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Background Osteogenesis imperfecta (OI) is a rare genetic disorder characterised by bone fragility and frequent fractures. Current treatments aim to preserve bone mass but are not curative. The Boost Brittle Bones Before Birth (BOOSTB4) trial was the first to investigate prenatal and postnatal mesenchymal stem cell therapy (SCT) for severe OI internationally. Understanding the experiences of parents and healthcare professionals involved in this pioneering trial provides vital insights for the design and delivery of future therapies in OI and other rare conditions.Aim To explore the attitudes and experiences of parents and healthcare professionals participating in the BOOSTB4 trial.Methods Semi-structured interviews were conducted with 16 parents and 13 healthcare professionals. Transcripts were analysed thematically to identify key experiences and perspectives.Results Three overarching themes were identified: (1) the role of the trial in shaping hope and expectations, (2) emotional and practical burden of participation, and (3) procedural and communication-related insights. Parents reported emotional benefits, though there were challenges in managing uncertainty about SCT efficacy. Decisions to participate were influenced by limited treatment alternatives and personal beliefs. Practical barriers included travel disruptions and procedural demands. Parental experiences were shaped by the quality of communication and access to specialist OI services. Healthcare professionals valued the trial’s innovation but noted difficulties in managing expectations and addressing logistical and ethical challenges.Conclusion Parents and healthcare professionals regarded BOOSTB4 as a promising and innovative approach to OI care. However, the emotional, ethical and logistical challenges emphasise the importance of transparent communication, patient-centred approaches and trial designs that accommodate individual families’ needs. These findings inform the development of future early-phase therapies for rare genetic conditions.Acknowledgements for Funding or Support The BOOSTB4 project received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 681045. Dr Melissa Hill is partially funded by the NIHR Great Ormond Street Hospital Biomedical Research Centre (BRC).