Q&A with Rocket Pharmaceutical's CTO Mayo Pujols and CEO Gaurav Shah

Rocket Pharmaceutical's CEO and CTO spoke with Pharm Exec about adding a new position 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 named Mayo Pujols as its first chief technical officer. Pujols, along with CEO Gaurav Shah, spoke with Pharm Exec about the new position.

Pharm Exec: What made rocket decide it was to add a CTO?

Mayo Pujols: The company has now reached a level of maturity where its right at the cusp of seeking approval for a few of our products, especially on the anti-viral side. With that milestone coming soon, the company felt that the timing was right to bring in a new leader to bring all of the tech-ops functions under one person and make it easier for the chief operating officer (COO) who oversees everything else. It was about dividing the work and the timing was perfect.

In addition, we’re about to open our new facility for internal production, so Rocket was looking for someone with that in mind for that team.

Gaurav Shah: Rocket recently completed our in-house AAV manufacturing readiness plan and are progressing on the path toward a Phase 2 pivotal study and potential commercialization for our therapy for Danon Disease.

Building an AAV manufacturing facility affords us greater control of supply, cost, quality, and timing and enables a more seamless path toward commercialization. Additionally, as we advance our three lentiviral vector-based gene therapy programs in human clinical studies, two of which have recently had positive top-line data readouts, Mayo will be instrumental in ensuring we have the technical capabilities to launch commercially.

Pharm Exec: As the first CTO, what are the first priorities?

Pujols: We’ve got several programs that are very close to applying for commercial approval, so ensuring that we have all of the data and studies to support those registrations is priority number one, on behalf of the company and the patients who are eagerly awaiting these products. The next priority is to get our in-house production capabilities up and running. That’s very important for some of our programs.

The third priority is ensuring that the company has the capabilities to scale up, launch these products, and be able to produce commercially. Also, ensuring that we have the capabilities organizationally and within all the labs. I’ve got my work cut out for me.

Guarav: Mayo will be the driving force behind the technical operations function and chemistry, manufacturing, and controls (CMC) for all lentiviral programs. As we grow closer to commercial approval, we will need to have processes in place to help patients waiting for these therapies.

Our first filing will be in Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. Additionally, Mayo will lead the Company’s in-house adeno-associated virus (AAV) manufacturing facility that recently achieved cGMP readiness for a planned Phase 2 pivotal study and potential commercialization in Danon Disease.

Pharm Exec: How will the CTO be interacting with the rest of the C-suite?

Pujols: I’m part of the C-suite and my onboarding has gone well in terms of integrating with that group. They took their time finding a candidate, so from a cultural fit, I felt like I’m in the right place. They see the value in what my role brings to the company, in terms of supply strategy and viability. It’s been a great integration and it’s now about all of us working together for the company.

Guarav: As Mayo leads the technical operations function, he will work closely with our leaders to ensure success across technical operations, quality operations, validation, process development and Good Manufacturing Practice (cGMP) manufacturing. The success of technical operations will help advance our integrated and sustainable pipeline of genetic therapies toward commercialization to deliver potentially curative treatments for patients who currently have no other options.

Pharm Exec: How is the role of CTO unique at Rocket?

Pujols: I’ve seen the CTO role be much narrower at other companies where it might be focused just on development or manufacturing. We’ve made it much broader, which means that under one umbrella, we have everything that we need to do to develop products that are ultimately going to be commercialized and reliable for patients. The role has been designed to keep this in mind.

The other thing that the company could have done was to create departments based on technology or type of therapy, and the fact that they decided to put it all under one umbrella is very helpful. It means that there is much more synergy, and things have been centralized to make it easier to bring products to market. The design helps the company accelerate, which helps with the sense of urgency that some of the therapies come with. It allows me the staffing as needed to ensure that we can reach the patients as quickly as possible.

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

Though gene therapy has been studied for decades, the science is at an exciting early stage. The doors are just starting to open, bringing treatments to a host of different diseases. Rocket is ideally positioned to be a leader and being at this pioneering stage provides a lot of room to improve processes, to create new technology, and improve patient access.

Pharm Exec: Did you have any say in the design of the role?

Pujols: As I was being considered and interviewed, I was asking a lot of questions about the scope and what was in and out of the role. I loved the fact that it was a very open team and wanted the input rather than saying “here’s the job, do you want it or not want it?”

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

Guarav: With Rocket recently completing our in-house AAV manufacturing readiness plan and progressing toward a Phase 2 pivotal study and potential commercialization for our gene therapy for Danon Disease, we knew it was a perfect time to bring in an experienced chief technical officer. We are very fortunate to have Mayo, one of the most seasoned cell and gene therapy technical operations and manufacturing leaders in the industry, as our CTO.