According to a story from BioPortfolio, the drug developer Rocket Pharmaceuticals recently announced that the first patient has been dosed in the company’s phase 2 clinical trial. This study is testing Rocket’s experimental drug candidate RP-L102 “Process B” as a treatment for Fanconi anemia. RP-L102 is a lentiviral vector gene therapy. Rocket is committed to the development of gene-based treatments for a variety of rare diseases that impact children.
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.
About The Trial
The clinical trial is small in size and is expected to include a dozen patients. These participants will be dosed with a single administration of RP-L102 and “Process B,” which is comprised of transduction enhancers, an improved process for stem cell enrichment, and commercial-grade vector. The primary endpoint for the trial will be improved resistance of mitomycin-C in cells responsible for bone marrow colony formation. Other endpoints will include genetic corrections in bone marrow and peripheral blood as well as stability or increases in various blood counts.
Fanconi anemia is a disease with very limited treatment options and though technically curable, the processes involve major and potentially lethal risks for the patient. Many patients do not survive for long and it is clear that new therapies are desperately needed. Hopefully, RP-L102 “Process B” will prove itself capable in this clinical study.