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Articles by Kadeja Johnson
Stem Cell Awareness Day 2026: exploring iPSCs, MSCs and tissue-specific stem cells
Stem Cell Awareness Day – the second Wednesday of October, is a global initiative dedicated to raising awareness about the use of stem cells in medicine. Last year, we looked at four ways stem cells are being applied in medicine, including treating genetic disorders, advancing care for neurodegenerative diseases, restoring insulin production and regenerating the heart.
Cell therapy weekly: US$9.6 million grant toward digital cell therapy manufacturing
This week: A contract development and manufacturing organization (CDMO) is a granted US$9.6 million towards supporting its digital cell therapy manufacturing operations, a clinical-stage company comes to the difficult decision to discontinue two of its allogeneic CAR-T programs, and a new partnership forms to support cell and gene therapy supply pathways in the EU and UK.
What’s coming up at Cell and Gene Therapy International Europe 2026?
Explore the diverse streams that Cell and Gene Therapy International Europe 2026 has to offer, located at the Hotel Palace Berlin (Berlin, Germany) from 3–5 November 2026. This year, the Cell and Gene Therapy International Europe event heads to Berlin, bringing two new streams through co-located events: Exosomes Europe and LNPs Europe.
“Plug-and-play” CAR-T cells offer flexibility against changing tumors
Created via canva.com Original story from City of Hope (CA, USA). Chimeric antigen receptor (CAR) T cell therapy genetically reprograms a patient’s immune cells to seek out and kill cancer. The personalized approach has revolutionized treatment, yet today’s therapies remain largely “hardwired” and unable to adapt after the rewired cells are restored to the patient’s bloodstream.
Cell therapy weekly: experimental solid cancer CAR-T approved for clinical testing
Credit: @ktsimage via canva.com This week: The US Food and Drug Administration (FDA; MD, USA) has approved clinical testing of an experimental CAR-T therapy in adults with colorectal cancer and children with solid tumors. Cellectis (Paris, France) has pivoted from allogeneic CAR-T therapies to focus on in vivo gene editing therapies for chronic diseases. Plus, Glidepath Health (NY, USA) has emerged from stealth mode with an AI-driven platform for managing cell and gene therapy logistics.
Novel prime assembly method achieves large-scale gene editing
Original story from Boston Children’s Hospital (MA, USA). A novel gene editing method, prime assembly, demonstrates precise insertion of gene-length DNA fragments into living cells, offering a potential universal therapeutic approach that could correct multiple disease-causing mutations with a single treatment. Genomic editing holds great potential yet continues to have its limitations.
What’s coming up at Biotech Week Boston 2026?
Explore carefully curated discussions, sessions, tracks and events brought together in one location: Biotech Week Boston will bring together world-leading biotech experts for one week. Biotech Week Boston is fast approaching – a week promising to be filled with industry experts, invaluable conversations and networking opportunities ready to be nurtured.
Tiny gene activation platform, TIGRa, easily packs into AAVs to deliver in vivo gene expression
Credit: Adapted from Vesvocrea via Canva.com TIGRa, a novel gene activation platform half the size of its CRISPR-based cousin, enables researchers to deliver gene activation into the cells of an animal model using AAVs. A team of researchers from Stanford Medicine (CA, USA) led by Yang Sun, has developed a gene activation platform, TIGRa, that is less than half the size of the Cas9-based CRISPR activator (CRISPRa) platforms, making it better suited for in vivo delivery.
Cell therapy weekly: regulatory applications submitted for Huntington’s gene therapy
Credit: @nitsuki via canva.com This week: After previously receiving Breakthrough Therapy and RMAT designations from the US Food and Drug Administration (FDA; MD, USA), uniQure (Amsterdam, The Netherlands) has submitted regulatory applications for its Huntington’s disease gene therapy in the US and UK.
In vivo CAR-T: emerging therapeutic programs
Explore in vivo CAR-T programs currently in clinical trials and several that have been acquired by major pharmaceutical companies, highlighting the growing interest in in vivo CAR-T. Previously, we examined the emergence of in vivo CAR-T therapies, exploring the differences between conventional ex vivo and in vivo CAR-T manufacturing.
CAR-T therapy shows promise against aggressive brain cancer
Created via Canva.com. Original story from Yale School of Medicine (CT, USA) A CAR-T therapy targeting B7-H3 showed promising results in treating recurrent glioblastoma, with over half of patients experiencing tumor stabilization or reduction. A new Phase I clinical trial has found that an emerging immunotherapy is promising for the treatment of recurrent glioblastoma, a highly aggressive and fatal brain cancer.
Cell therapy weekly: first patient dosed in Phase III geographic atrophy gene therapy trial
Credit: @berkay08 via Canva.com This week: Biotechnology company Ocugen, Inc. (PA, USA) announced that it has dosed the first patient in its global Phase III trial assessing a gene therapy for geographic atrophy (GA) secondary to dry AMD. Complement Therapeutics GmbH (Munich, Germany) has completed its Phase I/II Cohort 2 dosing of its gene therapy for GA, progressing to Cohorts 3 and 4. Plus, Genprex, Inc.
3D Printing at capillary scale using a hybrid approach
Created via Canva.com. Original story from the University of Notre Dame (IN, USA). A hybrid 3D printing technique using machine learning demonstrates potential for creating tiny blood vessels as small as capillaries, offering a step closer to printing fully functional organs for transplant patients. More than 100,000 people are awaiting an organ transplant in the United States, with a new candidate added to the list every 10 minutes.
Billion-dollar mergers and acquisitions in cell and gene therapy
Explore 2026's billion-dollar cell and gene therapy acquisitions, including in vivo CAR-T programs, epilepsy cell therapies, and in vivo gene placement systems. The cell and gene therapy landscape is constantly adapting, growing and finding its footing in a world where science and personalized medicine intersect so deeply. Conversations in the field have always emphasized harmonization and shared knowledge with the intention of bringing life-changing medicinal products to patients.
3D-printed spheroids enable dual bone and vascular tissue development
Original story from the Pennsylvania State University (PA, USA). An interdisciplinary Penn State team has developed a bioprinting technique using microRNA-modified stem cell spheroids that simultaneously regenerate bone tissue and facilitate vascularization, offering new treatment options for severe bone trauma and infections.
Cell therapy weekly: FDA approves glycogen storage disease type 1a gene therapy
This week: Sangamo Therapeutics (CA, USA) auctions off its main assets, Andelyn Biosciences (OH, USA) and Queen’s University (Ontario, Canada) partner to advance a lysosomal storage disorder gene therapy program and the US Food and Drug Administration (FDA; MD, USA) approves a gene therapy for glycogen storage disease type 1a (GSD1a).
Coral-inspired scaffold offers new strategy for bone regeneration
Original story from Sichuan University (Sichuan, China). Researchers have developed a coral-inspired 3D-printed scaffold that reprograms immune cells to promote bone regeneration in patients with steroid-induced bone death. Steroid-induced osteonecrosis of the femoral head (SONFH) is one of the leading causes of non-traumatic osteonecrosis.
SHIFTERS: using ultrasound to make solid tumors visible to immune cells
Original story from the University of Southern California Viterbi School of Engineering (CA, USA). Researchers have created a system that uses ultrasound to temporarily mark solid tumors, which have long resisted treatment, enabling CAR-T immune cells to recognize and attack the marked tumors. For more than a decade, CAR-T cell therapy has been one of the biggest breakthroughs in cancer medicine.
From first cases to real access: what Greece can teach cell and gene therapy
Drawing on expert perspectives from cell and gene therapy, health technology commercialization and European access, this column uses Greece as a case study for what it takes to deploy advanced therapies beyond the obvious centers. It is reasonably easy to talk about the future of cell and gene therapy from places where the infrastructure already exists or has at least been in place (for better or worse) for a while.
Cell therapy weekly: specialized partner launches to overcome cell therapy challenges
This week: IQVIA (London, UK) and Medera (London, UK) announced a collaboration aiming to accelerate the development of cardiac gene therapies and NueCell Bio (CA, USA) has launched as a specialized translational partner aiming to overcome challenges in advanced therapies. Plus, the US Food and Drug Administration (FDA; MD, USA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Aspen Neuroscience‘s (CA, USA) Parkinson’s disease cell therapy ANPD001.
Gene therapies in clinical trial: three promising candidates
From silencing disease-causing genes to restoring enzyme function and resetting retinal gene networks, three gene therapies in clinical trials are demonstrating how genetic medicine could transform outcomes for patients with inherited muscular dystrophy, metabolic disorders and progressive blindness. Gene therapies harness the ability to correct, replace or modify genes to address the underlying genetic causes of disease.
How jostling cells could offer a new approach to engineer blood vessel networks
Original story from Massachusetts Institute of Technology (MA, USA). The mechanical stretching of blood vessels could enable scalable production of functional artificial organs. Tissue engineers are finding ways to grow living organs and tissues from cells, with the aim of replacing diseased and damaged counterparts in the body. Scientists have successfully grown artificial muscles, livers, kidneys, skin, and other tissues.
Cell therapy weekly: US$1 billion acquisition to further foster cell therapy services
This week: A strategic partnership has been announced to support the development of a dual-targeting in vivo CAR-T therapy, and a developer and supplier of cell processing tools and services is to be acquired for US$1 billion. Plus, Immitra Bio (Plan-les-Ouates, Switzerland) secured US$3 million in pre-seed funding to support its in vivo scalable gene editing platform.
I never planned to work in regulatory affairs – and it’s the best career move I ever made
Share on X Share on Facebook Share on Linkedin Send to a friend Refer a friend Favourite Behind every safe and effective healthcare product stands the critical work of regulatory affairs professionals. In this column, Patrice Faloye, a regulatory affairs professional with a breadth of experience, shares her journey into regulatory affairs, discussing the pivotal moments that shaped her career path.
Cell therapy weekly: advances in personalized stem cell therapy for Parkinson’s disease
This week: A collaborative research project looks to advance the development of a gene therapy for a fatal lysosomal storage disorder and a trial assessing a personalized stem cell therapy for Parkinson’s disease demonstrates positive interim results. Plus, the US Food and Drug Administration (FDA; MD, USA) granted supplemental approval for Casgevy in pediatric sickle cell disease and β-thalassemia.
Promising preclinical safety profile for chronic spinal cord injury stem cell strategy
Original story from the International Society for Stem Cell Research. Preliminary findings presented at the International Society for Stem Cell Research annual meeting (Montréal, Canada; 8–11 July 2026; ISSCR) demonstrate promising preclinical safety profile for chronic spinal cord injury (SCI).
Four AI-based technologies being harnessed for regenerative medicine
Here, we explore the integration of artificial intelligence into regenerative medicine, showcasing four ways this advanced technology is being harnessed in the field. Previously, we have looked at ways machine-learning and artificial intelligence (AI) have been integrated in gene therapy developments.
Novel gene therapy harnesses brain’s glymphatic system for targeted delivery
Original story from the University of Rochester Medicine (URochester Medicine; NY, USA). Researchers have developed a gene therapy platform using the brain’s glymphatic system to deliver therapeutic genes to glial cells throughout the brain, bypassing the blood–brain barrier for safer, more effective treatments. A new study describes a gene therapy strategy that uses the brain’s own glymphatic transport system to distribute engineered viral vectors throughout the brain.
Phase I/II Parkinson’s disease cell therapy trial achieves primary endpoint
Original story from Lund University (Lund, Sweden). Researchers presented findings from the STEM-PD Phase I/II trial at the International Society for Stem Cell Research annual meeting (Montréal, Canada; 8–11 July 2026; ISSCR). The trial successfully achieved its primary endpoint, demonstrating the feasibility of transplanting stem cell-derived dopamine progenitor cells into the brain of Parkinson’s disease patients.
Cell therapy weekly: investigational AAV-based gene therapy for FSHD completes enrollment
This week: The US Food and Drug Administration (FDA; MD, USA) cleared an Investigational New Drug (IND) application for a novel adoptive cell therapy and a first-in-human trial assessing an investigational adeno-associated virus (AAV)-based epigenetic gene therapy, indicated for facioscapulohumeral muscular dystrophy (FSHD). This completes enrollment and dosing in its dose-escalation stage.
Lab-grown retinal cells may hold the potential to restore retinal function
Original story from Duke University Pratt School of Engineering (NC, USA). Biomedical engineers at Duke University have used induced pluripotent stem cells (iPSCs) to grow specialized blood vessel cells critical to retinal health for the first time. When injected into mouse models of retinal disease, these “retinal endothelial cells” integrated into the damaged tissue to regenerate blood vessels and restore retinal function.
Cell therapy weekly: association launches to address challenges in advanced cell therapies in Europe
This week: a biotechnology company has been awarded an US$8 million grant to support the clinical development an AAV immune-gene therapy for brain cancers, the US Food and Drug Administration (MD, USA) has approved an allogeneic cell therapy to address graft versus host disease (GVHD) in adult patients with hematologic malignancies.
Unlocking the potential of regenerative medicine in Europe: an interview with Tanja Xenia Pedersen
In this interview, Tanja Xenia Pedersen, Head of Biomedical Research at the Novo Nordisk Foundation (Hellerup, Denmark), dives into a recent report on the current state of regenerative medicine in Europe, the gaps, and the opportunities. Tanja discusses why Europe continues to struggle with translating strong science into therapies, and how a more coordinated, system-level approach could help unlock progress.
eBook | Viral and non-viral gene delivery approaches
Download peer-reviewed content and expert insights in our latest eBook. Gene therapies have revolutionized modern medicine by offering the potential to treat and cure diseases at their genetic root. Traditionally, these therapies have relied on viral vectors – including lentiviruses, adenoviruses and adeno-associated viruses (AAVs) – to deliver therapeutic genetic cargo to target cells and tissues.
Boosting CAR-T therapy success in difficult-to-treat blood cancers
Original story from the University of Maryland School of Medicine (UMSOM; MD, USA). Researchers have demonstrated how blocking a specific protein keeps CAR-T cells fighting cancer longer, potentially improving outcomes for blood cancer patients. UMSOM Researchers have identified a target that may improve the response of CAR T-cell therapy, a treatment for patients with recurrent or difficult-to-treat blood cancers.
Cell therapy weekly: BLA submission planned for Huntington’s gene therapy AMT-130
This week: uniQure (Amsterdam, Netherlands) has announced plans to submit a Biologics License Application (BLA) for AMT-130 in Huntington’s disease. Ernexa Therapeutics (MA, USA) has confirmed that it remains on track to submit an IND for its induced mesenchymal stem cell therapy and is well-positioned to initiate its first-in-human Phase I clinical trial.
A comprehensive report details strategic roadmap towards advancing regenerative medicine in Europe
Novo Nordisk Foundation (Hellerup, Denmark) releases a strategic roadmap to advance European regenerative medicine. The Novo Nordisk Foundation, an independent enterprise foundation, has released a comprehensive report outlining a strategic pathway to position Europe as a global leader in regenerative medicine.
Advanced iPSC bioprocessing: process improvements for scalable and quality-focused cell production
In this interview, Sarah Gilpin, Principal Scientist in Process Development Services at Sartorius (MA, USA), walks us through the early phases of stem cell therapy development – from proof-of-concept science to therapies that now offer hope to patients. She explores the critical considerations for developing a cell therapy, including ensuring functionality, navigating regulatory requirements, and defining quality standards.
World Sickle Cell Day 2026: current progress and developments
World Sickle Day, observed annually on June 19th, aims to raise awareness about sickle cell disease. Around 19,000 people in the UK and 100,000 people in the United States live with sickle cell disorder. In 2021, the World Health Organization reported that an estimated 7.74 million people live with sickle-cell disease globally.
Novel gene therapy platfrom shows promise in Duchenne muscular dystrophy preclinical model
Original story from The University of Texas MD Anderson Cancer Center (TX, USA). A novel platform utilizing extracellular vesicles (EVs) to deliver messenger RNA (mRNA) encoding the DMD gene safely restored dystrophin production and improved muscle function in preclinical models of Duchenne muscular dystrophy.
Novel gene therapy platform shows promise in Duchenne muscular dystrophy preclinical model
Original story from The University of Texas MD Anderson Cancer Center (TX, USA). A novel platform utilizing extracellular vesicles (EVs) to deliver messenger RNA (mRNA) encoding the DMD gene safely restored dystrophin production and improved muscle function in preclinical models of Duchenne muscular dystrophy.
New SMArT platform enhances safety of gene editing in hematopoietic stem cells
Original story from San Raffaele Telethon Institute for Gene Therapy (SR-Tiget; Milano, Italy). A team of researchers led by Luigi Naldini at the SR-Tiget has developed a new strategy to significantly improve the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells, potentially overcoming one of the major barriers limiting broader clinical application of genome editing therapies.
Exploring MSC-derived exosomes for tissue repair and regeneration
MSC-derived exosomes deliver stem cell benefits without the cells themselves – a curated review of recent research highlights their emergence as transformative next-generation therapeutics. MSCs are a crucial component of the body's self-healing mechanisms, responding to injury and infection through multiple mechanisms.
Cell therapy weekly: FDA draft guidance to help accelerate cell and gene therapies
This week: the US Food and Drug Administration (FDA; MD, USA) issued a draft guidance aiming to accelerate cell and gene therapies and a UK-based biotech company was awarded US$2.8 million in grants to support the development of its gene-control platform. Plus, Legend Biotech (NJ, USA) presented clinical data on two of its CAR-T therapies at the American Society of Clinical Oncology Annual Meeting (ASCO; May 26–June 2, 2026; IL, USA).
Macrophage cell therapy demonstrates success in advanced liver disease trial
Original story from The University of Edinburgh (Scotland). A new type of cell therapy shows promise as the first treatment for advanced liver disease, following results from a clinical trial. Patients with the condition who were treated with the cell therapy had a significantly lower risk of death or need for a liver transplant after four years compared with those who received standard medical care.
Experimental gene therapy boosts neuron resilience to neurodegeneration
Original story from the University of California San Diego School of Medicine (CA, USA).
3D-printed trays accelerate gut organoid production for tissue repair
Original story from Cincinnati Children’s Hospital Medical Center (OH, USA). A new 3D-printed scaffold tray could help accelerate the production of gut organoids, offering solutions for scalable tissue repair. Thanks to special 3D-printed scaffolding trays designed by experts at Cincinnati Children’s, researchers can now produce larger versions of functional human gut organoids twice as fast as previous methods − and these organoids grow their own nerve cells.
Advancing brain tumor treatment with cytokine-armoured CAR-T cells
Original story from UCLA Health Jonsson Comprehensive Cancer Center (CA, USA). A novel cytokine-armored CAR-T cell therapy for aggressive brain tumors has demonstrated reduced side effects in preclinical studies.
Cell therapy weekly: KMCAR™ T-cell therapy enters Phase I trial
This week: An Australian biotechnology company has opened its Phase I trial to assess a novel CAR-T therapy for multiple myeloma, while Protara Therapeutics (NY, USA) announced positive 12-month data from its Phase II trial evaluating a cell therapy for non-muscle invasive bladder cancer (NMIBC). Plus, a strategic partnership has been formed to support late-stage manufacturing of an AAV9 gene therapy for a neurodegenerative disorder.
Navigating biomarker discovery in translational research
A successful approach to biomarker discovery and analysis depends on multiple factors, including effective sample collection and advanced technologies for data interpretation. In this interview, Professor Melanie Bailey shares her expert perspective on the critical role of sample collection and explains how her lab tackles biomarker discovery in translational research.
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