Rare disease Archives

lysosomal storage disorders azafaros rare disease

News and Trends 10 Feb 2020

First-in-Class Drug For Inherited Metabolic Diseases Funded with €25M

The Dutch startup Azafaros has raised €25M in Series A funding to push its drug for inherited lysosomal storage disorders into phase I. The round was led by the Dutch VC firm Forbion. Other investors participating in the round included the Swiss firm BioMedPartners and the Dutch company BioGeneration Ventures, which was Azafaros’ founding investor […]

February 10, 2020 - 2 minutesmins - By Jonathan Smith

Interview 5 Feb 2020

Harnessing Genetic Suppression to Treat Rare Disease

Through quirks of genetics, some people are naturally resilient to heritable diseases. Thijn Brummelkamp, Managing Director and founder of the Dutch startup Scenic Biotech, explains how the company is using this genetic resilience to treat rare diseases and cancer. Heritable diseases result from genetic mutations that cause essential proteins in the body to malfunction. For […]

February 5, 2020 - 5 minutesmins - By Jonathan Smith

lupus autoimmune disease neutrophil citryll

More News! 26 Nov 2019

Rare Inflammatory Disease Drug Beats Immunosuppressants in Phase III

A small molecule drug developed by the US company ChemoCentryx and its Swiss partner Vifor Fresenius Medical Care Renal Pharma (VFMCRP) was more effective at treating a rare inflammatory vascular disease than current treatments in a phase III trial. The drug avacopan was trialed in 331 patients with anti-neutrophil cytoplasmic antibody-associated vasculitis. This life-threatening condition […]

November 26, 2019 - 3 minutesmins - By Jonathan Smith

mereo biopharma brittle bone disease osteogenesis imperfecta

More News! 11 Nov 2019

Mereo BioPharma’s Brittle Bone Disease Drug Stumbles in Phase II

The lead drug of the UK company Mereo BioPharma has achieved mixed results in a phase IIb trial in adults with the genetic condition osteogenesis imperfecta, also known as brittle bone disease. Mereo tested different doses of its antibody drug in 112 adults with osteogenesis imperfecta in a 12-month phase IIb trial. The drug failed […]

November 11, 2019 - 2 minutesmins - By Jonathan Smith

centogene rare diseases diagnostic ipo nasdaq

News and Trends 7 Nov 2019

€51M Nasdaq IPO Will Fund Centogene’s Rare Disease Diagnostics

The German company Centogene has raised €50.6M ($56M) in an IPO on the Nasdaq Stock Exchange to fund the development of diagnostics technology that could improve the treatment of rare diseases. The fundraise fell under Centogene’s target value of €54M ($60M). A part of the proceeds will fuel the development of biomarkers that Centogene will […]

November 7, 2019 - 2 minutesmins - By Jonathan Smith

News and Trends 16 Oct 2019

UK Firm Raises €51M to Repurpose Drugs for Rare Diseases Using AI

The Cambridge-based artificial intelligence company Healx has raised a Series B round of €50.8M to launch an AI tool that reduces the time it takes to develop treatments for rare diseases by five years. The Series B round was led by the UK VC firm Atomico and includes the returning investor Jonathan Milner, the founder […]

October 16, 2019 - 2 minutesmins - By Jonathan Smith

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

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