Rare disease Archives

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

News and Trends 2 Nov 2018

Gut Bacteria Treatment Shows Promise for Rare Genetic Kidney Condition

The Swedish biotech Oxthera reported positive preliminary Phase II results using its gut bacteria treatment, Oxabact, in patients with a rare genetic condition that causes kidney failure. The results came from an interim analysis one year into a three-year trial, where Oxthera is testing its gut microbiome treatment on patients with primary hyperoxaluria type […]

November 2, 2018 - 2 minutesmins - By Jonathan Smith

More News! 16 Oct 2018

Drug Combination for Rare Genetic Nerve Disorder Nails Phase III

A treatment developed by French biotech Pharnext has become the first clinically proven therapy for a rare genetic disorder that affects the nervous system. In a Phase III trial, the treatment reduced the level of disability of patients with Charcot-Marie-Tooth disease, a condition that affects peripheral nerves. This disease causes problems with walking, running and […]

October 16, 2018 - 2 minutesmins - By Jonathan Smith

News and Trends 9 Oct 2018

Hormone Therapy for Inherited Hormonal Disorder Fails at Phase III

A European Phase III trial has become a major stress for British biotech Diurnal. Its specially designed hormone therapy was no better than conventional therapies in treating an inherited hormonal disorder. Congenital adrenal hyperplasia is a rare genetic condition where patients lack crucial enzymes for making hormones. The most common form lacks 21-hydroxylase, the enzyme […]

October 9, 2018 - 3 minutesmins - By Jonathan Smith

In Depth 9 Oct 2018

How to Capitalize on Opportunities in Underserved Disease Areas

Developing treatments for underserved diseases can be surprisingly profitable for biotechs, we asked the experts to tell us more about how best to exploit these areas. We hear a lot about cancer therapies or drugs to target heart disease in the press, but overall a lot less about treatments for more underserved areas. Often successful […]

October 9, 2018 - 9 minutesmins - By Helen Albert

News and Trends 26 Sep 2018

Spanish Biotech Raises €21.3M To Advance Rare Disease Pipeline

Rare genetic diseases in the central nervous system may be less of a headache in future, as Barcelona-based biotech Minoryx has raised €21.3M in series B fundraising to test its lead drug candidate against other rare neurological conditions. According to Philippe Monteyne, partner at investor Fund+, “it is the largest investment in biotech in Spain […]

September 26, 2018 - 2 minutesmins - By Jonathan Smith

Knowledge hub

Categories

10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Approaches

Therapeutic areas

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

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

10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Rare disease

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

Gut Bacteria Treatment Shows Promise for Rare Genetic Kidney Condition

Drug Combination for Rare Genetic Nerve Disorder Nails Phase III

Hormone Therapy for Inherited Hormonal Disorder Fails at Phase III

How to Capitalize on Opportunities in Underserved Disease Areas

Spanish Biotech Raises €21.3M To Advance Rare Disease Pipeline

Subscribe to our newsletter to get the latest biotech news!

10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

10 oncology deals in 2025 spotlight where industry leaders are betting big

10 biotech companies leading the way in Denmark

Webinar: Advancements in antimalarial drug discovery and development

Six psychedelic drug companies aiming to revolutionize mental health treatment

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!