Rare disease Archives

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

granulomatous disease orchard therapeutics

News and Trends 18 Dec 2017

British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease

Orchard Therapeutics has licensed a gene therapy for the rare genetic disorder X-linked chronic granulomatous disease (X-CGD) from the non-profit Généthon. London-based Orchard Therapeutics is adding a new program to its pipeline of gene therapies. The company has obtained an exclusive license to a lentiviral vector to treat X-linked chronic granulomatous disease and the clinical trial data […]

December 18, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 14 Dec 2017

The FDA Fast Tracks Therapy for Rare Disease Causing Copper Accumulation

The FDA is speeding up the process for regulatory approval of WTX101, a therapy for Wilson’s disease that could significantly improve the standard of care. Wilson Therapeutics, in Stockholm, has convinced the FDA to include its drug WTX101 (bis-choline tetrathiomolybdate) in its Fast Track program. This move will speed up the approval of the drug, […]

December 14, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

Interview 11 Dec 2017

Edwin Moses on how Ablynx has made it to the top of European biotech

Ablynx CEO Edwin Moses discusses the technology and strategy that has led its company to join the ranks of the biggest European biotechs. Ablynx, based in Belgium, has just recently joined the European billion-euro biotech club with a market cap of €1.45Bn. With positive Phase III results for its lead compound and a $230M (€195M) […]

December 11, 2017 - 8 minutesmins - By Clara Rodríguez Fernández

wilsons disease treatment wilson therapeutics sweden 1

News and Trends 8 Dec 2017

Wilson’s Disease Treatment Gets a €24.4M Equity Boost in Sweden

Wilson Therapeutics has issued new shares on Nasdaq Stockholm, raising a total of SEK 244M (€24.4M) to help bring its Wilson’s disease treatment to the market. Stockholm-based biotech Wilson Therapeutics has closed a share issue that seems to have generated quite a high demand. The company’s main shareholders, Abingworth and MVM, had to sell SEK 156.75M […]

December 8, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

advicenne ipo rare disease children drta

News and Trends 6 Dec 2017

Update: French Biotech Closes €27M IPO to be the First to Treat Rare Disease in Children

Update (6/12/2017): Advicenne has raised a total of €27M on Euronext Paris, putting its market cap at €112M. Originally published on 21/11/2017 Advicenne is launching an IPO on Euronext Paris to raise the funds to bring the first treatment for dRTA, a rare disease of the kidneys, to the market. Advicenne, based in Nîmes, France, has officially […]

December 6, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

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Rare disease

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

British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease

The FDA Fast Tracks Therapy for Rare Disease Causing Copper Accumulation

Edwin Moses on how Ablynx has made it to the top of European biotech

Wilson’s Disease Treatment Gets a €24.4M Equity Boost in Sweden

Update: French Biotech Closes €27M IPO to be the First to Treat Rare Disease in Children

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10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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