Rocket Pharmaceuticals
New York, NY

Overall Rank: 51
Category: Biotechnology
Category Rank: 23


  • Top HealthTech Company of 2023


Rocket Pharmaceuticals pioneers turning innovative science into potential gene therapy cures by advancing a multi-platform approach and world-class pipeline of first-of-their-kind therapies for the betterment of our industry, science and humanity. Headquartered in New Jersey with nearly 300 employees, Rocket is a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need.

Since its inception in late 2015, Rocket has been tirelessly working to bring gene therapies to patients with rare diseases. The company is advancing a world-class pipeline of six programs across both AAV and LV platforms to address life threatening cardiac and hematologic rare diseases. Most recently, Rocket’s Biologics License Application (BLA) for its LV-based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I) was accepted and granted priority review by the FDA. Rocket also reached FDA alignment on its pivotal Phase 2 trial design for its gene therapy in Danon Disease. The company has received FDA Fast Track and Orphan Drug Designations across five of its programs, and EMA Priority Medicines (PRIME) and FDA Regenerative Medicine Advanced Therapy (RMAT) Designations across four of its programs.

These therapies represent a paradigm shift in healthcare, offering hope for patients with rare and devastating conditions who often have inadequate treatment options and providing insights for the scientific community that may be applied to other conditions.

Key Products

Rocket’s pipeline is comprised of first-in-class gene therapies for rare and devastating, inherited genetic diseases.

Rocket is platform-agnostic, meaning we choose the most practical gene therapy platform for the disease being targeted. Each program is intended to be transformative, enabling not only reversal of the disorder at molecular and cellular levels, but sustained relief from debilitating and potentially life-threatening symptoms.

To learn more, visit Rocket’s pipeline.


  • Of the 7,000 known rare diseases, only 5 percent have FDA approved therapies.

Key Executives

Gaurav Shah, MD, Chief Executive Officer

Gaurav Shah, a co-founder of Rocket Pharma, serves as Chief Executive Officer and Corporate Board Member. Prior to this role Gaurav was a Global Program Head in the Cell & Gene Therapies Unit at Novartis, where he had strategic oversight of 12 functions and helped spearhead pivotal trials with CART-19 for patients with leukemia and lymphoma. Earlier roles at Novartis included Global Clinical Program Head for CART-19 and for Biosimilars, and lead physician for Afinitor in several global oncology submissions. Gaurav started his career in industry at ImClone/Eli Lilly as a Medical Director overseeing oncology trials focused on monoclonal antibodies.

Kinnari Patel, PharmD, MBA, President & Chief Operating Officer

Kinnari Patel is President & Chief Operating Officer for Rocket Pharma. She oversees the regulatory, clinical execution, global program teams, alliance management, CMC, quality and development organizations. She has fifteen years of rare disease Research & Development experience including in Regulatory Science, Pharmacovigilance, Policy and Quality Compliance gained at leading pharmaceutical companies including AstraZeneca, Bristol-Myers Squibb, Novartis, Hoffmann La-Roche and Pfizer. She has led both small molecule and biologic development programs from Phase I through Phase IV across multiple therapeutic areas including immuno-oncology, oncology, respiratory, virology, transplantation and metabolism. Most notably, Kinnari has worked on Opdivo (nivolumab) from Phase I through BLA filing for metastatic melanoma, RCC and NSCLC, submitted and gained approval of Myalept (metreleptin) for ultra-rare lipodystrophy disease, and submitted Valcyte® (valganciclovir hydrochloride) NDA for prevention of cytomegalovirus disease in pediatric kidney and heart transplant patients.

Mayo Pujols, Chief Technical Officer, EVP

As Chief Technical Officer, Mayo Pujols leads Rocket’s state-of-the-art in-house Good Manufacturing Practice (cGMP) facility and Technical Operations function that includes Manufacturing, Validation Engineering, MS&T, Process Development, Analytical Development, Cell Therapy Development, Supply Chain, Project Management, Facilities Engineering and Environmental Health and Safety.

Isabel Carmona, JD, Chief Human Resources Officer, SVP

Isabel Carmona is the Chief Human Resources Officer for Rocket Pharma. Before joining Rocket, Isabel was Chief Human Resources Officer of Ichnos Sciences. There, she led the spin-off and set up of Ichnos Sciences as an independent oncology biotechnology company headquartered in New York City with research centers in Switzerland and India. Prior to that, she served as a member of the global operations and human resources leadership teams at Teva Pharmaceuticals and Shire Pharmaceuticals.

To learn more about their executive team, visit here

Board Members

Roderick Wong, MD, MBA, Chairman of the Board

Dr. Wong founded and serves as Managing Partner of RTW Investments. Rod has more than a dozen years of experience as a life sciences fund manager. Prior to RTW, he founded and served as a Managing Director and Portfolio Manager for the Davidson Kempner Healthcare Funds. Dr. Wong started his career in investment research, first as a Biotechnology Research Associate at Cowen & Company and then as a Healthcare Analyst at Sigma Capital. He graduated from the University of Pennsylvania Medical School, received an M.B.A. from Harvard Business School, and graduated Phi Beta Kappa with a B.S. in Economics from Duke University. Rod regularly serves on a variety of corporate boards, and has a keen interest in educating the next generation of life science entrepreneurs. He serves as an Adjunct Associate Professor of Finance at NYU Stern, and as an Advisor to the University of Pennsylvania Medical School’s HealthX program.

Elisabeth Bjork, MD, PhD, Independent Director

Dr. Bjӧrk is the Senior Vice President, Head of Late-stage Development, Cardiovascular, Renal and Metabolism (CVRM), BioPharmaceuticals R&D at AstraZeneca leading the global development of medicines within this area. Throughout her career at AstraZeneca, she has gained broad drug development experience covering clinical development phase I-IV, large outcomes programs, major global filings and health authority interactions (FDA, EMA, Japan) and commercial strategy/implementation. Dr Bjӧrk is an endocrinologist by training and an associate professor of medicine at Uppsala University, Sweden. She is also a board member of Chalmers University of Technology, Chalmers Ventures AB, Bjӧrks Matematik o Mera AB and rfidcompare europe AB.

Carsten Boess, MBA, Independent Director

Mr. Boess has served as a director of Inotek / Rocket Pharmaceuticals since January 2016. His most recent role has been EVP Corporate Affairs at Kiniksa Pharmaceuticals, a publicly traded biotechnology company. He previously served as Senior Vice President and Chief Financial Officer at Synageva Biopharma Corporation from 2011 until the company’s acquisition by Alexion Pharmaceuticals in 2015. Prior to his role at Synageva, Mr. Boess served in multiple roles with increasing responsibility with Insulet Corporation, including Chief Financial Officer from 2006 to 2009 and Vice President of International Operations from 2009 to 2011. Prior to that, Mr. Boess served as Executive Vice President of Finance for Serono, Inc., from 2005 to 2006. In addition, he was a member of the Geneva-based World Wide Executive Finance Management Team. Mr. Boess was also Chief Financial Officer at Alexion Pharmaceuticals, and was a finance executive at Novozymes of North America and Novo Nordisk in France, Switzerland and China. Mr. Boess received a Bachelor’s degree and Master’s degree in Economics and Finance, specializing in Accounting and Finance from the University of Odense, Denmark.

Pedro Granadillo, BS, Independent Director

Pedro is a highly-respected pharmaceutical business leader with more than 40 years of experience in the industry, primarily at Eli Lilly and Company, with a proven track record in human resources, manufacturing, quality and corporate governance. As a member of Lilly’s Executive Committee and as Lilly’s head of human resources, manufacturing and quality, he was responsible for policies affecting a global workforce of more than 45,000 employees, as well as a broad network of manufacturing facilities for its extensive line of products. He also oversaw more than 20 sites and 13,000 employees involved in the manufacturing of Lilly’s conventional “small-molecule” pharmaceuticals and “large-molecule” biotech therapies. Pedro has served on the Board of Directors of Haemonetics Corporation, Dendreon Corporation, Noven Pharmaceuticals, First Indiana Bank and most recently on the Board of Directors of NPS Pharmaceuticals, which sold to Shire for $5.2 Billion in 2015. Pedro earned his B.S. degree in Industrial Engineering from Purdue University.

Gotham Makker, MD, Independent Director

Dr. Makker has more than 17 years of healthcare industry experience. Since 2005, he has served as CEO of Simran Investment Group, LLC, a closely held equity investment fund. Prior to Simran, Dr. Makker was a healthcare portfolio manager and principal at Citadel Investment Group LLC, a position he held from 2002 to 2005. Prior to joining Citadel, Dr. Makker served as an analyst at Oracle Partners LP covering biotechnology and medical device sectors from 2000 to 2001. From 1999 to 2000, Dr. Makker was a senior analyst on the life sciences investment banking team at Hambrecht & Quist. Dr. Makker received an M.D. from the University of Nebraska Medical School and he completed the Sarnoff cardiovascular research fellowship at Columbia University, College of Physicians & Surgeons and at Harvard Medical School, Brigham & Women’s Hospital.

Gaurav Shah, MD, Chief Executive Officer

Dr. Shah was appointed Chief Executive Officer of Rocket in September 2015. Prior to joining Rocket, from 2011-2015, Dr. Shah held various leadership positions at Novartis including Global Program Head for CART-19, Global Clinical Program Head for CTL-019 and Biosimilars, and Global Clinical Leader for Afinitor. Prior to Novartis, he spent three years at Eli Lilly and Company as Medical Director overseeing clinical development of numerous programs including olaratumab. During his industry tenure, Dr. Shah has participated in several drug development programs resulting in successful regulatory approvals, such as CTL-019 in pediatric ALL, the first cell and gene therapy approved in the U.S., and successful commercial launches. Prior to joining industry, Dr. Shah was Assistant Professor of Medicine/Oncology at Columbia University. He holds a B.A. in Behavioral Neuroscience from Harvard University and an MD from Columbia University. Dr. Shah completed his internal medicine residency at Brigham and Women’s Hospital/Harvard Medical School and completed his hematology/oncology fellowship training at the Memorial-Sloan Kettering Cancer Center.

David Southwell, MBA, Director

Mr. Southwell is the President, Chief Executive Officer and Board member of TScan Therapeutics, an immuno-oncology company focused on T-cell receptors, a position he has held since 2018. Previously, he served as President, Chief Executive Officer, Board member of Inotek Pharmaceuticals from July 2014 until Inotek merged with Rocket in January 2018. From March 2010 to October 2012, Mr. Southwell served as Executive Vice President, Chief Financial Officer of Human Genome Sciences, Inc. until its merger with GlaxoSmithKline plc. Previously, Mr. Southwell served as Executive Vice President and Chief Financial Officer of Sepracor Inc. from July 1994 to July 2008, and as an investment banker at Lehman Brothers from 1984-1986 and 1988-1994. Mr. Southwell has served on the Board of Directors of PTC Therapeutics, Inc., since 2005, and has served on the Boards of Directors of Biosphere Medical (1998-2010), Human Genome Sciences Inc. (2008-2010), THL Credit Inc. (2007-2016), Inventiv Health (2016), and Spero Therapeutics (2017-2019). Mr. Southwell received a B.A. from Rice University and an M.B.A. from the Tuck School at Dartmouth College, where he has served as head of the MBA Advisory Board and currently serves on the Board of Advisers.

Naveen Yalamanchi, MD, Independent Director

Dr. Yalamanchi has over 15 years of healthcare investment and research experience. Since 2015, He has served as Partner and Portfolio Manager at RTW Investments, a healthcare-centered investment firm. Prior to RTW, Dr. Yalamanchi was Vice-President and co-portfolio manager at Calamos Arista Partners, a subsidiary of Calamos Investments, a position he held from 2012-2015. Prior to joining to Calamos Arista Partners, Dr. Yalamanchi held various healthcare investment roles at Millennium Management and Davidson Kempner Capital Management. Dr. Yalamanchi holds a B.S. in Biology from MIT and an M.D. from Stanford University. He completed his orthopedic surgery internship at UCLA medical center.

Fady Malik, M.D., Ph.D., Independent Director

Dr. Malik is Executive Vice President of Research and Development at Cytokinetics, a late-stage biopharmaceutical company, where he has worked in a variety of positions since he joined the founders to launch the company. At Cytokinetics, Dr. Malik has led R&D efforts resulting in multiple Investigational New Drug submissions, advancement of four programs to Phase 3, and the submission of the company’s first New Drug Application. Dr. Malik is also a Clinical Professor of Medicine in the Cardiology Division of the University of California, San Francisco, where he has held an appointment since 2000. Until 2019, he was a practicing Interventional Cardiologist at the San Francisco Veterans Administration Medical Center for over 18 years.

Key Milestones

  • October 2023: FDA BLA acceptance for RP-L201 for Severe Leukocyte Adhesion Deficiency-I (LAD-I)

  • September 2023: FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease

  • June 2023: FDA Fast Track and Orphan Drug Designations for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

  • FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 for Pyruvate Kinase Deficiency (PKD)

  • May 2023L FDA Clearance of IND for Clinical Trial of RP-A601 for PKP2-ACM

  • February 2023: FDA RMAT Designation for RP-A501 for Danon Disease

  • Presented Positive Data from LV Hematology and AAV Cardiovascular Gene Therapy

  • May 2023: Programs at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

  • January 2023: Unveiled PKP2-ACM program at the 41st Annual J.P. Morgan Healthcare Conference

News and Press Releases

  • Oct 2023: Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I) (Read)

  • Sep 2023: Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease (Read)

For more News and Press Releases, visit here.

Corporate Responsibility

Rocket’s unique set of core values—“Trust,” “Curiosity,” “Generosity” and “Elevate”—is a true beacon of hope within and outside the company. Trust is the bedrock, the ground upon which everything is built. Generosity and curiosity, derived from the same root words as “gene” and “cure,” describe a spirit of giving, aspiring for something greater than oneself, and humility in the search for excellence. Ultimately, the company’s associates and partners seek to elevate themselves, one another, and the lives of patients and families around the world through gene therapy.

To know more about their ESG Report, visit here.