Achieving market access for advanced therapy medicinal products (ATMPs) requires navigating national reimbursement routes. This is said to be particularly challenging for academic developers and small- and medium-sized enterprises, increasingly involved in ATMP development. We aimed to identify reimbursement routes for ATMPs in the Netherlands and assess how ATMPs obtained reimbursement.

Allogeneic mesenchymal stromal cell (ASC) therapy is a potential treatment option in patients with ischemic heart failure (HF). We aimed to investigate the effect of allogeneic Cardiology Stem Cell Center Adipose tissue derived mesenchymal Stromal Cell product (CSCC_ASC) by joining data from the international SCIENCE and the Danish ASC trial.

This review evaluates CRISPR-based strategies for myelin regeneration in peripheral demyelinating disorders, with a focus on Guillain - Barré syndrome (GBS) and Charcot - Marie - Tooth disease type 1A (CMT1A). It aims to identify current therapeutic approaches, delivery systems, and gaps in the literature.

Despite remarkable progress in the clinical management of hepatocellular carcinoma (HCC), complications such as heterogenicity of HCC cells and characteristics of cancer stem cells (CSCs) contribute to frequent relapse and treatment resistance. Lack of proper models has limited developing novel approaches to evaluate innovative therapeutic settings to overcome these challenges. To address current limitations for mimicking cancer microenvironments; various three-dimensional (3D) platforms have been developed, such as tumoroids, patient-derived xenograft (PDX) models, microfluidics-based cancer chip devices, and bio-printed microtissues. Notably, 3D bio-printing technology has enabled researchers to produce scalable complex multicellular tissue models with accurate matrix composition and cellular organization. These microtissues provide precise platforms studying liver regeneration pathways, fibrosis reversal, and cellular responses to therapeutic interventions. This paper, a systematic literature search of databases covering publications from 2000 to 2025, uniquely highlights how these advances enable precise recapitulation of tumor heterogeneity and microenvironmental complexity, thereby offering transformative platforms for personalized drug screening and elucidating mechanisms of liver tissue repair and regeneration. We discussed current challenges and future directions for translating 3D bio-printed liver models into clinically relevant tools, potentially accelerating therapeutic advances and their potential applications in regenerative medicine in terms of personalized medicine and drug screening.

Cell-based therapies have emerged as a promising frontier in regenerative medicine, offering potential treatments for various degenerative, autoimmune, and genetic disorders. However, their clinical translation is accompanied by complex ethical and regulatory challenges that must be addressed to ensure patient safety, equitable access, and scientific integrity. This review explores the intricate landscape of global regulatory frameworks governing cell-based therapies, highlighting key obstacles such as inconsistent international guidelines, prolonged approval timelines, and ethical considerations. The lack of regulatory harmonization often impedes innovation and delays the availability of advanced treatments to patients in need. In addition, stringent requirements can pose barriers for emerging biotech start-ups, limiting competition and technological advancements. Despite these challenges, regulatory frameworks also present opportunities to foster innovation, enhance patient safety, and streamline drug approval process. Collaborative efforts between policymakers, regulatory bodies, industry leaders, and research institutions are crucial to developing standardized yet adaptable guidelines that accommodate scientific progress while maintaining stringent safety protocols. By fostering a regulatory ecosystem that encourages innovation without compromising safety, stakeholders can unlock the potential of cell-based therapies, paving the way for their widespread adoption and integration into clinical practice.

Stroke-related long-term disability is primarily due to impaired motor function. Rehabilitation efforts have traditionally focused on central strategies while ignoring the affected muscles. Regenerative medicine approaches have emerged as a promising option for treating various conditions, including muscular disorders. The present study aims to compare the effects of intramuscular injections of mesenchymal stromal cells (MSCs) and Platelet-rich plasma (PRP) on motor recovery in poststroke survivors.

Latest developments in the field of Advanced Therapy Medicinal Products and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in September 2025.

Smart hydrogels have become precision platforms that interact with complex biological cues. We formalize a 2025 definition, materials that sense a clinically relevant cue and reproducibly execute a specified, reversible function under physiologic conditions, and introduce a unified, feature-based, three-tier framework: Responsive (open-loop cue and response), Adaptive (multi-cue or stateful), and Intelligent (closed-loop sense, decide, and act). This review captures momentum from 2020 to 2025, a period marked by clinical and innovative breakthroughs, FDA-cleared formulations, and integration of advanced technologies, including AI-assisted design, fourth-dimensional (4D) bioprinting, and biohybrid interfaces. We spotlight cutting-edge developments in programmable degradation, self-healing, and multi-stimuli responsiveness, alongside emerging hydrogel fabrication strategies such as nanoparticle (NP)-laden bioinks and light-activated crosslinking. Although barriers to regulation and translation remain, cross-disciplinary efforts with a sustainability- and ethics-first mind-set are redefining these materials' capabilities. Smart hydrogels are no longer just innovative, researchers in tissue engineering and regenerative medicine are actively redefining both their clinical potential and what it means for a material to be "smart."

Latest developments in the field of Advanced Therapy Medicinal Products and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in July 2025.

Clouston syndrome is a rare autosomal dominant form of hidrotic ectodermal dysplasia that presents significant challenges due to its multisystemic manifestations and limited therapeutic options. This review explores the functional role of stem cells in the treatment and management of Clouston syndrome, highlighting advancements in regenerative medicine and stem cell therapy. Stem cell types such as mesenchymal stem cells (MSCs), epidermal stem cells (EpSCs), and induced pluripotent stem cells (iPSCs) have shown promise in regenerating ectodermal derivatives like skin, hair, and nails. Gene-editing technologies, including Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR-Cas9), can correct pathogenic mutations in the gene. Emerging techniques in tissue engineering and three dimensional (3D) bioprinting are also discussed, focusing on their potential to create patient-specific, bioengineered constructs. Clinical trials in related genetic disorders provide evidence for the feasibility of these approaches in improving patient outcomes. However, challenges such as immune rejection, ethical concerns, and stem cell production scalability remain barriers. Addressing these issues requires interdisciplinary collaboration, sustained funding, and targeted research. This review underscores the transformative potential of regenerative medicine. It highlights the need for continued efforts to develop curative strategies for treatment, integrating stem cell-based therapies, gene editing, and bioengineering.

Latest developments in the field of Advanced Therapy Medicinal Products and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in October 2025.

Three-dimensional (3D) bioprinting has emerged as a promising biofabrication strategy for bone regeneration, offering unprecedented control over the spatial distribution of cells, biomaterials, and bioactive cues. By enabling the production of anatomically customized grafts with microarchitectural complexity and biological functionality, bioprinting holds potential to overcome limitations associated with autografts, allografts, and synthetic bone substitutes. This review provides a comprehensive synthesis of recent advances in bioprinting technologies, bioink design, and recent and studies targeting bone tissue engineering. Despite encouraging preclinical outcomes, the field faces significant challenges, including limited mechanical performance, control over cellular microenvironments, lack of standardization, and trade-offs between printing resolution and scalability. Notably, no clinical trials have yet investigated bioprinted bone constructs, reflecting the translational hurdles that persist. We critically discuss these gaps and propose strategic directions. Collectively, this review underscores the feasibility of 3D bioprinting for bone regeneration while highlighting the key scientific and technical milestones needed to transition from bench to bedside.

Mesenchymal stem/stromal cells (MSCs) are widely investigated for regenerative therapies, yet current expansion methods often compromise their stem-like properties, limiting large-scale clinical translation. We aimed to evaluate whether a novel hydrogel-based Bio-Block® platform preserved intrinsic adipose-derived MSC (ASCs) phenotype and secretome compared with conventional systems.

Hepatocyte transplantation has gained importance as an alternative treatment to orthotopic liver transplantation for end-stage liver disease. This study explores the modification of the hepatocyte matrix implant (HMI) procedure by replacing islet cells with conditioned medium (CM) derived from human umbilical cord-MSC (hUC-MSC) supplementation to alleviate liver cirrhosis in a rat model.

Mesenchymal stromal cell (MSC) therapy is an injectable, orthobiologic treatment for patients with knee osteoarthritis (OA). The purpose of this narrative review is to evaluate the literature on allogeneic MSCs used in knee OA patients and to report on clinical outcomes. A comprehensive literature search was performed using the following keywords: osteoarthritis, knee, stem cell transplantation, allogeneic or allogenic, human mesenchymal stem cells, and human mesenchymal stromal cells. There was no restriction on time, and subjective and objective clinical outcomes were reported. A total of 11 ( = 11) clinical trial studies were included. The most common allogeneic source was adipose tissue (AD-MSCs). Most studies included a control group and included participants with Kellgren-Lawrence grades of II-III or II-IV. The MSC dose used was highly variable. There were no significant adverse safety events. All studies reported an improvement in patient reported outcomes from baseline, with the most common observation being a durable reduction in pain for 6 to 24 months. There were positive effects of AD-MSCs on cartilage imaging in most studies, although outcomes were variable. Allogeneic MSC injections for knee OA resulted in improved OA related symptom scores, are safe, and provided patients with a reduction in pain at long-term follow-up.

Gene therapy has ushered in a new era for the treatment of inherited retinal diseases (IRDs). The approval of voretigene neparvovec-rzyl (Luxturna) for -associated retinal dystrophy marked a pivotal milestone, establishing proof-of-concept that gene addition can restore visual function in IRDs. However, the success of Luxturna is tempered by the reality that it applies to a narrow subset of IRDs, and that no other IRD gene therapy has thus far received regulatory approval. This review outlines the current landscape of IRD gene therapy, including trials for several forms of IRD including achromatopsia, choroideremia, Leber congenital amaurosis, X-linked retinitis pigmentosa, and X-linked retinoschisis. We highlight the central challenges facing the field: narrow gene- or variant-specific indications, vector limitations, and reliance on suboptimal clinical trial endpoints. The review also discusses emerging strategies - including dual AAV and split-intein vectors, non-viral delivery platforms, and precision gene editing technologies such as CRISPR, base editing, and prime editing. These innovations promise to expand therapeutic reach. Finally, we emphasize the need for improved regulatory frameworks and ethical considerations for gene-based therapies for IRD. The field now stands at a critical juncture, where the lessons of Luxturna can inform a more scalable, inclusive, and transformative future.

Despite the discovery, nearly 40 years ago, that mutations in the dystrophin gene were responsible for Duchenne muscular dystrophy (DMD), a cure for this devastating disease remains elusive. Considerable effort worldwide is focused on understanding DMD and devising treatments, including gene-, cell-, and pharmacologic-based therapies. More than 400 clinical trials for DMD and/or the related Becker muscular dystrophy (BMD) have been registered with clinicaltrials.gov, with many in various stages of completion, and more than 40 having been terminated or withdrawn. The failure of interventions in clinical trials represents a significant emotional burden for the entire DMD community. While some gene-based therapies are being approved, these can be expensive, and currently tend to target specific mutations. Several cell-based therapies and tissue engineering strategies are also currently in development. Of the many pharmacotherapies to address aspects of the pathophysiology of DMD, like preserving muscle fibers, enhancing regeneration, and increasing strength, glucocorticoids remain the most efficacious for attenuating the disease progression. Successful pharmacotherapies may enable patients to take advantage of perfected gene therapies when they eventually become available. Here, we explore the therapeutic merit of different pharmacotherapies currently under consideration and provide an update on recent advances in gene therapies for DMD.

Latest developments in the field of Advanced Therapy Medicinal Products and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in June 2025.

Latest developments in the field of Advanced Therapy Medicinal Products and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in August 2025.

Biomimetics offers promising tools to improve wound healing in difficult clinical conditions. Polynucleotides (PN) show high potential for tissue repair in oral and periodontal surgery, by relying on the body's inherent self-healing capabilities. The aim of the present study was to elucidate in vitro the effects of Odonto-PN (O-PN) and Regenfast (REG), two PN-based compounds, on oral tissue repair.

Latest developments in the field of Advanced Therapy Medicinal Products and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in May 2025.