About Us

About Us - Rocket employees at work

Mission, Vision & Values

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 meant “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, egolessness. No single one of us can do this job alone, and it is ok 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

History

July 14, 2015
October 2015
December 2015
H1 2016
March 2016
H1 2016
July 2016
February 2017
September 2017
H2 2017
January 4, 2018
May 2018
June 2018
July 2018
October 2018
November 2018
November 2018
January 2019
February 2019
October 2019
December 2019
H2 2020
July 2020
December 2020
February 2021
June 2021
December 2021
February 2022
March 2022
May 2022
July 2022
July 2022
September 2022
December 2022
December 2022
January 2023
February 2023
February 2023
Timeline Photo

Rocket becomes incorporated with initial idea and funding from RTW Investments

Timeline Photo

Gaurav Shah, MD becomes CEO of Rocket

Timeline Photo

Rocket closes Series A financing

Jonathan Schwartz, MD and Kinnari Patel, PharmD, MBA

Jonathan Schwartz, MD and Kinnari Patel, PharmD, MBA join Rocket team

Timeline Photo

Rocket licenses Pyruvate Kinase Deficiency (PKD) program

Timeline Photo

The growing team moves to the Alexandria Center of Life Sciences in NYC

Rocket licenses Fanconi Anemia (FA) program

Rocket licenses Danon Disease program

Timeline Photo

Rocket licenses Leukocyte Adhesion Deficiency-I (LAD-I) program

Rocket closes Series B financing

Timeline Photo

Rocket goes public with only 12 employees

Timeline Photo

Rocket announces first clinical data for Fanconi Anemia (FA) program at American Society of Gene and Cell Therapy (ASGCT) annual meeting

Rocket licenses Infantile Malignant Osteopetrosis (IMO) program

Rocket headquarters moves to Empire State Building

IND for Fanconi Anemia accepted by U.S. Food and Drug Administration

Rocket announces the Danon Disease program

IND for Leukocyte Adhesion Deficiency-I (LAD-I) accepted by U.S. Food and Drug Administration

IND for Danon Disease accepted by U.S. Food and Drug Administration

Rocket hosts inaugural Rare Disease Day 

IND for Pyruvate Kinase Deficiency (PKD) accepted by U.S. Food and Drug Administration

Timeline Photo

Rocket announces first clinical data for Leukocyte Adhesion Deficiency – I (LAD-I) program at American Society of Hematology (ASH) Annual Meeting

Rocket opens new facility in new jersey

Rocket opens state-of-the-art manufacturing facility in Cranbury, New Jersey

Rocket Celebrates 5 Years

Rocket Celebrates 5 Years

December 2020 News Release

Rocket presents updated, positive data in PKD, FA and LAD-1 at ASH and in Danon disease, representing possibly the first viable gene therapy approach for cardiac diseases.

February 2021 RARE Disease Month

Rocket again celebrates Rare Disease Day by lighting up iconic buildings and hosting webinar featuring rare disease community advocates, including Abbey Meyers.

Rocket hosts its more than 130 employees for a summer townhall and team building event at its state-of-the-art manufacturing facility in Cranbury, NJ.

Dec 2021 Press Release - Positive Clinical Data

Rocket presents updated, positive data in Danon Disease, FA, LAD-I and PKD
Rocket Celebrates Rare Disease Day 2022

Rocket celebrates Rare Disease Day by hosting 250+ members of the rare disease community and Rocket team at the NASDAQ Tower in Times Square and virtually.

Rocket holds spring townhall featuring inspirational LAD-I patient family and professional development workshops.

May-2022-Cell-Therapy-Annual Meeting

Rocket presents top-line pivotal data from FA and LAD-I programs at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

July 2022 Wall of Stars

Rocket hosts summer townhall headlined by pediatric patient living with Danon Disease and unveiling of the Wall of Stars.

Rocket achieves Current Good Manufacturing Practice (cGMP) readiness milestone for state-of-the-art AAV manufacturing facility in Cranbury, N.J.

Sept-2022-HFSA

Rocket presents positive data from Phase 1 trial in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting, including updated safety and efficacy data from patients in the pediatric and adult cohorts.
Dec 2022

Rocket completes acquisition of Renovacor, extending leadership in AAV-based cardiac gene therapy
December 2022 - ASH Presentation - Timeline photo

Rocket presents positive updated data from the Phase 2 pivotal study for Fanconi Anemia and longer-term follow-up from the Phase 2 study for PKD at the 64th American Society of Hematology (ASH) Annual Meeting.

January 2023 - Press Release - Timeline photo

Rocket expands cardiac gene therapy portfolio with addition of RP-A601 for PKP2-ACM and announces positive updated data for RP-A501 in Danon Disease at the 41st Annual J.P. Morgan Healthcare Conference in San Francisco.
(RMAT) Designation for RP-A501

FDA grants RMAT designation to RP-A501 for Danon Disease. RP-A501 is the first cardiac gene therapy to receive RMAT designation.
Rocket celebrates Rare Disease Day

Rocket celebrates Rare Disease Day by hosting inspiring event at Make A Wish New Jersey and advancing Light Up for Rare initiative.

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, and a 2019 Grammy nomination (Falu’s Bazaar) and 2022 Grammy win (A Colorful World).

Kinnari Patel, PharmD, MBA

Kinnari Patel, PharmD, MBA

President & Chief Operating Officer

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.

Kinnari 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. 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.

In her spare time, Kinnari has a passion for mentoring, teaching and volunteering for non-profit organizations.

Mayo Pujols

Mayo Pujols

Chief Technical Officer, EVP

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.

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.

Isabel Carmona, JD

Isabel Carmona, JD

Chief Human Resources Officer, SVP

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.

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 Officer

Carlos Martin

Chief Commercial Officer
Carlos Martin is the Chief Commercial Officer of Rocket Pharma. Prior to joining Rocket in April 2021, 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, SVP

Raj Prabhakar, MBA

Chief Business Officer, SVP

Raj Prabhakar is the Chief Business Officer and SVP 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.

Gayatri R. Rao, MD, JD

Chief Development Officer of LVV, SVP

Gayatri R. Rao, MD, JD

Chief Development Officer of LVV, SVP

Gayatri Rao is the Chief Development Officer of LVV, which includes the FA, LAD-I and PKD Programs. Prior to this role, she served as a Vice President for Regulatory Policy and Patient Advocacy at Rocket Pharma.  Gayatri most recently served as Director of the Office of Orphan Products Development (OOPD) within the US Food and Drug Administration (FDA), where she was responsible for implementing statutory programs focused on promoting the development of medical products for rare diseases.  At the FDA, Gayatri served as the FDA’s rare disease lead for interactions with Congress, international and national regulatory organizations, industry sponsors and patient advocacy groups. During her tenure, she oversaw a dramatic rise in the number of orphan drug designation requests, granted over 2000 designations, and issued over 250 orphan exclusivity decisions.  In addition, she worked to successfully implement the Rare Pediatric Disease Priority Review Voucher program and launched a $2M Orphan Product Natural History Grant Program to complement the office’s longstanding $15M Orphan Product Clinical Trial Grant Program and $6M Pediatric Device Consortia Grant Program.

Prior to heading FDA’s OOPD, Gayatri was an Associate Chief Counsel in the FDA’s Office of Chief Counsel and began her career at an international law firm in Washington, D.C., where she focused on 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.

Jonathan Schwartz, MD

Chief Gene Therapy Officer, SVP

Jonathan Schwartz, MD

Chief Gene Therapy Officer, SVP

Jonathan Schwartz is Chief Gene Therapy Officer for Rocket Pharma, focused on 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, 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 BA 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.

Jonathan enjoys running, cooking and gardening and is a foremost authority on 1970s soul and funk music.

Mark White, MB.ChB

Chief Medical Officer, SVP

Mark White, MB.ChB

Chief Medical Officer, SVP

Mark White, MB.ChB, is Chief Medical Officer for Rocket Pharma. 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. 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 spearheaded approvals and launches to achieve mega-brand status in severe eosinophilic asthma, as well as in the design and delivery of a large lifecycle management program across multiple conditions including rare diseases, with the granting of five Orphan Drug Designations (ODDs) and one Breakthrough Therapy Designation (BTD).

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 in the design and delivery of clinical pharmacology trials and 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 and Chief Compliance Officer, SVP

Martin Wilson, JD

General Counsel and Chief Compliance Officer, SVP

Martin Wilson is the General Counsel and Chief Compliance Officer for Rocket Pharma. 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 his Juris Doctorate 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.

John Militello, CPA

VP, Principal Accounting Officer

John Militello, CPA

VP, Principal Accounting Officer

John Militello joined the Company in January 2018 as Controller and serves as principal accounting officer. Prior to joining Rocket Pharma, John worked at Immune Pharmaceuticals, Inc., a publicly traded biotechnology company, where he most recently served as VP of Finance and Controller and served as the principal financial officer.  Prior to that, John was an Assistant Controller with Retrophin, Inc., a San Diego based biotech company, and the Manager, External Reporting & Compliance at Volt Information Sciences, Inc., a publicly traded staffing company. Prior to Volt Information Sciences, Inc., John was a Senior Manager with BDO USA, LLP serving multi-national SEC registrants and a member of the Life Sciences Practice.

Mr. Militello is a Certified Public Accountant and earned his Bachelor of Science degree in Accounting from St. Joseph’s College.

Jessie Yeung, MBA

VP, Investor Relations and Corporate Finance

Jessie Yeung, MBA

VP, Investor Relations and Corporate Finance
Jessie joined Rocket as VP of Investor Relations and Corporate Finance in 2022. Most recently, Jessie was the Head of Corporate Finance and Investor Relations at Legend Biotech. During her two and a half years at Legend, she raised over $1 billion from high-quality investors through private placements and helped launch the biggest biotech IPO of 2020. In addition, she led Financial Planning & Analysis, as well as Corporate Communications. Prior to this role, she was an equity research analyst at Bank of America Merrill Lynch covering the large-cap biotechnology sector. Jessie was the financial lead on the biotechnology research team where she built and maintained over 30 dynamic financial models. She also gained significant biotech IPO exposure as she was involved in over 10 IPOs, establishing cash flow and profitability projections and conducting due diligence for IPOs.
 
She has developed an extensive network of investors during her 4 years of equity research experience - prior to Bank of America, Jessie was with Wells Fargo equity research covering the Pharmaceuticals sector and also at J.P.Morgan in equity research covering consumer retail companies.
 
Prior to equity research, Jessie was with J.P.Morgan for over 10 years, where she held a number of corporate finance roles including financial planning and analysis, product and valuation control, business development as well as risk management. She was also responsible for annual budgeting, financial performance reporting, headcount, and expense management.
 
Jessie holds a Bachelor’s Degree in Business Administration / Finance from Carnegie Mellon University, and an MBA in Finance from Columbia Business School.

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 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

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

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 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

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

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

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

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

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.

Scientific Advisors

Eric Adler, MD
Danon Advisory Board
Director of Cardiac Transplant and Mechanical Circulatory Support, UC San Diego

Juan Bueren, PhD
Fanconi & LAD Advisory Board
CIEMAT Gene Therapy Head

Hans-Peter Kiem, MD
Fanconi Advisory Board
Fred Hutchinson Cell & Gene Therapy Director

Donald Kohn, MD, UUC, BS, MS
LAD and Osteopetrosis Advisory Board
UCLA Human Gene Medicine Program Director

Johan Richter, MD, PhD
Osteopetrosis Advisory Board
Lund University Hospital Gene Therapy Head

Maria Grazia Roncarolo, MD
George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, Director of the Center for Definitive and Curative Medicine, and Co-director of the Institute for Stem Cell Biology and Regenerative Medicine, Stanford University

Axel Schambach, PhD
Lecturer on Pediatrics, Boston Children’s Hospital

Jose Carlos Segovia, PhD
PKD Advisory Board
CIEMAT Investigator

Julian Sevilla, MD, PhD
Clinical Investigator, Hospital Infantil Universitario Niño Jesús

Matthew Taylor, MD, PhD
Danon Advisory Board Director, Adult Clinical Genetics, University of Colorado Anschutz

Adrian Thrasher, MD, PhD
Fanconi and LAD Advisory Board UCL Institute of Child Health, Great Ormond Street Hospital (GOSH), NHS Trust

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

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

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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
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