Rare disease Archives

Scenic biotech lysosomal storage disorders

News and Trends 10 Mar 2022

Emerging Startups Take Aim at Lysosomal Storage Disorders

Lysosomal storage disorders such as Niemann-Pick type C have proved difficult to tackle in the biotech industry. With recent funding rounds in the bag, Scenic Biotech and Maze Therapeutics strive to treat these conditions by activating protective genes. Lysosomal storage disorders are inherited conditions caused by mutations in metabolic enzymes found inside our cells, leading […]

March 10, 2022 - 4 minutesmins - By Jonathan Smith

Medical anca associated vasculitis avacopan

News and Trends 1 Dec 2021

Targeted Drugs for Rare Autoimmune Disease Hit Regulatory Milestones

New, more targeted treatments are on the horizon for the rare autoimmune disease ANCA-associated vasculitis, with a nod towards European approval for a first-in-class drug and positive phase II results from a rival treatment. In November, the Swiss firm Vifor Fresenius Medical Care Renal Pharma (VFMCRP) received a thumbs-up from the EMA for avacopan, a […]

December 1, 2021 - 4 minutesmins - By Anita Chakraverty

treatments, biotech, genes, biomanufacturing, infographic

Infographics by 15 Nov 2021

Gene therapy: Making Rare Disease Therapies Less Rare

While individually uncommon, rare diseases collectively impact the lives of over 300 million people worldwide. Many rare conditions are chronic, progressive, and life-threatening with limited treatment options available. Advances in gene therapy provide hope to actual cures and could revolutionize treatment prospects for patients with rare genetic diseases. This infographic shows how we can unlock […]

November 15, 2021 - 2 minutesmins - By External Contributor

Expert Advice 27 May 2020

Why Listening to Patient Organizations is Key in Drug Development

As the biotech industry flourishes and therapies become more targeted and personalized, the need for drug developers to interact with patients is increasing as well. While patient organizations took a backseat in the past, they are continuously gaining importance in drug development today. Nowadays, talking with patient organizations has become paramount for successful drug development, […]

May 27, 2020 - 11 minutesmins - By Larissa Warneck-Silvestrin

novartis spinal muscular atrophy gene therapy

News and Trends 19 May 2020

Update: Novartis’ Gene Therapy Zolgensma Gets Nod from EU

Update (19/05/2020): The gene therapy Zolgensma, developed by the big pharma Novartis and approved in the US last year, has received conditional approval in Europe for the treatment of spinal muscular atrophy (SMA). The gene therapy is approved to treat patients with a clinical diagnosis of SMA type 1 or SMA patients that have up […]

May 19, 2020 - 5 minutesmins - By Jonathan Smith

News and Trends 8 May 2020

Phase III Failure Sinks Drug for Rare Neurological Disorder

The Italian biotech Newron is halting the development of the experimental drug sarizotan after it failed to meet efficacy endpoints in phase III, dashing hopes for an approved drug for Rett syndrome. Expectations had been high that the oral drug might be able to treat breathing difficulties associated with the rare genetic disorder after promising […]

May 8, 2020 - 4 minutesmins - By Anita Chakraverty

novartis avexis zolgensma fda data manipulation

News and Trends 2 Apr 2020

Update: FDA Imposes No Penalties for Novartis Data Manipulation Scandal

Update (02/04/2020): The FDA has concluded an investigation into a data manipulation scandal surrounding the approval of Novartis’ gene therapy Zolgensma, and has decided against sanctions. The FDA launched an investigation into the manipulated data surrounding the gene therapy application last year, and recently reported that the investigated data violations didn’t meet the threshold for […]

April 2, 2020 - 3 minutesmins - By Jonathan Smith

News and Trends 31 Mar 2020

Ipsen Resumes Phase III Testing for Rare Disease Drug and Abandons Pediatric Trial

The French pharmaceutical company Ipsen has restarted the dosing of its treatment for a rare bone disease in a phase III trial after obtaining questionable interim results. At the same time, the company has terminated a phase II trial of the same drug in pediatric patients with a different bone disorder following a partial clinical […]

March 31, 2020 - 4 minutesmins - By Karen O’Hanlon Cohrt

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

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Rare disease

Emerging Startups Take Aim at Lysosomal Storage Disorders

Targeted Drugs for Rare Autoimmune Disease Hit Regulatory Milestones

Gene therapy: Making Rare Disease Therapies Less Rare

Why Listening to Patient Organizations is Key in Drug Development

Update: Novartis’ Gene Therapy Zolgensma Gets Nod from EU

Phase III Failure Sinks Drug for Rare Neurological Disorder

Update: FDA Imposes No Penalties for Novartis Data Manipulation Scandal

Ipsen Resumes Phase III Testing for Rare Disease Drug and Abandons Pediatric Trial

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

Harnessing Genetic Suppression to Treat Rare Disease

Rare Inflammatory Disease Drug Beats Immunosuppressants in Phase III

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

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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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