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

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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

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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

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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

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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

Diversity, Equity and Inclusion at Rocket

Rocket celebrates diversity and our team is a testament to that. We foster an open environment where all ideas are welcome at the table, and every team member’s voice is valued.

Rocket is firmly committed to fostering a diverse workforce and advancing gender equality.

  • About half (47%) of the Rocket team identifies as female*
  • Over half of Rocket’s senior leadership team (64%) and employee base (48%) are BIPOC (Black, Indigenous and People of Color)*

Ensuring a diverse workforce and gender equality is something we take seriously here at Rocket. We’re committed to creating a collaborative space where differences are celebrated and unique perspectives are appreciated, united behind our mission of seeking gene therapy cures.

All team members are encouraged to embrace opportunities for learning and professional growth, regardless of experience or titles. We have a genuine commitment to lifelong learning and believe everyone, even the most accomplished among us, has room to grow.

*Based on the latest data available from March 2024.

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

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

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September 2024
MikaelDoisten

Mikael Dolsten, M.D., Ph.D., joins Board of Directors

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September 2024
may-2024-orphan-medical-product-designation-1

Rocket announces completion of enrollment the Phase 2 pivotal trial of RP-A501 for Danon disease

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May 2024
may 2024 orphan medical product designation

Received Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2-ACM

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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).

Kinnari Patel, PharmD, MBA

Kinnari Patel, PharmD, MBA

President, Head of R&D and Chief Operating Officer

Kinnari Patel, PharmD, MBA

President, Head of R&D and Chief Operating Officer

Dr. Kinnari Patel is President, Head of R&D and Chief Operating Officer for Rocket Pharma, overseeing the global R&D and Technical Operations leadership team with oversight spanning discovery through late-stage development and manufacturing. This includes regulatory, clinical, global program teams, CMC, quality and the development organization. Dr. Patel’s prior responsibilities at Rocket included IR, IT, HR, finance and corporate compliance. Dr. Patel has more than 20 years of R&D experience in rare diseases and across several therapeutic areas including immuno-oncology, oncology, respiratory, virology, transplantation, cardiology and metabolism.

Dr. Patel has played a pivotal role in raising approximately $1 billion in financing, overseeing the transition of Rocket to a publicly traded company and facilitating the acquisition of Renovacor. She has helped grow Rocket from a three to about 300-person company with a diverse and inclusive culture. She maintains a strong focus on nurturing talent, leading the creation of Rocket University and the Gene Therapy Drug Development Certificate Program. Dr. Patel also spearheaded the development of the Rocket manufacturing site headquartered in Cranbury, NJ from its inception to a fully operational facility. In addition to her current role, Dr. Patel serves as the Rocket board observer and member of the Audit & Compensation Committee.

Before joining Rocket, Dr. Patel gained deep industry experience at leading pharmaceutical companies, including AstraZeneca, Bristol-Myers Squibb, Novartis and Hoffmann La-Roche. She has led both small molecule and biologic development programs from Phase I through Phase IV. Most notably, Dr. Patel has worked on eight drugs that have been submitted for review and approvals across the U.S. and EU, including Opdivo® (nivolumab) for metastatic melanoma, RCC and NSCLC and Myalept® (metreleptin) for ultra-rare lipodystrophy disease. 

Dr. Patel received the dual degrees of B.S. in Biology and Doctorate of Pharmacy from the USciences in Philadelphia, PA. She also completed a two-year Post-Doctoral Regulatory Affairs Fellowship through Rutgers University with Johnson & Johnson and Pfizer. Additionally, she received her Executive MBA from NYU Stern School of Business with specialization in Corporate Finance, Leadership and Strategy. Most recently, she graduated from the C-Suite Harvard Business School Advanced Management Program.

Beyond her dedication to Rocket, Dr. Patel is a member of the Alliance for Regenerative Medicine’s (ARM) Board of Directors and serves on the Healthcare Businesswomen’s Association (HBA) Global Advisory Board. She has devoted her life to helping others through fostering a diverse, equitable and inclusive culture and advocating for female business leaders. In addition, Dr. Patel is deeply committed to making a positive impact in advancing science for rare diseases and has been widely recognized as a fearless advocate for patients and their loved ones impacted by these devastating conditions across the globe.

Jonathan Schwartz, MD

Chief Medical & Gene Therapy Officer

Jonathan Schwartz, MD

Chief Medical & Gene Therapy Officer

Jonathan Schwartz is Chief Medical & 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. In this position, he oversees Medical Directors, Clinical Operations, Clinical Scientists, Biometrics, Translational Science and Pre-clinical Research and Development. 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.

Aaron Ondrey

Aaron Ondrey

Chief Financial Officer

Aaron Ondrey,

Chief Financial Officer

Aaron Ondrey is Chief Financial Officer for Rocket Pharma, leading the company’s Finance, Investor Relations, and Corporate Communications functions. Aaron is a seasoned finance executive with more than 20 years of experience in financial management, strategic planning, commercial finance, capital allocation and mergers and acquisitions across multiple therapeutic areas.

Previously, Aaron served as CFO of Mirati Therapeutics, overseeing all aspects of Finance, IT, and Facilities through the launch of KRAZATI® and the company’s acquisition by Bristol Myers Squibb in January 2024. Prior to joining Mirati as Senior Vice President, FP&A in July of 2022, Aaron was the Vice President, Finance, at Arena Pharmaceuticals and oversaw FP&A, Corporate Planning and Strategic sourcing.

Prior to Arena, Aaron served as the Head of U.S. and Global Commercial Finance at Alexion Pharmaceuticals with responsibility for financial management of approximately $5 billion in revenue across more than 50 countries. He previously held leadership positions of increasing responsibility supporting multiple product launches at Regeneron Pharmaceuticals, most recently as Head of Commercial Finance, Business Planning. Aaron received a Bachelor of Sciences in finance from Case Western Reserve University.

In his spare time, Aaron enjoys spending time with his family, including his wife, three kids and three dogs. He is an avid soccer fan and loves to cook. Sunday morning pancakes and midnight noodles are family favorites. Aaron balances his time between the countryside of Connecticut and the coast of Maine.

Isabel Carmona, JD

Isabel Carmona, JD

Chief People Officer

Isabel Carmona, JD

Chief People Officer

Isabel Carmona is the Chief People Officer for Rocket Pharma, overseeing all aspects of the company’s People strategy and operations through a period of rapid growth as the company plans its transition to commercial stage. 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.

Isabel has developed her career across the United States and Europe, where she has held leadership positions in legal, compliance, operational excellence, and human resources in multiple industries including life sciences, information technology and financial services.

Isabel earned her JD degree with a specialty in business and international law from the Universidad de Sevilla and is also an avid listener of classical music and operas particularly of those set in her hometown of Sevilla, Spain.

Carlos Martin

Chief Commercial Operations and Revenue Officer

Carlos Martin

Chief Commercial Operations and Revenue Officer

Carlos Martin is the Chief Commercial Operations and Revenue Officer of Rocket Pharma, leading the Marketing, Commercial Operations and Business Insights & Analytics functions. He joined Rocket in April 2021 and established the company’s early commercial strategy and infrastructure as Chief Commercial Officer.

Prior to joining Rocket, Carlos was the U.S. Commercial Head of Advanced Accelerator Applications (AAA), a Novartis Oncology Company. His responsibilities included leading commercial strategy and new product launches as well as transforming their go-to-market model.

Before joining AAA, Carlos held roles of increasing responsibility at Novartis, where he developed his career across the U.S., Europe, Latin America and Asia. He brings in-depth background and experience globally and locally in commercial strategy and operations across a wide range of modalities and disease areas, including oncology, cardiovascular, metabolism and complex therapeutics.

Carlos earned a bachelor’s degree in economics from ESADE & the Polytechnic University of Barcelona, Spain and an MBA from the Thunderbird School of Global Management at Arizona State University. He is passionate about extending and improving patients’ lives through scientific and technological advancements.

Raj Prabhakar, MBA

Chief Business Officer

Raj Prabhakar, MBA

Chief Business Officer

Raj Prabhakar is the Chief Business Officer for Rocket Pharma. Raj has over twenty years of broad experience, including the last 19 years at clinical development-stage biotechnology companies. Raj most recently comes to Rocket from Caladrius Biosciences, which through the subsidiary PCT as well as proprietary Regulatory T-cell and CD34 cell therapy programs, had direct hand-on business, manufacturing and operating experience in clinical-stage autologous ex-vivo cell and cell-based gene therapy programs. Prior to Caladrius Raj spent nearly ten years at Celsion Corporation leading multiple oncology candidates from Phase I through Phase III pivotal programs, spanning platforms including small and large molecules, including oligonucleotides and in-vivo non-viral gene therapy approaches to cancer. Prior to holding strategic planning and business development roles, Raj has also worked in clinical program management, manufacturing and operations. Raj has also worked at non-profit organizations such as PATH Vaccine Development Program, and private-staged companies such as Osiris Therapeutics and Protiveris.

Raj holds dual Bachelor of Sciences degrees in biology and mechanical engineering from MIT and an MBA from the Harvard Business School.

Mayo Pujols

Mayo Pujols

Chief Technical Officer

Mayo Pujols

Chief Technical Officer

Mayo Pujols is Chief Technical Officer for Rocket Pharma, leading 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.

Mayo has nearly three decades of experience, working in leadership roles across technical operations, quality operations, validation, process development and cGMP. Prior to joining Rocket in July 2022, Mayo was Chief Executive Officer of Andelyn Biosciences, where he led their expansion to a full-service gene therapy CDMO. Over the course of his career, he has also held key roles at Novartis, Celgene, Merck, Advaxis, MedImmune and Schering-Plough.

Mayo 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.

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.

Mark White, MB.ChB

General Manager, Commercial Affairs

Mark White, MB.ChB

General Manager, Commercial Affairs

Mark White, MB.ChB, is General Manager, Commercial Affairs for Rocket Pharma, overseeing the Medical Affairs, Marketing, Commercial Operations, and Value & Access functions. He previously served as Chief Medical Officer, guiding the Medical, Clinical, Pharmacovigilance, and Biometrics organizations through the company’s first product filing and period of rapid growth as the company plans its transition to commercial stage.

Mark is a passionate and seasoned drug developer with more than 25 years of industry experience and rich expertise in late-stage drug development, global regulatory submissions and commercial strategy and launches. Prior to joining Rocket, Mark was Global Franchise Head for Fasenra (benralizumab) at AstraZeneca, where he led a large, multidisciplinary team through Phase 3 delivery, including global regulatory approvals. Additionally, he led the team through approvals and launches to achieve mega-brand status in severe eosinophilic asthma, as well as in the design and delivery of a large evidence generation program in severe asthma and in lifecycle management across multiple conditions including rare diseases, with the granting of five orphan drug designations and one Breakthrough Therapy designation.

At AstraZeneca, Mark was previously the program lead for Myalept (metreleptin) at the time of U.S. approval and launch for the ultra-rare condition of generalized lipodystrophy, the program lead for Saphnelo (anifrolumab) in systemic lupus erythematosus (SLE), and at MedImmune was the program lead for the influenza vaccine portfolio. In his earlier career, Mark spent time in global marketing, portfolio strategy, clinical development including as a late-stage clinical lead for multiple Phase 3 trials and regulatory submissions.

Mark joined the industry from clinical practice in the United Kingdom with post-graduate qualifications in acute medicine (MRCP), anesthesiology and intensive care (FRCA), as well as in pharmaceutical medicine (MFPM). He obtained his Bachelor of Medicine and Bachelor of Surgery (MB.ChB) at the University of Manchester School of Medicine.

Outside of work, Mark’s natural habitat is high in the mountains, but he also enjoys road and mountain biking, skiing, running and traveling with his family.

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 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.

Pedro Granadillo, BS

Independent 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.

Gotham Makker, MD

Independent Director

Gotham Makker, MD

Independent Director

Dr. Makker has more than 17 years of healthcare industry experience. Dr. Makker is a partner and head of strategic investments at RTW. Prior to joining RTW, he 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 MD 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

David Southwell, MBA

Director

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 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.

Naveen Yalamanchi, MD

Independent Director

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 Bachelor of Science in biology from MIT and an MD from Stanford University. He completed his orthopedic surgery internship at UCLA Medical Center.

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.

R. Keith Woods

Independent Director

R. Keith Woods

Independent Director

Mr. Woods is a seasoned executive with more than 30 years of industry experience who most recently served as Chief Operating Officer of argenx, where he led its global commercial organization, including marketing, market access, medical affairs, program management, and supply chain, during the company’s successful transition to commercial stage. He helped launch VYVGART®, a treatment for a rare autoimmune condition that causes muscle weakness, generating more than $1 billion in sales. Prior to argenx, Mr. Woods was the Senior Vice President of North America Business Operations at Alexion Pharmaceuticals, where he oversaw the U.S. and Canada business and was responsible for more than $1 billion in annual sales. He previously served as Vice President and Managing Director of Alexion UK, Vice President of U.S. Commercial Operations and Executive Director of Sales, leading the launch of SOLIRIS® in atypical hemolytic uremic syndrome. Mr. Woods received a Bachelor of Science in marketing from Florida State University.

Mikael Dolsten, M.D, PhD

Independent Director

Mikael Dolsten, M.D, PhD

Independent Director

Dr. Dolsten is a seasoned accomplished industry executive with extensive global pharmaceutical management experience who currently serves 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.

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.

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