• The manuscript includes data of the first four trial participants that were treated with RP-L102 under the Phase 1/2 FANCOLEN-I study.
   
• First RP-L102 trail data demonstrates increase in bone marrow engraftment levels, which further leads to stabilization as well as restored bone marrow function.

Rocket Pharmaceuticals, Inc., an American multi-platform clinical stage biotechnology firm, reportedly announced long-term data from the company’s ongoing Phase 1/2 clinical trial of RP-L102, gene therapy which is based lentiviral vector (LVV) for Fanconi Anemia (FA).

The manuscript includes data of the first four trail participants that were treated with RP-L102 under the Phase 1/2 FANCOLEN-I study, which used first-generation “Process A” without using any conditioning regimen. Follow-up for all of the primary four patients was kept as 18-30 months from RP-L102 administration.

Senior Vice President as well as Chief Medical Officer of Rocket Pharmaceuticals, Jonathan Schwartz, M.D. stated that the data from the company’s first study of RP-L102 demonstrates increase in bone marrow engraftment levels, which further leads to stabilization as well as restored bone marrow function.

Jonathan added that all gathered data highlights the unique natural selective advantage that exists in Fanconi Anemia for stem cells that are gene corrected over stem cells that are diseased, this potentially removes the conventional need for conditioning.

Jonathan also stated that by the end of 2019, the company would have a first look at the primary data from the company’s Phase 1 trial RP-L102 of ‘Process B’. This trial uses new cells and uses a modified process of stem cell enrichment, final drug product, commercial-grade vector as well as transduction enhancers.

The information printed in the manuscript is from four pediatric patients aged between 3-6 years. These patients received RP-L102 using cryopreserved or fresh CD34+ cells which were collected as well as transduced.

Source credit: https://rocketpharmaceuticals.gcs-web.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-publication-data-phase-12-trial