CRANBURY, NJ–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces the appointment of Mayo Pujols as Chief Technical Officer (CTO) and Executive Vice President. Mr. Pujols brings nearly three decades of experience, from leadership roles across technical operations, quality operations, validation, process development and Good Manufacturing Practice (cGMP) manufacturing. He most recently served as Chief Executive Officer of Andelyn Biosciences, a leading gene therapy contract development and manufacturing organization (CDMO) and prior to Andelyn was the Head of Global Cell and Gene Technical Development and Manufacturing for Novartis Pharmaceuticals. As Rocket’s first CTO, Mr. Pujols will lead the technical operations function and chemistry, manufacturing and controls (CMC) for all lentiviral programs. Additionally, he will lead the Company’s state-of-the-art adeno-associated virus (AAV) manufacturing facility that has recently achieved cGMP readiness for a planned Phase 2 pivotal study in Danon Disease.
“The arrival of Mayo, one of the leading cell and gene therapy technical operations experts in the industry, comes at an exciting time, as we have 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,” said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma. “Mayo’s proven track record of success and leadership in gene therapy and CAR-T cell therapy at both Novartis and Celgene adds to our team’s rich experience as we prepare our first regulatory filings for our lentiviral platform and advance our AAV platform in our pursuit of gene therapy cures for patients living with rare and devastating diseases.”
Prior to joining Rocket, Mr. Pujols was Chief Executive Officer of Andelyn Biosciences, a CDMO affiliated with Nationwide Children’s Hospital, where he led its expansion to a full-service gene therapy CDMO. Previously, he served as Head of Global Cell and Gene Technical Development and Manufacturing for Novartis Pharmaceuticals, where he was responsible for the global development and internal and external manufacturing for the cell and gene therapy franchise. He also served as Vice President of global CAR‐T operations and technology at Celgene, where he led global manufacturing and technical operations of CAR-T products and built the company’s commercial manufacturing capabilities. Throughout his distinguished career, he has also held key roles at Merck, Advaxis, MedImmune and Schering-Plough.
“Rocket’s progress moving four gene therapy programs to clinical proof of concept and achieving the primary endpoint for two of them in a short time is remarkable,” said Mr. Pujols. “Additionally, its forward-looking approach to in-house AAV manufacturing will help ensure patients with rare genetic disorders, like Danon Disease, have reliable access to quality therapies for which there are currently no other viable options. I’m grateful and excited to join this passionate group of visionaries who are dedicating their careers to making a difference in the lives of patients and family members facing these devastating diseases.”
Mr. Pujols earned his bachelor’s degree in chemical engineering from Stevens Institute of Technology and his master’s in chemical engineering and applied chemistry from Columbia University’s The Fu Foundation School of Engineering and Applied Science.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket’s clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and potential cancer, Leukocyte Adhesion Deficiency-I (LAD- I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections that are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia . Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket’s future expectations, plans and prospects, including, without limitation, Rocket’s expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA ), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, Rocket’s plans for the advancement of its Danon Disease program following the lifting of the FDA’s clinical hold and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, and Rocket’s plans for the advancement of its Danon Disease, FA and PKD programs based on the data presented at ASGCT and the potential for therapeutic benefit related thereto, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litiga tion Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “will give,” “estimate,” “seek, “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients , families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligations to update or refise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.