Rocket Pharmaceuticals RCKT announced that the EMA accepted the marketing authorization application (MAA), seeking approval for RP-L102, an investigational gene therapy for fanconi anemia (FA).

Fanconi anemia (FA) is a rare genetic disorder impairing bone marrow's blood cell production, leading to severe health issues. Early diagnosis and treatment, especially through blood stem cell ...

Its clinical program is an LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer.

Our work on genomic integrity ranges from the Fanconi Anemia pathway, which responds to DNA interstrand crosslinks, to the ATR and ATM checkpoint kinases that coordinate the cell’s response to ...