Rare disease Archives

metabolic disorders chaperones gain thx 1

News and Trends 20 Feb 2018

A New Biotech Hopes to Bring Us the Next Generation of Rare Metabolic Disorder Drugs

Barcelona-based biotech, Minoryx, has spun out Gain Therapeutics to develop the next generation of drugs for rare metabolic disorders like GM1 gangliosidosis. Gain Therapeutics is a new biotech that will focus on developing new pharmacological chaperones for the treatment of rare diseases. It was set up by Minoryx, a biotech that has so far raised over €24M for […]

February 20, 2018 - 3 minutesmins - By Alex Dale

News and Trends 20 Dec 2017

British Gene Therapy Gets a Huge £85M Boost in Series B

Orchard Therapeutics has raised a big round of financing to gene therapy options for many rare diseases for which there are no treatments. London-based Orchard Therapeutics has managed to raise £85M (€96M) in Series B, an amount that will certainly give an important boost to its pipeline of gene therapy treatments for rare diseases. The […]

December 20, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

granulomatous disease orchard therapeutics

News and Trends 18 Dec 2017

British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease

Orchard Therapeutics has licensed a gene therapy for the rare genetic disorder X-linked chronic granulomatous disease (X-CGD) from the non-profit Généthon. London-based Orchard Therapeutics is adding a new program to its pipeline of gene therapies. The company has obtained an exclusive license to a lentiviral vector to treat X-linked chronic granulomatous disease and the clinical trial data […]

December 18, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 14 Dec 2017

The FDA Fast Tracks Therapy for Rare Disease Causing Copper Accumulation

The FDA is speeding up the process for regulatory approval of WTX101, a therapy for Wilson’s disease that could significantly improve the standard of care. Wilson Therapeutics, in Stockholm, has convinced the FDA to include its drug WTX101 (bis-choline tetrathiomolybdate) in its Fast Track program. This move will speed up the approval of the drug, […]

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

Interview 11 Dec 2017

Edwin Moses on how Ablynx has made it to the top of European biotech

Ablynx CEO Edwin Moses discusses the technology and strategy that has led its company to join the ranks of the biggest European biotechs. Ablynx, based in Belgium, has just recently joined the European billion-euro biotech club with a market cap of €1.45Bn. With positive Phase III results for its lead compound and a $230M (€195M) […]

December 11, 2017 - 8 minutesmins - By Clara Rodríguez Fernández

wilsons disease treatment wilson therapeutics sweden 1

News and Trends 8 Dec 2017

Wilson’s Disease Treatment Gets a €24.4M Equity Boost in Sweden

Wilson Therapeutics has issued new shares on Nasdaq Stockholm, raising a total of SEK 244M (€24.4M) to help bring its Wilson’s disease treatment to the market. Stockholm-based biotech Wilson Therapeutics has closed a share issue that seems to have generated quite a high demand. The company’s main shareholders, Abingworth and MVM, had to sell SEK 156.75M […]

December 8, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

advicenne ipo rare disease children drta

News and Trends 6 Dec 2017

Update: French Biotech Closes €27M IPO to be the First to Treat Rare Disease in Children

Update (6/12/2017): Advicenne has raised a total of €27M on Euronext Paris, putting its market cap at €112M. Originally published on 21/11/2017 Advicenne is launching an IPO on Euronext Paris to raise the funds to bring the first treatment for dRTA, a rare disease of the kidneys, to the market. Advicenne, based in Nîmes, France, has officially […]

December 6, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

dystrophic epidermolysis bullosa proqr rna therapy

News and Trends 30 Nov 2017

RNA Therapy for Rare Skin Disease Gets Orphan Status

The EMA has granted orphan drug designation to ProQR’s QR-313, a possible first disease-modifying treatment for the rare disease dystrophic epidermolysis bullosa. Dystrophic epidermolysis bullosa (DEB) is a genetic disease that causes the skin to easily blister, severely affecting the quality of life of patients since a very early age. Current treatment is limited to […]

November 30, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

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

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Rare disease

A New Biotech Hopes to Bring Us the Next Generation of Rare Metabolic Disorder Drugs

British Gene Therapy Gets a Huge £85M Boost in Series B

British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease

The FDA Fast Tracks Therapy for Rare Disease Causing Copper Accumulation

Edwin Moses on how Ablynx has made it to the top of European biotech

Wilson’s Disease Treatment Gets a €24.4M Equity Boost in Sweden

Update: French Biotech Closes €27M IPO to be the First to Treat Rare Disease in Children

RNA Therapy for Rare Skin Disease Gets Orphan Status

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

French Gene Therapy for Wilson’s Disease gets Orphan Status

New Dutch Spin Out will Treat Stroke with RNA Therapy

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

Subscribe to our newsletter to get the latest biotech news!

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Advertise with us

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