Pharma Companies Merge to Advance Gene Therapy Pipeline

Mathew Shanley

Earlier today, Inotek Pharmaceutical announced that it has agreed to merge with Rocket Pharmaceutials Ltd. as the result of its dedication to the continued advancement of its pipeline of gene therapies.

The proposed therapies are currently in-development and based on lentiviral virus (LVV) and adeno-associated virus (AAV) gene therapy platforms, with a focus on treating devastating rare diseases. Under the terms of the agreement, Rocket shareholders are expected to own an estimated 81% of the mega-company, and Inotek shareholders will be the owners of the remaining 19%

Since its inception, Rocket’s development of LVV gene therapies has been the effort of treating bone marrow diseases resulting from gene mutations. Its approach is intended to be curative and could potentially replace the need for allogeneic transplant procedures, which typically lack donors.

Additionally, Rocket has a lentiviral-based gene therapy for infantile malignant osteopetrosis in development. The genetic bone disorder is characterized by increased bone density due to impaired bone resorption by osteoclasts.

“Rocket is a leader in developing first-in-class gene therapies for patients with rare genetic diseases with complex and challenging treatment options, such as bone marrow and organ transplants. We believe the combined company will be well-funded, and it will be led by Gaurav Shah, MD, who was formerly a CART-19 Global Program Head in the Cell and Gene Therapies Unit at Novartis. In addition to Gaurav, Rocket has a seasoned team of gene therapy and rare disease drug development experts,” said David Southwell, President and CEO of Inotek in a press release. “The proposed transaction will provide significant and immediate value to accelerate the development of Rocket’s five distinct programs.”

Rocket has also announced that the company will be moving forward with an AAV-based program for an undisclosed rare pediatric disease with an approximate patient population of 15,000+ in the United States/Europe.

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