Niche played a pivotal role in the successful registration of Strimvelis, the world’s first ex vivo autologous gene therapy to gain regulatory approval. As a trusted partner in this landmark achievement, we provided critical expertise in regulatory strategy, data integration and preparation of the clinical and technical documentation that met the rigorous standards of the European Medicines Agency. Our deep understanding of the complexities surrounding advanced therapy medicinal products (ATMPs), was instrumental in securing the historic approval of Strimvelis for ADA-SCID.
Following the acquisition of Strimvelis and associated gene therapy assets by Orchard Therapeutics, our contributions were formally acknowledged as foundational to the success of the regulatory submission. Our involvement included:
"I have always been impressed by the standard of the work delivered by the Niche teams. In addition, the contracting process is simple and efficient, communication is open, frequent and proactive, with any issues getting resolved quickly and efficiently.."
Being a small biotech company, Orchard focuses on key competencies internally, while engaging with bespoke service providers to deliver important aspects of their business. Niche has helped Orchard with this agile business model over several years, by generating submission-ready regulatory documents such as clinical study reports for Orchard’s HSC-based gene therapies. This ongoing collaboration underscores our proven track record in navigating the unique regulatory and clinical challenges of gene therapy development and our shared dedication to bringing transformative treatments to patients with rare and life-threatening diseases.
From the outset, Niche's expertise aligned well with Orchard's streamlined approach to operations. Our contracting process is simple and efficient, allowing both Orchard and Niche to focus on what’s important – clinical development of pioneering gene therapies. Frequent, open, proactive communication enabled operational efficiency. Moreover, deliverables are generated according to tight regulatory timelines. The Niche team is passionate and dedicated, with extensive in-house knowledge of gene therapy. This combination of efficiency and expertise has helped Orchard to drive forward the gene therapy specialism, one of the most revolutionary fields in biotechnology.
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