Rare disease Archives

News and Trends 17 May 2016

New Italian Drug is FDA approved for trials in Rare Neurological Disease

Newron sees its Investigational New Drug application approved by the FDA. It can now begin clinical trials for Rett Syndrome patients – a rare neurological disorder. Based near Milan (Italy), Newron is a Biopharma developing therapies for diseases of the central nervous system (CNS). Last year it raised over €23M to advance its pipeline, which features candidates […]

May 17, 2016 - 2 minutesmins - By Denise Neves Gameiro

News and Trends 6 May 2016

Stem cell Biotech gets €26M to Start-off Gene Therapy Development for Children

Orchard Therapeutics has just been launched with a €26M investment led by a major investor. It will be developing gene therapies for a range of blood-related disorders in children. Based in London, Orchard Therapeutics may have just been founded, but it’s based on a long scientific track record. Its founding scientists have been pioneering the area […]

May 6, 2016 - 3 minutesmins - By Denise Neves Gameiro

News and Trends 6 May 2016

Interview with Two Key People of the Fight Against Lupus

I was in Paris this week in the building of the Rare Disease Alliance, an association helping patients affected by a rare disease. The association lend their space to Neovacs, a biotech company from Paris, which was organising a gathering of experts of lupus. Lupus is one of the most common rare disease and affects more than 5 million […]

May 6, 2016 - 1 minutemin - By Joachim Eeckhout

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

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

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

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Rare disease

New Italian Drug is FDA approved for trials in Rare Neurological Disease

Stem cell Biotech gets €26M to Start-off Gene Therapy Development for Children

Interview with Two Key People of the Fight Against Lupus

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

Subscribe to our newsletter to get the latest biotech news!

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

Subscribe to our newsletter

Advertise with us

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