Rare disease Archives

advicenne distal renal tubular acidosis rare disease

News and Trends 6 Nov 2017

Update: The First Drug for This Rare Kidney Disease May Come From a French Biotech

Update (6/11/2017): ADV7103 has maintained its efficacy in a 6-month extension of the Phase III study. In 79% of the patients, the blood bicarbonate levels were kept within the normal range after 6 months of treatment, further supporting its push to become the first treatment for the disease on the market. Originally published 11/9/2017 Advicenne has […]

November 6, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 27 Sep 2017

French Gene Therapy for Wilson’s Disease gets Orphan Status

Vivet Therapeutics has been granted Orphan Drug Designation for its gene therapy for Wilson’s disease, VTX-801, by both the FDA and the European Commission. The French biotech Vivet Therapeutics is developing what could eventually become the first gene therapy for Wilson’s disease. Seeing its potential, both the FDA and the EC have decided to grant […]

September 27, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

More News! 14 Sep 2017

New Dutch Spin Out will Treat Stroke with RNA Therapy

ProQR has spun out Amylon Therapeutics, a company that will treat central nervous system disorders with a lead candidate based on RNA technology. Amylon Therapeutics, a brand new spin-out from RNA company ProQR, has announced its first seed investment round. The funds, of an undisclosed amount, will help the new company kick off the development of its drug candidates. Based in Leiden, Amylon […]

September 14, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

Orchard Therapeutics Gene Therapy ADA SCID FDA

News and Trends 23 Aug 2017

Update: British Biotech’s Gene Therapy for Rare ‘Bubble Baby’ Disease set on UK Fast-Track

UPDATE (23/08/2017): The UK has also prioritized OTL-101 for review as a ‘Promising Innovative Medicine.’ This is the first step on the path to the Early Access to Medicine Scheme to bring it to market faster. Original Publication 24/07/2017 Orchard Therapeutics has received rare pediatric disease designation for OTL-101, a gene therapy for the treatment of […]

August 23, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

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

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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

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