Holoclar (GPLSCD01) / Holostem, Chiesi - LARVOL DELTA

Correlation of the Eosinophilic Esophagitis Histology Scoring System With Peak Esophageal Eosinophil Counts: Pooled Analysis From the EoE KIDS and LIBERTY EoE TREET Studies (CAP 2026) - P3 | "Falk Pharma, Ferring, GI Reviewers, GSK, Holoclara, Invea, Knightpoint, LucidDx, Nexstone Immunology/Uniquity, Nutricia, Parexel/Calyx, Phathom, Regeneron, Revolo, Robarts/Alimentiv, Roivant, Sanofi, Shire/Takeda, Target RWE, Third Harmonic Bio, and Upstream Bio; and received research support from Adare/Ellodi, Allakos, Arena/Pfizer, AstraZeneca, Celgene/Receptos/BMS, Celldex, Dr. In this pooled analysis of 2 phase-3 EoE studies, strong correlations between EoEHSS grade/stage total scores and PEC were identified at W16/24 and W52. The EoEHSS also captured important histopathologic changes unrelated to eosinophil inflammation, valuable for disease diagnosis and monitoring in children and adults with EoE receiving dupilumab. Supported Collins received consulting fees from Alimentiv, Arena/Pfizer, AstraZeneca, Calypso Biotech, EsoCap Biotech, GSK, Receptos/Celgene/Bristol Myers Squibb, Regeneron Pharmaceuticals Inc, Sanofi, and Shire (a Takeda company)."

Retrospective data • Eosinophilic Esophagitis • Gastrointestinal Disorder • Immunology • Inflammation

Dupilumab Improves Histopathologic Aspects of Eosinophilic Esophagitis: Results of a Pooled Analysis From the EoE KIDS and LIBERTY EoE TREET Phase 3 Studies (CAP 2026) - P3 | "Falk Pharma, Ferring, GI Reviewers, GSK, Holoclara, Invea, Knightpoint, LucidDx, Nexstone Immunology/Uniquity, Nutricia, Parexel/Calyx, Phathom, Regeneron, Revolo, Robarts/Alimentiv, Roivant, Sanofi, Shire/Takeda, Target RWE, Third Harmonic Bio, and Upstream Bio; and research support from Adare/Ellodi, Allakos, Arena/Pfizer, AstraZeneca, Celgene/Receptos/BMS, Celldex, Dr. In this pooled analysis of 2 phase-3 studies, dupilumab improved histopathologic abnormalities of EoE during treatment initiation, compared with placebo (W16/24) and maintenance (W52) in patients with EoE. Supported Collins received a consulting fee from Alimentiv, Arena/Pfizer, AstraZeneca, Calypso Biotech, EsoCap Biotech, GSK, Receptos/Celgene/Bristol Myers Squibb, Regeneron Pharmaceuticals Inc, Sanofi, and Shire (a Takeda company). Chehade received a consulting fee from Adare Pharma Solutions/Ellodi Pharmaceuticals, Allakos, Apogee Therapeutics, AstraZeneca, Bristol Myers Squibb, Nexstone..."

P3 data • Retrospective data • Eosinophilic Esophagitis • Gastrointestinal Disorder • Immunology

Current Landscape and Future Prospects of Corneal Regenerative Medicine. (PubMed, Ophthalmol Ther) - "Regulatory approval of commercial products, such as Holoclar and Nepic, confirms the potential of standardized regenerative products...Overall, regenerative medicine is reshaping corneal therapeutics, offering effective alternatives to conventional transplantation with reduced donor dependence and improved safety. Future work must focus on long-term safety, cost-effectiveness, and equitable global access to realize its full clinical potential."

Journal • Review • Dry Eye Disease • Fibrosis • Gene Therapies • Immunology • Inflammation • Ocular Inflammation • Ophthalmology • Transplantation

Selection of qualified treatment centrum for cell therapy (ESGCT 2024) - "Cell therapy Holoclar was developed by such a smaller company and was approved in EU for use in adult patients with moderate-to-severe limbal stem-cell deficiency caused by trauma (burns, including chemical burns to the eyes)...The flexibility to find solutions during challenging times of COVID or team changes (maternity leave, sickness, relocation) is required to maintain accessibility to treatment. Management of more complex processes and a broader team was effectively managed and coordinated by the support team, Medasol."

Gene Therapies • Mood Disorders • Novel Coronavirus Disease • Thermal Injury

HOLOCORE lessons: biological and clinical insights from a pan-European phase IV clinical trial of Holoclar® for treating Limbal Stem Cell Deficiency (ESGCT 2025) - No abstract available

Clinical • P4 data

SELECTION OF QUALIFIED TREATMENT CENTRUM FOR CELL THERAPY (ASGCT 2025) - "In ophthalmology, Holoclar, a tissue-engineered therapy for moderate-to-severe limbal stem-cell deficiency (LSCD) due to trauma, is an example of a start-up innovation, which lacks wider acknowledgement... Effective QTC selection requires evaluating treatment capabilities, patient pathways, and institutional support. Strong center management and adaptability are essential for overcoming logistical and operational challenges. The structured coordination by Medasol facilitated a streamlined approach to patient access and treatment continuity."

Corneal Abrasion • Mood Disorders • Ophthalmology

Status and prospects for the development of regenerative therapies for corneal and ocular diseases. (PubMed, Regen Ther) - "Based on these advances, four regenerative therapy products for corneal epithelial disease, each employing distinct cell sources and culture techniques, have been commercialized since the regulatory approval of Holoclar® in Italy as a regenerative therapy product for corneal epithelial disease in 2015...The product was approved in Japan as Vyznova® in 2023. The development of regenerative therapies for retinal and ocular surface diseases is actively being pursued, and these therapies use somatic stem cells and pluripotent stem cells (PSCs), especially induced pluripotent stem cells (iPSCs). Accordingly, the eye field is anticipated to play a pioneering role in regenerative therapy development going forward."

Journal • Review • Ophthalmology

Illustrative Potency Assay Examples from Approved Therapies. (PubMed, Adv Exp Med Biol) - "In this chapter, we present and discuss actual potency assays used for the release of representative commercial ATMP from each category of products (namely, KYMRIAH (tisagenlecleucel), Holoclar (limbal epithelial stem cells), and PROCHYMAL/RYONCIL™ (remestemcel-L)). We also examine concerns related to the biological relevance of selected potency assays and challenges ahead for harmonization and broader implementation in compliance with current quality standards and regulatory guidelines."

Journal • Gene Therapies

HOLOCORE-FU: Follow-up Study After ACLSCT for Restoration of Corneal Epithelium in Patients With LSCD Due to Ocular Burns (clinicaltrials.gov) - P=N/A | N=47 | Completed | Sponsor: Holostem Terapie Avanzate s.r.l. | Active, not recruiting ➔ Completed

Trial completion • Thermal Injury • Transplantation

HOLOCORE-FU: Follow-up Study After ACLSCT for Restoration of Corneal Epithelium in Patients With LSCD Due to Ocular Burns (clinicaltrials.gov) - P=N/A | N=48 | Active, not recruiting | Sponsor: Holostem Terapie Avanzate s.r.l. | Recruiting ➔ Active, not recruiting | N=70 ➔ 48

Enrollment change • Enrollment closed • Thermal Injury • Transplantation

Regenerative medicine for the corneal epithelium : Cell therapy from bench to bedside (PubMed, Ophthalmologie) - "In these situations, transplantation of cultured limbal epithelial cells on an amniotic membrane or fibrin gel as substrate (Holoclar®) can help to regenerate the corneal surface...The reprogramming of such cells can be achieved with the help of transcription factors. In addition, work is being done on biosynthetic or synthetic matrices, which not only serve as substrate material for the transplantation but also support the functional properties of these cells (self-renewal, corneal epithelial-typical phenotype)."

Journal • Review • Bone Marrow Transplantation • Thermal Injury • Transplantation

HOLOCORE: Efficacy and Safety of Autologous Cultivated Limbal Stem Cells Transplantation (ACLSCT) for Restoration of Corneal Epithelium in Patients With Limbal Stem Cell Deficiency (clinicaltrials.gov) - P4 | N=70 | Completed | Sponsor: Holostem Terapie Avanzate s.r.l. | Active, not recruiting ➔ Completed

Trial completion • Thermal Injury • Transplantation