BioMarin launches kNOWyourDuchenne, comprehensive program for patients with Duchenne Muscular Dystrophy to understand their genetic mutationPublished : Tuesday, September 1, 2015
BioMarin Pharmaceutical Inc. announced the introduction of kNOWyourDuchenne, a program to help families and physicians obtain and interpret genetic testing for patients with Duchenne muscular dystrophy. This novel program will identify specific genetic mutations leading to Duchenne, which may help guide clinicians and caregivers to potential treatments in development. Interpretation of the test will be performed by an independent lab director.
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 represent all Leiden scientists and connects them with societal and industrial partners with one aim: to impact society with Leiden research and technologies.
From the Horizon 2020 program the European Union made 15 million euros available for a pharmacogenetics LUMC study: medication tailored to the DNA of the patient. With this grant an infrastructure will be rolled out in seven countries that enables patients to receive medication that takes their DNA variations into account. As a result, medication can be used more effectively, with fewer side effects.
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BioMarin receives rare pediatric disease designation from FDA for drisapersen for the potential treatment of Duchenne Muscular DystrophyPublished : Wednesday, August 19, 2015
BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for drisapersen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment. The FDA has previously granted drisapersen Orphan and Fast Track Status, Breakthrough Therapy Designation, as well as Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.
DARWIN 2 24-week monotherapy data in RA confirm previous results and support best-in-class potential for filgotinibPublished : Monday, August 10, 2015
Galapagos NV announced that the selective JAK1 inhibitor filgotinib as once-daily monotherapy at week 24 showed further improvements in signs and symptoms of moderately to severe, active rheumatoid arthritis (RA) in the DARWIN 2 Phase 2B study. Filgotinib was well tolerated in this study. Hemoglobin levels increased. These final 24-week study results are consistent with the efficacy and safety profile of filgotinib observed in prior clinical studies. With these final results, the data package for AbbVie is complete, which triggers the start of the licensing decision period.
Galapagos NV announced that the last patient has been randomized in the FITZROY Phase 2 clinical study. The study evaluates the efficacy and safety of filgotinib, a selective JAK 1 inhibitor, during 20 weeks of treatment in 175 patients with Crohn's disease. Galapagos is eligible to receive $50 M fee from AbbVie if AbbVie elects to in-license filgotinib after receipt of the full RA DARWIN 1 and 2 data and elects to move forward with filgotinib in Crohn's disease.
The Netherlands Embassy in Japan announced that the Representative Director & Chairman of Astellas Pharma Inc., Mr. Masafumi NOGIMORI, has been appointed with the Royal Netherlands decoration of ‘Officer in the Order of Oranje-Nassau’. The honor is in recognition of Mr. Nogimori’s and Astellas’ contributions to the relationship between the Netherlands and Japan.
Galapagos' selective JAK1 inhibitor filgotinib meets key efficacy endpoints, shows ACR70 responses up to 39%, and maintains safety profile after 24 weeks of treatment in DARWIN 1 Phase 2B studyPublished : Wednesday, July 29, 2015
Galapagos NV announced that at week 24, patients treated with the selective JAK1 inhibitor filgotinib showed further improvement in signs and symptoms of rheumatoid arthritis activity, as demonstrated by improved ACR responses, DAS28(CRP), and other scores, compared to week 12 in the DARWIN 1 Phase 2B methotrexate add-on study.
On Thursday the 2nd of July, Rabobank Bollenstreek presented a check to Generade: The Centre of Expertise Genomics at the TOPlab in Leiden. Starting September, Generade will conduct research on how to prevent the development of fungus on tulip bulbs. The Innovation fund of Rabobank Bollenstreek donates 100,000 euros to help this research blossom in the near future. This contribution is an important step in the battle against the fungus and the damage it causes to the tulip bulb sector each year.
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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.
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