Rare disease Archives

khondrion netherlands melas syndrome inherited diseases

Startup Scout 20 Sep 2019

This Biotech Targets Mitochondria to Treat Inherited Diseases

As we come to the end of the global Mitochondrial Disease Awareness Week, it’s time to visit Khondrion. This Dutch company is developing drugs to treat rare diseases such as MELAS syndrome by targeting defective mitochondria. Mission: To develop small molecule drugs able to treat Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) syndrome. This […]

September 20, 2019 - 3 minutesmins - By Jonathan Smith

More News! 11 Sep 2019

Dutch Company Raises €60M to Treat Inherited Inflammatory Disease

The Dutch pharmaceutical company Pharvaris has raked in €60M in a Series B round to fund the clinical development of the first oral treatment for hereditary angioedema, a condition that causes swelling in the limbs and airways. The Series B round will fuel the clinical progress of Pharvaris’ oral small molecule treatment for hereditary angioedema, […]

September 11, 2019 - 2 minutesmins - By Jonathan Smith

bluebird bio gene therapy beta thalassemia

News and Trends 4 Jun 2019

First Gene Therapy for Beta Thalassemia Approved in Europe

The US biotech company bluebird bio has obtained EU conditional market approval for a gene therapy that could remove the need for blood transfusions for people with the blood disorder beta-thalassemia. Called Zynteglo, the gene therapy is aimed at treating people with beta-thalassemia who are over 12 years of age and have any but the […]

June 4, 2019 - 3 minutesmins - By Jonathan Smith

pure biologics wroclaw devics disease 1 1

Startup Scout 31 May 2019

This Biotech Treats a Neurological Autoimmune Disorder Using DNA

We’re jumping into Poland this week. Pure Biologics is a biotech based in Wrocław developing DNA molecules that could treat the incurable neurological condition Devic’s disease with fewer side effects than existing treatments. Mission: To develop a treatment that makes an existing treatment for Devic’s disease, plasmapheresis, more targeted and with fewer side effects than […]

May 31, 2019 - 4 minutesmins - By Jonathan Smith

achondroplasia pfizer therachon acquisition

More News! 9 May 2019

Pfizer Pays up to €700M to Acquire Swiss Biotech Restoring Bone Growth in Achondroplasia

Pharma giant Pfizer is acquiring the Swiss biotech Therachon, which is developing a protein drug to boost bone growth in the genetic condition achondroplasia. Pfizer will pay €303M ($340M) upfront, and up to €420M ($470M) dependent on Therachon reaching milestones in the development of its drug improving bone growth in achondroplasia. This genetic condition inhibits […]

May 9, 2019 - 2 minutesmins - By Jonathan Smith

orchard therapeutics cell therapy stem cell rare disease

News and Trends 28 Mar 2019

Gene Therapy Exceeds Expectations in Treating Children’s Neurological Disease

A gene therapy developed by the UK company Orchard Therapeutics has greatly improved the motor symptoms of children with an incurable neurological disease. The 20 children in the phase II trial suffered from metachromatic leukodystrophy, a rare genetic disease caused by the mutation of a protein that breaks down sulfur-containing molecules in cells. This leads […]

March 28, 2019 - 3 minutesmins - By Jonathan Smith

biogen nightstar therapeutics gene therapy ophthalmology

News and Trends 4 Mar 2019

Biogen Acquires UK Biotech Developing Gene Therapy for Blindness

The big US biotech Biogen has agreed to acquire Nightstar Therapeutics for €773M ($877M), including Nightstar’s candidate gene therapies for inherited blindness. Expected to be complete by mid-2019, Biogen’s acquisition of Nightstar bags it the UK company’s most advanced program. Currently in phase III, the therapy targets choroidemia, an incurable genetic condition which causes progressive […]

March 4, 2019 - 2 minutesmins - By Jonathan Smith

orphazyme niemann pick type c disease rare metabolic disease molecules

News and Trends 30 Jan 2019

Danish Biotech’s Drug for Rare Metabolic Disease Aces Clinical Trial

A drug, developed by the Danish biotech Orphazyme for a rare metabolic disease, has shown promising results in a phase II/III trial, reducing the disease’s progression by 74%. The 50 patients recruited into the trial suffered from Niemann-Pick disease type C, a metabolic disease in which proteins crucial for storing fat molecules such as cholesterol […]

January 30, 2019 - 3 minutesmins - By Jonathan Smith

amryt skin gene therapy epidermolysis bullosa

News and Trends 8 Jan 2019

Topical Gene Therapy Shows Early Promise for Inherited Skin Disease

A gene therapy applied to the skin, developed by UK company Amryt Pharma, showed positive preclinical results for the treatment of a rare genetic skin disease. The disease, called recessive dystrophic epidermolysis bullosa (RDEB), is one of a group of diseases in which sufferers have mutations in proteins vital to the integrity of the […]

January 8, 2019 - 3 minutesmins - By Jonathan Smith

News and Trends 7 Dec 2018

Orchard’s Gene Therapy for Rare Immune Disease Works for Over a Year

A single dose of a gene therapy developed by Orchard Therapeutics can restore immune function for at least a year in patients with X-linked chronic granulomatous disease (X-CGD). The gene therapy was trialed in patients between the ages of 2 and 27 with the rare disease, which is caused by a rare genetic mutation that […]

December 7, 2018 - 3 minutesmins - By Clara Rodríguez Fernández

Interview 28 Nov 2018

Navigating the Rare Disease Space: Insights from a Danish CEO

Developing drugs for rare diseases is a tough nut for biotech companies to crack. Labiotech’s Helen Albert interviewed the CEO of the Danish company Orphazyme, Anders Hinsby, at the recent BIO-Europe conference in Copenhagen, who told her about the appeals and challenges of making it in the rare disease space. Traditionally, the low number of […]

November 28, 2018 - 5 minutesmins - By Jonathan Smith

More News! 22 Nov 2018

First Drug for Rare Inflammation Syndrome Approved by FDA

The FDA has approved the first drug for the treatment of a rare condition in which the body produces too many active immune cells, developed in a Swiss-Swedish partnership. The antibody drug, emapalumab, is approved to treat primary hemophagocytic lymphohistiocytosis. This condition is a rare, often fatal syndrome in which the immune system produces too […]

November 22, 2018 - 2 minutesmins - By Jonathan Smith

Knowledge hub

Categories

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Approaches

Therapeutic areas

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

R&D

BD

World biotech leaders

Connect with decision-makers and researchers worldwide for collaborative opportunities

Company

Join Us

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

Rare disease

This Biotech Targets Mitochondria to Treat Inherited Diseases

Dutch Company Raises €60M to Treat Inherited Inflammatory Disease

First Gene Therapy for Beta Thalassemia Approved in Europe

This Biotech Treats a Neurological Autoimmune Disorder Using DNA

Pfizer Pays up to €700M to Acquire Swiss Biotech Restoring Bone Growth in Achondroplasia

Gene Therapy Exceeds Expectations in Treating Children’s Neurological Disease

Biogen Acquires UK Biotech Developing Gene Therapy for Blindness

Danish Biotech’s Drug for Rare Metabolic Disease Aces Clinical Trial

Topical Gene Therapy Shows Early Promise for Inherited Skin Disease

Orchard’s Gene Therapy for Rare Immune Disease Works for Over a Year

Navigating the Rare Disease Space: Insights from a Danish CEO

First Drug for Rare Inflammation Syndrome Approved by FDA

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

Subscribe to our newsletter

Get the latest biotech news directly to your inbox!

Newsletter Signup - Footer

Subscribe to our newsletter to get the latest biotech news!