The flight path towards a first approved product for Rocket Pharmaceuticals has been hit with turbulence, after the biotech culled a gene therapy programme for the treatment of a rare blood disorder.
Despite nearing the end of its regulatory pathway, Rocket opted to withdraw the rolling approval application to the US Food and Drug Administration (FDA) for RP-L102 (mozafancogene autotemcel). The biotech had been hoping to finish the submission process this year.
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Investors were largely unmoved by the announcement, with shares in Rocket opening 0.6% down at $3.19 at market open on 3 October compared to the previous day’s market close. The US-listed biotech has a market cap of $348m.
The gene therapy, also known as Fanskya, was being developed to treat patients with Fanconi anaemia, a disorder in which impaired bone marrow reduces blood cell production. There are no therapies specifically approved for the condition.
Rocket’s decision to discontinue the candidate’s development is part of a broader company restructuring announced in July 2025. At the time, the biotech laid off 30% of its staff and pivoted to focus on its cardiovascular disease programmes. RP-L102 has now become the first pipeline casualty amid the reprioritisation.
“Rocket is focusing its resources on programs with the clearest regulatory and commercial pathways,” the company said in a SEC filing. Rocket added that it could look to offload the asset via an external partnership.
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By GlobalDataIn the same filing, the biotech announced it had already ceased internal investment in RP-L102 in July 2025. A marketing authorisation application (MAA) with the European Medicines Agency (EMA) was pulled in the same month.
RP-L102 has been in somewhat of a developmental limbo. Rocket announced early Phase I/II data (NCT03157804) with the therapy in Fanconi anaemia patients as far back as September 2019. The application acceptances in Europe and the US have been based on results produced by this study.
Rocket was keen to highlight that its decision was purely strategic, adding that there were no safety concerns with the therapy.
A major safety issue plagued Rocket earlier this year when a patient in its pivotal Danon disease gene therapy trial died. The FDA swiftly placed RP-A501 on clinical hold, which was then lifted in August with a restriction on higher doses.
The biotech has, however, been inching closer to approval for Kresladi, a gene therapy to treat severe leukocyte adhesion deficiency-I. Despite a rejection in 2024, the FDA is now reviewing supplemental data it requested at the time. Rocket expects to submit a full biologics licence application (BLA) for Kresladi to resolve the complete response letter (CRL) this year.
Rocket’s pipeline pivot is part of a broader revaluation trend in the cell and gene therapy sector. On 2 October, Takeda pulled the plug on its cell therapy programme. Former cell and gene therapy stalwart Bluebird bio, which was sold for less than $30m in 2024, underwent a major rebranding last month in an effort to charter a new chapter in the market.
Cell and gene therapies are still expected to play a major role in the pharma industry going forward, given their innovative modality. A report by GlobalData forecasts the global market could be worth $76bn by 2030.
GlobalData is the parent company of Pharmaceutical Technology.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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