Rare disease Archives

News and Trends 2 Nov 2018

Gut Bacteria Treatment Shows Promise for Rare Genetic Kidney Condition

The Swedish biotech Oxthera reported positive preliminary Phase II results using its gut bacteria treatment, Oxabact, in patients with a rare genetic condition that causes kidney failure. The results came from an interim analysis one year into a three-year trial, where Oxthera is testing its gut microbiome treatment on patients with primary hyperoxaluria type […]

November 2, 2018 - 2 minutesmins - By Jonathan Smith

More News! 16 Oct 2018

Drug Combination for Rare Genetic Nerve Disorder Nails Phase III

A treatment developed by French biotech Pharnext has become the first clinically proven therapy for a rare genetic disorder that affects the nervous system. In a Phase III trial, the treatment reduced the level of disability of patients with Charcot-Marie-Tooth disease, a condition that affects peripheral nerves. This disease causes problems with walking, running and […]

October 16, 2018 - 2 minutesmins - By Jonathan Smith

News and Trends 9 Oct 2018

Hormone Therapy for Inherited Hormonal Disorder Fails at Phase III

A European Phase III trial has become a major stress for British biotech Diurnal. Its specially designed hormone therapy was no better than conventional therapies in treating an inherited hormonal disorder. Congenital adrenal hyperplasia is a rare genetic condition where patients lack crucial enzymes for making hormones. The most common form lacks 21-hydroxylase, the enzyme […]

October 9, 2018 - 3 minutesmins - By Jonathan Smith

In Depth 9 Oct 2018

How to Capitalize on Opportunities in Underserved Disease Areas

Developing treatments for underserved diseases can be surprisingly profitable for biotechs, we asked the experts to tell us more about how best to exploit these areas. We hear a lot about cancer therapies or drugs to target heart disease in the press, but overall a lot less about treatments for more underserved areas. Often successful […]

October 9, 2018 - 9 minutesmins - By Helen Albert

News and Trends 26 Sep 2018

Spanish Biotech Raises €21.3M To Advance Rare Disease Pipeline

Rare genetic diseases in the central nervous system may be less of a headache in future, as Barcelona-based biotech Minoryx has raised €21.3M in series B fundraising to test its lead drug candidate against other rare neurological conditions. According to Philippe Monteyne, partner at investor Fund+, “it is the largest investment in biotech in Spain […]

September 26, 2018 - 2 minutesmins - By Jonathan Smith

News and Trends 3 Sep 2018

Oxford Biotech Raises €40M to Deliver Drugs to Hard-to-Reach Targets

Evox Therapeutics has raised £35.5M (€39.4M) in a Series B fundraising that has been backed, among others, by the venture arm of Google’s parent company. A spin-out from the University of Oxford and the Karolinska Institute, Evox Therapeutics has raised a large round that will fund the development of its drug delivery technology to […]

September 3, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 26 Jul 2018

AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever

Cambridge-based Healx has raised $10M (€8.6M) in a Series A round to scale up its AI technology, which could significantly reduce the time and cost of developing drugs for rare diseases. Backed, among other investors, by the founder of Abcam, Healx plans to use the fundraising to double its workforce from 15 to over 30 […]

July 26, 2018 - 4 minutesmins - By Clara Rodríguez Fernández

Interview 12 Mar 2018

A Dutch Company on the Quest Against Cystic Fibrosis

ProQR is the result of a personal quest against cystic fibrosis started by its CEO, Daniel de Boer. Interested in his story and the company’s unique approach to treating the disease using RNA technology, I decided to speak with him. Daniel de Boer founded ProQR in 2012 following a strong determination to improve the lives […]

March 12, 2018 - 6 minutesmins - By Clara Rodríguez Fernández

News and Trends 28 Feb 2018

Like Finding a Needle in a Haystack: How Are Biotechs Fighting Rare Diseases?

Rare diseases affect fewer than 1 in 2,000 people in Europe and less than 200,000 Americans at any given time. A lack of financial incentive has put pharma off but biotechs are keen to step in and develop treatments and cures. Over 6,000 rare diseases affect up to 30 million people in the EU alone, which highlights […]

February 28, 2018 - 8 minutesmins - By Alex Dale

News and Trends 20 Feb 2018

Microsoft Joins Shire to Diagnose Rare Diseases Much Faster

Microsoft has partnered with Shire and EURORDIS to accelerate the diagnosis of rare disease, a process that often takes years and takes a big toll on the health of millions. There are over 6,000 different rare diseases, most of them beginning in childhood. However, diagnosing them takes an average of 5 years, delaying the start […]

February 20, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

metabolic disorders chaperones gain thx 1

News and Trends 20 Feb 2018

A New Biotech Hopes to Bring Us the Next Generation of Rare Metabolic Disorder Drugs

Barcelona-based biotech, Minoryx, has spun out Gain Therapeutics to develop the next generation of drugs for rare metabolic disorders like GM1 gangliosidosis. Gain Therapeutics is a new biotech that will focus on developing new pharmacological chaperones for the treatment of rare diseases. It was set up by Minoryx, a biotech that has so far raised over €24M for […]

February 20, 2018 - 3 minutesmins - By Alex Dale

News and Trends 20 Dec 2017

British Gene Therapy Gets a Huge £85M Boost in Series B

Orchard Therapeutics has raised a big round of financing to gene therapy options for many rare diseases for which there are no treatments. London-based Orchard Therapeutics has managed to raise £85M (€96M) in Series B, an amount that will certainly give an important boost to its pipeline of gene therapy treatments for rare diseases. The […]

December 20, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

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Rare disease

Gut Bacteria Treatment Shows Promise for Rare Genetic Kidney Condition

Drug Combination for Rare Genetic Nerve Disorder Nails Phase III

Hormone Therapy for Inherited Hormonal Disorder Fails at Phase III

How to Capitalize on Opportunities in Underserved Disease Areas

Spanish Biotech Raises €21.3M To Advance Rare Disease Pipeline

Oxford Biotech Raises €40M to Deliver Drugs to Hard-to-Reach Targets

AI Company Raises €8.6M to Find Drugs for Rare Diseases Faster and Cheaper Than Ever

A Dutch Company on the Quest Against Cystic Fibrosis

Like Finding a Needle in a Haystack: How Are Biotechs Fighting Rare Diseases?

Microsoft Joins Shire to Diagnose Rare Diseases Much Faster

A New Biotech Hopes to Bring Us the Next Generation of Rare Metabolic Disorder Drugs

British Gene Therapy Gets a Huge £85M Boost in Series B

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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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