Rare disease Archives

News and Trends 15 Oct 2015

Spanish connection: VC leads Biotech series A Fund to cure Rare Neurodegenerative Disease

Minoryx Therapeutics is a Barcelona based Rare-disease specialist who has a panel of orphan-drug experts for their discovery program. Now backed in a €19.4M Series A round from leading VC firms Ysios (No 15 in our top EU VCs) and Roche Venture Fund, Minoryx will have the well-needed cash to push for further R&D of their […]

October 15, 2015 - 2 minutesmins - By Dani Bancroft

News and Trends 2 Oct 2015

Merck relinquishes Rare Disease license to instead focus on Immuno-oncology

Merck has opted to return its Phenylketonuria (PKU) candidates Kuvan and Peg-Pal back to BioMarin from January 2016. Merck will instead focus on other R&D areas, and will receive €340M in compensation (5 times the annual sales of Kuvan) with €180M for future Peg-Pal milestones. This appears to support the back-peddling trend of major Biotechs […]

October 2, 2015 - 2 minutesmins - By Dani Bancroft

News and Trends 1 Oct 2015

Neil Woodford’s Latest €25M UK Biotech pay-out is to AMO Pharma

As we already well know, Neil Woodford is an Economist investor listed as one of the most influential figures in the European biotech industry. From London (UK), his private equity firm Woodford Patient Capital Trust is now planning to dish out €25M to AMO Pharma, to further their phase II pipeline therapies for CNS disorders such […]

October 1, 2015 - 2 minutesmins - By Dani Bancroft

News and Trends 30 Sep 2015

Apitope relocates to Wales after €12M series B financing round

Now, as we’ve recently discussed here at Labiotech, there doesn’t appear to be many Welsh biotechs…however, this is all about to change, with major Autoimmune disease focused Apitope relocating it’s HQ to Wales. This is following a €12M series B fundraiser led by the Wales Lifesciences Fund for its monoclonal antibody pipeline. Aptiope are currently […]

September 30, 2015 - 2 minutesmins - By Dani Bancroft

News and Trends 29 Sep 2015

Could an Alpaca’s monoclonal antibodies be used to treat an ultra-rare blood disease?

Ablynx‘s Nanobody platform has used the smaller than human monoclonal antibodies from an alpaca to develop the phase III Orphan designated drug Caplacizumab. A phase III study ‘Hercules’ is now pioneering Caplacizumab as the first ever anti-Von Willebrand factor (vWF) nanobody for treating an ultra-rare blood clotting disorder. Caplacizumab is a pioneering monoclonal antibody (mAb) […]

September 29, 2015 - 3 minutesmins - By Dani Bancroft

News and Trends 22 Sep 2015

UniQure’s cure for rare Lysosomal disease ready for phase III trial

UniQure from the Netherlands has promising results for its enzyme replacement therapy phase Ib/II trial targeting a rare Lysosomal disease. This has granted fast-track approval to launch its phase III trial for children with Sanfilippo Type B (MPS-III) in collaboration with France’s Institut Pasteur. UniQure is the Amsterdam gene-therapy biotech with a lot going on. Last year […]

September 22, 2015 - 3 minutesmins - By Dani Bancroft

News and Trends 2 Sep 2015

Alexion launches a duo of new therapies for ultra-rare diseases in the EU

Thanks to Alexion Pharmaceuticals, two ultra-rare diseases finally got the first treatment in the European Union, namely Lysosomal acid lipase deficiency (LAL-D) and Pediatric-onset hypophosphatasia. Alexion ramps up its product-pipeline. Until now, the Connecticut-based company, which comprises a market cap of about $38.5Bn (€34,2Bn), has lived mostly on sales of Soliris, the most expensive drug on […]

September 2, 2015 - 2 minutesmins - By Philip Hemme

News and Trends 14 Jul 2015

Newron’s ressurection story continues with a FDA Orphan Drug designation

New success for Newron Pharmaceuticals. The FDA approved Orphan Drug designation to its new chemical entity Sarizotan, designed to treat patients suffering from Rett syndrome. This decision fits the one from the European Medicine Agency, whose Committee for Orphan Medical Products positively reviewed the drug last month. The Italian pharma company Newron with a market […]

July 14, 2015 - 2 minutesmins - By Philip Hemme

News and Trends 1 Jul 2015

Two of Alexion’s rare-disease drugs get the thumbs up from the EU

The European Medicines Agency (EMA) is about to approve a couple of rare diseases drugs developed by Alexion. The two enzymes, Strensiq and Kanuma, received positive feedback from the EMA’s Committee for Medicinal Products for Human Use, which normally leads to the approval of the drug. The Committee for Medicinal Products for Human Use has […]

July 1, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 12 May 2015

Alexion will invest €450 million to expand to Dublin

Alexion Pharmaceuticals will travel across the ocean to build its first ever biologics manufacturing facility outside the United States. This €450 million, four-year project, will be constructed at the Alexion’s College Park site in Blanchardstown (Ireland) and should create approximately 200 additional full-time jobs. This will bring Alexion’s total workforce in Ireland to almost 500 employees. […]

May 12, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 16 Apr 2015

Wilson Therapeutics on its way to cure Wilson disease

Wilson Therapeutics has closed a €37.4M Series B financing to advance its late-stage development program for Wilson’s disease. The company will present its ongoing Phase II trial of WTX101-201, the company’s treatment for this disease, at the upcoming 50th International Liver Congress 2015. Approximately 1 in 15000 people worldwide have Wilson Disease, a rare genetic disorder that prevents the […]

April 16, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 23 Mar 2015

AOP Orphan and Confluence Pharmaceuticals, together for Fragile X Syndrome

AOP Orphan Pharmaceuticals and Confluence Pharmaceuticals, signed today a definitive agreement to co-develop and market Confluence’s lead asset, a product for the treatment of Fragile X Syndrome in Europe and the Middle East. Confluence is an American biopharmaceutical company focused on developing therapeutic treatments for Fragile X Syndrome and Autism Spectrum Disorders. The company has […]

March 23, 2015 - 2 minutesmins - By Lourdes Riquelme

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Rare disease

Spanish connection: VC leads Biotech series A Fund to cure Rare Neurodegenerative Disease

Merck relinquishes Rare Disease license to instead focus on Immuno-oncology

Neil Woodford’s Latest €25M UK Biotech pay-out is to AMO Pharma

Apitope relocates to Wales after €12M series B financing round

Could an Alpaca’s monoclonal antibodies be used to treat an ultra-rare blood disease?

UniQure’s cure for rare Lysosomal disease ready for phase III trial

Alexion launches a duo of new therapies for ultra-rare diseases in the EU

Newron’s ressurection story continues with a FDA Orphan Drug designation

Two of Alexion’s rare-disease drugs get the thumbs up from the EU

Alexion will invest €450 million to expand to Dublin

Wilson Therapeutics on its way to cure Wilson disease

AOP Orphan and Confluence Pharmaceuticals, together for Fragile X Syndrome

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The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Advertise with us

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