Rocket Pharmaceuticals CEO Gaurav Shah said the remote-working policies that have resulted from the global COVID-19 pandemic have created a challenging time for the pharmaceutical industry, as well as the rest of the global community, but believes a silver lining will be forthcoming.
Rocket Pharmaceuticals Chief Executive Officer Gaurav Shah said the remote-working policies that have resulted from the global COVID-19 pandemic have created a challenging time for the pharmaceutical industry, as well as the rest of the global community, but believes a silver lining will be forthcoming.
Sometimes, going through difficult times brings out the best in people and creates a strong sense of community, Shah said.
The new working conditions are causing Shah and his team to assess the status of the ongoing clinical trials the company is conducting, including its Phase II trial in Fanconi Anemia, as well as its three Phase I programs for Danon disease, Leukocyte Adhesion Deficiency-1 (LAD-1), and Pyruvate Kinase Deficiency. In an interview with BioSpace, Shah said Rocket Pharma is talking to physicians and clinicians running the gene therapy trials at the various sites on a daily basis regarding the changing needs of health care facilities during the pandemic. Shah noted that the trials are spread over a number of centers, which provides some redundancies, particularly of patient needs to be shifted.
Shah said he anticipates some case-by-case operational delays, but, as of now, believes those will be near-term only – barring an escalation of the COVID-19 threat. Shah said the company continues to anticipate data readout for each of these programs in the second half of the year. As a result, he said there is no material change to the company’s guidance.
“Our guidance has been pretty conservative,” Shah said. He added that the company will be transparent if the coronavirus situation continues longer than late spring or early summer and causes change to its guidance.
The company’s adeno-associated virus (AAV) and lentiviral-based gene therapy programs are aimed at rare diseases that are similar in nature, with three being hematological diseases and the fourth, Danon disease, a form of heart failure. Shah noted that the only treatments for these diseases are bone marrow and heart transplants that have a number of toxicity risks and fail rates.
“These are fatal diseases, each and every one of them. The only options are transplants. There’s a need for a curative approach,” Shah said.
Because of the limited options for these patients, Shah said gene therapy provides hope for a better tomorrow. The different gene therapy approaches the company is taking have a successful track record laid by trailblazing companies such as AveXis, a Novartis company, and bluebird bio. Shah said the company is applying its gene therapies at the different disease types so they will have a meaningful impact on the patients. And it’s a dedication to the well-being of patients that is such a driving force at Rocket Pharmaceuticals. Shah said everyone at the company is driven by the patient-centric mission to provide hope and a healthy future for the patients their therapies can benefit.
“The goal is to build something that isn’t a starting point, but a long term solution to these extremely devastating diseases,” Shah said.
Looking ahead, beyond the readout of the ongoing clinical trials and the decline in cases of COVID-19, Shah pointed to the company’s current construction of a new manufacturing facility in Cranbury, N.J. Shah said the new facility will allow the company to build out its manufacturing capabilities for its gene therapy products. He anticipates the site will come online next year and, when it is complete, the facility will house the company’s R&D and manufacturing groups. As requirements demand, Shah said Rocket Pharmaceuticals will be hiring in support of those needs.
“Slowly but definitively, we’re becoming a long term, sustainable integrated company,” Shah said.
We’ll have discovery, research, manufacturing, clinical, regulatory and commercialization all under one umbrella.”
The company will continue to maintain a presence in New York. Shah said its current offices will continues to support financing, legal and business development for the company.
“We’re trying to really build something here and right now is the right time in gene therapy,” Shah said.
