Rare disease Archives

News and Trends 31 Aug 2022

Emactuzumab designated as an orphan medicinal product for TGCT in Europe

SynOx Therapeutics Limited says its novel monoclonal antibody, emactuzumab, in development for the treatment of tenosynovial giant cell tumor (TGCT) and other diseases, has been designated as an orphan medicinal product for both localized and diffuse types of the disease by the European Medicines Agency (EMA). No systemic treatments have been approved for TGCT in […]

August 31, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 24 Aug 2022

Amgen announces top-line results from biosimilar phase 3 study

Amgen has announced positive top-line results from its DAHLIA study. This was a randomized, double-blind, active-controlled, two-period crossover phase 3 study evaluating the efficacy and safety of ABP 959, a biosimilar candidate to SOLIRIS (eculizumab), compared with SOLIRIS in adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Amgen said the study met its primary endpoints, demonstrating […]

August 24, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 23 Aug 2022

Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome

Ocelot Bio, Inc., a clinical-stage biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead candidate OCE-205 for the treatment of hepatorenal syndrome. Ocelot Bio is currently enrolling patients in a phase 2 clinical trial of OCE-205 in hepatorenal syndrome with acute kidney injury (HRS-AKI), […]

August 23, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 22 Aug 2022

Vaderis emerges from stealth to start HTT trial

Vaderis Therapeutics AG, a clinical stage biotech company focused on developing treatments for rare diseases associated with vascular malformations, has emerged from stealth. The company has also started its INSIGHT proof-of-concept clinical trial in patients suffering from hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome. About HHT HHT is a disorder where some blood […]

August 22, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 19 Aug 2022

Emtora to use $16.9M grant to develop treatment for rare genetic disease

Emtora Biosciences, a Texas-based clinical-stage biopharmaceutical company, has been awarded a $16.9 million non-dilutive grant from the Cancer Prevention & Research Institute of Texas (CPRIT). The funding will be used to advance the clinical and product development of its lead program, eRapa, an encapsulated form of rapamycin, for the treatment of familial adenomatous polyposis (FAP). […]

August 19, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 19 Aug 2022

Aeglea BioTherapeutics’ arginine 1 deficiency treatment gets EMA ok

Aeglea BioTherapeutics, Inc. says its marketing authorization application (MAA) for pegzilarginase for the treatment of arginase 1 deficiency (ARG1-D) has been validated by the European Medicines Agency (EMA). The MAA was submitted by Immedica Pharma AB, Aeglea’s commercialization partner in Europe and the Middle East. Pegzilarginase is a novel, recombinant human arginase 1 enzyme that […]

August 19, 2022 - 3 minutesmins - By Jim Cornall

In Depth 22 Jul 2022

The coming of age story of RNAi technology

The first RNA interference (RNAi) therapy was approved in 2018, after 20 years in development. What can we expect to see in the RNAi field in the future? After two decades of scientific effort, multiple devastating clinical trial setbacks, and billions of dollars spent on research and development, RNAi therapies are finally establishing themselves in […]

July 22, 2022 - 7 minutesmins - By Farhan Mitha

News and Trends 21 Jul 2022

Almirall launches new call to find innovative therapies for skin diseases

A new call for new therapies to treat skin diseases has been launched by global Spanish-based biopharmaceutical company, Almirall, S.A. This is the seventh call through AlmirallShare – an open innovation platform for proposals to establish collaborations in dermatological research. The aim of this year’s edition is to find new partnership opportunities for assets in […]

July 21, 2022 - 3 minutesmins - By Liza Laws

News and Trends 22 Mar 2022

Biotechs Carve out Niches in Cystic Fibrosis Treatment Space

Cystic fibrosis has proved resistant to treatments that provide long-lasting respite from the sticky mucus that affects the lungs and other organs. Emerging biotechs such as Kither Biotech as well as the old guard including Vertex are finding new ways to tackle the inherited disease. Cystic fibrosis is best known for causing thick mucus in […]

March 22, 2022 - 5 minutesmins - By Anita Chakraverty

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

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Rare disease

Emactuzumab designated as an orphan medicinal product for TGCT in Europe

Amgen announces top-line results from biosimilar phase 3 study

Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome

Vaderis emerges from stealth to start HTT trial

Emtora to use $16.9M grant to develop treatment for rare genetic disease

Aeglea BioTherapeutics’ arginine 1 deficiency treatment gets EMA ok

The coming of age story of RNAi technology

Almirall launches new call to find innovative therapies for skin diseases

Biotechs Carve out Niches in Cystic Fibrosis Treatment Space

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

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

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Advertise with us

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