Rocket Pharmaceuticals has decided to stop developing a gene therapy treatment for a rare blood disorder called Fanconi anaemia. This decision is part of a larger company restructuring that was announced in July 2025. The company had been working on this therapy, called RP-L102 or Fanskya, and was close to completing the approval process with the US Food and Drug Administration (FDA). However, they chose to withdraw their application. Investors did not react strongly to this news, and the company’s stock price dropped slightly. Rocket Pharmaceuticals has a market value of $348 million. The gene therapy was being developed to help patients with Fanconi anaemia, a condition where the bone marrow does not produce enough blood cells. There are currently no approved treatments specifically for this disorder. As part of the restructuring, Rocket Pharmaceuticals laid off 30% of its staff and decided to focus more on its cardiovascular disease programs. The gene therapy, RP-L102, is the first project to be discontinued as a result of this change in focus. The company stated that they are focusing their resources on programs that have clearer paths to regulatory approval and commercial success. They also mentioned that they might look for a partner to take over the development of this therapy. Rocket Pharmaceuticals stopped investing in RP-L102 internally in July 2025 and also withdrew its application for approval in Europe around the same time. The therapy has been in development since 2019, and the data from early trials has been used to support its applications for approval. The company emphasized that their decision was based on strategy and not due to any safety concerns with the therapy. Earlier this year, there was a safety issue with another one of Rocket’s gene therapies, which led to a temporary halt in its clinical trial. However, the company is making progress with another gene therapy called Kresladi, which is being developed to treat a different rare disorder. Despite a setback in 2024, the FDA is currently reviewing additional data for Kresladi, and Rocket plans to submit a complete application for approval this year. This shift in focus by Rocket Pharmaceuticals is part of a broader trend in the cell and gene therapy sector. Other companies, such as Takeda and Bluebird bio, have also made significant changes to their programs and strategies. Despite these challenges, cell and gene therapies are expected to play a major role in the future of the pharmaceutical industry, with a projected market value of $76 billion by 2030.