Renovacor, Inc.

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 .

NEW YORK and LONDON, July 19, 2023 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, today announced the appointment of experienced life sciences executive Wendy DiCicco as interim Chief Financial Officer (CFO). Ms. DiCicco has more than 25 years of experience in the life sciences industry, currently serving as an independent financial and board advisor to companies, often in the role of interim CFO. “Wendy’s broad and deep experience in both strategic and operational aspects of finance will be invaluable assets to our management team as we work to bring nomacopan through its key milestones, create value for our investors, and make a meaningful difference for patients who have significant unmet needs,” said Rachelle Jacques, President and CEO of Akari. Ms. DiCicco served as CFO for Renovacor, a pre-clinical biopharmaceutical company developing a gene therapy for cardiovascular disease. Initially as interim CFO, transitioning to permanent, she led the company through Initial Public Offering (IPO) in 2021 and sale to Rocket Pharmaceuticals in December 2022. She also served as interim CFO for FerGene, Inc. a Phase 3 urologic oncology gene therapy company from January 2020 through May 2022. Previously, she was CFO and Chief Operating Officer (COO) of Centinel Spine and the President and COO of Camber Spine, both developers of best-in-class spinal implants. She has held CFO roles for several pre-IPO, private equity backed medical device and biotech companies, in varying stages of commercialization. Her first CFO role was with Kensey Nash Corporation, a publicly traded developer and manufacturer of biologics and medical devices in the cardiovascular and orthopedics industries, where she advanced the company from a pre-revenue IPO stage to approximately $100 million in global revenue across multiple product platforms. Her career started in public accounting at Deloitte & Touche. Ms. DiCicco currently serves on the public company Board of Directors of EyePoint Pharmaceuticals, Inc. where she serves as the Audit Committee Chair. In addition, she serves on the boards of Imvax, Inc. and ExpressCells. Ms. DiCicco has also served on the boards of SWK Holdings Corp, II-VI, Inc. (now Coherent Corp), Sincerus Pharmaceuticals, Carmell Therapeutics, SynCardia Systems and CannaPharma Rx. She is currently on the Board of Directors of the Philadelphia Chapter of the National Association of Corporate Directors. Ms. DiCicco received a B.S. in accounting from Philadelphia College of Textiles and Science and is a licensed Certified Public Accountant (CPA). She also is an appointed Board Leadership Fellow and Corporate Governance Fellow of the National Association of Corporate Directors (NACD). About Akari Therapeutics Akari Therapeutics, plc (Nasdaq: AKTX) is a biotechnology company developing advanced therapies for autoimmune and inflammatory diseases. Akari’s lead asset, investigational nomacopan, is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. Akari’s pipeline includes a Phase 3 clinical trial program investigating nomacopan for severe pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). Akari has been granted Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA for nomacopan for the treatment of pediatric HSCT-TMA. Akari’s pipeline also includes a clinical program developing nomacopan for adult HSCT-TMA and pre-clinical research of long-acting PAS-nomacopan in geographic atrophy (GA). For more information about Akari, please visit akaritx.com. Cautionary Note Regarding Forward-Looking Statements Certain statements in this press release constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies, and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies, and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control. Such risks and uncertainties for our company include, but are not limited to: needs for additional capital to fund our operations, our ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; an inability or delay in obtaining required regulatory approvals for nomacopan and any other product candidates, which may result in unexpected cost expenditures; our ability to obtain orphan drug designation in additional indications; risks inherent in drug development in general; uncertainties in obtaining successful clinical results for nomacopan and any other product candidates and unexpected costs that may result there; difficulties enrolling patients in our clinical trials; failure to realize any value of nomacopan and any other product candidates developed and being developed in light of inherent risks and difficulties involved in successfully bringing product candidates to market; inability to develop new product candidates and support existing product candidates; the approval by the FDA and EMA and any other similar foreign regulatory authorities of other competing or superior products brought to market; risks resulting from unforeseen side effects; risk that the market for nomacopan may not be as large as expected risks associated with the impact of the COVID-19 pandemic; inability to obtain, maintain and enforce patents and other intellectual property rights or the unexpected costs associated with such enforcement or litigation; inability to obtain and maintain commercial manufacturing arrangements with third party manufacturers or establish commercial scale manufacturing capabilities; the inability to timely source adequate supply of our active pharmaceutical ingredients from third party manufacturers on whom the company depends; unexpected cost increases and pricing pressures and risks and other risk factors detailed in our public filings with the U.S. Securities and Exchange Commission, including our most recently filed Annual Report on Form 20-F filed with the SEC. Except as otherwise noted, these forward-looking statements speak only as of the date of this press release and we undertake no obligation to update or revise any of these statements to reflect events or circumstances occurring after this press release. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. For more information Investor Contact:Mike MoyerLifeSci Advisors(617) 308-4306mmoyer@lifesciadvisors.com Media Contact:Eliza SchleifsteinSchleifstein PR (917) 763-8106eliza@schleifsteinpr.com

Advanced pipeline of four clinical programs across AAV cardiology and LV hematology portfolios delivering strong results for Danon Disease, Fanconi Anemia, LAD-I and PKD Expanded leadership position in AAV cardiac gene therapy, with significant opportunities for value creation across Danon Disease, PKP2-ACM and BAG3-DCM programs Established in-house cGMP manufacturing for AAV, poised to support Danon pivotal trial in Q2’23 and subsequent commercialization Cash, cash equivalents and investments of approximately $400M; expected operational runway through 2024 CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2022. “Rocket closed 2022 with positive results across four gene therapy programs spanning both our AAV cardiology and LV hematology therapeutic areas in an unprecedented fashion for a gene therapy company, validating our approach to strong science and R&D, carefully selected assets and smart execution. We are thrilled to begin 2023 with the FDA recently granting RMAT designation to RP-A501 for Danon Disease, validating the strength of our results and underscoring RP-A501's potential as a transformative therapy for Danon patients,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. Dr. Shah continued, “At the same time, we expanded our leadership position in AAV cardiac gene therapy with the recent unveiling of compelling preclinical proof of concept for RP-A601 for the treatment of PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) and addition of the BAG3-associated dilated cardiomyopathy (DCM) asset following the acquisition of Renovacor. Taken together, these two diseases along with Danon Disease affect more than 100,000 patients in the U.S. and EU. Further, our LV hematology portfolio delivered stellar results as we now advance towards our first regulatory filings for Leukocyte Adhesion Deficiency (LAD-I) in the second quarter of 2023 and Fanconi Anemia (FA) in the fourth quarter of 2023, and subsequent commercialization.” “I am also pleased that we brought in additional funds of $197.7 million in 2022. We begin this year in a strong financial position to execute on near and long-term milestones, anticipating that our cash runway of approximately $400 million will fund operations through 2024,” said Dr. Shah. “I am grateful to our growing Rocket team of multi-disciplinary experts, scientific collaborators and the critical voices of the patient community who were essential to this progress and continue to help advance our mission of seeking gene therapy cures. I look forward to building off the successes of 2022 and continuing our progress in 2023.” Key Pipeline and Operational Updates Announced positive clinical data from Phase 1 trial of RP-A501 for Danon Disease. The Company provided an update at the 41st Annual J.P. Morgan Healthcare Conference that continued to demonstrate RP-A501 was generally well tolerated with evidence of restored expression of the deficient LAMP2 protein and durable improvement or stabilization of clinical parameters in the adult and pediatric Danon Disease patients treated in the Phase 1 study. The Company previously presented positive data from the study at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022. The FDA recently granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501 that will provide the benefits of added intensive FDA guidance and expedited review through the program’s development. The initiation of the Phase 2 pivotal trial is on track for the second quarter of 2023. As previously disclosed, the Company anticipates pursuing a single arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator. Successfully completed two in-house AAV production runs at state-of-the-art, Current Good Manufacturing Practice (cGMP) manufacturing and R&D facility in Cranbury, N.J. In-house production runs of AAV drug product for the planned Phase 2 pivotal study in Danon Disease resulted in high-quality drug substance enabling an approximately threefold increase in the number of patients treatable per batch, a significantly improved full versus empty particle ratio, and promising product comparability data generated to date compared to the Phase 1 material manufactured externally. Announced positive top-line data from Phase 2 pivotal trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). Positive top-line data presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) and at the 64th American Society of Hematology (ASH) Annual Meeting demonstrated 100% overall survival at 12 months post-infusion via Kaplan Meier estimate and a statistically significant reduction in all hospitalizations, infection- and inflammatory-related hospitalizations and prolonged hospitalizations for all nine LAD-I patients with three to 24 months of available follow-up. Data also showed evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The Company recently presented data at the Tandem Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR) in February 2023 demonstrating all patients sustained neutrophil CD18 restoration and expression of more than 10% (range: 15%-89%, median: 51%), as of the November 2, 2022, cut-off date. At the congress, the RP-L201 program received first place in the abstract awards competition. Based on the data from the Phase 2 pivotal LAD-I trial, Rocket anticipates submitting a Biologics License Application (BLA) in the second quarter of 2023. Announced positive top-line data from Phase 2 pivotal trial of RP-L102 for Fanconi Anemia (FA). Data presented at the 64th ASH Annual Meeting (cut-off October 26, 2022), indicated that RP-L102 demonstrated phenotypic correction in at least six of 10 evaluable patients with ≥12 months of follow-up as demonstrated by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells, concomitant genetic correction and hematologic stabilization. A seventh patient has displayed evidence of progressively increasing genetic correction as demonstrated by peripheral blood and BM vector copy numbers (VCNs), with recent development of MMC resistance and indicators of hematologic stability after 36 months of follow-up. Data demonstrated increased resistance to MMC in five patients at least at two timepoints, ranging from 74% to 94% at the most recently evaluated timepoint. The safety pro RP-L102 was highly favorable, and the treatment, administered without any cytotoxic conditioning, was well tolerated. The Company recently presented these data at the Tandem Meetings of ASTCT and CIBMTR in February 2023 and the RP-L102 program received second place in the abstract awards competition. Based on the data from the Phase 2 pivotal Fanconi Anemia trial, Rocket anticipates submitting a BLA in the fourth quarter of 2023. Announced positive clinical data from Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD). Data presented at the 64th ASH Annual Meeting (cut-off October 26, 2022) demonstrated that both patients at 24 months post-RP-L301 infusion had robust and sustained efficacy demonstrated by normalized hemoglobin (from baseline levels in the 7.0-7.5 g/dL range), improved hemolysis parameters, independence from red blood cell transfusions and improved quality of life both reported anecdotally and as documented via formal quality of life assessments. The safety pro highly favorable, with no RP-L301-related serious adverse events through 24 months post-infusion in both adult patients. Adult and pediatric enrollment is completed in the Phase 1 study. The company recently presented these data at the Tandem Meetings of ASTCT and CIBMTR in February 2023. Phase 2 pivotal trial initiation is anticipated in the fourth quarter of 2023. Raised $197.7M to develop full pipeline of assets. During 2022, the Company completed a follow-on offering of common stock for net proceeds of $108.1 million, sold shares of common stock for net proceeds of $46.6 million pursuant to the at-the-market offering program and acquired cash and cash equivalents of $43.0 million as part of the acquisition of Renovacor. Acquired Renovacor, extending leadership in AAV-based cardiac gene therapy. In December 2022, Rocket completed the acquisition of Renovacor, bringing in $43.0 million of non-dilutive capital. The acquisition also provided Rocket access to an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. Additionally, Rocket gained access to world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. Strengthened Board of Directors and leadership team with new appointments. Broadened cardiovascular experience with the appointment of Fady Malik, M.D., Ph.D. to Rocket’s Board of Directors. Dr. Malik brings nearly 25 years of experience as an internationally recognized cardiovascular physician-scientist and highly successful biopharmaceutical executive. In addition, Rocket appointed Mayo Pujols as Executive Vice President, Chief Technical Officer. Mr. Pujols has nearly 30 years of experience in leadership roles across technical operations, quality operations, validation, process development and cGMP manufacturing. Rocket also appointed Carlos Martin as Senior Vice President, Chief Commercial Officer. Mr. Martin brings over 20 years of global commercial leadership gained at Novartis, Schering Plough and Eli Lilly. Recognized Rare Disease Day with an event at NASDAQ Tower in New York City. On February 28, 2022, Rocket hosted its annual Rare Disease Day celebration highlighting the theme, “Rare, But Not Alone.” More than 250 members of the global rare disease community and Rocket team gathered in person and virtually to hear about the impact of rare disease and clinical research from patients, families, advocacy groups and scientific collaborators and innovators – including Dr. Moris Danon, who first identified Danon Disease. The event concluded with the lighting of the Empire State Building, as well as other global landmarks, in Rare Disease Day colors. Upcoming Investor Conferences Cowen’s 43rd Annual Health Care Conference: March 6-8, 2023 Needham 21st Annual Virtual Healthcare Conference: April 17-20, 2023 Fourth Quarter and Full Year 2022 Financial Results Cash position. Cash, cash equivalents and investments as of December 31, 2022, were $399.7 million. R&D expenses. Research and development expenses were $50.0 million and $165.6 million for the three and twelve months ended December 31, 2022, compared to $32.2 million and $125.5 million for the three and twelve months ended December 31, 2021. The increase in R&D expenses was primarily driven by increases in manufacturing and development costs of $26.3 million, laboratory supplies of $6.6 million, compensation and benefits expense of $11.5 million due to increased R&D headcount and direct materials of $3.6 million. Increases noted were offset by a decrease in license fees of $12.9 million attributable to the expense in connection with warrants to purchase shares of common stock recorded for the year ended December 31, 2021. G&A expenses. General and administrative expenses were $19.0 million and $58.8 million for the three and twelve months ended December 31, 2022, compared to $12.2 million and $41.8 million for the three and twelve months ended December 31, 2021. The increase in G&A expenses was primarily driven by increases in commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $4.9 million, compensation and benefits of $4.4 million due to increased G&A headcount and acquisition related expense of $3.2 million related to the Renovacor acquisition which closed on December 1, 2022. Net loss. Net loss was $66.7 million and $221.9 million or $0.92 and $3.26 per share (basic and diluted) for the three and twelve months ended December 31, 2022, compared to $44.2 million and $169.1 million or $0.69 and $2.67 per share (basic and diluted) for the three and twelve months ended December 31, 2021. Shares outstanding. 79,123,312 shares of common stock were outstanding as of December 31, 2022. Financial Guidance Cash position. As of December 31, 2022, Rocket had cash, cash equivalents and investments of $399.7 million. Rocket expects such resources will be sufficient to fund its operations through 2024, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of our six clinical and/or preclinical programs. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LV)-based gene therapy are for the treatment of 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 rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has preclinical AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit . Rocket Cautionary Statement Regarding Forward-Looking Statements Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, 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, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as “aim,” “anticipate,” "believe," “can,” “continue,” “design,” “estimate,” "expect," "intend," “may,” "plan," “potential,” "will give," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. 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 ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, our ability to submit regulatory filings with the U.S. Food and Drug Administration (FDA) and to obtain and maintain FDA or other regulatory authority approval of our product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, our integration of an acquired business, which involves a number of risks, including the possibility that the integration process could result in the loss of key employees, the disruption of our ongoing business, or inconsistencies in standards, controls, procedures, or policies, our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire and any unexpected expenditures, 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, 2021, filed February 28, 2022 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. Three Months Ended December 31, Twelve Months Ended December 31, 2022 2021 2022 2021 Operating expenses: Research and development $ 50,037 $ 32,161 $ 165,570 $ 125,476 General and administrative 19,044 12,171 58,773 41,772 Total operating expenses 69,081 44,332 224,343 167,248 Loss from operations (69,081 ) (44,332 ) (224,343 ) (167,248 ) Research and development incentives 500 500 500 1,000 Interest expense (467 ) (463 ) (1,862 ) (2,977 ) Interest and other income, net 1,245 849 3,889 3,068 Amortization of premium on investments - net 1,081 (801 ) (47 ) (2,912 ) Total other income (expense), net 2,359 85 2,480 (1,821 ) Net loss $ (66,722 ) $ (44,247 ) $ (221,863 ) $ (169,069 ) Net loss per share attributable to common stockholders - basic and diluted $ (0.92 ) $ (0.69 ) $ (3.26 ) $ (2.67 ) Weighted-average common shares outstanding - basic and diluted 72,889,548 64,470,930 68,148,925 63,235,417 December 31, December 31, 2022 2021 Cash, cash equivalents, and investments $ 399,670 $ 388,740 Total assets 551,807 497,020 Total liabilities 62,121 42,296 Total stockholders' equity 489,686 454,724