daiichi Sankyo, and Aclaris Therapeutics. Since inception, Rigel Pharmaceuticals h...
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