Rare disease Archives

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

cannabidiol cannabis epilepsy davet syndrome epidiolex gw pharmaceuticals cbd

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

News and Trends 21 Mar 2017

A Potential First Treatment for a Rare Genetic Disease clears Phase I

Minoryx Therapeutics has successfully completed a Phase I trial for its lead compound MIN-102 targeting X-linked adrenoleukodystrophy (X-ALD). The Spanish biotech Minoryx is specialized on orphan diseases and its lead program is a PPAR-gamma agonist, MIN-102, which is in development for X-ALD. The company now announced the successful results of its Phase I trial with MIN-102 revealing the […]

March 21, 2017 - 2 minutesmins - By Melanie De Almeida

Allergan Editas Medicine CRISPR eye disease

News and Trends 15 Mar 2017

Allergan makes a deal with Editas to License CRISPR Programs for Eye Disease

Allergan has signed a deal with Editas Medicine to access its CRISPR technology and license up to five of its programs in ocular disease. The Irish Allergan has a certain tendency to go on shopping sprees. Its latest deal is a research and development alliance with the American Editas Medicine, which is on the side of […]

March 15, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

Knowledge hub

Categories

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

Top biotech deals of February 2025

Approaches

Therapeutic areas

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

These 7 biotechs are shaping the next generation of endometriosis care

R&D

BD

World biotech leaders

Connect with decision-makers and researchers worldwide for collaborative opportunities

Company

Join Us

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

Rare disease

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?

A Potential First Treatment for a Rare Genetic Disease clears Phase I

Allergan makes a deal with Editas to License CRISPR Programs for Eye Disease

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

Subscribe to our newsletter

Get the latest biotech news directly to your inbox!

Newsletter Signup - Footer

Subscribe to our newsletter to get the latest biotech news!