News and Press Releases
Data from GNT-018-IDES trial supports feasibility of imlifidase as pretreatment in gene therapy treatment for patients with Crigler–Najjar syndrome who are immune to AAV
Results presented today at ESGCT 2025 10 October 2025 -- Paris, France and Lund, Sweden -- Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare...
Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: October 10, 2025
Relief Therapeutics and NeuroX, Successor to MindMaze, Sign Definitive Agreement for Business Combination
Companies sign definitive agreement following previously announced binding term sheet Transaction to create an expanded, SIX-listed, commercial-stage, AI-driven, scalable health tech company Combined company to be renamed MindMaze Therapeutics Holding...
Category: Biotechnology, Drug Discovery, Other, Pharmaceutical
Posted: October 8, 2025
Terumo Blood and Cell Technologies Supports Expanded Access to Rare Disease Therapies in Latin America
Regulatory approvals in 14 countries expand treatment options for sickle cell disease, Guillain-Barré syndrome and myasthenia gravis Approvals align with the historic World Health Assembly resolution on rare diseases. 16...
Category: Biotechnology, Drug Delivery, Other, Pharmaceutical
Posted: September 16, 2025
Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines
Designation granted for warm autoimmune hemolytic anemia and IgG4-related disease Rilzabrutinib is currently under regulatory review in the US, EU and China for potential use in immune thrombocytopenia. 3 April...
Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: April 3, 2025
PacBio and Radboud UMC Announce Remarkable Study Results Using HiFi Long Read Sequencing to Help Advance Rare Disease Diagnostics
14 January 2025 – Menlo Park, California US – PacBio (NASDAQ: PACB), a leading provider of highly accurate long-read sequencing solutions, announced a significant publication from Radboud University Medical Center...
Category:
Posted: January 16, 2025
PTC Therapeutics to Participate at Upcoming Investor Conferences
4 November 2024 -- New Jersey, US -- PTC Therapeutics, Inc today announced that the company will present a company overview at the following conferences: UBS Global Healthcare ConferenceWednesday, Nov....
Category: Biotechnology, Clinical Trials, Other, Pharmaceutical
Posted: November 4, 2024
PTC Therapeutics Inc. 100 Corporate Court South Plainfield, NJ 07080-2449
Sterling Pharma Solutions Announces Partnership with Soligenix for the Manufacture of Oncology-Targeted API
3 October 2024 -- Cramlington, UK -- Sterling Pharma Solutions, a global contract development and manufacturing organisation, and Soligenix, a late-stage biopharmaceutical company focused on developing and commercialisation of products...
Category: BioManufacturing, Clinical Trials, Pharmaceutical
Posted: October 3, 2024
Sterling Place, Dudley Cramlington, Northumberland, NE23 7QG, United Kingdom
AzurBio Pharma Launches to Fast-Track European Market Entry for Biopharma Companies with Breakthrough Treatments
1 October 2024 -- Paris, France -- AzurBio Pharma, specializing in bringing innovative pharmaceutical products to European markets and focusing on rare and serious diseases with unmet medical needs, is...
Category: Biotechnology, Clinical Trials, Drug Discovery, Pharmaceutical
Posted: October 1, 2024
Sarepta Therapeutics Announces Recipients of LGMD Grant Awards for 2024; Program Supports Early Genetic Testing and Awareness
Recipients awarded a total of more than $100,000 in grants Funding will support education and activities to shorten diagnostic journey. 30 September 2024 -- Massachusetts, US -- Sarepta Therapeutics, Inc, the...
Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: September 30, 2024
Hill House, 1 Little New Street London, EC4A 3TR United Kingdom
Mogrify appoints Dr Jonathan Appleby as chief scientific officer
Strategic hire bolsters executive leadership team as company progresses multiple therapeutic programmes toward and into clinical development 15 July 2024 – Cambridge, UK – Mogrify, a regenerative medicine company developing...
Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: July 15, 2024
Biogen Receives European Commission Approval for QALSODY (tofersen), the First Therapy to Treat a Rare, Genetic Form of ALS
SOD1-ALS is a devastating, uniformly fatal and ultra-rare genetic form of ALS estimated to affect less than 1,000 people in Europe QALSODY is Biogen’s third rare disease therapy to be...
Category: Clinical Trials, Drug Discovery, Pharmaceutical
Posted: May 30, 2024
TMC Pharma champions holistic approach to medical services in rare disease drug development
13 March 2024 – In a recent webinar, rare disease specialist and global clinical research organisation (CRO) TMC Pharma gathered esteemed speakers to share insights into the scope of medical...
Category: Drug Discovery, Pharmaceutical
Posted: March 13, 2024
Lodge Farm Barn Elvetham Park Estate, Fleet Road, Hartley Wintney, Hampshire RG27 8AS, UK
Clinigen launches ‘What is Possible?’ campaign to accelerate access to medicines on Rare Disease Day
With over 7,000 rare diseases that impact over 300 million people globally, just 5% of rare diseases have a licensed treatment 29 February 2024 -- London, UK -- Clinigen, the...
Category: Other
Posted: February 29, 2024
