Leukocyte Adhesion Deficiency Type 1

CRANBURY, N.J.--( BUSINESS WIRE )-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease. After the two-patient safety run-in, followed by harmonized global site activations, the remaining 10 patients were enrolled across the United States (U.S.) and European Union within three months. Given the prevalence of Danon disease across regions, the Company plans to pursue regulatory filings concomitantly in the U.S. and ex-U.S. “ From a clinical perspective, the important thing is that we are moving closer to the goal of having a treatment for patients with Danon disease,” said Barry H. Greenberg, MD, FHFSA, Director of the Advanced Heart Failure Treatment Program and Distinguished Professor of Medicine at UC San Diego Health. “ I can attest to the excitement and anticipation within the Danon patient community for this novel, one-time treatment designed to improve cardiac abnormalities associated with Danon disease and help preserve normal cardiac function by delivering functional LAMP2B genes to the heart tissue. The rapid recruitment of the Phase 2 trial signifies the positive views of the study clinicians regarding this investigational therapy.” RP-A501 Phase 2 Pivotal Trial Overview The global, single-arm, multi-center Phase 2 pivotal trial evaluates the efficacy and safety of RP-A501 in 12 patients with Danon disease, including a pediatric safety run-in (n=2), and a dose level of 6.7 x 10 13 GC/kg. To support accelerated approval, the study assesses the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression, and reductions in left ventricular mass. Key secondary endpoint is change in troponin. Additional secondary endpoints include natriuretic peptides, Kansas City Cardiomyopathy Questionnaire, New York Heart Association class, event free survival to 24 months and treatment emergent safety events. These endpoints could support full approval with longer-term follow-up. A global natural history study is running concurrently with the Phase 2 pivotal trial. The pediatric run-in enrolled two patients in a sequential manner with a minimum three-month follow-up prior to subsequent enrollment. In addition, all patients enrolled in the trial are required to have a three-months observational pre-treatment run-in to enable an assessment of troponin (and other biomarker) trajectories to optimally assess this key secondary endpoint. Details about the Phase 2 study can be found at www.clinicaltrials.gov under NCT identifier NCT06092034. About RP-A501 RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. Danon disease is caused by mutations in the LAMP2 gene. RP-A501 consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene (AAV9.LAMP2B) which, when inserted into heart cells harboring mutations in the endogenous LAMP2 gene, has the potential to fully restore cardiac function at its root. RP-A501 represents a single dose treatment and is administered as an intravenous infusion. In preclinical and clinical studies, AAV9.LAMP2B has been shown to target cardiac cells (cardiomyocytes) and deliver the functional LAMP2B gene to heart tissue, which ultimately leads to improved cardiac structure and function in patients. In 2023, the U.S. Food and Drug Administration granted regenerative medicine advanced therapy designation, and the European Medical Agency granted PRIority MEdicines (PRIME) eligibility to RP-A501. About Danon Disease Danon disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon disease is cardiac transplantation, which is associated with substantial complications and is not considered curative, representing the high unmet medical need for patients with Danon disease. In 2023, Rocket secured an ICD-10 code from the Centers for Medicare and Medicaid Services (CMS) to document patients with LAMP2 deficiency in Danon disease. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket’s lentiviral vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s adeno-associated viral vector-based cardiovascular portfolio includes a late-stage program for Danon disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn , YouTube, and X . Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as “could,” “believe,” “expect,” “anticipate,” “intend,” “plan,” “will give,” “estimate,” “seek,” “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, including the timing and outcome of the FDA’s review of the additional CMC information that Rocket will provide in response to the FDA’s request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket’s ability to establish key collaborations and vendor relationships for its product candidates, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket’s ability to transition to a commercial stage pharmaceutical company, and Rocket’s expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into 2026. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2023, filed February 27, 2024 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Industry veteran Doug Williams is back with a new approach to CAR-T cell therapies that he expects to take into the clinic in the US by year’s end. After a brief stint at Sana Biotechnology , leaving his R&D chief post in the spring, Williams is returning to the role of CEO at a little-known biotech. In recent weeks, he took the top spot at China-born and now California-headquartered biotech TriArm Therapeutics . It’s a position he held at Codiak BioSciences , a startup that tried turning exosomes into medicines, but the relatively nascent idea couldn’t survive on the public markets in 2023. Now, he’s driving TriArm’s mission to “democratize” CAR-T cell therapies using what it calls a “fast-in-time” (FIT) and non-viral manufacturing process, Williams told Endpoints News . He said TriArm’s technology platform has led to a two-day manufacturing process. “There’s also some potential advantages to cell fitness, the actual fitness of the CAR, the composition of cells that we’re reinfusing back into the patient,” Williams said. “We’re preserving a population of cells using this FIT process that may end up being more fit from a biologic perspective because they’re enriched in STEM and naïve T cells,” he added. TriArm has been relatively quiet since launching in 2019. Its financial backers include Panacea Venture , MSD Capital and Lombard Odier , and it employs about 100 people across Shanghai, Taipei and San Mateo, CA. The company aims to speed up the testing of CAR-T therapies and make faster go/no-go R&D decisions by employing an investigator-initiated model in China and then moving into clinical studies in the US, Williams said. “As somebody that is a drug developer by choice and by training, to be able to get a glimpse of what is arguably real data in a population of patients as sort of a preamble to filing an IND and really moving ahead in larger, much more expensive trials, it’s a fantastic setup to have available,” Williams said. Its first US-based trial, testing a CD19-directed autologous therapy, is slated to start later this year, Williams said. A gene therapy could soon enter a similar patient setting with the emergence this week of a new biotech called Vironexis . — Kyle LaHucik → Biogen ’s board continues to evolve under chair Caroline Dorsa , who replaced Stelios Papadopoulos last year. On Jan. 1, ex- AstraZeneca BioPharmaceuticals R&D chief Mene Pangalos will take a seat on the board, and Lloyd Minor — the dean of Stanford University’s School of Medicine — will become a board member much earlier on Oct. 1. After the hubbub surrounding the departure of board member Alex Denner and the appointment of his romantic partner Susan Langer in June 2023, Biogen then elected ex- 3M president and CFO Monish Patolawala to the board in November. Richard Mulligan and William Jones also left the board last year. Pangalos retired from AstraZeneca in April and was replaced by Sharon Barr . → Vir Biotechnology ’s revamp took the spotlight in early August, as CEO Marianne De Backer made the decision to prop up its hepatitis B and D development and wind down its Covid-19, flu and T cell-based viral vector programs. On Wednesday, Vir said that Jason O’Byrne will succeed Sung Lee as CFO on Oct. 2. O’Byrne had the same title at Caribou Biosciences since February 2021 and is the former SVP of finance at Audentes Therapeutics . He spent 13 years with Genentech in a number of roles, including head of commercial finance for the Asia-Pacific region. To accommodate the makeover, Vir laid off about a quarter of its staff. → BridgeBio Oncology Therapeutics has selected Yong Ben as chief medical and development officer. Readers may know Ben from BeiGene , where he was CMO, solid tumors from 2019-22, and he’s been a venture partner at Eight Roads for the past two years. Ben was the global clinical leader of immuno-oncology clinical development at AstraZeneca when Imfinzi received its first approval for advanced bladder cancer in May 2017, and a week later, he became CMO of BioAtla . BridgeBio’s oncology spinout debuted in May with a $200 million infusion from Cormorant Asset Management , Omega Funds , GV and others. → As Endpoints News reported on Monday, Eli Lilly ended a three-month search by promoting Lucas Montarce to CFO. He replaces current Alphabet finance chief Anat Ashkenazi , with an interim stint by controller Gordon Brooks sandwiched in between. Montarce’s career at Lilly began in 2001 and has held enough positions to scroll through ever since. He was elevated to president and general manager for the Spain, Portugal and Greece hub in February after nearly three years as group VP, corporate controller and chief financial officer for Lilly Research Laboratories . He takes over as CFO at a time when the Indianapolis pharma’s market cap has exceeded $800 billion. → Pfizer CSO Mikael Dolsten has nabbed a spot on the board of directors at Rocket Pharmaceuticals . Dolsten announced in July that he would leave Pfizer after 15 years, but he’s sticking around until the company finds a successor. In late June, questions about manufacturing resulted in a CRL for marnetegragene autotemcel ( marne-cel ), Rocket’s gene therapy to treat an ultra-rare disease called severe leukocyte adhesion deficiency-I. → Meg Alexander has been promoted to president and COO of Ovid Therapeutics , which is on the verge of starting a Phase 2 study of its Graviton -partnered ROCK2 inhibitor in cerebral cavernous malformations. This is Alexander’s third appearance in as many years after she was named chief strategy officer last year and corporate affairs chief in 2022. “Her extensive experience, operational expertise, and business acumen position her well to lead the next phase of the company’s evolution,” Ovid CEO Jeremy Levin said in a statement . → Sana Biotechnology finance chief Nate Hardy will resign “for personal reasons” on Oct. 4, according to an SEC filing . Hardy was on the finance team at Juno Therapeutics with CEO Steve Harr before he took the CFO job at Sana in 2018. The Seattle-based biotech just hired UCB ’s Dhaval Patel as its CSO. → After five years on the job, Forge Biologics CEO Tim Miller is stepping down , making way for commercial chief John Maslowski to take over Oct. 1. Maslowski initially joined Forge as chief commercial officer in June 2021. Before that, he was president and CEO of Castle Creek Biosciences and Fibrocell Science . → Matt Shaulis is headed to BeiGene as general manager of North America on Sept. 25, while Josh Neiman — the chief commercial officer for North America and Europe — has stepped down “to pursue a new entrepreneurial opportunity at a healthcare technology startup,” according to a release . For the last year and a half, Shaulis has been chief commercial officer and US president for Swedish drug developer Hansa Biopharma , and he took on the role of president, inflammation and immunology for the international developed markets at Pfizer from 2018-21. At the end of his seven-year tenure with the pharma giant, he was elevated to SVP for global commercial and medical go-to-market model transformation. → Jeff Hartness has been named chief commercial officer at I&I biotech Apogee Therapeutics , which hit the Nasdaq with Sagimet Biosciences on July 14, 2023. Hartness spent more than 15 years with Sanofi before jumping to Bausch Health in 2016 as VP, strategic account management. Last year, he was promoted to EVP and head of Bausch Health’s market access, commercial operations, policy and government affairs teams, while serving as general manager of the neurology portfolio and generics businesses. Chaired by ex- Prometheus Biosciences chief and current Mirador Therapeutics CEO Mark McKenna , Apogee is trying to distinguish itself in the atopic dermatitis space with its monoclonal antibody APG777 . → The team at non-opioid pain biotech Latigo Biotherapeutics is quickly taking shape under new CEO Nima Farzan , who succeeded Westlake Village BioPartners ’ Desmond Padhi in late July. After bringing in medical chief Neil Singla last month, Latigo has tapped William Blair analyst Tim Lugo as CFO. Lugo joined William Blair as a senior associate in 2005 and would eventually become a partner, leading the biotechnology equity research team. Latigo also added Westlake managing director Beth Seidenberg and Foresite Capital ’s Jim Tananbaum to the board of directors. → Now that Rytelo ( imetelstat ) is being sold in the US for lower-risk myelodysplastic syndromes with transfusion dependent anemia, Geron Corporation has recruited Jim Ziegler as chief commercial officer. Ziegler had been EVP, commercial for Iovance , which secured the long-awaited FDA approval of its TIL therapy Amtagvi for melanoma in February. He also spent a decade in two separate stints with Gilead , culminating in his role as VP of the cardiopulmonary and inflammation business unit. → Alan List is retiring as CMO of Precision BioSciences , and 12-year Merck vet Murray Abramson has jumped on board to lead clinical development. Abramson left Merck in 2011 to become VP of global clinical operations at Biogen, a position he held for nine years. From 2020-22, Abramson was SVP of clinical innovation for Tempus . Precision also said in a release that ex- Aligos Therapeutics EVP John Fry will be a strategic clinical advisor for its hepatitis B program. Following his resignation as president and CEO of Moffitt Cancer Center over his connections to China, List reemerged as Precision’s medical chief in 2021. → Rami Daoud has made his way to gene therapy developer Affinia Therapeutics as chief business and financial officer. Daoud comes off a yearlong stint as president of GeneScience Pharmaceuticals USA and was a corporate development exec with Amarin . Rick Modi ’s crew was an Endpoints 11 winner in 2021 and had IPO aspirations that ultimately faded in late 2022. → HC Bioscience has recruited Simon Tsang to replace Christine Foster as CBO. Tsang is an Amgen corporate development alum who was VP, business development for Tesaro from 2016-19. After GSK bought Tesaro for $5.1 billion, he landed at Pivotal bioVenture Partners as an operating partner. HC Bioscience is in a small-but-emerging group of biotechs working on tRNA-based drugs, along with Flagship ’s Alltrna and Tevard Biosciences . → Rivus Pharmaceuticals , which claimed a Phase 2a win last month with its drug for obesity-related heart failure with preserved ejection fraction, has appointed Tom O’Neil as CFO and ex- ProfoundBio COO/CFO Erin Lavelle to the board of directors. As finance chief, O’Neil helped lead Satsuma Therapeutics to its IPO in 2019 and its sale to Shin Nippon Biomedical Laboratories last year. Lavelle orchestrated ProfoundBio ’s $112 million Series B in February, and Genmab would agree to buy the ADC biotech less than two months later. She also picked up another board seat this week at Aviceda Therapeutics. → We have an impressive lineup of CMO appointments: First, Eli Lilly’s obesity and metabolic disease partner HAYA Therapeutics has named Jordan Shin as CMO. Shin had been SVP of clinical development and translational science (cardiology) for gene therapy player Lexeo Therapeutics , which went public last November with a $100 million IPO. Earlier, he worked for Renovacor as SVP of clinical development and translational science until it was sold to Rocket Pharmaceuticals . Up to $1 billion is on the line with HAYA and Lilly’s recent drug discovery pact . → Canadian psychedelic biotech Reunion Neuroscience has brought in Mark Pollack as medical chief. Pollack briefly worked for Sage as SVP for global medical affairs and was part of a leadership shakeup that also involved the departures of CSO Al Robichaud and development chief Jim Doherty . Reunion went private last summer with the help of MPM BioImpact , and a $103 million Series A followed in May from MPM, Novo Holdings and several other investors. → A week after Reunion’s Series A financing, San Diego upstart Aardvark Therapeutics raised $85 million in a Series C to bankroll late-stage trials for its Prader-Willi syndrome asset ARD-101 . And like Reunion, Aardvark has found a new medical chief. Manasi Sinha Jaiman was promoted to the same position at stem cell therapy maker ViaCyte before it was sold to Vertex in 2022 for $320 million , and she would stay at Vertex as a VP. In the same release, Aardvark included the appointment of regulatory affairs leader Tom Bicsak , who came on board in July. → Milan-based AAVantgarde Bio is picking up Jayashree Sahni as CMO. Sahni is taking over the reins from Naveed Shams , who had served in the role since 2022. Sahni joins the team from Novartis , where she was senior global program clinical lead. Earlier in her career, Sahni was with Roche , where she worked on the Phase 1 and 2 clinical trials for Vabysmo . → Elsewhere, RA Capital -backed Tyra Biosciences has recruited Doug Warner as CMO. Warner joins the California-based crew from eFFECTOR Therapeutics , where he served in the same role. Previously, Warner had an 18-year stint at Amgen, where he led development of drugs like Vectibix , Xgeva and Prolia . Warner’s appointment comes after Tyra picked up $200 million in PIPE financing in February to progress its oral FGFR3-selective inhibitor, TYRA-300 . → Longwood ’s Solu Therapeutics , which debuted in August 2023 with a $31 million seed round, has welcomed Sergio Santillana as CMO. Peer Review told you about Santillana’s resignation from the same position at Ikena Oncology in February, and he’s also the ex-CMO at Merrimack Pharmaceuticals and Ariad Pharmaceuticals . Earlier in his career, Santillana had a string of roles at Eli Lilly, GSK and Takeda . → Maud Brandely is retiring after eight years as CMO of French cancer biotech Transgene , and Sanofi Genzyme vet Emmanuelle Dochy has replaced her . Dochy just completed a seven-year run at Bayer , where she recently served as VP of global medical affairs for the tyrosine kinase inhibitor (TKI) oncology franchise and scientific partnerships head. Meanwhile, Transgene has also enlisted Roche oncology alum Maurizio Ceppi as CSO. → KalVista Pharmaceuticals has named Brian Piekos as CFO. Far from his first run as finance chief, Piekos served in the role at Elicio Therapeutics , Gemini Therapeutics and AMAG Pharmaceuticals . In May, KalVista announced that it was dropping its preclinical program in hereditary angioedema in favor of focusing on its HAE pill sebetralstat . At the time, KalVista said it would find a partner for its dropped program. → Beverley Carr has taken the CBO post at London-based Charm Therapeutics , a startup co-founded by the University of Washington’s David Baker that uses its 3D deep learning tech DragonFold to discover new drugs. Carr is a GSK business development vet who had been chief business and operating officer for Amphista Therapeutics and then stepped into the role of interim CEO when Nicki Thompson left. → In July, Cue Biopharma announced that it was prioritizing its autoimmune pipeline and slashing 25% of its staff. Now, J&J vet Lucinda Warren has joined the Boston team as CBO. Warren’s 10-year stint at J&J culminated in her role as VP of business development for neuroscience and Japan regionally. Earlier in her career, Warren was with Janssen Cilag Australia and Centocor / Janssen Biologics . → Verily , the healthcare company spun out of Google parent Alphabet , has rolled out the welcome mat for Michael Radwin as its new head of data science and AI. Radwin joins from the CDC, where he was a founding member of the Office of Public Health Data, Surveillance, and Technology. Radwin’s résumé also includes roles at Intuit and Yahoo . Earlier in June, Verily announced that it was jumping into the GLP-1 business with the launch of a new virtual program to help patients manage cardiometabolic diseases with care teams and medications. → Dutch biotech VarmX , which is developing a reversal agent for blood thinners, has promoted Martijn Negen to the role of COO. Negen joined VarmX last September as SVP of global commercial strategy & business development from AM-Pharma . He also has experience from Portola Pharmaceuticals , Teva Global Specialty Medicines and Genentech. → Opthea has announced a slew of changes to its leadership team. First, the company is losing commercial chief Judy Robertson , CFO Peter Lang and SVP, global clinical operations Bruno Gagnon , effective Sept. 9. Filling in for Lang and Robertson in the interim will be Danforth Advisors managing director Daniel Geffken and Genentech and Novartis vet Mike Campbell , respectively. Meanwhile, Jen Watts will succeed Gagnon. That’s not all: Opthea is also picking up Dayong Li as SVP, biometrics and Anthony Bonifazio as VP, market access. → UK respiratory disease biotech Enterprise Therapeutics has introduced Annabella Amatulli as head of regulatory affairs. The Janssen vet was the global regulatory sciences leader for Alfasigma , the Italian company that bought Ocaliva maker Intercept Pharmaceuticals . → Tonix Pharmaceuticals has brought aboard Thomas Englese as EVP of commercial operations. Englese was formerly chief commercial officer of Tris Pharmaceuticals and Aziyo Biologics . Before that, he had an 11-year run with Mallinckrodt , culminating in his role as SVP and general manager of North America hospital therapies. → A handful of VCs have been making some moves. First up: Abingworth has turned to SFJ Pharmaceuticals founder and executive chair Robert DeBenedetto to tackle the role of managing director, clinical co-development; Sofinnova Partners has added a new partner. Karl Naegler just completed a four-year run as managing partner with Wellington Partners Life Sciences in June; and finally, Frazier Healthcare Partners has named Willis Chandler as an executive-in-residence on the growth buyout team. Chandler left Cencora (formerly AmerisourceBergen ) in June after nearly three years as president of global pharma services and six years overall. → Co-founded by Jim Wilson , Passage Bio has elected Ultragenyx CBO Tom Kassberg to the board of directors. One of Wilson’s new companies, GEMMA Biotherapeutics , licensed a trio of gene therapy candidates from Passage Bio in early August. → Enoch Kariuki has joined the board of directors at Cyrus Mozayeni ’s ADC shop Pheon Therapeutics , which is chaired by ex- Blueprint Medicines chief Jeff Albers . Kariuki became president of Endeavor BioMedicines this year and ran Lengo Therapeutics until it was purchased by Blueprint in 2021. → Yoshiharu Mizui now has a seat on the board of directors at SEED Therapeutics , the biotech near Philadelphia that struck a “molecular glue” deal with Eisai last month. Mizui is the president of Eisai’s VC arm, Eisai Innovation , and he also juggles two other roles as head of collaboration & incubation in the DHBL (Deep Human Biology Learning) office and head of academic & industry alliance. → Creyon Bio has made room for DCVC Bio operating partner Serge Messerlian and Lux Capital principal Shaq Vayda on the board of directors. Messerlian has board seats at Auron Therapeutics and Plexium , and Vayda’s numerous board credits include Atavistik Bio and Enveda Biosciences .

CRANBURY, N.J.--( BUSINESS WIRE )-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of Mikael Dolsten, M.D., Ph.D., as an independent director to its Board of Directors. Dr. Dolsten is an accomplished industry executive with extensive global pharmaceutical management experience. “ As an industry-leading pharmaceutical executive, I am very pleased to welcome Mikael to our Board of Directors,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “ His research and development experience will help advance our leading scientific capabilities and contribute to our mission of developing and delivering potentially transformative treatments for patients.” Throughout his career, Dr. Dolsten has held several positions and is currently the Chief Scientific Officer, President, Research & Development (R&D) at Pfizer Inc. (NYSE: PFE), focused on advancing gene therapies, small-molecule medicines, biotherapeutics, and vaccines. Dr. Dolsten’s 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. He 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. “ Rocket has an impressive pipeline of clinical programs which have shown tremendous promise potentially curing genetic disease,” said Dr. Mikael Dolsten, Chief Scientific Officer and President, Pfizer Research & Development. “ I am excited to bring my passion and experience to Rocket as it becomes a commercial organization and strives to reach more patients in need.” About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn , YouTube, and X . Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as “could,” “believe,” “expect,” “anticipate,” “intend,” “plan,” “will give,” “estimate,” “seek,” “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, including the timing and outcome of the FDA’s review of the additional CMC information that Rocket will provide in response to the FDA’s request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket’s ability to establish key collaborations and vendor relationships for its product candidates, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket’s ability to transition to a commercial stage pharmaceutical company, and Rocket’s expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into 2026. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2023, filed February 27, 2024 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.