Rare disease Archives

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 The World’s First Gene Therapy will be Withdrawn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

Interview 6 Mar 2017

How did this Biotech seal a €1B Deal with AstraZeneca? The CEO Explains

We talked to the CEO of Bicycle Therapeutics, Kevin Lee, about how his company’s technology could cure not just cancer but also a raft of other diseases. Bicycle Therapeutics made a splashy debut in 2014 with a €25M fundraising round, only to outdo it with a €1B deal with AstraZeneca last fall. What’s the fuss? […]

March 6, 2017 - 7 minutesmins - By Evelyn Warner

News and Trends 28 Feb 2017

It’s Global Rare Disease Day! What Cures is Biotech Cooking Up?

We’ve compiled a list of some of the latest advances of biotech in the field of rare disease to contribute to Rare Disease Day 2017. The European Organization for Rare Diseases (EURORDIS) is once again coordinating Rare Disease Day, which takes place on the last day of February every year. The aim is to raise awareness of […]

February 28, 2017 - 5 minutesmins - By Clara Rodríguez Fernández

News and Trends 15 Feb 2017

French Biotech gets a Boost in the NASH Race with a Successful IPO

Inventiva has completed an IPO on Euronext Paris raising €48M that will support the advancement of its pipeline, which includes big indications such as NASH. Inventiva, a drug discovery company from France, is the protagonist today of a successful biotech IPO on Euronext Paris despite the financial difficulties of past years. The company has raised €48M, which will […]

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

lysogene ipo euronext paris e1486638993948

News and Trends 9 Feb 2017

Gene Therapy Biotech Founded to Save the CEO’s Daughter Goes Public in France

Lysogene has raised €22.6M from its IPO on Euronext Paris that will contribute to the completion of clinical trials to treat the rare genetic diseases. Karen Aiach, CEO of Lysogene, started the company back in 2009 to develop a life-saving treatment for her daughter Ornella with Sanfilippo A. This rare genetic disease severely affects the nervous system of […]

February 9, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 13 Jan 2017

UPDATE: J&J and Actelion settle on a price, R&D assets will be spun out

UPDATE (12/01/2017): J&J and Actelion have reportedly settled on a price: $260 (€245) per share, totaling more than $28 (€26) billion. The Swiss biotech’s R&D assets will be spun out into a new company that will remain independent. UPDATE (03/01/2017): The deal has veered towards splitting Actelion into two companies, one for its commercialized portfolio […]

January 13, 2017 - 4 minutesmins - By Clara Rodríguez Fernández

News and Trends 5 Jan 2017

Shire sells its RNA platform to a US-based Biotech

Shire has sold its RNA therapy program to a smaller biotech that wants to accelerate the development of products from the platform. The Irish drug giant Shire is giving its MRT program, which includes two mRNA platforms and 12 employees, to US-based RaNA Therapeutics. In exchange, Shire will receive an undisclosed share of equity in the biotech as well […]

January 5, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

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