Rare disease Archives

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

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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

Screenshot 2016 12 16 at 4.59.08 PM e1481904006793

News and Trends 19 Dec 2016

How is Biotech responding to Patient Advocacy in Rare Disease?

A panel of experts in rare disease discussed some of the latest trends at Labiotech Refresh, focusing on the growing patient involvement and how it is benefiting this field. Rare diseases are attracting biotech and pharma more and more since they are typically less crowded areas with unmet clinical needs. However, the lower numbers of […]

December 19, 2016 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 7 Dec 2016

UPDATE: More Good News on a Cannabis Derivative for Epilepsy

Update (07/12/2017): GW Pharma has announced more positive Phase III results for Epidiolex, pushing the drug closer to the market and a win for supporters of legalizing marijuana. Originally published on 27/09/2016 GW Pharmaceuticals just released the results of its third successful Phase III clinical trial for Epidiolex (cannabidiol), a drug that can reduce the frequency of […]

December 7, 2016 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 29 Nov 2016

New British Partnership set to Cure Rare Diseases with Gene Therapy

Oxford BioMedica and Orchard Therapeutics are partnering up to bring gene therapy to the market and treat rare diseases for which no cure is available yet. Oxford BioMedica develops lentiviral vectors for gene and cell therapy applications. The company just announced a partnership with another biotech from the UK’s golden triangle, Orchard Therapeutics. The deal is […]

November 29, 2016 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 2 Nov 2016

NHS adopts Next-Gen Sequencing to Test for Birth Defects

Despite a lawsuit from Illumina, Premaitha forged ahead with its NGS-based non-invasive prenatal test; the NHS just adopted it as a standard screen. With its seemingly infinite applications, next-generation sequencing (NGS) is a hot area, especially for diagnostics biotechs. A number have been founded in recent years to develop prenatal tests for birth defects, and […]

November 2, 2016 - 3 minutesmins - By Evelyn Warner

News and Trends 17 May 2016

New Italian Drug is FDA approved for trials in Rare Neurological Disease

Newron sees its Investigational New Drug application approved by the FDA. It can now begin clinical trials for Rett Syndrome patients – a rare neurological disorder. Based near Milan (Italy), Newron is a Biopharma developing therapies for diseases of the central nervous system (CNS). Last year it raised over €23M to advance its pipeline, which features candidates […]

May 17, 2016 - 2 minutesmins - By Denise Neves Gameiro

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News and Trends 6 May 2016

Stem cell Biotech gets €26M to Start-off Gene Therapy Development for Children

Orchard Therapeutics has just been launched with a €26M investment led by a major investor. It will be developing gene therapies for a range of blood-related disorders in children. Based in London, Orchard Therapeutics may have just been founded, but it’s based on a long scientific track record. Its founding scientists have been pioneering the area […]

May 6, 2016 - 3 minutesmins - By Denise Neves Gameiro

News and Trends 6 May 2016

Interview with Two Key People of the Fight Against Lupus

I was in Paris this week in the building of the Rare Disease Alliance, an association helping patients affected by a rare disease. The association lend their space to Neovacs, a biotech company from Paris, which was organising a gathering of experts of lupus. Lupus is one of the most common rare disease and affects more than 5 million […]

May 6, 2016 - 1 minutemin - By Joachim Eeckhout

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Rare disease

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

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

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

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

Shire sells its RNA platform to a US-based Biotech

How is Biotech responding to Patient Advocacy in Rare Disease?

UPDATE: More Good News on a Cannabis Derivative for Epilepsy

New British Partnership set to Cure Rare Diseases with Gene Therapy

NHS adopts Next-Gen Sequencing to Test for Birth Defects

New Italian Drug is FDA approved for trials in Rare Neurological Disease

Stem cell Biotech gets €26M to Start-off Gene Therapy Development for Children

Interview with Two Key People of the Fight Against Lupus

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

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