The Latest Cardiac and Vascular News
IRVINE, Calif., Nov. 10, 2025 (GLOBE NEWSWIRE) — JenaValve Technology, Inc., developer and manufacturer of the Trilogy™ Transcatheter Heart Valve (THV) System, today announced highlights from the 2025 Transcatheter Cardiovascular Therapeutics (TCT) Annual Scientific Symposium in San Francisco, CA with a live case broadcast from the ARTIST study as well as late-breaking data from PREVUE-VALVE.
SUNNYVALE, Calif., Nov. 10, 2025 (GLOBE NEWSWIRE) — BioCardia, Inc. [NASDAQ: BCDA], a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, today announced the first patient enrolled at Henry Ford Health in its ongoing Phase 3 CardiAMP® HF II clinical trial for patients with ischemic heart failure of reduced ejection fraction (“HFrEF”). “The CardiAMP-HF trial, in which Henry Ford Health participated, was groundbreaking for the field of heart failure. Although the primary endpoint was not met, the study showed impressive results with an autologous cell therapy in patients with HFrEF, particularly those with elevated biomarkers of heart stress,” said Dr. Gerald Koenig, M.D., Director of the Cardiac Catheterization Laboratory at Henry Ford West Bloomfield Hospital and Director of Clinical Research in the Cardiac Catheterization Laboratory at Henry Ford Health System, and Principal Investigator in the Phase 3 CardiAMP HF II trial. “Our team is actively offering patients the opportunity to participate in this important confirmatory study intended to enhance microvascular function and improve patient outcomes, including quality of life.” “We greatly value our collaboration with Dr. Koenig and the world class Henry Ford Health team in the CardiAMP HF II Trial,” said Peter Altman, PhD, CEO of BioCardia. “Between the U.S. and the EU, there are roughly 2 million ischemic HFrEF patients with NYHA Class II and III symptoms. Many of these patients have a prognosis worse than many cancers and few remaining options. This places a terrible burden on patients, their families, and the healthcare system. We are excited by the CardiAMP HF results showing that our cell therapy system reduced both mortality and major adverse cardiac events, improved quality of life, and showed positive impacts to other study endpoints in patients with HFrEF. These results suggest that our investigational cell therapy could address important clinical needs.” About the CardiAMP Heart Failure II Study CardiAMP HF II is a 250-patient randomized multicenter procedure placebo-controlled study of the CardiAMP autologous cell therapy as a one-time treatment for patients with ischemic heart failure with reduced ejection fraction (HFrEF) on guideline directed medical therapy having elevated NTproBNP. The study is intended to confirm the safety and efficacy results in these patients observed in the CardiAMP HF study. The CardiAMP HF II study uses a similar three-tier composite primary outcome measure to the CardiAMP HF study consisting of all cause death, nonfatal major adverse cardiac events, and a validated quality of life measure. In CardiAMP HF, this composite efficacy endpoint was achieved with statistical significance in the patients with elevated NTproBNP who are the focus of the CardiAMP HF II study. Advances in this therapeutic approach for this trial include using the cell population analysis at screening to define treatment doses and improvements to the Helix system including the FDA approved Morph DNA steerable platform. About CardiAMP Autologous Cell Therapy Granted FDA Breakthrough designation, CardiAMP Cell Therapy uses a patient’s own bone marrow cells delivered to the heart in a minimally invasive, catheter-based procedure intended to increase capillary density and reduce tissue fibrosis, treating microvascular dysfunction. The CardiAMP Cell Therapy clinical development for heart failure is supported by the Maryland Stem Cell Research Fund and is reimbursed by Centers for Medicare and Medicaid Services (CMS). CAUTION – Limited by United States law to investigational use. About BioCardia® BioCardia, Inc., headquartered in Sunnyvale, California, is a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary disease. CardiAMP® autologous and CardiALLO™ allogeneic cell therapies are the Company’s biotherapeutic platforms with three clinical stage product candidates in development. These therapies are enabled by its Helix™ biotherapeutic delivery and Morph® vascular navigation product platforms. For more information visit: https://www.BioCardia.com. Forward Looking Statements This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include, among other things, references to our investigational product candidates, the potential benefits and mechanism of actions of the CardiAMP cell therapy, future regulatory approvals, enrollment in our clinical trials, and the safety and efficacy of our product candidates and therapies. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements. We may use terms such as “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately” or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in BioCardia’s Form 10-K filed with the Securities and Exchange Commission on March 26, 2025, under the caption titled “Risk Factors,” and in our subsequently filed Quarterly Reports on Form 10-Q. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law. CONTACT: Media Contact:
Miranda Peto, Investor Relations
Email: mpeto@BioCardia.com
Phone: 650-226-0120
Investor Contact:
David McClung, Chief Financial Officer
Email: investors@BioCardia.com
Phone: 650-226-0120
-Positive Phase 1 data for CTX310® presented in a late-breaking presentation at the American Heart Association (AHA) Scientific Sessions and simultaneously published in The New England Journal of Medicine- -CASGEVY® momentum accelerating; nearly 300 patients have been referred to Authorized Treatment Centers (ATCs), approximately 165 patients have completed their first cell collection and 39 have received infusions across all regions; Vertex expects clear line of sight to over $100 million in total CASGEVY revenue this year and significant growth expected in 2026- -Pediatric development of exa-cel is advancing in SCD and TDT, with enrollment in two global Phase 3 studies completed; initial data will be presented at the upcoming American Society of Hematology (ASH) annual meeting- -Clinical trials ongoing for CTX112™, targeting CD19, across multiple indications; broad updates for CTX112 in autoimmune disease and oncology expected by year-end- -Preclinical data for CTX460™, presented at the European Society of Gene and Cell Therapy (ESGCT) annual congress, demonstrated in vivo gene correction of alpha-1 antitrypsin deficiency (AATD) with a potential best-in-class profile; clinical trial initiation planned for mid-2026- -Phase 2 clinical trial ongoing for SRSD107, targeting Factor XI for the prevention of thromboembolic disorders; first patient dosed and trial expanded to additional sites in Europe- -Strong balance sheet with approximately $1.9 billion in cash, cash equivalents, and marketable securities as of September 30, 2025- ZUG, Switzerland and BOSTON, Nov. 10, 2025 (GLOBE NEWSWIRE) — CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the third quarter ended September 30, 2025. “This has been another strong quarter of execution and progress across our portfolio,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “CASGEVY® momentum continues to build globally, reflecting growing patient engagement and clinical advancement. Enrollment has been completed in two global Phase 3 pediatric studies, and dosing is on track to complete this quarter. Additionally, positive Phase 1 data for CTX310® presented in a late-breaking presentation at the American Heart Association Scientific Sessions and published in The New England Journal of Medicine, highlight the breadth and potential of our platform to address serious cardiovascular disease. We continue to advance our broader pipeline, including dosing the first patient in the Phase 2 clinical trial of SRSD107 and unveiling our novel SyNTase™ editing platform with CTX460™, highlighting continued innovation and expansion of our therapeutic toolkit. With strong execution and growing momentum across our programs, CRISPR Therapeutics is well positioned to lead the next wave of gene editing innovation and deliver potentially transformative therapies to patients.” Recent Highlights and Outlook Hemoglobinopathies and CASGEVY® (exagamglogene autotemcel [exa-cel]) CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), designed to eliminate both vaso-occlusive crises (VOCs) and transfusion requirements. CASGEVY is approved in the U.S., Great Britain, the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain (Bahrain), Qatar, Canada, Switzerland and the United Arab Emirates (UAE) for the treatment of both SCD and TDT. Across these markets, there are more than 60,000 eligible patients in these countries, including approximately 37,000 in North America and Europe and more than 23,000 in the Middle East. With a well-established treatment pathway and a growing number of patients progressing through each stage, CASGEVY has strong momentum heading into late 2025 and beyond. In September, a reimbursement agreement was reached in Italy for patients with SCD and TDT. Italy represents the largest population of individuals with TDT in Europe, with approximately 5,000 people aged 12 years and older with TDT and approximately 2,300 with SCD.Globally, since launch through September 30th, 2025, approximately 165 patients with SCD or TDT have completed their first cell collection, including 50 people in the third quarter. 39 patients have received infusions of CASGEVY, including 10 infused in the third quarter of 2025. Nearly 300 patients have been referred by their physicians to an authorized treatment center (ATC) to begin the treatment process. This includes 110 cell collections in the first nine months of 2025, double the total for all of 2024. Enrollment of children 5 to 11 years of age with SCD or TDT in two global Phase 3 studies of CASGEVY has been completed, and dosing is expected to be completed this quarter. Initial data from these studies will be presented at the American Society of Hematology (ASH) annual meeting on December 6th, 2025.The number of ATCs initiating patients continues to increase in the U.S., Europe, and the Middle East. Through the end of September, 25 ATCs had initiated more than 5 patients, with at least one ATC in each region initiating 20 or more patients.Momentum continues to build through the final months of 2025. With continued uptake and reimbursement progress across major regions, Vertex expects a clear line of sight to over $100 million in total CASGEVY revenue this year with significant growth expected in 2026. In Vivo Liver Editing CRISPR Therapeutics is advancing a pipeline of in vivo gene editing candidates addressing major unmet needs in cardiovascular, metabolic and rare diseases using its proprietary, de-risked lipid nanoparticle (LNP) delivery platform.CTX310®, targeting ANGPTL3, is in an ongoing Phase 1 clinical trial in patients with homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias. Phase 1 data were presented in a late-breaking session at the American Heart Association (AHA) Scientific Sessions and published simultaneously in The New England Journal of Medicine (NEJM). Results from the Phase 1 clinical trial highlight the potential of CTX310 to safely and durably lower both triglycerides (TG) and low-density lipoprotein (LDL) following a single-course IV administration. CRISPR Therapeutics is advancing CTX310 into Phase 1b clinical trials, prioritizing development in sHTG and mixed dyslipidemia.CTX320™ is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with an increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels affect up to 20% of the global population and remain unaddressed by current therapies. The Company plans to provide an update in the first half of 2026.CRISPR Therapeutics continues to advance its preclinical in vivo programs: CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD); CTX340™, targeting AGT for the treatment of refractory hypertension; and CTX450™, targeting ALAS1 for the treatment of acute hepatic porphyria (AHP). CTX460 is the first investigational candidate using the Company’s novel SyNTase editing platform, unveiled in October. SyNTase is designed to enable precise, in vivo gene correction, and represents an important expansion of CRISPR Therapeutics’ toolkit. Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress demonstrated >90% mRNA correction, a 5-fold increase in total AAT levels, and >99% serum M-AAT:Z-AAT ratio in AATD disease models. These findings provide preclinical proof-of-concept for precise, single-dose in vivo gene correction using the SyNTase editing platform and support the potential best-in-class profile of CTX460. CRISPR Therapeutics expects to initiate a clinical trial of CTX460 in mid-2026.Preclinical data from CTX340 were presented in a late-breaking poster presentation at the American Heart Association (AHA) Scientific Sessions. In a spontaneous hypertensive rat model, CTX340 showed >70% liver editing and mean arterial pressure reduction of 53 mmHg compared to vehicle that was durable throughout the study (~8.5 months). Furthermore, in non-human primates, CTX340 showed greater than 90% AGT reduction with a two-dose regimen showing the additive effects of repeat dosing and enabling dose titration. CTX340 was well tolerated with no hypotension or hypokalemia observed. CTX340 is currently in IND/CTA-enabling studies. Autoimmune Disease and Immuno-Oncology CTX112™, targeting CD19, is being developed for autoimmune disease and hematologic malignancies and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma. A Phase 1 clinical trial in autoimmune disease is underway in systemic lupus erythematosus (SLE), systemic sclerosis and inflammatory myositis. In parallel, a Phase 1 clinical trial of CTX112 in relapsed or refractory B-cell malignancies is ongoing. The Company plans to provide a broad update on CTX112 by year-end. CTX131™, targeting CD70, was previously in development for both solid tumors and hematologic malignancies. While the Phase 1 data are encouraging, the Company has strategically redirected resources away from this program to advance other programs with the greatest potential for long-term value creation.CRISPR Therapeutics is leveraging its expertise and proprietary lipid nanoparticle (LNP) delivery platform, mRNA, and conjugation capabilities to advance an in vivo CAR T platform with the ability to address autoimmune disease and oncology.CRISPR Therapeutics’ autoimmune disease and immuno-oncology programs are supported by a wholly-owned, GMP manufacturing facility located in Framingham, Massachusetts, which provides end-to-end production capabilities for its cell therapy portfolio and supports both clinical and future commercial supply. siRNA In September, CRISPR Therapeutics and its partner Sirius Therapeutics announced that the first patient was dosed in Europe in the Phase 2 clinical trial of SRSD107, a long-acting Factor XI (FXI) small interfering RNA (siRNA) for thromboembolic disorders. The trial is evaluating the safety and efficacy of SRSD107 in preventing venous thromboembolism (VTE) following total knee arthroplasty (TKA) and will inform dose selection for future pivotal trials. SRSD107 has the potential to be a best-in-class FXI inhibitor, showing deep reductions in FXI via semi-annual subcutaneous injection. SRSD107 is being co-developed by CRISPR Therapeutics and Sirius Therapeutics as part of a broader strategic collaboration to advance RNA-based medicines.CRISPR Therapeutics and Sirius Therapeutics have expanded the Phase 2 clinical trial with additional centers in Europe. SRSD107 is being developed for a range of thromboembolic and clotting-related indications, including arterial fibrillation (AF), cancer-associated thrombosis (CAT), chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD), ischemic stroke and VTE. Regenerative Medicine CRISPR Therapeutics continues to advance its regenerative medicine efforts for Type 1 diabetes (T1D). Beyond CTX211™, the Company is developing next-generation programs that leverage induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors. These approaches aim to achieve insulin independence in T1D patients without requiring chronic immunosuppression. The Company expects to provide an update this year. Upcoming Events The Company will participate in the following events in November: Guggenheim 2nd Annual Healthcare Innovation ConferenceDate: Wednesday, November 12, 2025Time: 11:30 a.m. ET Jefferies Global Healthcare ConferenceDate: Wednesday, November 19, 2025Time: 1:00 p.m. GMT Third Quarter 2025 Financial Results Cash Position: Cash, cash equivalents, and marketable securities were $1,944.1 million as of September 30, 2025, compared to $1,903.8 million as of December 31, 2024. The increase in cash was primarily driven by proceeds from the issuance of common shares, option exercise activity and interest income, offset by operating expenses, as well as the $25.0 million upfront cash payment made as part of the Sirius Agreement. R&D Expenses: R&D expenses were $58.9 million for the third quarter of 2025, compared to $82.2 million for the third quarter of 2024. The decrease in R&D expense was primarily driven by a decrease in variable external research and manufacturing costs, as well as a decrease in employee-related expenses, including stock-based compensation expenses.G&A Expenses: General and administrative expenses were $16.9 million for the third quarter of 2025, compared to $17.4 million for the third quarter of 2024.Collaboration Expense: Collaboration expense, net, was $57.1 million for the third quarter of 2025, compared to $11.2 million for the third quarter of 2024. In the third quarter of 2024, we exercised our option to defer specified costs under the CASGEVY program in excess of the deferral limit of $110.3 million under the A&R Vertex JDCA, as amended. The increase in collaboration expense, net, was primarily attributable to the timing of when we reached the deferral limit in 2024, as no such limit was applicable in 2025.Net Loss: Net loss was $106.4 million for the third quarter of 2025, compared to a net loss of $85.9 million for the third quarter of 2024. About CASGEVY® (exagamglogene autotemcel [exa-cel])CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CRISPR Therapeutics – Vertex Collaboration for CASGEVY CRISPR Therapeutics and Vertex established a strategic research collaboration in 2015 to discover and develop therapies using CRISPR/Cas9 technology to address the underlying genetic causes of human disease. CASGEVY is the first approved therapy to emerge from this collaboration. Under an amended collaboration agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY and shares program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY. About CTX112 CTX112 is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications. CTX112 is being investigated in ongoing clinical trials in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis and in adult patients with relapsed or refractory B-cell malignancies. About In Vivo ProgramsCRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technologies. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include: CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD); CTX340™, targeting AGT for the treatment of refractory hypertension; and CTX450™, targeting ALAS1 for the treatment of acute hepatic porphyria (AHP). About SRSD107SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA). SRSD107 specifically targets the human coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, thereby blocking the intrinsic coagulation pathway and promoting anticoagulant/anti-thrombotic effects. SRSD107 is being co-developed by CRISPR Therapeutics and Sirius Therapeutics as part of a strategic collaboration to advance innovative treatments for cardiovascular and clotting-related diseases. About the CRISPR Therapeutics and Sirius Therapeutics CollaborationCRISPR Therapeutics and Sirius Therapeutics entered into a strategic collaboration in 2025 to develop and commercialize novel small interfering RNA (siRNA) therapies for thromboembolic disorders and other serious diseases. The lead program, SRSD107, is a long-acting siRNA targeting Factor XI (FXI) with the potential to offer best-in-class efficacy and safety. Under the agreement, the companies will co-develop SRSD107 and share costs and profits equally. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will lead in Greater China. The collaboration also provides CRISPR Therapeutics with the option to license up to two additional siRNA programs. This partnership expands CRISPR Therapeutics’ therapeutic portfolio into RNA-based medicines, complementing its ongoing efforts in gene editing and broadening its impact across serious and chronic diseases. For Sirius, the collaboration marks a major milestone in its mission to deliver innovative RNA-based therapies globally, leveraging deep expertise in siRNA design and delivery. About CTX211 CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of type 1 diabetes (T1D), which incorporates gene edits that aim to make cells hypoimmune and enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose. About CRISPR Therapeutics Founded over a decade ago, CRISPR Therapeutics is a leading gene editing company focused on developing transformative medicines for serious diseases. The Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, approved for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. CRISPR Therapeutics is advancing a broad and diversified pipeline across hemoglobinopathies, oncology, regenerative medicine, cardiovascular and autoimmune, and rare diseases. The Company continues to expand its leadership in gene editing through the development of SyNTase™ editing, a novel and proprietary gene-editing platform designed to enable precise, efficient, and scalable gene correction. To accelerate and expand its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit www.crisprtx.com.CRISPR THERAPEUTICS® standard character mark and design logo, SyNTase™, CTX112™, CTX131™, CTX211™, CTX310®, CTX320™, CTX340™, CTX450™ and CTX460™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. CRISPR Special Note Regarding Forward-Looking StatementsStatements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) CRISPR Therapeutics strategy, goals, anticipated financial performance and the sufficiency of its cash resources; (iv) plans and expectations for the commercialization of, and anticipated benefits of, CASGEVY, including anticipated patient access to CASGEVY; (v) regulatory submissions and authorizations, including timelines for and expectations regarding additional regulatory agency decisions; (vi) the expected benefits of its collaborations; and (vii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9 and SyNTase, as well as other technologies. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This press release also contains information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. This press release discusses investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. Investor Contact: +1-617-307-7503 ir@crisprtx.com Media Contact: +1-617-315-4493 media@crisprtx.com CRISPR Therapeutics AG Condensed Consolidated Statements of Operations (Unaudited, In thousands except share data and per share data) Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Revenue: Collaboration revenue $— $— $— $— Grant revenue 889 602 2,646 1,623 Total revenue $889 602 $2,646 $1,623 Operating expenses: Research and development 58,902 82,160 201,280 238,498 Acquired in-process research and development — — 96,253 — General and administrative 16,931 17,419 55,143 54,853 Collaboration expense, net 57,115 11,153 159,777 110,250 Total operating expenses 132,948 110,732 512,453 403,601 Loss from operations (132,059) (110,130) (509,807) (401,978)Total other income, net 26,237 25,064 61,841 75,924 Net loss before income taxes (105,822) (85,066) (447,966) (326,054)Provision for income taxes (619) (876) (3,020) (2,887)Net loss (106,441) (85,942) (450,986) (328,941)Foreign currency translation adjustment (28) 76 94 66 Unrealized gain on marketable securities 973 13,368 3,052 8,586 Comprehensive loss $(105,496) $(72,498) $(447,840) $(320,289)Net loss per common share — basic $(1.17) $(1.01) $(5.12) $(3.92)Basic weighted-average common shares outstanding 91,305,337 85,234,926 88,124,241 83,988,063 Net loss per common share — diluted $(1.17) $(1.01) $(5.12) $(3.92)Diluted weighted-average common shares outstanding 91,305,337 85,234,926 88,124,241 83,988,063 CRISPR Therapeutics AGCondensed Consolidated Balance Sheets Data(Unaudited, in thousands) As of September 30, 2025 December 31, 2024 Cash and cash equivalents $286,497 $298,257 Marketable securities 1,629,213 1,605,569 Marketable securities, non-current 28,412 — Working capital 1,810,135 1,849,350 Total assets 2,245,308 2,242,034 Total shareholders’ equity 1,915,982 1,932,080
BOULDER, Colo., Nov. 10, 2025 /PRNewswire/ — Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions, today announced the appointment of Michael Nofi, as Chief…
Three Late Breaking Science Presentations from MAPLE-HCM Provide Additional Data Including Responder Analyses, Patient Reported Outcomes, and Cardiac Biomarkers SOUTH SAN FRANCISCO, Calif., Nov. 10, 2025 (GLOBE NEWSWIRE) — Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that additional data from MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM) were presented in three Late Breaking Science sessions at the Hypertrophic Cardiomyopathy Medical Society Scientific Sessions and the American Heart Association Scientific Sessions 2025 in New Orleans, LA. Two of the presentations were simultaneously published in the Journal of the American College of Cardiology.1,2 “These additional analyses from MAPLE-HCM expand on the primary finding that aficamten is superior to metoprolol on exercise capacity, with new insights into the overall treatment effect of aficamten as well as its effect on symptoms and biomarkers in comparison to metoprolol,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “What’s notable is patients treated with aficamten achieved a significantly greater number of clinical response categories compared with metoprolol, and that nearly 40% achieved significant improvements in patient reported symptoms.” Responder Analysis Shows Significantly More Patients on Aficamten Achieved Positive or Complete Response Compared to Metoprolol Andrew Wang, M.D., Cardiologist and Professor of Medicine, Duke University School of Medicine presented a pre-specified responder analysis from MAPLE-HCM evaluating five clinically relevant measures of disease burden: complete hemodynamic response, symptom improvement, cardiac biomarker response, enhanced exercise capacity and favorable cardiac remodeling. These results were also simultaneously published in the Journal of the American College of Cardiology.1 After 24 weeks of treatment, aficamten was associated with greater improvements than metoprolol in all outcome measures (all p
GOTHENBURG, Sweden, Nov. 10, 2025 /PRNewswire/ — Cereno Scientific (Nasdaq First North: CRNO B), an innovative biotech pioneering treatments to enhance and extend life for people with rare cardiovascular and pulmonary diseases, today announced the submission of the clinical trial…
Multicenter outcomes FISH&CHIPS study of nearly 8,000 patients confirms total plaque volume as a powerful independent predictor of long-term cardiovascular events
Heartflow Plaque Staging Personalizes Individual Cardiac Risk
*Heartflow Plaque Analysis is an FDA-cleared device. Heartflow Plaque Staging is an investigational only framework and its safety and effectiveness have not been reviewed by the FDA.
Overall Considerations
3. Tzimas, et al. JACC. 2023. https://doi.org/10.1016/j.jcmg.2023.05.011
NEW ORLEANS, Nov. 09, 2025 (GLOBE NEWSWIRE) — Heartflow, Inc. (Heartflow) (Nasdaq: HTFL), the leader in AI technology for coronary artery disease (CAD), today announced late-breaking data from the FISH&CHIPS Study presented at the American Heart Association (AHA) Scientific Sessions 2025. The new data add to the robust and growing body of evidence supporting AI-powered Heartflow Plaque Analysis with Heartflow Plaque Staging* — the most clinically validated framework for actionable CAD care. The retrospective analysis, which evaluated nearly 8,000 symptomatic patients from a cohort of the FISH&CHIPS Study, represents the largest validation to date of the Heartflow Plaque Staging framework based on total plaque volume (TPV) measurement.1 Key findings include: Patients in the highest TPV stage experienced more than a 5x greater risk of major cardiovascular events compared with patients in the lowest stage (hazard ratio 5.10, p-value < 0.0001).Higher plaque volume stages were independently associated with significantly increased rates of cardiovascular death and myocardial infarction over a median 3.3 years of follow-up.Associations remained significant after adjustment for coronary stenosis, FFRCT values and cardiovascular risk factors. “This study provides strong validation of TPV-based staging measured with Heartflow Plaque Analysis as a predictor of future heart attacks or cardiovascular death,” said Timothy Fairbairn, Ph.D., principal investigator for the FISH&CHIPS study, Liverpool Heart and Chest Hospital, and Associate Professor at the University of Liverpool, UK. “The ability to accurately measure plaque will enable cardiologists to better predict which patients are most at risk above the traditional risk factors, and thus personalize treatment, in order to prevent heart attacks or death in the future.” The findings build on results from the DECIDE Registry presented at the Society of Cardiovascular Computed Tomography (SCCT) 2025 Annual Scientific Meeting in July. DECIDE data showed that Heartflow Plaque Analysis with Plaque Staging led to changes in medical management for more than half of patients, resulting in an average reduction in LDL cholesterol of 18.7 mg/dL at 90 days. These results indicate management changes guided by Heartflow Plaque Staging result in an expected 15% decrease in risk of a cardiac event.2,3,4 “We are demonstrating how AI-powered Heartflow Plaque Analysis with Heartflow Plaque Staging can fundamentally change the way we manage CAD,” said Campbell Rogers, M.D., F.A.C.C., Chief Medical Officer at Heartflow. “These latest findings show that by embedding plaque insights directly into the diagnostic pathway, we can help physicians make more confident decisions to guide personalized and precise treatment for their patients.” *Heartflow Plaque Analysis is an FDA-cleared device. Heartflow Plaque Staging is an investigational-only framework, and its safety and effectiveness have not been reviewed by the FDA. About Heartflow’s Technology and ResearchHeartflow’s technology is redefining precision cardiovascular care through clinically-proven AI and the world’s largest coronary imaging dataset. Heartflow has been adopted by more than 1,400 institutions globally and continues to strengthen its commercial presence to make this cutting-edge solution more widely available to an increasingly diverse patient population. Backed by ACC/AHA guidelines and supported by more than 600 peer-reviewed publications, Heartflow has redefined how clinicians manage care for nearly 500,000 patients worldwide. Key benefits include: Proprietary data pipeline: Built from more than 110 million annotated CTA images, Heartflow’s data foundation powers advanced AI models that deliver highly accurate, reproducible insights across diverse patient populations.Extensive clinical and real-world validation: Heartflow’s AI-driven solutions have been validated through clinical evidence in over 100 studies assessing over 365,000 patients. Proven in real-world practice with reproducibility and accuracy, Heartflow’s coronary CTA image acceptance rates exceed 96%.Seamless clinical integration via upgraded workflow: Heartflow delivers final quality-reviewed analyses instantly upon order, enabling clinicians to move from diagnosis to decision without delay.Quality system, global security and patient-data integrity compliance: Heartflow meets or exceeds leading international standards, including HITRUST, SOC 2 Type 2, GDPR, HIPAA, CCPA, ISO 13485, and ISO 27001. About Heartflow, Inc.Heartflow is transforming coronary artery disease from the world’s leading cause of death into a condition that can be detected early, diagnosed accurately, and managed for life. The Heartflow One platform uses AI to turn coronary CTA images into personalized 3D models of the heart, providing clinically meaningful, actionable insights into plaque location, volume, and composition and its effect on blood flow — all without invasive procedures. Discover how we’re shaping the future of cardiovascular care at heartflow.com. Media ContactElliot Levyelevy@heartflow.com Investor ContactNick Laudiconlaudico@heartflow.com Photos accompanying this announcement are available at https://www.globenewswire.com/NewsRoom/AttachmentNg/4702aa38-c48c-4291-91aa-937325c8206f https://www.globenewswire.com/NewsRoom/AttachmentNg/a2a0e458-a265-4790-90b4-33f7403b0b2e 1 Fairbairn et al. AHA 2025.2 Rinehart SJ, et al. DECIDE Primary Outcomes. J Cardiovasc Comput Tomogr. 2025; 19(4):S78-79. doi.org/10.1016/j.jcct.2025.05.1853 Collins et al. Lancet 2016. DOI: 10.1016/S0140-6736(16)31357-54 Fairbairn et al. HEART. 2025. doi:10.1136/heartjnl-2025-BSCI.5
BOSTON, Nov. 09, 2025 (GLOBE NEWSWIRE) — Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, today announced that positive interim data from its ongoing MUSIC-HFpEF Phase 1/2a clinical trial was presented at the American Heart Association (AHA) Scientific Sessions 2025 taking place November 7-10, 2025, in New Orleans, Louisiana. The late-breaking presentation, titled “First-in-Human Gene Therapy Trial for Patients with Heart Failure with Preserved Ejection Fraction (HFpEF): MUSIC-HFpEF – Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction,” was delivered by Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center, during the Featured Science session on Biological and Pragmatic Interventions in Heart Failure: From Present to Future on Sunday, November 9, 2025, at 8:00 AM CST. The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene therapy treatment for heart failure with preserved ejection fraction (HFpEF) delivered through a proprietary minimally invasive intracoronary infusion methodology. SRD-002 utilizes an adeno-associated type 1 virus vector carrying the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a) to directly target the molecular pathways underlying the core pathology of HFpEF by enhancing myocardial relaxation and reducing stiffness. The presentation highlighted data from the ongoing trial, which, as of the interim data cutoff date of October 15, 2025, has treated five patients in Cohort A with a low dose of 3E13 viral genomes (vg) who completed the twelve-month follow-up and five patients in Cohort B with a high dose of 4.5E13 vg. SRD-002 demonstrated a favorable safety profile and early clinical benefits. With follow-up ranging from 6 to 12 months, no gene therapy-related serious adverse events have been reported. Patients in the low-dose cohort have shown improvements in New York Heart Association (NYHA) heart failure classification and Kansas City Cardiomyopathy Questionnaire at both 6 and 12 months, with clinically meaningful stabilizations and improvements in pulmonary capillary wedge pressure (PCWP) at rest and peak exercise. The data support progression to the Phase 2 portion of the ongoing trial to test durable disease-modification in HFpEF by SRD-002. Patients in Cohort A and B received higher optimized doses of 3.0E13 vg and 4.5E13 vg, respectively—substantially higher than in prior studies, yet still significantly lower than systemic intravenous (IV) infusion. The dosing strategy was rationally designed and optimized using Medera’s proprietary human-based mini-Heart™ technology platform, including its HFpEF disease model co-developed with AstraZeneca, which has contributed to a US Food and Drug Administration (FDA) Investigational New Drug (IND) clearance and Fast Track Designation. “The data presented today at the AHA Scientific Sessions demonstrate continued safety and clinical benefits with SRD-002,” said Marat Fudim, MD, MHS. “These results reinforce the potential of gene therapy to address the fundamental pathophysiological mechanisms in HFpEF and provide hope for patients with this challenging condition.” “We are pleased to announce the completion of enrollment for this innovative clinical trial and are encouraged by the outcomes observed in the low dose cohort,” said Ronald Li, PhD, CEO and Founder of Medera. “We remain dedicated to advancing this investigational gene therapy, which targets a condition characterized by significant unmet medical needs.” The AHA Scientific Sessions is the premier global event in cardiovascular health and science offering unmatched access to groundbreaking research, dynamic learning, and career-defining connections. For additional information about the MUSIC-HFpEF trial, visit ClinicalTrials.gov using the study identifier NCT06061549. About Heart Failure with Preserved Ejection Fraction (HFpEF) Heart failure (HF) is a global pandemic with an estimated 64.3 million cases worldwide and a rising prevalence trend. Accounting for 50% or more of the overall HF population, HFpEF is an age-related condition that has become increasingly prevalent in recent years. This surge is partly due to better awareness and identification of the condition and partly due to lifestyle changes affecting cardiac myocytes. Individuals affected by HFpEF experience similar morbidity and mortality to patients with HF with reduced ejection fraction (HFrEF). Despite the growing epidemic of this emerging syndrome, HFpEF-focused interventional trials have had little success, except for the use of sacubitril-valsartan (Entresto™) and the sodium glucose transporter-2 (SGLT-2) inhibitor empagliflozin (Jardiance™) for reducing cardiovascular mortality and heart failure hospitalization. However, these agents are not disease-modifying, highlighting the critical need for therapeutic interventions targeting the physiological mechanisms involved in HFpEF. About Medera Inc. Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics. Medera operates via its two preclinical and clinical business units, Novoheart and Sardocor, respectively. Novoheart capitalizes on the world’s first and award-winning “mini-Heart” Technology for revolutionary disease modelling and drug discovery, uniquely enabling the modelling of human-specific diseases and discovery of therapeutic candidates free from species-specific differences in accordance to the FDA Modernization Act 2.0. Novoheart’s versatile technology platform provides a range of state-of-the-art automation hardware and software as well as screening services, for human-specific disease modelling, therapeutic target discovery and validation, drug toxicity and efficacy screening, and dosage optimization carried out in the context of healthy and/or diseased human heart chambers and tissues. Global pharmaceutical and academic leaders are using Novoheart’s technology platform for their drug discovery and development purposes. The Novoheart platform has facilitated and accelerated the development of Sardocor’s lead therapeutic candidates that are currently in clinical trials. Sardocor is dedicated to the clinical development of novel next-generation therapies for Medera. Leveraging Novoheart’s human-based drug discovery and validation platforms, Sardocor aims to expedite drug development and regulatory timelines for its gene and cell therapy pipeline. Sardocor has received Investigational New Drug (IND) clearances from the FDA for three ongoing AAV-based cardiac gene therapy clinical trials targeting Heart Failure with Reduced Ejection Fraction (HFrEF), Heart Failure with Preserved Ejection Fraction (HFpEF) with the Fast Track Designation, and Duchenne Muscular Dystrophy-associated Cardiomyopathy (DMD-CM) with the Orphan Drug Designation. Additionally, Sardocor’s pipeline includes four preclinical gene therapy and three preclinical small molecule candidates targeting various cardiac, pulmonary, and vascular diseases. For more information, please visit www.medera.bio. Contacts Ally StubinPublic RelationsICR HealthcareAlly.stubin@icrhealthcare.com 646.667.1861 Stephanie CarringtonInvestor RelationsICR HealthcareStephanie.carrington@icrhealthcare.com 646.277.1282
Oral presentation highlights sotagliflozin’s unique benefits to HFpEF patients in significantly improving cardiac and physical performance, and quality of life Oral presentation highlights sotagliflozin’s unique benefits to HFpEF patients in significantly improving cardiac and physical performance, and quality of life
Data support NEK7 as a potential novel and differentiated therapeutic approach to modulate the NLRP3 inflammasome in multiple cardiovascular and cardiometabolic diseases, including pericarditis and atherosclerosis Initial data from a Phase 1 study of MRT-8102 in healthy volunteers and elevated CVD-risk subjects on track for first half of 2026 Poster presentation on November 8 at 10:30 a.m. CST BOSTON, Nov. 08, 2025 (GLOBE NEWSWIRE) — Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, today announced the company will present preclinical data on the potential of MRT-8102, a first-in-class, NEK7-directed MGD for inflammatory diseases driven by the NLRP3 inflammasome, at the American Heart Association’s Scientific Sessions 2025, held November 7-10 in New Orleans, LA. “These promising findings reinforce our belief in the highly differentiated profile of MRT-8102, the only clinical-stage degrader targeting NEK7, as a potential treatment for cardiovascular and cardiometabolic diseases such as pericarditis, atherosclerosis, and others,” said Sharon Townson, Ph.D., Chief Scientific Officer of Monte Rosa Therapeutics. “By modulating the NLRP3/IL-1/IL-6 pathway upstream of other approaches, MRT-8102 potently inhibited pyroptotic cell death and inhibited the release of multiple inflammatory cytokines. Furthermore, MRT-8102 has the potential to block cholesterol crystal-induced cardiovascular inflammation characterized by pyroptosis and cytokine release that leads to atherosclerotic plaque pathogenesis. We are encouraged by the growing interest in targeting the NLRP3/NEK7 inflammasome to treat cardiovascular disease, and we believe we have a unique approach to achieve this. We continue to enroll our Phase 1 study of MRT-8102 and look forward to presenting initial data in healthy volunteers and elevated CVD-risk subjects in the first half of 2026.” The poster, entitled, “Selective Degradation of NIMA-related kinase 7 (NEK7) via a Molecular Glue Degrader Inhibits IL-1 Downstream of NLRP3 Inflammasome Activation: A Novel Therapeutic Approach for Cardiovascular Inflammation” (Poster Number #Sa4063), will be displayed on Saturday, November 8, 2025 from 10:30 to 11:30 a.m. CST in a poster session entitled, “Novel Cellular Stress Sensors in Cardiovascular Pathology: Metabolic, Mechanical, and Immune Interactions.” The poster will be presented by Daric Wible, Ph.D., Senior Scientist II, Biology, Monte Rosa Therapeutics. Summary of key findings: MRT-8102 is a selective, potent, and durable NEK7 degrader. Activation of the NLRP3 inflammasome critically depends on NEK7.Administration of MRT-8102 led to inhibition of NLRP3 inflammasome in vitro and in vivo and subsequently inhibited production of multiple inflammatory cytokines.In in vitro assays, MRT-8102 inhibited pyroptotic membrane permeabilization in stimulated human monocyte-derived macrophages (hMDM), unlike anti-IL-1 and anti-IL-6 therapies. Additionally, only MRT-8102 inhibited release of multiple cytokines from stimulated hMDM.In vitro, NEK7 degradation inhibited cholesterol crystal-induced NLRP3 inflammasome activation, a key driver of atherosclerotic plaque pathogenesis, more potently than selnoflast, an NLRP3 inhibitor currently in development.In a mouse peritonitis model, MRT-8102 led to potent inhibition of the cytokines IL-1β, IL-1α, IL-6, and TNF in peritoneal lavage.MRT-8102 demonstrated near-complete suppression of IL-1β and Caspase-1 activity in ex vivo-stimulated whole blood from orally dosed cynomolgus monkeys.Degrading NEK7 to modulate the inflammasome represents a novel and differentiated approach with potential therapeutic application in multiple cardiovascular and cardiometabolic diseases, including pericarditis and atherosclerosis. About MRT-8102MRT-8102 is a potent, highly selective, and orally bioavailable investigational molecular glue degrader (MGD) that targets NEK7 for the treatment of inflammatory diseases linked to NLRP3, IL-1β, and IL-6 dysregulation. NEK7 has been shown to be required for NLRP3 inflammasome assembly, activation and IL-1β release both in vitro and in vivo. Aberrant NLRP3 inflammasome activation and the subsequent release of active IL-1β and interleukin-18 (IL-18) has been implicated in multiple inflammatory disorders, including cardiovascular disease, gout, osteoarthritis, neurologic disorders including Parkinson’s disease and Alzheimer’s disease, and metabolic disorders. In a non-human primate model, MRT-8102 was shown to potently, selectively, and durably degrade NEK7, and resulted in near-complete reductions of IL-1β and caspase-1 following ex vivo stimulation of whole blood. MRT-8102 has demonstrated a considerable safety margin ( >200-fold exposure margin over projected human efficacious dose) in GLP toxicology studies. MRT-8102 is currently being investigated in a Phase 1 study (clinicaltrials.gov identifier NCT07119125) in healthy participants and participants at elevated cardiovascular disease risk. About Monte RosaMonte Rosa Therapeutics is a clinical-stage biotechnology company developing highly selective molecular glue degrader (MGD) medicines for patients living with serious diseases. MGDs are small molecule protein degraders that have the potential to treat many diseases that other modalities, including other degraders, cannot. Monte Rosa’s QuEEN™ (Quantitative and Engineered Elimination of Neosubstrates) discovery engine combines AI-guided chemistry, diverse chemical libraries, structural biology, and proteomics to rationally design MGDs with unprecedented selectivity. Monte Rosa has developed the industry’s leading pipeline of first-in-class and only-in-class MGDs, spanning autoimmune and inflammatory diseases, oncology, and beyond, with three programs in the clinic. Monte Rosa has ongoing collaborations with leading pharmaceutical companies in the areas of immunology, oncology and neurology. For more information, visit www.monterosatx.com. Forward-Looking Statements This communication includes express and implied “forward-looking statements,” including forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that are not historical facts and in some cases, can be identified by terms such as “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue,” “ongoing,” or the negative of these terms, or other comparable terminology intended to identify statements about the future. Forward-looking statements contained herein include, but are not limited to, statements around the potential of the Company’s NEK7-directed MGD, referred to as MRT-8102, to address inflammatory diseases driven by the NLRP3 inflammasome, including cardiovascular disease and cardiometabolic disease, including pericarditis and atherosclerosis, the Company’s belief that MRT-8102 could offer a differentiated approach to treating multiple inflammatory diseases based on the potency, selectivity, and durable pharmacodynamics seen in its preclinical studies, the Company’s belief in the potential for MRT-8102 to treat cardiovascular disease by blocking cholesterol crystal-induced cardiovascular inflammation characterized by pyroptosis and cytokine release that leads to atherosclerotic plaque pathogenesis, our expectations for the continuing advancement of our Phase 1 study and the timing thereof, including updates related to status, safety data, pharmacokinetics, NEK7 protein degradation, and key downstream pharmacodynamic markers and the timing of any clinical data read-outs, including the potential readout of initial data in healthy volunteers and elevated CVD-risk subjects expected in the first half of 2026, as well as our expectations of success for our programs, including for MRT-8102, among others. By their nature, these statements are subject to numerous risks and uncertainties, including those risks and uncertainties set forth in our most recent Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission on March 20, 2025, and any subsequent filings, that could cause actual results, performance or achievement to differ materially and adversely from those anticipated or implied in the statements. You should not rely upon forward-looking statements as predictions of future events. Although our management believes that the expectations reflected in our statements are reasonable, we cannot guarantee that the future results, performance, or events and circumstances described in the forward-looking statements will be achieved or occur. Recipients are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date such statements are made and should not be construed as statements of fact. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, any future presentations, or otherwise, except as required by applicable law. Certain information contained in these materials and any statements made orally during any presentation of these materials that relate to the materials or are based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party studies, publications, surveys and other data to be reliable as of the date of these materials, we have not independently verified, and make no representations as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, no independent source has evaluated the reasonableness or accuracy of our internal estimates or research and no reliance should be made on any information or statements made in these materials relating to or based on such internal estimates and research. Investors Andrew Funderburkir@monterosatx.com Media Cory Tromblee, Scient PRmedia@monterosatx.com
