Rare disease Archives

pharnext pleodrug charcot marie tooth cmt1a

News and Trends 27 Apr 2016

French Pleodrug for Rare Neurological Disease opens up phase III trial in US

Pharnext is opening their first US study sites for a phase III trial in a rare neuropathy – a milestone in the development of Pharnext’s leading candidate. Based in Paris (France), Pharnext is developing new therapies for neurological conditions, including rare diseases such as Charcot-Marie-Tooth Disease. Pharnext uses network pharmacology to discover synergistic combinations of drugs […]

April 27, 2016 - 2 minutesmins - By Denise Neves Gameiro

saphetor ngs lausanne university hospital endocrine

News and Trends 22 Apr 2016

Swiss Biotech to Investigate rare Endocrine Disorders with NGS

Saphetor is developing genome-scale analysis of next generation sequencing (NGS) results – and will now ‘play’ with real data of Endocrine patients at the Lausanne University Hospital. Based in Lausanne’s EPFL Innovation Park (Switzerland), Saphetor was founded only in 2014 and completed a seed financing round this February, raising around €1M (1.2M CHF). This startup develops proprietary […]

April 22, 2016 - 2 minutesmins - By Denise Neves Gameiro

genzyme sanofi enzyme replacement therapy pompe disease neogaa neo1

News and Trends 8 Mar 2016

Sanofi Genzyme goes to Phase III with Rare Neuromuscular Disease

Sanofi Genzyme (Boston and France) have obtained good phase I/II results with a candidate for the rare Pompe Disease and expects to move to phase III in the second quarter of 2016. Sanofi Genzyme continues to bet on rare diseases and neurodegenerative disorders, now with a therapy for late-onset Pompe Disease. Pompe Disease is a […]

March 8, 2016 - 2 minutesmins - By Denise Neves Gameiro

News and Trends 29 Feb 2016

Our Top 5 Articles on Biotech & Rare Disease

Today is Rare Disease Day 2016. Because it is an important field for Biotech, we went back to review some of our best articles on the topic from the Labiotech archives. Here are some of our best – in addition to an overview of 7 Rare diseases and who in Biotech is working on them. Nº […]

February 29, 2016 - 2 minutesmins - By Denise Neves Gameiro

News and Trends 29 Feb 2016

Exploiting the Power of NGS to Diagnose Rare Diseases

Diploid (Belgium) is using genome sequencing data to diagnose rare diseases – and is now charging only if its algorithms can match the genome to a specific rare disease. Patients of rare diseases often struggle with the lack of therapies options – an empty space where Biotechs seem to thrive. But therapy availability is not the […]

February 29, 2016 - 3 minutesmins - By Denise Neves Gameiro

News and Trends 29 Feb 2016

It’s Rare Disease Day 2016: Here are 7 Indications which Biotech is Fighting

According to the European Organisation for Rare Disease, there are 20–30 million people in Europe alone who are affected by one of an estimated 6,000 rare diseases. These include many rare genetic disorders, such as Duchenne’s muscular dystrophy, but also rare forms of cancer and auto-immune disease. The term rare can, therefore, be misleading as many diseases are entering […]

February 29, 2016 - 6 minutesmins - By Dani Bancroft

News and Trends 26 Jan 2016

2011-2015: What a Fantastic 5 Years for European Biotech

The crisis of 2008 hit the Biotech sector hard, and it wasn’t clear how the Biotech industry would get back on track. But now, end of 2015, we can definitely say the Biotech industry did extremely well and probably better than most of the analysts would have predicted… So what were the major news stories […]

January 26, 2016 - 5 minutesmins - By Philip Hemme

News and Trends 1 Dec 2015

Rare Diseases: Still a ‘Blue Ocean’ for Biotechs?

We all know the success stories linked to Rare Disease such as Genzyme. But I wanted to get a deeper understanding on what is today considered as Rare disease, who is acting the field and the reasons for so many Biotech companies to dive into the field. Last week was the RARE 2015 congress in […]

December 1, 2015 - 6 minutesmins - By Philip Hemme

News and Trends 27 Oct 2015

Meet the French Woman who started a Biotech to Save her Daughter from a Rare Disease

Karen Aiach had no background in science or the biotech industry, yet when her daughter Ornella was diagnosed with the super-rare Sanfilippo syndrome this inspired her to co-found and become the CEO of Lysogene, today a clinical-stage biotech based in Paris, France. Now Ornella is participating in the ongoing clinical trials for a new gene […]

October 27, 2015 - 7 minutesmins - By Dani Bancroft

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

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Rare disease

French Pleodrug for Rare Neurological Disease opens up phase III trial in US

Swiss Biotech to Investigate rare Endocrine Disorders with NGS

Sanofi Genzyme goes to Phase III with Rare Neuromuscular Disease

Our Top 5 Articles on Biotech & Rare Disease

Exploiting the Power of NGS to Diagnose Rare Diseases

It’s Rare Disease Day 2016: Here are 7 Indications which Biotech is Fighting

2011-2015: What a Fantastic 5 Years for European Biotech

Rare Diseases: Still a ‘Blue Ocean’ for Biotechs?

Meet the French Woman who started a Biotech to Save her Daughter from a 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

Subscribe to our newsletter to get the latest biotech news!

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

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

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

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

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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