The UK should look to become a European leader in rare disease medicine, a new report says. The report, ‘Investing in Rarity: The Economic and Personal Value of Improving Treatment for Rare Disease’, shows that if the UK became the best-performing European country on… Read More
News round-up for 16 – 20 March February by Bruno Quinney, Content Team at DDW. This week, a pioneering method is being used to make drugs for Parkinson’s disease. Elsewhere, advances have been made in both research and trials for common cancers. The top stories: Edinburgh team… Read More
The US Food and Drug Administration (FDA) has granted Fast Track Designation to a treatment for the rare paediatric disease late-infantile neuronal ceroid lipofuscinosis (LINCL/CLN2 disease). PLX-200, developed by biotechnology company Polaryx Therapeutics, will now undergo an expedited review with the potential to address the paediatric disease with unmet… Read More
Rare disease therapies could generate over $400 billion in global sales by 2032, a new report reveals. Evaluate’s ‘2026 Orphan Drug Report’ predicts the sales of orphan drugs will continue to rise steadily, despite policy changes and pricing turmoil. According to the forecast, orphan drugs will account for… Read More
The latest episode of the DDW Highlights Podcast is now available to listen to below. DDW’s Bruno Quinney narrates five key stories of the previous week to keep DDW subscribers up-to-date on the latest industry news. Last week, researchers made a key discovery into cancer survival. Elsewhere, a global research effort… Read More
Biopharmaceutical company BOOST Pharma has appointed Hans Schambye as Chief Executive Officer. BOOST, a company developing novel, first–in–class off-the-shelf cell therapies for rare, debilitating paediatric skeletal diseases, announced Schambye has transitioned from his role on the Board of Directors with immediate effect into his new role as… Read More
News round-up for 9 – 13 February by Bruno Quinney, Content Team at DDW. This week in DDW, researchers have made a key discovery into cancer survival. Elsewhere, a global research effort into kidney disease is underway. The top stories: Researchers identify RNA linked to cancer… Read More
The US Senate has reauthorised the US Food and Drug Administration (FDA)’s Priority Review Voucher (PRV), helping biotech companies to develop rare disease drugs. The PRV programme, which incentivises rare disease drug development, was reauthorised on 30 January, with President Donald Trump signing the bill. … Read More
UCB’s Kygevvi (doxecitine and doxribtimine) has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending marketing authorisation under exceptional circumstances. The recommendation is for the treatment of paediatric and adult patients with genetically confirmed thymidine kinase… Read More
Cell technology company iXCells Biotechnologies has partnered with Rosebud Biosciences to advance organoid-based models for rare diseases. The partnership will set out to develop a personalised, human-based approach for predicting drug safety and informing translational decision-making in rare diseases. iXCells will integrate Rosebud’s organoid generation and characterisation expertise into its existing… Read More
The BioIndustry Association (BIA) has called for a new, reformed pathway to give UK patients access to innovative rare disease medicines. According to the BIA’s new report, while the UK’s rare disease ecosystem has a solid foundation, more work is needed to build a connected, flexible and risk-proportionate access pathway to… Read More
The rulebook for rare disease therapies will be overhauled to make it quicker and easier to get these therapies tested, manufactured and approved in the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) has announced. The announcement came in a new paper supporting the ambitions of… Read More
The UK Government has announced that every newborn baby in England will have their genetic code mapped to test for a wide range of conditions, part of a plan that will also see £650m invested in DNA research by 2030. The Department for Health and Social Care… Read More
BIAL, a biopharmaceutical company focused on neurosciences and rare diseases, will give an oral presentation on its potential disease-modifying compound, BIA 28-6156, at the GBA1 Meeting 2025 being held 5-7 June 2025, in Montreal, Canada. The GBA1 Meeting 2025 is hosted by McGill University, focusing on all aspects of GBA1… Read More
Dan Williams, CEO of SynaptixBio, explains the science behind antisense oligonucleotides (ASOs) and the challenges and opportunities in using them to tackle rare diseases such as H-ABC. Gene silencing’ is a term used to describe the process of stopping a specific gene from making its associated protein. It is different… Read More
