Roswell Park Comprehensive Cancer Center and Generate Biomedicines have dosed the first patient in a first-in-human clinical trial evaluating GB-5267, an autologous IL‑18–armoured CAR T-cell therapy. The therapy is designed to overcome key barriers that have limited the success of cellular therapies in solid tumours. … Read More
Hosted by Drug Discovery World and supported by Bio-Techne this webinar will explore the advances and challenges taking place within the cell and gene therapy field. You will hear from Hans Schambye CEO at BOOST Pharma, Lucas Arruda, CSO at CuraCell, and Brandon Cieniewicz, Director, Research & Development at CERo… Read More
Skylark Bio has revealed it is expanding its pipeline with SKY-AS, a differentiated gene therapy candidate designed to restore UBE3A expression in individuals with Angelman syndrome (AS). SKY-AS represents the company’s first central nervous system (CNS) development programme and expands Skylark’s portfolio of precision, locally delivered… Read More
Circio Holding has successfully completed a private placement raising gross proceeds to the company of approx. NOK 200 ($21) million, through the allocation of 16,865,000 new shares. The Offer Price corresponds to a 9% discount to the volume weighted average share price (VWAP) quoted for trade in… Read More
Novartis and Bristol Myers Squibb (BMS) have both paused clinical trials of CAR-T cell therapies for lupus and other autoimmune diseases due to concerning safety findings. Novartis has placed holds on trials of rapcabtagene autoleucel (rap-cel), stopping screening, randomisation, and treatment administration in trials in lupus, myasthenia… Read More
A new study has presented an innovative electromagnetic field (EMF)-responsive gene switch that enables fully reversible, safe, and precise control over gene expression. “Gene switches” hold significant potential for understanding gene expression and for therapeutic applications, particularly for the non-invasive treatment or management of genetic disorders. … Read More
Genespire has commenced patient enrolment in an observational study, InforMMA, in children with methylmalonic acidemia (MMA) caused by mutations in the MMUT gene. The study is designed to follow patients aged up to 16 years who live with severe MMA, with the aim of better characterising disease… Read More
The first patient has been dosed with PMCC-COE-KMA, an investigational CAR T-cell therapy being developed by Peter MacCallum Cancer Centre’s Centre of Excellence in Cellular Immunotherapy (CoE) and licensed by Australian biotechnology company HaemaLogiX. The KOALA trial will evaluate the safety and efficacy of PMCC-COE-KMA in patients… Read More
In June 2026, China’s National Medical Products Administration cleared satricabtagene autoleucel (satri-cel), CARsgen’s Claudin18.2 CAR-T, for advanced gastric cancer. It became the world’s first CAR-T cell therapy approved for a solid tumour. The science is genuinely impressive. But the geography is the story. Satri-cel is not a Chinese… Read More
The US Food and Drug Administration (FDA) granted accelerated approval for Ultragenyx Pharmaceutical’s Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and paediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). Dr Eric Crombez, Chief Medical Officer at Ultragenyx, commented: “As our… Read More
Pancreatic tumours can resist immunotherapy by recruiting nearby immune cells, according to new research, which has revealed a potential new strategy for overcoming resistance in cell therapies. CAR T-cell therapy has transformed treatment for several blood cancers but has repeatedly stalled against solid tumours such as pancreatic… Read More
Skylark Bio has dosed the first patient in the SONIX Phase I/II clinical trial evaluating SKY-GJB2, its investigational inner-ear-targeted gene therapy for GJB2-mediated hearing loss. “Dosing the first patient in SONIX brings us one step closer to a future where children born with genetic hearing loss have new… Read More
Hosted by Drug Discovery World and supported by BioIVT, this webinar examines the advances, challenges and opportunities that exist in neuroscience drug discovery and development. You will hear from a number of experts who will discuss how both technology and scientific approaches are helping enable the development of… Read More
Circio Holding and Avenue Biosciences have entered a research collaboration to combine their synergistic technologies to improve long-term expression of secreted proteins, relevant for treatment of a broad range of diseases. Secreting proteins from cells into surrounding tissue, or into circulation, opens new treatment opportunities beyond classical gene therapy for… Read More
Hosted by Drug Discovery World and supported by BioIVT, this webinar examines the advances, challenges and opportunities that exist in neuroscience drug discovery and development. You will hear from a number of experts who will discuss how both technology and scientific approaches are helping enable the development of… Read More
