News and Trends 8 Dec 2017 Wilson’s Disease Treatment Gets a €24.4M Equity Boost in Sweden Wilson Therapeutics has issued new shares on Nasdaq Stockholm, raising a total of SEK 244M (€24.4M) to help bring its Wilson’s disease treatment to the market. Stockholm-based biotech Wilson Therapeutics has closed a share issue that seems to have generated quite a high demand. The company’s main shareholders, Abingworth and MVM, had to sell SEK 156.75M […] December 8, 2017 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 6 Dec 2017 Update: French Biotech Closes €27M IPO to be the First to Treat Rare Disease in Children Update (6/12/2017): Advicenne has raised a total of €27M on Euronext Paris, putting its market cap at €112M. Originally published on 21/11/2017 Advicenne is launching an IPO on Euronext Paris to raise the funds to bring the first treatment for dRTA, a rare disease of the kidneys, to the market. Advicenne, based in Nîmes, France, has officially […] December 6, 2017 - 3 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 30 Nov 2017 RNA Therapy for Rare Skin Disease Gets Orphan Status The EMA has granted orphan drug designation to ProQR’s QR-313, a possible first disease-modifying treatment for the rare disease dystrophic epidermolysis bullosa. Dystrophic epidermolysis bullosa (DEB) is a genetic disease that causes the skin to easily blister, severely affecting the quality of life of patients since a very early age. Current treatment is limited to […] November 30, 2017 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 6 Nov 2017 Update: The First Drug for This Rare Kidney Disease May Come From a French Biotech Update (6/11/2017): ADV7103 has maintained its efficacy in a 6-month extension of the Phase III study. In 79% of the patients, the blood bicarbonate levels were kept within the normal range after 6 months of treatment, further supporting its push to become the first treatment for the disease on the market. Originally published 11/9/2017 Advicenne has […] November 6, 2017 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 27 Sep 2017 French Gene Therapy for Wilson’s Disease gets Orphan Status Vivet Therapeutics has been granted Orphan Drug Designation for its gene therapy for Wilson’s disease, VTX-801, by both the FDA and the European Commission. The French biotech Vivet Therapeutics is developing what could eventually become the first gene therapy for Wilson’s disease. Seeing its potential, both the FDA and the EC have decided to grant […] September 27, 2017 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
More News! 14 Sep 2017 New Dutch Spin Out will Treat Stroke with RNA Therapy ProQR has spun out Amylon Therapeutics, a company that will treat central nervous system disorders with a lead candidate based on RNA technology. Amylon Therapeutics, a brand new spin-out from RNA company ProQR, has announced its first seed investment round. The funds, of an undisclosed amount, will help the new company kick off the development of its drug candidates. Based in Leiden, Amylon […] September 14, 2017 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 23 Aug 2017 Update: British Biotech’s Gene Therapy for Rare ‘Bubble Baby’ Disease set on UK Fast-Track UPDATE (23/08/2017): The UK has also prioritized OTL-101 for review as a ‘Promising Innovative Medicine.’ This is the first step on the path to the Early Access to Medicine Scheme to bring it to market faster. Original Publication 24/07/2017 Orchard Therapeutics has received rare pediatric disease designation for OTL-101, a gene therapy for the treatment of […] August 23, 2017 - 3 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 26 Jul 2017 Dutch Biotech Reports Positive Phase II Data on Rare Inflammatory Disease Pharming has published Phase II data in The Lancet supporting its lead candidate as a treatment for the rare genetic disease hereditary angioedema. Pharming, one of the top biotechs in Leiden, specializes in recombinant protein technology. Its lead compound, ruconest, is a recombinant human C1 inhibitor protein. The results of a 32-patient Phase II study published […] July 26, 2017 - 1 minutemin - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 12 Jul 2017 French Biotech and Canadian University to treat Rare Disease with Red Blood Cells Erytech Pharma has entered a partnership with Queen’s University to treat the rare disease arginase-1 deficiency using engineered red blood cells. Erytech, based in Lyon, is known for its unique technology for drug delivery. Its most advanced program, in Phase IIb for metastatic pancreatic cancer, employs red blood cells engineered to produce the enzyme L-asparaginase, which depletes […] July 12, 2017 - 1 minutemin - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
Interview 10 Jul 2017 Meet the CEO of Minoryx, the Spanish Biotech Fighting Rare Disease I spoke with Marc Martinell, CEO of Minoryx, to learn first-hand how his company in Barcelona is bringing new hope for patients suffering from rare diseases. Having previously worked in Crystax Pharmaceuticals and Oryzon Genomics, Marc Martinell is now CEO of one of Spain’s most successful biotechs. Minoryx Therapeutics is developing what could be the first […] July 10, 2017 - 6 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 13 Jun 2017 German Biotech Raises €25M to Fuel Diagnostics of Rare Diseases Centogene has raised €25M in a Series A round to support the global expansion of its genome analysis platform, which is specialized in rare diseases. The German biotech Centogene is pioneering the development of genetic diagnostic tests to address the challenges of personalized medicine in order to match therapies more effectively to patients. Founded in 2005, the […] June 13, 2017 - 2 minutesmins - By Melanie De Almeida Share WhatsApp Twitter Linkedin Email
News and Trends 5 Jun 2017 New Dutch Biotech Gets Funding to Find Genetic Off Switches Scenic Biotech is applying a technology to identify first-in-class therapeutic targets that has secured it €6.5M in Series A. Scenic Biotech, founded in Amsterdam just this year, has raised €6.5M to exploit the potential of a technology to discover new types of disease-related genes with applications in cancer and rare genetic diseases. The company, created in […] June 5, 2017 - 3 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email