Q&A with Mayo Pujols, CTO of Rocket Pharmaceutical, and Gaurav Shah, CEO of Rocket Pharmaceutical

The CEO and CTO of Rocket Pharmaceutical spoke with Pharm Exec about adding a new role to the company’s C-suite.

One of the most important decisions a growing company can make is to expand the C suite and add more leadership positions. Rocker Pharmaceuticals recently appointed Mayo Pujols as its first technical director. Pujols, along with CEO Gaurav Shah, spoke with Pharm Exec about the new position.

Pharmaceutical Director: What prompted Rocket to add a CTO?

Mayo Pujols: The company has now reached a level of maturity where it is about to seek approval for a few of our products, particularly on the antiviral side. As this important milestone approached, the company felt the time was right to bring in a new leader to consolidate all technical functions under one person and ease the task of the chief operating officer (COO) who oversees everything else. It was about dividing up the work and the timing was perfect.

Additionally, we are about to open our new facility for in-house production, so Rocket was looking for someone with that in mind for this team.

Gaurav Shah: Rocket recently completed its in-house AAV manufacturing readiness plan and is progressing towards a pivotal Phase 2 study and potential commercialization of our Danon disease treatment.

Building an AAV manufacturing facility gives us better control over supply, cost, quality and schedule and allows for a more transparent path to commercialization. Additionally, as we move forward with our three lentiviral vector-based gene therapy programs in human clinical studies, two of which have recently had positive data reads, Mayo will be instrumental in ensuring we have the capabilities techniques necessary for the commercial launch.

Pharmaceutical Director: As the first CTO, what are the first priorities?

Pujol: We have several programs that are in the process of seeking commercial approval, so ensuring that we have all the data and studies to support these records is the number one priority, on behalf of the company and the patients who look forward to it. these products. . The next priority is to build our in-house production capabilities. This is very important for some of our programs.

The third priority is to ensure that the company has the capacity to grow, to launch these products and to be able to produce commercially. Also, to ensure that we have the organizational capacities and within all the laboratories. I have cut out.

Guarav: Mayo will drive the Technical Operations and Chemistry, Manufacturing and Controls (CMC) function for all lentiviral programs. As we move closer to commercial approval, we will need to put processes in place to support patients awaiting these therapies.

Our first filing will be for Leukocyte Adhesion Deficiency-I (LAD-I), a serious pediatric genetic disease. Without successful bone marrow transplantation, mortality in patients with severe LAD-I is 60-75% before the age of 2 years and survival beyond 5 years is rare. In addition, Mayo will lead the company’s in-house adeno-associated virus (AAV) manufacturing facility which recently achieved cGMP preparation for planned pivotal Phase 2 study and potential commercialization in Danon’s disease.

Pharmaceutical Director: How will the CTO interact with the rest of the C suite?

Pujol: I’m part of the C suite and my onboarding went smoothly in terms of onboarding with this group. They took their time finding a candidate, so from a cultural point of view, I felt I was in the right place. They see the value of what my role brings to the business, in terms of sourcing strategy and sustainability. It was a great integration and now it’s all about working together for the company.

Guarav: As Mayo leads the Technical Operations function, he will work closely with our leaders to ensure the success of Technical Operations, Quality Operations, Validation, Process Development and GMP Manufacturing. (cGMP). Successful technical operations will help advance our integrated and sustainable gene therapy pipeline toward commercialization to deliver potentially curative treatments to patients who currently have no other options.

Pharmaceutical Director: How is the role of the CTO unique at Rocket?

Pujol: I have seen the CTO role be much narrower in other companies where he could focus only on development or manufacturing. We’ve made it much broader, which means that under one umbrella, we have everything we need to do to develop products that will ultimately be marketed and trusted for patients. The role was designed with that in mind.

The other thing the company could have done was create departments based on technology or type of therapy, and the fact that they decided to put everything under one umbrella is very helpful. This means that there is a lot more synergy and that things have been centralized to facilitate the marketing of products. The design helps the company to accelerate, which contributes to the sense of urgency associated with certain therapies. This allows me to have the necessary staff to ensure that we can reach patients as quickly as possible.

Guarav: Rocket’s cross-platform approach sets us apart by enabling our team to develop therapies based on high unmet needs. We select the platform that makes the most sense for the disease, and then adapt our strategy to the needs of the disease community. Ensuring the success of our AAV and LVV manufacturing processes is critical to the role of the CTO and ultimately to making potential gene therapy a reality for patients with rare genetic diseases.

Although gene therapy has been studied for decades, the science is at an exciting early stage. The doors are just beginning to open, bringing cures for a host of different ailments. Rocket is uniquely positioned to be a leader and being at this pioneering stage offers plenty of room to improve processes, create new technologies and improve patient access.

Pharmaceutical Director: Did you have any say in the design of the role?

Pujol: As I was being considered and interviewed, I asked a lot of questions about the scope and what was in and out of the role. I loved that it was a very open team and wanted to get the opinion rather than say “here’s the job, do you want it or don’t you want it?”

There were a lot of questions about what should be added to the role and what maybe shouldn’t. I had a say in that, which I liked.

Guarav: As Rocket recently completed our in-house AAV manufacturing readiness plan and is moving towards a pivotal Phase 2 study and potential commercialization of our gene therapy for Danon’s disease, we knew it was the perfect time. to call on an experienced technical director. We are very fortunate to have Mayo, one of the most experienced technical operations and manufacturing leaders of cell and gene therapies in the industry, as Technical Director.