Rare disease Archives

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

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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

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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

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Rare disease

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

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

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

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Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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