Long-Term Results for Fanconi Anemia Treatment Indicate Efficacy


Fanconi anemia (FA) is a rare disease in which the bone marrow in the body essentially fails. It is a genetic condition where the body progressively becomes deficient in red blood cells, white blood cells, and platelets within the bone marrow. In the most severe cases it can lead to various cancers, which have both solid and liquid tumors. Half of FA patients are diagnosed as children, before they reach the age of 10.


RP-L102 is an investigational therapy for FA which has recently shown extreme promise in a Phase I/II study. It is a gene therapy, but uniquely it is lentiviral based as opposed to currently marketed adeno-associated viral vector-based gene therapies.

RP-L102 was created by Rocket Pharmaceuticals. They just presented long-term follow-up data from their clinical trial for FA. This data was presented at the European Society of Cell and Gene Therapy which was held in Spain.

The data represents the very first long-term results utilizing the therapy. Thankfully, these results showed significant improvement for patients. Blood counts stabilized as did durable mosaicism. Additionally, MMC improved, which indicates durable phenotypic correction. Conditioning regimes which are typically used for allogeneic stem cell transplants were not used for these patients.

The company had a 10% threshold. With four of the nine patients in the trial experiencing these results, the Rocket Pharmaceuticals far exceeded this percentage. This was the number agreed upon by the FDA and EMA for a Phase II Registration Study. This new investigation will begin enrolling participants before the end of 2019.

As a whole, the positive results indicated for RP-L102 could mean big things for the FA patient community. It means that this gene therapy may not only benefit patients, but it may be able to eliminate their need for an allogeneic transplant and the severe risks and side effects which can accompany it.

It will be exciting to see the future of this therapy and how it may benefit Fanconi anemia patients.

You can read more about this treatment here.

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