BioMarin announces FDA accepts drisapersen NDA for treatment of Duchenne Muscular Dystrophy amenable to exon 51 skippingPublished : Monday, June 29, 2015
BioMarin Pharmaceutical Inc. announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee Act (PDUFA) goal date for a decision is December 27, 2015. The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.
BioMarin announces EMA validates MAA for drisapersen for treatment of Duchenne Muscular Dystrophy amenable to exon 51 skippingPublished : Thursday, June 25, 2015
BioMarin Pharmaceutical Inc. announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA's standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016.
With the Leiden Bio Science Park being home to a vibrant startup community several organizations choose to host their finance information & match making events here in Leiden and on our park. They come with seed capital, soft money, crowd funding, business angels, informal investors, venture capital, … and they are looking for starters and young companies, ready to scale up! You can pitch your business case, you can seek advice, you can strengthen your network, you can catch up on the latest financing news. Come and have a look for yourself!
Leiden University and Leiden University Medical Center (LUMC) are seeking commercial partners for collaborative development or licensing on a number of innovative technologies. Luris is the Knowledge Exchange Office for both organisations. Luris represents the interests of academics while at the same time helping societal partners, industry and users of Leiden research and technology to become more successful and enhance Leiden’s impact on the world.
The 2015 edition of the Night of Art and Science will take place on September 19th and revolves around art and science from Leiden. This year Leiden Bio Science Park will organize the exposition MICROmania! showcasing fluorescence microscopy images of beautiful stained cells originating from research at companies and research institutes at Leiden Bio Science Park.
ISA Pharmaceuticals B.V., a clinical-stage immunotherapy company focusing on rationally designed immunotherapeutics against cancer and persistent viral infections, announced it has strengthened the IP position of its lead compound ISA101. The European Patent Office has granted three patents (EP2267132, EP2112157, EP2468763) which relate to the use of HPV antigen-based peptides. ISA101, a novel immunotherapeutic consisting of synthetic long peptides (SLP®s), targets severe diseases triggered by human papillomavirus (HPV) infection.
Galapagos completes recruitment for ORIGIN Phase 2 trial with GLPG1205 in ulcerative colitis patientsPublished : Thursday, June 18, 2015
Galapagos NV announced it has completed recruitment for the Phase 2 Proof-of-Concept study in ulcerative colitis with GPR84 inhibitor GLPG1205, a potential novel treatment for inflammatory bowel diseases. Topline results from this study are now expected in Q1 2016. GPR84 was identified as a target for these diseases through Galapagos' target discovery platform and GLPG1205 is fully proprietary to Galapagos.
Galapagos NV, a clinical stage biotechnology company focused on developing novel mode of action medicines, announces that the last RA patients in its DARWIN 1 and 2 dose finding studies with filgotinib have completed their final visit. This triggers the clinical research organization's process of last 24 week data collection from both studies, to be followed by final database lock and analysis. Galapagos expects to announce topline results in late July (DARWIN 1) and in August (DARWIN 2) 2015.
CTMM and TI Pharma, the two largest public-private top institutes in the Dutch top sector Life Sciences & Health, presented their plans and ambition for their joint future during the CTMM + TI Pharma Launch Meeting at the Muntgebouw in Utrecht. CTMM (Center for Translational Molecular Medicine) and TI Pharma (Top Institute Pharma) also announced that the new merged organization will from now on be known as Lygature. Lygature will focus on partnerships for medical innovation.
BioMarin submits Drisapersen MAA to EMA for the treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingPublished : Monday, June 8, 2015
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed globally each year.
Toxys heeft de BioBusiness Masterclass Award 2015 in de wacht gesleept tijdens het HollandBIO Dutch Life Sciences SME Event. Het bedrijf, een spin-off van het Leids Universitair Medisch Centrum (LUMC), ontwikkelde de ToxTracker test. Deze in vitro assay van Toxys is gebaseerd op embryonale stamcellen en maakt het mogelijk om verschillende kankerverwekkende effecten van stoffen op een eenvoudige en betrouwbare manier te identificeren.
ttopstart and HollandBIO have launched the Life Sciences Funding Database at the Dutch Life Sciences SME Event in Rijswijk. Access to funding is a crucial factor for innovation in life sciences. The Life Sciences Funding Database shows entrepreneurs and academia the ropes in identifying funding opportunities and includes all relevant national and European funding instruments: subsidies, grants and attractive fiscal arrangements.
Pharming Group NV announced that it has entered into an exclusive distribution agreement with Cytobioteck S.A.S. (“Cytobioteck”), a privately owned Bogota, Colombia based specialty healthcare company, for the distribution of RUCONEST® (recombinant human C1 inhibitor) for the treatment of acute attacks of Hereditary Angioedema (HAE) in Colombia and Venezuela.
Organ-on-chip technology is used in drug research and customized treatments for patients. Research Institutes and industry join forces within the Institute for human Organ and Disease Model Technologies (hDMT) that was officially launched on May 18. Combining facilities, people and expertise will allow the Netherlands to become a global player in this technology. Leiden is very active in novel disease models, innovative drug development and screening solutions and thus providing for alternative to animal tests. In the new institute Leiden University/LACDR, LUMC, and Galapagos take part. Young Leiden based companies like Mimetas, Ocello, Toxys and Pluriomics are also active in organ-on-a-chip services and in vitro essays.
Pharming Group N.V. and Clinigen Group plc have entered into an international global access collaboration for HAEi, the International Patient Organization for C1- Inhibitor Deficiencies. The “HAEi GAP” program will provide access to RUCONEST® (conestat alfa) to eligible patients with hereditary angioedema (HAE), who currently do not have access to effective medication to treat acute attacks of the disease.
New Peer-Reviewed Publication Provides Further Insights into Mechanism of ISA Pharmaceuticals’ SLP® ImmunotherapeuticsPublished : Tuesday, May 12, 2015
ISA Pharmaceuticals B.V., a clinical-stage immunotherapy company focusing on rationally designed immunotherapeutics against cancer and persistent viral infections, announced the publication of a new peer-reviewed paper* demonstrating a beneficial effect of Synthetic Long Peptide (SLP®s) immunotherapeutics on intratumoral macrophages in cancers induced by human papilloma virus 16 (HPV16).
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