News and Press Releases
CRISPR Therapeutics Presents New Preclinical Data for CTX460 Demonstrating In Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase Editing Platform
Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) highlight a potential best-in-class profile The AATD program, CTX460, is the first investigational candidate to emerge from the SyNTase editing platform...
Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: October 10, 2025
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
Editas Medicine Reports In Vivo Proof-of-Concept Data for EDIT-401 at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress
Preclinical data demonstrates proof-of-concept for EDIT-401 with ≥90% mean LDL-C reduction in non-human primates and mouse models 9 October 2025 -- Massachusetts, US -- Editas Medicine, Inc, a pioneering gene editing company,...
Category: Biotechnology, Drug Discovery, Other, Pharmaceutical
Posted: October 9, 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
Yourgene Health Launches IONA Care+ Non-invasive Prenatal Testing Service
Offers expectant parents fully comprehensive prenatal screening test for genetic conditions, including microdeletions 13 February 2025 -- Manchester, UK -- Yourgene Health (part of the Novacyt group of companies), a leading...
Category: Biotechnology, Other, Pharmaceutical
Posted: February 13, 2025
Skelton House Lloyd Street North Manchester Science Park M15 6SH United Kingdom
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
Biofidelity and CellCarta partner to deploy Aspyre Lung in global clinical trials
9 January 2025 -- Cambridge, UK and Montreal, Canada -- Biofidelity, a leader in innovative genomic solutions, and CellCarta, the global contract research organization (CRO) partner of choice for biopharmaceutical...
Category: Biotechnology, Clinical Trials, Drug Discovery, Pharmaceutical
Posted: January 9, 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
PacBio Announces the HiFi Solves Sub-fertility Consortium in Asia Pacific
Collaboration Aims to Harness HiFi Sequencing to Improve Diagnosis and Treatment of Subfertility and Recurrent Pregnancy Loss HiFi Solves Sub-fertility Consortium, an innovative collaboration involving five leading centers across the...
Category: Biotechnology, Other
Posted: September 12, 2024
Broken String Biosciences appoints Steve Becker as Chief Commercial Officer
Appointment brings extensive experience to lead commercial operations and accelerate growth in key global markets Cambridge, UK, 24 July 2024: Broken String Biosciences (“Broken String”), a genomics company driving development...
Category: BioManufacturing
Posted: July 24, 2024
Unit AB3-04, Level 3 BioData Innovation Centre Wellcome Genome Campus Hinxton Cambridge, CB10 1DR
