The discussion focuses on how an Italian nonprofit organization navigated scientific development, manufacturing readiness, and regulatory engagement to achieve U.S. Food and Drug Administration approval for a gene therapy. It reflects broader questions about alternative development models beyond traditional commercial biotech and how regulatory frameworks can accommodate nonprofit-led innovation in rare diseases. 👉 View the full video: EP 29 - How an Italian Non Profit Got a

Publisher name Pharmaceutical Technology The commentary discusses systemic pressures facing the regenerative medicine sector beyond scientific development, including fragile business models, reimbursement uncertainty, and limited manufacturing scalability. It argues that without coordinated policy, financing, and infrastructure solutions, many advanced therapies may struggle to transition from clinical promise to sustainable real-world availability. 👉 Read the full article:

Publisher name CBS News, 60 Minutes The article examines how approved genetic therapies can offer curative outcomes for rare and severe diseases while carrying price tags that reach several million dollars per dose. It highlights the structural drivers behind these costs, including complex manufacturing, small patient populations, and reimbursement challenges, raising questions about long-term sustainability and equitable access within healthcare systems. 👉 Read the full art

Publisher name World Economic Forum Global investment in regenerative medicine infrastructure is increasingly framed as an economic and health system priority rather than a niche scientific endeavor. The article highlights how manufacturing capacity, skilled workforce development, regulatory readiness, and cross-border coordination are now critical bottlenecks for scaling cell and gene therapies worldwide. It positions infrastructure planning as essential to ensuring equitabl

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 in Cytotherapy (Review Article) Asia’s CGT sector continues to expand with fast-track pathways in Japan, Korea, China and Singapore while India and Thailand advance through flexible access models and domestic innovation. Approvals for CAR-T, stem cell and gene therapies are rising and pricing differences highlight how local manufacturing and regulatory maturity shape affordability and access. Regional collaboration and shared infrastructure are identified as key lev

Published on: University of Colorado Boulder Scientists have found that manipulating cellular storage hubs called P bodies can rewind mature cells to a more flexible state and push them toward hard-to-generate lineages such as germ-cell-like or totipotent-like cells. The study shows how releasing specific RNAs from these structures reshapes cell identity, opening new possibilities for fertility treatment, organ regeneration, and early-stage disease modeling. 👉 Read the full

Published on Endpoints News The FDA has unveiled a new “plausible mechanism pathway” to speed access to personalized gene editing therapies, inspired by the Baby KJ case. The approach aims to support rare disease treatment by allowing alternative trial designs while maintaining regulatory oversight. 👉 Read the full article: FDA unveils new path to speed personalized therapies, inspired by Baby KJ Disclaimer Images used are for illustrative purposes only and do not depict act

Published on Fierce Biotech A patient in Intellia’s phase 3 trial of the CRISPR therapy nex-z has died following severe liver toxicity, prompting the FDA to keep both studies on hold. The company is investigating the liver signal and developing risk mitigation steps while monitoring all previously treated patients. 👉 Read the full article: Patient dies after receiving Intellia's CRISPR therapy Disclaimer Images used are for illustrative purposes only and do not depict actual

Published on University of Colorado Boulder CU Boulder scientists have discovered that P bodies act as organized RNA storage hubs that steer how stem cells decide their identity. By unlocking these structures, researchers were able to revert mature cells to earlier developmental states and generate germ-cell-like and totipotent-like cells with high efficiency. 👉 Read the full article: Scientists Discover New Way to Shape What a Stem Cell Becomes Disclaimer Images used are fo

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

By Dr. Samuel Ting Chuo Yew President, Sarawak Society for Cell and Gene Therapy Research (SSCTR) The Ministry of Health Malaysia and the National Pharmaceutical Regulatory Agency (NPRA) have recently issued two landmark documents that define the national framework governing the use, research, and regulation of advanced cellular and genetic therapies in Malaysia. These are the “Guidance Document and Guidelines for Registration of Cell and Gene Therapy Products (CGTPs)” publis

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,...