Rocket Pharmaceuticals Announces Data from Phase 1/2 Trial of RP-L102

Rocket Pharmaceuticals (NASDAQ:RCKT) has announced the publication of long-term data from its ongoing Phase 1/2 trial of RPL-102, the company’s lentiviral vector (LVV)-based gene therapy in the journal Nature Medicine.

As quoted in the press release:

The data included in the manuscript are from the first four patients treated with RP-L102 in the Phase 1/2 FANCOLEN-I trial that utilized first-generation “Process A” without the use of any conditioning regimen. Follow-up for each of the initial four patients was 18-30 months from administration of RP-L102.

“Data from our first trial of RP-L102 demonstrate increasing levels of bone marrow engraftment, leading to stabilization and restored bone marrow function. These data highlight the natural selective advantage that uniquely exists in FA for gene corrected stem cells over diseased stem cells, which potentially obviates the need for conditioning,” said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “At the end of the year, we will have a first look at initial data from our Phase 1 trial of ‘Process B’ RP-L102, which utilizes fresh cells and incorporates a modified stem cell enrichment process, transduction enhancers, and commercial-grade vector and final drug product. We are also excited by the prospect of starting our global registrational trial incorporating recent alignment on endpoints from both the U.S. Food and Drug Administration and European Medicines Agency.”

“There is an increased and urgent need for new therapies for patients and families suffering from FA as current treatments are limited to toxic and burdensome bone marrow transplant,” said Paula Río, Ph.D., Senior Scientist, División de Terapias Innovadoras en el Sistema Hematopoyético, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundación Jiménez Díaz, and co-first author of the manuscript. “We are very pleased to see long-term follow-up data that further support our thesis for RP-L102 gene therapy without any conditioning to serve as an innovative, low-toxicity treatment for the hematologic component of this devastating disease.”

Click here to read the full press release.