RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood. Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart.

An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. and EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months.

Phase 2 Trial Sites

For more details about the RP-A501 for Danon Disease clinical trial, visit clinicaltrials.gov.

Frequently Asked Questions

The Natural History of a Disease is the typical progression of the disease over a person’s lifetime starting from its onset

Natural History studies are observational studies that gather comprehensive data on the course of the disease to gain a better understanding of its progression and impact on patients. Data captured may include information on the physical signs and symptoms patients experience, test results (for example blood tests or imaging), genetic information, and health-related quality of life, over a period of time. This information helps describe the disease course and identify important factors (e.g. demographic, genetic, and/or lab test results), that may contribute to better or worse disease outcomes.

Travel, accommodation, and food stipend would be provided. Study assessments are provided free of charge.

Natural History Studies are particularly important for rare diseases. Given the diversity of symptoms and the small size of the patient population, these studies are often the only opportunity for researchers and physicians to address significant knowledge gaps and obtain a robust understanding of the disease and patient experience.

Engaging in a Natural History Study offers numerous benefits to the community, such as:

  • Raising awareness in medical, academic and pharmaceutical sectors
  • Empowering rare disease patient communities with knowledge and support for advocating better healthcare policies and support services
  • Enhancing patient care with improved insights into disease progression, allowing for more personalized treatment plans and potentially earlier detection of complications
  • Contributing to advancements in healthcare

These studies provide various avenues to help inform and design clinical trials, such as:

  • Highlighting areas of unmet medical need
  • Understanding characteristics of the patient population to define a treatment benefit from a patient’s perspective.
  • Describing disease progression, enabling the identification of clinical outcome measures and biomarkers
  • Provide patient-centric data for health authorities such as the FDA, and other public health institutions to use in their review processes of investigational treatments.
Learn more about Rocket’s clinical trial for FA

Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@nullrocketpharma.com

View more details about the Phase 2 pivotal trial of RP-A501 and natural history study on clinicaltrials.gov.

To read our Expanded Access statement, click here.

About Us

team photos - about us

Mission, Vision & Values

Our mission is to seek gene therapy cures for patients with devastating rare diseases through the most innovative science and platforms to fulfill the promise of gene therapy for the betterment of our industry, science and humanity.

Our vision is to free every patient and the world from rare genetic diseases.

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.

Trust

+
Trust is given and trust is earned – it’s a balance. The word “trust” comes from the “Proto-Indo-European word “deru” which means “to be firm, solid, steadfast.” Trust is the ground and foundation for everything we do.

Generosity

+
Being generous means following up, sharing our best ideas, forgiving ourselves and others, asking who needs us, treating our word as gold, taking time to truly see others, and so many other things. The word “generous” has the same root as the word “gene” – which means “to beget.” Genes thrive on the generosity of others. What more is there to say?

Curiosity

+
The wonder of a child staring up at the night sky. Humility, selflessness. No single one of us can do this job alone and it is okay to ask for help. Curiosity is derived from the Latin word, “cura” which gave birth to the word “care” as well as “cure.” Generosity is to curiosity what gene is to cure.

Elevate

+
Derived from the Latin word “levis,” which means “light” as opposed to heavy. How can we bring trust, generosity and curiosity to elevate ourselves, each other, the pipeline and ultimately the life experience of patients and their families?

“For the first time in the history of our species, we are discussing not just effective treatments, but potentially total cures at the genetic level, which is the deepest essence of who we are as physical living beings.”

— Gaurav Shah, MD, Chief Executive Officer

Advocacy and Partnerships

Rocket cultivates trusted partnerships with patients and families, advocacy organizations, scientists, healthcare providers and health authorities to help guide us on our mission—and we execute on every commitment we make. 

Patients with rare diseases are our North Star—our constant reminder keeping us centered on our path seeking cures through gene therapy. Only by understanding their journey can we move forward in ours.

Learn More Here

History

July 2025
Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A601 Gene Therapy for PKP2-Arrhythmogenic Cardiomyopathy poster

RP-A601 gene therapy received FDA RMAT Designation for the treatment of PKP2 Arrhythmogenic Cardiomyopathy

Read More

June 2025
Rocket Pharmaceuticals Announces FDA IND Clearance of RP-A701 for the Treatment of BAG3-associated Dilated Cardiomyopathy poster

FDA IND clearance received for RP-A701 for the treatment of BAG3-associated Dilated Cardiomyopathy

Read More

May 2025
Rocket Pharmaceuticals Announces FDA IND Clearance of RP-A701 for the Treatment of BAG3-associated Dilated Cardiomyopathy (2)

Preliminary data from Phase 1 clinical trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy at 28th Annual ASCGT

Read More

November 2024
RP-A501

Long-term data from the Phase 1 study of RP-A501 for Danon disease published in the New England Journal of Medicine and presented at American Heart Association conference

Read More

Load More

Leadership

Gaurav Shah, MD

Chief Executive Officer

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.

Gaurav graduated from Harvard College (summa cum laude, Phi Beta Kappa) with a degree in behavioral neuroscience. He received his MD from Columbia (AOA), completed his internal medicine residency at Brigham and Women’s Hospital, and hematology/oncology fellowship training at Memorial-Sloan Kettering. After receiving board certification in medical oncology, he served as an Adjunct Assistant Professor of Oncology at Columbia.

To find balance, Gaurav also has a passion for Indian classical music and has toured extensively including performances at Carnegie Hall and on National Geographic TV, a feature in the Smithsonian Grammy nominations in 2019 (Falu’s Bazaar) and 2024 (Abundance in Millets) and 2022 Grammy win (A Colorful World).

Sarbani Chaudhuri

Chief Commercial & Medical Affairs Officer

Sarbani Chaudhuri

Chief Commercial & Medical Affairs Officer

Sarbani Chaudhuri is the Chief Commercial & Medical Affairs Officer at Rocket Pharma, leading the Marketing, Value & Access, Business Excellence & Operations, and Medical Affairs functions. She is a biopharma executive leader who has built global and U.S. organizations to deliver pioneering therapeutics that have radically transformed patient outcomes and been central to enterprise growth.

With more than 20 years of experience, Sarbani has been at the forefront of disruptive innovation in cell and gene therapies, T-cell engagers, antibody drug conjugates (ADC), and precision therapeutics, through building disease area powerhouses with multiple blockbuster launches, shaping R&D strategies, and business development.

Prior to joining Rocket in April 2025, Sarbani was Vice President at Johnson & Johnson Innovative Medicine leading hematology, where she was a driving force in J&J becoming the top hematology company worldwide in 2024. She led exponential growth of the multi-billion-dollar business, building an industry-leading multiple myeloma powerhouse with DARZALEX® and launching pioneering cell therapy and bispecifics CARVYKTI®, TECVAYLI®, TALVEY®, while expanding into lymphoma and leukemia with a next-generation pipeline.

In her prior role, as Global Head of ENHERTU® and Breast Cancer at AstraZeneca, she established an unparalleled leadership foundation through unlocking multiple HER2-directed tumors. Previously, at Pfizer, she cemented oncology as the company’s growth engine with IBRANCE® and created a rare cardiac and neuromuscular franchise with VYNDAQEL®. Sarbani also held several leadership roles within Novartis’ U.S. sales and marketing organization, playing a key role in strengthening the U.S. go-to-market model.

She received a Bachelor of Engineering degree from VJTI, University of Mumbai, a Master of Business Management from XLRI, Institute of Management Studies, and an MBA from the University of Pennsylvania, with a focus on healthcare management.

Sarbani lives in New Jersey with her son and enjoys inventive cuisine, creative writing, and global travel adventures.

Meg E. Dodge, J.D., LL.M

Senior Vice President, Head of External Affairs

Meg E. Dodge, J.D., LL.M

Senior Vice President, Head of External Affairs

Meg Dodge is the Senior Vice President, Head of External Affairs at Rocket Pharmaceuticals (NASDAQ: RCKT), where she provides strategic leadership for investor relations, corporate communications, government affairs, and executive operations. As a member of the Executive Leadership Team, Meg serves as a trusted strategic partner to the CEO and C-suite, shaping enterprise-level priorities and aligning stakeholders around Rocket’s mission to deliver curative gene therapies for rare and devastating diseases.

Prior to Rocket, Meg served as Vice President of Investor Relations and Communications at Krystal Biotech (NASDAQ: KRYS), where she oversaw all aspects of investor relations and communications strategy through the approval and launch of VYJUVEK® in 2023. Earlier in her career, she held senior leadership roles at Équilibre Biopharmaceuticals and Graviton Bioscience as Chief of Staff, at Indigo 8 as Managing Partner, at Morgan Stanley as Executive Director in the Private Bank, and at Goldman Sachs as Senior Vice President in the Investment Management Division.

Meg holds a J.D. from Syracuse University College of Law, an LL.M. from New York University School of Law, and a B.A. magna cum laude from Colby College. She serves on the Kent School Advisory Council, is a Newport Trust and Investment Commissioner, and is an active advocate for women’s leadership in biotech and policy reform in genetic medicine.

Gayatri R. Rao, MD, JD

Chief Regulatory Officer & SVP, Clinical Safety

Gayatri R. Rao, MD, JD

Chief Regulatory Officer & SVP, Clinical Safety

Gayatri Rao is the Chief Regulatory Officer & SVP, Clinical Safety at Rocket Pharma, overseeing the Regulatory, Pharmacovigilance, Real World Evidence and Medical Writing functions across the pipeline of LV hematology and AAV cardiovascular programs. Previously, Gayatri served as Rocket’s Chief Development Officer, LV, and Vice President, Regulatory Policy and Patient Advocacy. Since joining the company, Gayatri has played an instrumental role in advancing the LAD-I, Fanconi Anemia, and PKD programs through clinical development.

Prior to Rocket, Gayatri served as Director of the Office of Orphan Products Development (OOPD) within the U.S. Food and Drug Administration (FDA), where she was responsible for implementing statutory programs focused on promoting the development of medical products for rare diseases including the orphan drug and rare pediatric disease designation programs and overseeing more than $20M in grants to support natural history and clinical trial development. At the FDA, Gayatri served as the agency’s rare disease lead for interactions with Congress, international regulatory organizations, industry and patient advocacy groups.

Prior to heading FDA’s OOPD, Gayatri was an Associate Chief Counsel in the FDA’s Office of Chief Counsel with a focus on combination products, clinical trials, and medical devices, and began her career at an international law firm in Washington, D.C., specializing in healthcare and food and drug law related matters. She holds a JD from the University of Pennsylvania Law School, a Master of Bioethics from the University of Pennsylvania School of Medicine, and an MD from Rutgers New Jersey Medical School.

Syed Rizvi, MD

Chief Medical Officer

Syed Rizvi, MD

Chief Medical Officer

Syed Rizvi is the Chief Medical Officer (CMO) of Rocket Pharma, providing strategic leadership for clinical development and operations while overseeing medical directors, regulatory strategy, pharmacovigilance, biostats, and real-world evidence.

Syed brings over 20 years of experience across all stages of drug development, including clinical strategy, execution, and commercialization. Syed has a proven track record in the cell and gene therapy field including guiding the development and commercialization of multiple cell therapies for cancer treatments, including three approved autologous CAR-T cell therapies: CARVYKTI® and ABECMA® in myeloma and BREYANZI® in lymphoma.

Prior to joining Rocket in September 2025, Syed served as Chief Medical Officer at Poseida Therapeutics, an allogeneic cell therapies and genetic medicines company, where he advanced Poseida’s promising pipeline of allogeneic CAR-T and gene therapy programs to the point of the company’s acquisition by Roche in January 2025.

Before Poseida, Syed was Chief Medical Officer at Caribou Biosciences, an allogeneic cell therapy company, where he was responsible for overall strategy and execution of all clinical development programs. Prior to this, he served as Chief Medical Officer at Chimeric Therapeutics, where he helped build the company's pipeline by licensing programs from leading universities and progressing existing programs through the clinical development process. Previously, Dr. Rizvi held a series of global clinical leadership roles of increasing responsibility at Legend Biotech, Celgene, Novartis, Merck, and Genta Inc.

Syed received his M.D. from Dow Medical College and spent several years in direct patient care before joining Saint Vincent’s Comprehensive Cancer Center in New York. He has authored multiple scientific publications in peer-reviewed journals and is a member of the American Society of Clinical Oncology, the American Society of Hematology, the American Society for Transplantation and Cellular Therapy, and the European Hematology Association, among other professional organizations.

Jonathan Schwartz, MD

Chief Science and Gene Therapy Officer

Jonathan Schwartz, MD

Chief Science and Gene Therapy Officer

Jonathan Schwartz is Chief Science and Gene Therapy Officer for Rocket Pharma, responsible for advancing Rocket’s gene therapies through critical stages in development and enhancing the Company’s dedicated focus on strategic application of gene therapy technologies to current and future therapeutic areas. Jonathan joined Rocket as founding Chief Medical Officer in 2016 and has been instrumental in crafting and advancing an industry-leading pipeline of gene therapy programs across AAV cardiology and LV hematology portfolios.

Prior to Rocket, Jonathan was Vice President for Clinical Development at Stemline Therapeutics where he oversaw early development efforts for anticancer immunoconjugate, vaccine and small-molecule platforms. Before his role at Stemline, Jonathan had a seven-year tenure at ImClone/Eli Lilly, where he oversaw the development of the antiangiogenic monoclonal antibody Ramucirumab (CYRAMZA®) from end-of-Phase-1 through a Phase 3 program resulting in stomach, liver, lung and colorectal cancer approvals by the FDA and international health authorities. Jonathan has also participated in multistage development of several additional monoclonal antibodies.

Prior to joining the industry, Jonathan was Associate Professor of Medicine at the Mount Sinai Medical Center in New York, specializing in the treatment and translational research of hepatobiliary malignancies; he was also Director for the Hematology-Oncology Fellowship training program. Jonathan received a Bachelor of Arts in American Civilization from Brown University and an MD from Washington University in St. Louis. He completed post-graduate Internal Medicine and Hematology-Oncology training at the Mount Sinai and New York Presbyterian Hospitals.

Chris Stevens

Chief Operating Officer

Chris Stevens

Chief Operating Officer, Rocket Pharma

Chris Stevens is the Chief Operating Officer (COO) of Rocket Pharma, responsible for shaping corporate strategy, scaling operations, and driving business growth. As COO, Chris leads the quality, manufacturing, EHS, and corporate facilities organizations.

Chris is a seasoned executive with 25 years of experience across technical operations, product strategy, and general management. Prior to Rocket, Chris most recently served as Executive Vice President and Chief Patient Supply Officer at Spark Therapeutics, where he successfully led teams across manufacturing, supply chain, quality, compliance, engineering, EHS, and facilities, playing a key role in delivering gene therapies to patients globally.

During his tenure at Spark, he was also Head of Manufacturing through the approval and launch of Luxturna, the first gene therapy approved for a genetic disease in the U.S. and EU. He later served as Asset General Manager for the Hemophilia A program and subsequently Head of Product Strategy, overseeing program development for Spark’s entire portfolio.

Prior to Spark, Chris led teams at GlaxoSmithKline focused on strategy, operations, and planning, as well as GMP clinical manufacturing operations including process transfer and cell banking. Earlier in his career he held roles of increasing responsibility at Bristol-Myers Squibb in process engineering, manufacturing and supply chain in Massachusetts, Indiana and Puerto Rico.

Chris received his M.S. in Industrial Management from the University of Southern Indiana and a B.S. in Chemical Engineering from Purdue University.

Martin Wilson, JD

General Counsel & Chief Corporate Officer

Martin Wilson, JD

General Counsel & Chief Corporate Officer

Martin Wilson is the General Counsel & Chief Corporate Officer for Rocket Pharma. Martin joined the company in December 2021 as its founding General Counsel & Chief Compliance Officer. In his current position, Martin leads the integrated Legal, Compliance, Finance, IT, People, and Business Operations functions as the company plans its expected transition to commercial stage.

Prior to joining Rocket, Martin was General Counsel and Chief Corporate Officer at Ichnos Sciences, where he oversaw Legal, Compliance, Business Development, Human Resources and IT. He helped establish Ichnos as an independent entity and set up its corporate structure.

Before Ichnos, Martin served as General Counsel, Chief Compliance Officer, Corporate Secretary and Head of Human Resources at Teligent, Inc. Prior to Teligent, he was Vice President and Assistant General Counsel at Endo Pharmaceuticals following the acquisition of Par Pharmaceuticals. Martin held multiple roles of increasing responsibility at Par over the course of 11 years, including Chief Compliance Officer.

Earlier in his career, Martin worked in the Licensing group at Schering-Plough. He earned JD from Villanova University Charles Widger School of Law. In his spare time, Martin is an avid skier and basketball player and is involved in youth sports with his two young children.

Board of Directors

Roderick Wong, MD, MBA

Chairman of the Board

Roderick Wong, MD, MBA

Chairman of the Board

Dr. Wong founded and serves as Managing Partner of RTW Investments. Rod has more than 12 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 MBA from Harvard Business School, and graduated Phi Beta Kappa with a Bachelor of Science 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 Björk, MD, PhD

Independent Director

Elisabeth Björk, MD, PhD

Independent Director

Dr. Bjӧrk was previously Senior Vice President, Head of Late-stage Development, Cardiovascular, Renal and Metabolism (CVRM), BioPharmaceuticals R&D at AstraZeneca, where she led the global development of medicines within this area. Throughout her career at AstraZeneca, she 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

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 Worldwide 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.

Mikael Dolsten, MD, PhD

Director

Mikael Dolsten, MD, PhD

Director

Dr. Dolsten is a seasoned accomplished industry executive with extensive global pharmaceutical management experience who previously served as Chief Scientific Officer, President, Research & Development (R&D) at Pfizer, focusing on advancing gene therapies, small-molecule medicines, biotherapeutics, and vaccines. Previous roles include President of Research at Wyeth and Executive Vice President, Head of Worldwide Research at Boehringer Ingelheim, and senior R&D positions at AstraZeneca, Pharmacia, and Upjohn. Dr. Dolsten is a member of the Board of Overseers for the Scripps Research Institute and a Foreign Member of The Royal Swedish Academy of Engineering Sciences. He also advised the Obama administration on regulatory and drug development issues and then Vice President Biden’s Cancer Moonshot Initiative. He is a named inventor on several patents and has published approximately 150 articles in international journals, with contributions in molecular cell biology, immunology, and oncology. Dr. Dolsten received a PhD in tumor immunology and MD from the University of Lund in Sweden.

Peter Fong, PhD

Director

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 bachelor’s degree in industrial engineering from Purdue University.

Fady Malik, MD, PhD

Independent Director

Fady Malik, MD, PhD

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.

Piratip Pratumsuwan

Independent Director

Piratip Pratumsuwan

Independent Director

Piratip brings over a decade of experience in healthcare investment and research. As Managing Director, Research Analyst at RTW Investments, a healthcare-focused investment firm, he has been integral to guiding the firm’s investment strategy since joining in 2014 directly from academia. Piratip specializes in researching transformative technologies, with a particular focus on gene therapy and gene editing. He leads RTW’s comprehensive efforts in these fields, which encompass investments in both public and private companies as well as within academic institutions. Piratip holds an M.A. in Biotechnology from Columbia University and a B.S. in Biochemistry from McGill University.

Gaurav Shah, MD

Chief Executive Officer

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.

Gaurav graduated from Harvard College (summa cum laude, Phi Beta Kappa) with a degree in behavioral neuroscience. He received his MD from Columbia (AOA), completed his internal medicine residency at Brigham and Women’s Hospital, and hematology/oncology fellowship training at Memorial-Sloan Kettering. After receiving board certification in medical oncology, he served as an Adjunct Assistant Professor of Oncology at Columbia.

To find balance, Gaurav also has a passion for Indian classical music and has toured extensively including performances at Carnegie Hall and on National Geographic TV, a feature in the Smithsonian Grammy nominations in 2019 (Falu’s Bazaar) and 2024 (Abundance in Millets) and 2022 Grammy win (A Colorful World).

David Southwell, MBA

Independent Director

David Southwell, MBA

Independant Director

Mr. Southwell is an experienced executive with over 30 years of experience leading biotechnology and pharmaceutical companies and is currently the Chief Executive Officer of Volastra. Previously, he served as President, Chief Executive Officer, Board member of TScan Therapeutics from 2018 to 2023 and 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 Bachelor of Arts from Rice University and an MBA 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.

Scientific Advisors

Juan Bueren, PhD
Head of the Hematopoietic Innovative Therapies Division at CIEMAT and CIBERER

Hans-Peter Kiem, MD, PhD
Deputy Director and Professor, Translational Science and Therapeutics Division, Fred Hutch Cancer Center

Donald Kohn, MD, UUC, BS, MS
Director of the UCLA Human Gene and Cell Therapy Program

Axel Schambach, PhD
Professor and Director of the Institute of Experimental Hematology

Jose Carlos Segovia, PhD
Divisional Head, Cell Technology Division, CIEMAT

Matthew Taylor, MD, PhD
Clinical Genetics and Genomics, UCHealth University of Colorado Hospital

John Wagner, MD
Director of the Institute of Cell, Gene and Immunotherapeutics, University of Minnesota

Barry Byrne, MD, PhD
Associate Chair of Pediatrics, Director of the Powell Gene Therapy Center, University of Florida

Partners

Children’s Hospital of Philadelphia (CHOP)

1 of 13

Temple University

2 of 13

REGENXBIO

3 of 13

Memorial Sloan Kettering Cancer Center

4 of 13

NYU

5 of 13

CIRM

6 of 13

University of Minnesota

7 of 13

Stanford Medical School

8 of 13

Forty Seven, a Gilead company

9 of 13

University of Colorado

10 of 13

University of California, Los Angeles

11 of 13

University of California, San Diego

12 of 13

Fred Hutchinson Cancer Research Center

13 of 13

CIBER

CIBERER

CIEMAT

1 of 5

University College London Great Ormond Street Hospital for Children

2 of 5

Lund University

3 of 5

MolMed

4 of 5

University of Hannover

5 of 5

Discover how Rocket Pharmaceuticals is addressing environmental, social, and governance (ESG) factors in our business operations.

Click below to view reports.

View 2024 ESG Report
View 2023 ESG Report
View 2022 ESG Report
Scroll to Top