Congenital Bone Marrow Failure Syndromes

MANHASSET, N.Y.--(BUSINESS WIRE)-- Red cell disorders, such as Diamond-Blackfan anemia (DBA) and sickle cell disease (SCD), affect more than 1 billion people worldwide and are a significant cause of chronic illness and mortality. To further our understanding and potential treatments of these disorders, The Feinstein Institutes for Medical Research has been awarded a $6.1 million grant from the National Institutes of Health (NIH). This press release features multimedia. View the full release here: Dr. Lionel Blanc will lead the new NIH-funded red cell disorders research (Credit: Feinstein Institutes) Led by Lionel Blanc, PhD, professor in the Institute of Molecular Medicine at the Feinstein Institutes, the seven-year initiative aims to build off previous research and will shed light on DBA, SCD and anemia. Specifically, Dr. Blanc and his team will attempt to unravel the process and malfunction of erythropoiesis, or the production of red blood cells, in those disorders. “Understanding red cell blood disorders, how they develop and progress, is essential given their global impact on millions,” said Dr. Blanc. “This grant from the National Heart, Lung and Blood Institute will help pave the way for innovative treatments and improve the quality of life for those affected.” The grant will help fund three research projects: Study of ribosome and cell cycle length regulation in fetal vs. adult erythropoiesis. Explore the erythromyeloblastic island associated with DBA and SCD. Focus on identifying and testing novel drugs for these hematologic disorders. “Dr. Blanc’s work in uncovering the fundamental molecular mechanisms of red blood cell disorders paves a path towards novel therapeutic approaches,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. “His translational research forms the foundation for identifying innovative treatments for complex blood disorders.” Dr. Blanc is a recognized leader in hematology research. In 2022, he published research in Blood showing that high mobility group box-1 protein (HMGB1) prevents the body’s ability to produce sufficient oxygen-rich red blood cells. In 2019, he received a $2.5 million grant from the NIH to study treatment for erythropoietic disorders, including DBA. About the Feinstein Institutes The Feinstein Institutes for Medical Research is the home of the research institutes of Northwell Health, the largest health care provider and private employer in New York State. Encompassing 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health system science, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine – a new field of science that has the potential to revolutionize medicine. For more information about how we produce knowledge to cure disease, visit and follow us on LinkedIn. View source version on businesswire.com: Contacts Matthew Libassi 631-793-5325 mlibassi@northwell.edu Source: The Feinstein Institutes for Medical Research Smart Multimedia Gallery Photo Dr. Lionel Blanc will lead the new NIH-funded red cell disorders research (Credit: Feinstein Institutes) Logo View this news release and multimedia online at:

REDWOOD CITY, Calif., April 02, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CIndU), today announced that its management will present at the 23rd Annual Needham Virtual Healthcare Conference at 11am EDT on April 8, 2024. A live webcast of the presentation will be available on the Events & News – Presentations page of Jasper's Investor Relations website. An archived replay of the presentation will be available on Jasper's website for 30 days following the live broadcast. Inducement Grant On April 1, 2024, four new employees were awarded grants of options to purchase an aggregate of 22,400 shares of voting common stock (the “Options”). Each Option was granted pursuant to the Jasper Therapeutics, Inc. Amended and Restated 2022 Inducement Equity Incentive Plan, as approved by the compensation committee of Jasper’s board of directors on March 14, 2022 and as amended and restated on June 2, 2023, and was granted as an inducement material to such employee’s employment with Jasper in accordance with Nasdaq Listing Rule 5635(c)(4). The exercise price of each Option is $28.30. Each Option will vest over four years, with 25% of the total number of shares vesting on the one year anniversary of the date of commencement of such employee’s employment with Jasper and 1/48th of the total number of shares subject to each Option vesting monthly thereafter, subject in each case to such employee’s continued service to Jasper on each vesting date. Jasper is providing this information in accordance with Nasdaq Listing Rule 5635(c)(4). About Jasper Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria and lower to intermediate risk myelodysplastic syndromes (MDS) and as a conditioning agent for stem cell transplants for rare diseases such as sickle cell disease (SCD), Fanconi anemia (FA) and severe combined immunodeficiency (SCID). To date, briquilimab has a demonstrated efficacy and safety pro more than 145 dosed participants and healthy volunteers, with clinical outcomes as a conditioning agent in SCID, acute myeloid leukemia, MDS, FA, and SCD. For more information, please visit us at . Forward-Looking Statements Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding Jasper’s presentation at the 23rd Annual Needham Virtual Healthcare Conference, Jasper’s employees and equity plans and briquilimab’s potential to address mast cell driven diseases such as CSU and CIndU. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jasper’s product candidates may not be beneficial to patients or successfully commercialized; patients’ willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper’s business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jasper’s filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper’s assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements. Contacts: Joyce Allaire (investors) LifeSci Advisors 617-435-6602 jallaire@lifesciadvisors.com Alex Gray (investors) Jasper Therapeutics 650-549-1454 agray@jaspertherapeutics.com Lauren Walker (media) Real Chemistry 646-564-2156 lbarbiero@realchemistry.com

Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined with hematologic stabilization extending out to 42 months after treatment with RP-L102 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 the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102, its lentiviral (LV) vector-based investigational gene therapy for Fanconi Anemia (FA), complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. “The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder. Currently, there are no existing options to potentially prevent BMF for patients with FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma. “We are appreciative of the patients, their families, and researchers who helped reach this meaningful milestone and continue participating in the clinical development program. We look forward to partnering closely with the EMA throughout the review process to make RP-L102 available to patients with FA who are in need of new treatment options.” MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial. RP-L102 demonstrated sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization. The safety pro highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated. There were no signs of bone marrow dysplasia, clonal dominance, or insertional mutagenesis related to RP-L102. In the absence of allogeneic hematopoietic stem cell transplant (HSCT), the primary cause of death among patients with FA is BMF, which typically occurs during the first decade of life. Although allogeneic transplants can cure the hematologic component of FA, they confer significant side effects and substantially increase the risk of solid organ malignancies, which have become the most frequent cause of FA-related death. The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024. About RP-L102 RP-L102 is an investigational gene therapy that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to contain a functional copy of the FANCA gene. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions for the program. RP-L102 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. About Fanconi Anemia Fanconi Anemia (FA) is a rare genetic disorder characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. In the absence of allogeneic hematopoietic stem cell transplant (HSCT), the primary cause of death among patients with FA is BMF, which typically occurs during the first decade of life. Allogeneic HSCT, when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Both chemotherapy conditioning and graft-versus-host disease, a known complication of allogeneic HSCT, are associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene lead to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a “gold standard” test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as “natural gene therapy” provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells. There is a high unmet medical need for patients with FA. 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. Our 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 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 “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, Rocket’s plans for the advancement of its DD program, including its planned pivotal trial, and the safety, effectiveness and timing of related 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 and Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies. 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, 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. View source version on businesswire.com: Contacts Media Kevin Giordano media@rocketpharma.com Investors Brooks Rahmer investors@rocketpharma.com Source: Rocket Pharmaceuticals, Inc. View this news release online at: