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 tre ...

Fanconi anemia is a rare genetic disease in which essential DNA repair pathway genes are mutated, disrupting the DNA damage response. Patients with Fanconi anemia experience hematological ...

Patients in the EU with Fanconi anaemia (FA) could soon have the first gene therapy option for the disorder, as the EMA starts a review of Rocket Pharma’s RP-L102 candidate. FA is an inherited ...

preimplantation genetic diagnosis (PGD) made headlines when a Colorado couple used assisted reproductive technology (ART) to have a baby named Adam, whose umbilical cord stem cells could cure his ...

In 2023, Autumn was diagnosed with Fanconi anemia, a rare and incurable genetic disorder. For the past month, 18 local high school and college basketball programs have teamed up to raise awareness ...

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.