A gene therapy developed by an Italian nonprofit, Fondazione Telethon, known as Waskyra (etuvetidigene autotemcel), has received U.S. Food and Drug Administration approval for treating Wiskott-Aldrich syndrome, a rare immune disorder with limited treatment options. The decision marks the first time the FDA has authorized a gene therapy from a nonprofit applicant and reflects a flexible regulatory approach that considered diverse clinical data from small open-label studies and

Published on Alliance for Regenerative Medicine A large coalition of rare disease organizations is pushing Congress to advance two bipartisan bills that would expand pediatric access to specialized care and sustain incentives for developing rare disease treatments, including CGTs. The proposals focus on strengthening early-access pathways and improving out-of-state provider credentialing for children with complex conditions. 👉 Read the full article: ARM and the Rare Disease

Published on ISCT Telegraft Hub ISCT highlights how its Telegraft Hub is expanding global engagement with new working groups, member-driven education, and stronger cross-regional collaboration. The update emphasizes community-building across cell and gene therapy professionals as ISCT prepares for major 2026 initiatives. 👉 Read the full article: Connecting People and Growing Science – ISCT Telegraft Hub Update Disclaimer Images are for illustrative purposes only and may not

Published on Fierce Biotech Roche’s obinutuzumab regimen has gained FDA approval for adults with active lupus nephritis, offering a faster infusion option and improving complete renal responses compared to standard therapy in Phase III. 👉 Read the full article: Gazyva/Gazyvaro FDA approval for lupus nephritis Disclaimer Images used are for illustrative purposes only and do not depict actual persons, products, or facilities. For full detail, refer to the original article via

Published on Cell & Gene New research highlights how allogeneic natural killer (NK) cell platforms are shaping the next phase of cancer immunotherapy. These off-the-shelf cell therapies promise broader access, faster deployment, and strong safety profiles across multiple tumor types. 👉 Read the full article: Building the Future of Cancer Immunotherapy Through Allogeneic NK Cell Innovation Disclaimer Images used are for illustrative purposes only and do not depict actual pers

Published on SciTechDaily Researchers report that transplanted stem cells may regenerate brain tissue and restore lost function after stroke. The study highlights early evidence of neuronal regrowth and recovery, marking a breakthrough in regenerative neurology. 👉 Read the full article: Stroke Damage Reversed as Stem Cells Regrow the Brain Disclaimer Images used are for illustrative purposes only and do not depict actual persons, products, or facilities. For full detail, ref

Published by ASGCT & Citeline The ASGCT Q2 2025 report shows sustained CGT momentum with new approvals in gene and mRNA therapies, steady deal activity, and rising oncology focus. M&A volume increased sharply, led by major pharma consolidations in advanced therapies. 👉 Read the full report: ASGCT Gene, Cell, & RNA Therapy Landscape Report Q2 2025 Disclaimer Images used are for illustrative purposes only and do not depict actual persons, products, or facilities. For full deta

Published on LinkedIn (via Spencer Knight) Novo Nordisk has announced its plan to acquire Akero Therapeutics for up to $5.2 billion,...

Published on LinkedIn (via Spencer Knight) Bristol Myers Squibb announced a $1.5 billion acquisition of Orbital Therapeutics, advancing...

Published on LinkedIn (via Spencer Knight) The FDA has approved INLEXZO™ (gemcitabine intravesical system) from Johnson & Johnson,...

Published on Technology Networks Researchers at USC Norris and City of Hope engineered CAR T cells that deliver IL-12 fused to a PD-L1...

Published on Bayer Bayer’s AskBio has randomized the first European participants in its Phase II trial of AB-1005, an investigational...

Published on BlueRock Therapeutics BlueRock Therapeutics has treated the first Parkinson’s disease patient in its pivotal Phase III trial...

Published on Alliance for Regenerative Medicine (LinkedIn) Two landmark therapies, Zolgensma and Yescarta, highlight how cell and gene...

Published on LinkedIn (via Spencer Knight) The FDA has approved Inliruqo™ (imlunestrant) from Eli Lilly, the first oral estrogen receptor...

Published on Mayo Clinic News Network Mayo Clinic researchers report that fat-derived mesenchymal stem cells can help prevent vein...

Published on Sky News A breakthrough gene therapy called AMT-130 has slowed progression of Huntington’s disease by as much as 75% in a...

Published on Drug Target Review USC Stem Cell scientists have created kidney assembloids that function like real organs, combining...

Published on LinkedIn (via Spencer Knight) Pfizer announced plans to acquire Metsera for up to $4.9B, adding GLP-1 and amylin-based...

Published on LinkedIn (via Spencer Knight) Roche announced a $3.5B acquisition of 89bio, adding pegozafermin, a late-stage therapy for...