Imlifidase as pretreatment in gene therapy for patients with Crigler–Najjar syndrome who are immune to AAV On October 10, 2025, Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare genetic diseases, and Hansa Biopharma, a Sweden-based leader in IgG cleaving enzyme technology announced that…..
[caption id="attachment_11913" align="aligncenter" width="1024"] Blarney's facility Opening[/caption] Merck KGaA opens climate-neutral €150 million filter manufacturing facility in Ireland On September 18, 2025Merck, a leading science and technology company, has opened today its €150 million filter manufacturing facility in Blarney Business Park, Cork, Ireland. The site is Merck’s first…..
www.mitogether.com, the reference website for professionals and families affected by genetic mitochondrial diseases [caption id="attachment_11905" align="aligncenter" width="1024"] Mitogether consortium[/caption] There are more than 400 forms of genetic—or primary—mitochondrial diseases linked to mutations in genes directly involved in the functioning or maintenance of mitochondria. Mitochondria are unique in that they have…..
Texas Children’s provides new breakthrough treatment for patient with AADC deficiency [caption id="" align="alignnone" width="700"] Texas Children's neurosurgeon Dr. Daniel Curry leads first-ever FDA-approved gene therapy treatment for AADC deficiency. ©Texas Children's Hospital[/caption] On August 19, 2025, Texas Children's, a non-profit healthcare organization, has announced that a three-year-old girl has…..
Gene therapy improves blood flow in the brain in patients with sickle cell disease [caption id="attachment_11887" align="aligncenter" width="700"] Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy talks to St. Jude patient enrolled on the reported clinical trial, Corey Luellen. ©St. Jude Children's Research Hospital[/caption]…..
Genome of Europe launched public awareness survey on genomics across five EU countries The Genome of Europe (GoE) – co-funded by the European Union’s Digital Europe Programme (101168231) – is a pan-European research project uniting 27 countries to create a comprehensive reference database of genetic data from people of diverse…..
First vascularized model of stem cell islet cells [caption id="attachment_11877" align="aligncenter" width="1024"] Engineered vasculature induces functional maturation of pluripotent stem cell-derived islet organoidsResearchers have devised the right conditions to grow vascularized stem cell islets. The image shows vasculature (red) tightly wrapped around insulin-producing cells (green) in the islets (blue). ©…..
TreeFrog Therapeutics secures €30 million from EIB TreeFrog Therapeutics, a French biotech specializing in cell therapy, has secured a €30 million financing from the European Investment Bank (EIB). The financing will support the advancement of their lead cell therapy program in Parkinson’s Disease to the clinic. This cell therapy has…..
CellProthera Selects CELLforCURE by SEQENS for Phase 3 GMP Manufacturing of its stem cell-based therapy First clinical batches of ProtheraCytes® are planned for 2026 CellProthera, a French regenerative cell therapy developer specializing in ischemic diseases, has chosen CELLforCURE by SEQENS, as its contract development and manufacturing organization (CDMO) partner for its…..
CRISPR Therapeutics and Sirius Therapeutics Announce Multi-Target Collaboration to Develop Novel siRNA Therapies CRISPR Therapeutics, a Swiss biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Sirius Therapeutics, a sino-american clinical stage biotech company developing innovative small interfering RNA (siRNA) therapies for global markets, have concluded a…..
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