Saturday, January 20, 2018 10:46:54 AM
BIOTECH
January 19, 2018
Rocket Pharmaceuticals, Ltd., a major multi-platform gene therapy company in the U.S mainly enaged in addressing challenging rare diseases has announced that it has completed merging with Inotek Pharmaceuticals Corporation. The new company will now be called Rocket Pharmaceuticals Inc (NASDAQ:RCKT). Rocket will mainly focus on developing a pipeline of gene therapy programs that will mainly target undertreated and rare diseases. The company’s common stock will trade on NASDAQ under the symbol “RCKT”. Rocket has its headquarters in New York City and is headed by Gaurav Shah, M.D as the President and Chief Executive Officer. Before joining he worked as Global Program Head in the Cell & Gene Therapies Unit at Novartis.
In a statement, Dr. Shah said the transaction got a lot of support from both Inotek and Rocket shareholders. He added that this was a clear proof of the two companies’ long-term commitment to grow into a fully integrated multi-platform gene therapy company. He added that the completion of the merger will offer an opportunity to grow its operations, implement clinical development strategies and expand its manufacturing and analytics abilities. Dr. Shah added that they will be focusing more on driving the value of the new company.
Rocket uses a multi-platform development program that leverages the well-developed adeno-associated viral vector (AAV) and lentiviralvector (LVV) gene delivery methods and is currently targeting deadly rare diseases in children which lead to early mortality in the absence of bone marrow transplant.
The lead program of the company is a Phase 1/2 LVV-based gene therapy for Fanconi Anemia (FA) and is currently undergoing clinical studies with academic partners in Europe and the U.S. FA causes instability of the genetic system due to DNA mutations during gene repair leading to early bone marrow failure and malignancy. Initial results in people with FA have shown clinical engraftment of ex vivo-transduced autologous hematopoietic stem cells (HSCs). The rate of gene-corrected cells raise over time, indicating the selective advantage of gene-corrected cells in the bone marrow without the need for conditioning. Functional correction as well as clinical proof of idea have also been observed.
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