Rare disease Archives

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

AOP Orphan Pharmaceuticals Confluence Pharmaceuticals Fragile X

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

News and Trends 5 Mar 2015

Baxter’s Bioscience Business Acquires SuppreMol

Baxter has acquired SuppreMol for €200M before working capital and other adjustments. The acquisition includes SuppreMol’s early-stage development portfolio of novel biologic immunoregulatory therapeutics for the treatment of autoimmune diseases. SuppreMol is a privately held biopharmaceutical company based in Martinsried, Germany developing treatment options for autoimmune and allergic diseases, focusing on the modulation of Fc […]

March 5, 2015 - 2 minutesmins - By Lourdes Riquelme

News and Trends 18 Feb 2015

Highway to Brain

Researchers in Barcelona have developed a shuttle to transport drugs into the brain, running through the blood-brain barrier. If there is an irreplaceable organ, for the moment, that’s the brain. The gray matter is not only protected by the skull, but also by a barrier of cells that tightly regulates the transport of substances into this […]

February 18, 2015 - 2 minutesmins - By Lourdes Riquelme

Resources

Categories

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Approaches

Therapeutic areas

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

R&D

BD

World biotech leaders

Connect with decision-makers and researchers worldwide for collaborative opportunities

Company

Join Us

Subscribe to our newsletter

Advertise with us

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Rare disease

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

Baxter’s Bioscience Business Acquires SuppreMol

Highway to Brain

Subscribe to our newsletter to get the latest biotech news!

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Subscribe to our newsletter

Get the latest biotech news directly to your inbox!

Newsletter Signup - Footer

Subscribe to our newsletter to get the latest biotech news!