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ClinicalTrials.gov Updates Phase 2 Trial Registration for iPSC-Derived Retinal Pigment Epithelium Cell Transplantation for Age-Related Macular Degeneration
ClinicalTrials.gov USA Overview Information regarding a Phase 2 clinical trial for iPSC-derived Retinal Pigment Epithelium (RPE) cell transplantation targeting Age-related Macular Degeneration (AMD) has been updated or newly registered o... -
Exosome Therapeutics Firm Unveils Advanced Platform, Pipeline Progress Targeting Regenerative Medicine and Enhanced Drug Delivery
Company Disclosures USA Overview An exosome-based therapeutics company has detailed significant pipeline advancements and its cutting-edge technology platform at a recent investor briefing. The presentation particularly emphasized exosom... -
Precision Gene Editing with Adenine Base Editors Restores Muscle Function in Childhood FSHD Model, Advancing Gene Therapy
bioRxiv Australia Overview This study presents a novel therapeutic strategy for childhood-onset Facioscapulohumeral Muscular Dystrophy (FSHD), an intractable genetic disorder. Researchers leveraged an iPSC-derived skeletal muscle model t... -
Century Therapeutics Pivots iPSC Platform to Type 1 Diabetes Beta Cell Therapy, Targeting 2025 IND with Off-the-Shelf Approach
Century Therapeutics USA Overview Century Therapeutics has announced a strategic realignment of its induced pluripotent stem cell (iPSC) platform, now exclusively dedicated to developing beta islet cell therapy for Type 1 Diabetes. The c... -
ARPA-H Launches AEGIS Project: Affordable Gene-Editing Therapies for Pediatric Immune Diseases via CRISPR and LNP Delivery
Princeton University USA Overview The ARPA-H THRIVE-funded AEGIS project is pioneering the development of affordable gene-editing therapies for children suffering from rare inborn errors of immunity (IEIs). By leveraging advanced CRISPR ... -
Ultra-Homotypic Targeting: Lanthanide-Modified EVs Boost Cancer Detection Sensitivity by Over 25-Fold
University of Tokyo Japan Overview Researchers at the University of Tokyo, led by Professor Keisuke Goda, have unveiled a groundbreaking 'ultra-homotypic targeting' technology. By engineering extracellular vesicles (EVs) with lanthanide ... -
Children’s Hospital of Philadelphia Develops CAR T-Cell Therapy for Pediatric Solid Tumors: First Human Trial Targets PHOX2B-Derived Peptides
Children's Hospital of Philadelphia (CHOP) USA Overview Researchers at Children's Hospital of Philadelphia (CHOP) are leading the development of CAR T-cell therapies for pediatric solid tumors like neuroblastoma. A 'peptide-centric' (PC-... -
Crystalys Therapeutics Secures $35M Series A Funding to Advance Gene Therapy Development
BioBucks USA Overview Crystalys Therapeutics, a gene therapy developer, has raised $35 million in a Series A funding round. This capital will accelerate the research and development of its innovative gene therapy pipeline, targeting unme... -
Scribe Therapeutics Achieves $129M IPO, Targeting Cardiometabolic Diseases with Reversible Epigenetic Gene Silencing
BioPharma Dive USA Overview Scribe Therapeutics successfully completed a $129 million IPO, marking the first gene editing company to go public in over two years. The company develops an epigenetic approach that silences gene expression w... -
MD Anderson Reports 70% Response Rate with Novel Claudin-18.2-Targeted CAR-T Cell Therapy for Intractable Kidney Cancer
MD Anderson Newsroom USA Overview Researchers at MD Anderson Cancer Center announced a 70% objective response rate (ORR) in an early-phase clinical trial of a novel CAR-T cell therapy targeting Claudin-18.2 for patients with metastatic r...
