Gene Therapy for Fanconi Anemia Gains IND Clearance From the FDA

According to a story from BioSpace, the gene therapy company Rocket Pharma recently announced that its investigational product candidate RP-L102 recently saw its Investigational New Drug (IND) application gain approval from the US Food and Drug Administration (FDA). RP-L102 is in development for the treatment of Fanconi anemia. This means that the product is cleared to begin clinical trials testing. This marks the company’s first IND clearance from the agency. Rocket Pharma is committed to the development of gene therapies for rare, severe diseases.

About Fanconi Anemia

Fanconi anemia is a rare genetic disease which is most characterized by a deficient response to DNA damage. Research surrounding this rare disorder has helped further the understanding of cancer development and the function of bone marrow. The disease is caused by genetic mutations affecting the protein normally responsible for DNA repair. Symptoms of Fanconi anemia include developmental abnormalities and deficiencies, short stature, fatigue, pale skin, easy bruising, infections, and acute myeloid leukemia. Bone marrow failure means that the body cannot produce blood cells. Treatment may include androgens and hematopoietic growth factors. A stem cell transplant can permanently cure the condition. The risk of serious complications and cancer means that many patients do not survive into their adult years. Fanconi anemia commonly appears alongside other genetic conditions and is more prevalent in the Ashkenazi Jewish population. To learn more about Fanconi anemia, click here.

Future Development

The upcoming clinical trial, which is slated to begin some time in 2019, will test a formulation of RP-L102 known as “Process B.” This is essentially an upgraded and modified variant of RP-L102. This trial is expected to consist of 12 patients and it will test the effectiveness, tolerability, and overall safety of Process B RP-L102.

RP-L102 has been cooperatively developed between Rocket and another company based out of Spain. The experimental gene therapy has been given Orphan Drug designation in both the US and Europe as a treatment for Fanconi anemia type A.


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