Rocket Pharmaceuticals’ strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was developing for Fanconi anemia.
Rocket Pharmaceuticals has pulled the biologics license application for its investigational gene therapy mozafancogene autotemcel, which it was proposing for the treatment of a rare blood disorder.
In an SEC filing on Friday, the New Jersey biotech said the withdrawal was driven by “business and strategic considerations,” and that it follows Rocket’s “previously announced corporate reprioritization” initiative in July, in which it is focusing its efforts on programs with the clearest regulatory and commercial pathways.
Rocket emphasized in its SEC filing, however, that the withdrawal “does not reflect concerns regarding the safety or efficacy profile” of mozafancogene autotemcel, also dubbed fanca-cel. Data to date “continue to support” a favorable risk-benefit profile for the program, with no “significant” safety concerns documented, the company added.
Rocket has also pulled its marketing authorization application with the European Medicines Agency.
Fanca-cel is an investigational gene therapy that uses hematopoietic stem cells derived from the patients themselves, and which have been modified to contain a functional copy of the FANCA gene. The asset is being trialed for Fanconi anemia, a rare blood disease caused by mutations in this gene and characterized by bone marrow failure, congenital defects and a heightened risk for cancer.
After a patient death in May led the FDA to place its pivotal Phase II Danon disease program on clinical hold, Rocket launched a sweeping strategic realignment initiative in July. The Danon asset, a gene therapy called RP-A501, was released from its hold in August, allowing the biotech to restart mid-stage development.
Still, the July initiative left Rocket 30% smaller—some 80 employees were laid off—and forced the biotech to narrow its pipeline focus, ultimately pulling money from earlier-stage assets, including fanca-cel.
Rocket “will consider external partnership opportunities” that can support the advancement of fanca-cel, according to the company’s SEC filing, and the biotech retains the “ability to re-engage with regulators” in the future if it finds a sustainable pathway to take the gene therapy forward.
The past year has been turbulent for Rocket. Aside from the Danon clinical hold, the biotech last year suffered a regulatory setback when the FDA rejected its gene therapy Kresladi for severe leukocyte adhesion deficiency-I. In its complete response letter, the regulator asked for additional clarity on Kresladi’s manufacturing.
In its second-quarter 2025 report, Rocket noted that it “continues to work with senior leaders and reviewers” from the FDA’s Center for Biologics Evaluation and Research for its resubmission. The biotech expects to refile before the end of the year.
