Rare disease Archives

News and Trends 26 Jul 2017

Dutch Biotech Reports Positive Phase II Data on Rare Inflammatory Disease

Pharming has published Phase II data in The Lancet supporting its lead candidate as a treatment for the rare genetic disease hereditary angioedema. Pharming, one of the top biotechs in Leiden, specializes in recombinant protein technology. Its lead compound, ruconest, is a recombinant human C1 inhibitor protein. The results of a 32-patient Phase II study published […]

July 26, 2017 - 1 minutemin - By Clara Rodríguez Fernández

Erytech pharma Queens university red blood cells

News and Trends 12 Jul 2017

French Biotech and Canadian University to treat Rare Disease with Red Blood Cells

Erytech Pharma has entered a partnership with Queen’s University to treat the rare disease arginase-1 deficiency using engineered red blood cells. Erytech, based in Lyon, is known for its unique technology for drug delivery. Its most advanced program, in Phase IIb for metastatic pancreatic cancer, employs red blood cells engineered to produce the enzyme L-asparaginase, which depletes […]

July 12, 2017 - 1 minutemin - By Clara Rodríguez Fernández

Minoryx Therapeutics Marc Martinell interview

Interview 10 Jul 2017

Meet the CEO of Minoryx, the Spanish Biotech Fighting Rare Disease

I spoke with Marc Martinell, CEO of Minoryx, to learn first-hand how his company in Barcelona is bringing new hope for patients suffering from rare diseases. Having previously worked in Crystax Pharmaceuticals and Oryzon Genomics, Marc Martinell is now CEO of one of Spain’s most successful biotechs. Minoryx Therapeutics is developing what could be the first […]

July 10, 2017 - 6 minutesmins - By Clara Rodríguez Fernández

Centogene Rare Disease Diagnostics Series A

News and Trends 13 Jun 2017

German Biotech Raises €25M to Fuel Diagnostics of Rare Diseases

Centogene has raised €25M in a Series A round to support the global expansion of its genome analysis platform, which is specialized in rare diseases. The German biotech Centogene is pioneering the development of genetic diagnostic tests to address the challenges of personalized medicine in order to match therapies more effectively to patients. Founded in 2005, the […]

June 13, 2017 - 2 minutesmins - By Melanie De Almeida

Scenic Biotech Genetic Off Switch Cancer Rare Disease

News and Trends 5 Jun 2017

New Dutch Biotech Gets Funding to Find Genetic Off Switches

Scenic Biotech is applying a technology to identify first-in-class therapeutic targets that has secured it €6.5M in Series A. Scenic Biotech, founded in Amsterdam just this year, has raised €6.5M to exploit the potential of a technology to discover new types of disease-related genes with applications in cancer and rare genetic diseases. The company, created in […]

June 5, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 26 May 2017

UPDATE: UK Cannabis Biotech ready for a Blockbuster in Rare Epilepsy

UPDATE (26/05/2017): GW has released more positive results in The New England Journal of Medicine demonstrating that Epidiolex dramatically reduces convulsive seizures in children with Dravet syndrome. The results have primed the drug for its NDA submission to the FDA and led some to forecast blockbuster success. Original Publication 30/03/2016 GW Pharmaceuticals (UK) has announced positive results […]

May 26, 2017 - 4 minutesmins - By Ekaterina Perets

In Depth 24 May 2017

What Happens when the Immune System Attacks Life-Saving Drugs?

Immunogenicity has been largely disregarded despite its huge impact on the performance of biological drugs. What is it and how can we fight it? Immunogenicity can be a huge problem. Affecting especially protein-based drugs, this process consists in the development of anti-drug antibodies (ADAs) against a molecule that, in most cases, is saving the life […]

May 24, 2017 - 4 minutesmins - By Clara Rodríguez Fernández

vivet therapeutics gene therapy fundraising

News and Trends 4 May 2017

French Gene Therapy Biotech Raises a Huge €37.5M in Series A

Vivet Therapeutics has raised a huge €37.5M in Series A to help bring a new gene therapy technology for rare liver diseases to the clinic. Vivet Therapeutics has managed to attract some of the hottest biotech investors in Europe in its Series A. Led by Novartis Venture Fund and Columbus Venture Partners, and also counting […]

May 4, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 20 Apr 2017

Goodbye Glybera! The World’s First Gene Therapy will be Withdrawn

The excitement about the therapy was undercut when it became clear Glybera would be a commercial failure. In October, it will officially be dead. uniQure has announced that it will not go for the renewal of its marketing authorization for Glybera in Europe, which is set to expire in October this year. Since Glybera missed approval in the […]

April 20, 2017 - 3 minutesmins - By Evelyn Warner

More News! 29 Mar 2017

More “FDA Love” for Cannabis Company with Orphan Drug Designation

Last year was a doozy for biotech, but it was spectacular for GW Pharmaceuticals. Its streak continues with more special treatment. GW Pharmaceuticals, which is developing epilepsy treatments from marijuana, is making a name for itself not just as “that company” turning a recreational drug into medical ones; the company was also one of 2016’s surprise successes — its […]

March 29, 2017 - 2 minutesmins - By Evelyn Warner

Lysogene gene therapy Sanfillipo A obs study

News and Trends 29 Mar 2017

French Gene Therapy Biotech Gets Ready for Phase II/III

Lysogene has crunched the numbers on its observational study for Sanfillipo A – now the company can move into the final phase for its Sanfilippo A therapy. Lysogene is a French biotech specializing in gene therapy of rare CNS diseases. Its lead asset is a gene therapy for the treatment of the rare genetic disorder […]

March 29, 2017 - 3 minutesmins - By Melanie De Almeida

News and Trends 28 Mar 2017

Why did Biotech Beat Pharma to Bring the First Gene Therapy to Market?

Pharma would seemingly best biotech in any competition. A few CEOs explain why it left the honor of “The First Gene Therapy” to biotech. On July 20, 2012, the western world’s first gene therapy, Glybera, received marketing authorization from the EMA. The treatment was designed and developed by uniQure as one-off treatment for Lipoprotein Lipase Deficiency (LPLD), a […]

March 28, 2017 - 5 minutesmins - By Evelyn Warner

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Rare disease

Dutch Biotech Reports Positive Phase II Data on Rare Inflammatory Disease

French Biotech and Canadian University to treat Rare Disease with Red Blood Cells

Meet the CEO of Minoryx, the Spanish Biotech Fighting Rare Disease

German Biotech Raises €25M to Fuel Diagnostics of Rare Diseases

New Dutch Biotech Gets Funding to Find Genetic Off Switches

UPDATE: UK Cannabis Biotech ready for a Blockbuster in Rare Epilepsy

What Happens when the Immune System Attacks Life-Saving Drugs?

French Gene Therapy Biotech Raises a Huge €37.5M in Series A

Goodbye Glybera! The World’s First Gene Therapy will be Withdrawn

More “FDA Love” for Cannabis Company with Orphan Drug Designation

French Gene Therapy Biotech Gets Ready for Phase II/III

Why did Biotech Beat Pharma to Bring the First Gene Therapy to Market?

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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