EventsCliniciansPacientsResearchersATMPsCell therapyGen TherapyRegulationCTGCTNICRodrigo Ortiz 20 September 2024 Bridging Gaps: ERN ITHACA and SATB2 Europe Visit the Center for Technologies of Gene and Cell Therapy The CTGCT hosted representatives from ERN ITHACA and SATB2 Europe to discuss collaborative efforts in rare disease research and treatment. The meeting focused on developing innovative therapies and strengthening connections between researchers, clinicians, and ... More
EventsCliniciansPacientsResearchersATMPsCell therapyGen TherapyBusiness ModelDrug DiscoveryEconomicsInvestmentMarketaccessRegulationResearchCTGCTGregor Cuzak 12 September 2024 Event Date: 6 September 2024 • 09:00 do: 6 September 2024 • European Commission Hosts Sixth Regional HTA Event in Budapest The European Commission recently held its sixth regional event in Budapest, focusing on the upcoming Health Technology Assessment (HTA) regulation. Set to launch in January 2025, this new framework aims to streamline the evaluation ... More
ArticlesPacientsResearchersCell therapyGen TherapyResearchCTGCTNICRodrigo Ortiz 30 August 2024 Exciting news in Sonogenetics: A New Frontier for Deep Tissue Treatment Researchers have made a discovery that could change how we treat some diseases deep within the human body. This technique, known as sonogenetics, combines the power of ultrasound with genetically engineered cells to deliver ... More
CliniciansPacientsResearchersBusiness ModelCharite BerlinCTGCTNICUCLUMCUtrechtGregor Cuzak 8 July 2024 The Promise and Price of Advanced Therapies Here’s what Claude Sonnet 3.5 has to answer when we question it: why do ATMPs cost so much? Advanced Therapy Medicinal Products (ATMPs), including gene and cell therapies, represent a revolutionary frontier in medicine, ... More
ArticlesCliniciansPacientsResearchersDrug DiscoveryMarketaccessRegulationResearchCTGCTGregor Cuzak 27 June 2024 Bespoke Gene Therapy Consortium – cooperation for rare disease therapies At the Bespoke Gene Therapy Consortium (BGTC, website), a consortium for the treatment of rare monogenetic diseases, they aim to treat neglected diseases with a very small number of patients with a focus solely ... More
EventsCliniciansPacientsResearchersGen TherapyGMPClinic studyDatamanagementDrug DiscoveryEthicalcommitteeInvestmentLaboratoryLogisticsManufacturing ProcessModalitetadelovanjazdravilaPharmacodynamicsPharmacokineticsPre ClinicalRaw MaterialRegulationResearchCTGCTNICGregor Cuzak 26 June 2024 Event Date: 14 June 2024 • 09:00 − 17:00 CTNNB1 Conference – One Year to Cure One year to cure is the goal shared by parents of children with the CTNNB1 Syndrome rare disease. Out of approximately 400 known cases worldwide, 82 families with children gathered at the conference in ... More
