Latest news with #RegenerativeMedicineAdvancedTherapy
Business Wire
17-07-2025
- Business
- Business Wire
Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A601 Gene Therapy for PKP2-Arrhythmogenic Cardiomyopathy
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 U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601, the Company's investigational adeno-associated virus (AAV)-based gene therapy for the treatment of PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A601 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review through the program's development. 'The FDA's RMAT designation for RP-A601 represents a meaningful advancement for Rocket and for patients living with PKP2-ACM, a life-threatening genetic heart disease characterized by ventricular arrhythmias and sudden cardiac death,' said Kinnari Patel, PharmD, MBA, President, Head of R&D of Rocket Pharmaceuticals. 'This marks the fifth RMAT designation in our history and underscores our commitment to developing potentially curative gene therapies for patients with rare and inherited cardiovascular diseases. The early clinical data for RP-A601 are highly encouraging, and we look forward to continued collaboration with the FDA throughout the program's development.' RMAT designation was established under the 21 st Century Cures Act to expedite the development and review of promising therapeutic candidates, including gene therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation provides several benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product's biologics license application (BLA). Preliminary results from the ongoing Phase 1 clinical trial of RP-A601 presented at the 2025 Annual Meeting of the American Society of Gene and Cell Therapy demonstrated encouraging early safety and efficacy. All three adult patients treated with a single dose of RP-A601 (8x10¹³ GC/kg) showed increased PKP2 protein expression, including 110% and 398% increases in the two patients with low baseline levels, as well as improved desmosomal integrity with relocalization of key structural proteins. Improvements or stabilization were observed across clinically meaningful endpoints, including right ventricular function, ventricular arrhythmias, and quality of life highlighted by increases of 34–41 points in KCCQ-12 scores and improvements in NYHA classification from Class II to Class I. The safety profile was favorable, with RP-A601 generally well-tolerated, no dose-limiting toxicities, and most adverse events being mild or moderate and self-limited. About RP-A601 RP-A601 is an investigational gene therapy for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). RP-A601 consists of a recombinant adeno-associated serotype rh74 capsid containing a functional version of the human PKP2 transgene ( which is administered as a single intravenous (IV) infusion. RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment that may improve survival and quality of life for patients affected by PKP2-ACM. Rocket holds Fast Track designation in the U.S. and Orphan Drug designation in the U.S. and Europe for the program. About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) PKP2-ACM is an inherited heart disease caused by mutations in the PKP2 gene and characterized by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. PKP2-ACM affects approximately 50,000 adults and children in the U.S. and Europe. Patients living with PKP2-ACM have an urgent unmet medical need, as current medical, implantable cardioverter defibrillator (ICD), and ablation therapies do not consistently prevent disease progression or arrhythmia recurrence, are associated with significant morbidity including inappropriate shocks and device and procedure-related complications, and do not address the underlying pathophysiology or genetic mutation. 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 adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. 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 'could,' 'believe,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'will give,' 'estimate,' 'seek,' 'will,' 'may,' 'suggest' or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to fund its operations into 2027. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled 'Risk Factors' in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 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.
Business Wire
16-07-2025
- Health
- Business Wire
Genascence Announces U.S. Food and Drug Administration Grants Regenerative Medicine Advanced Therapy Designation to GNSC-001 for Knee Osteoarthritis (OA)
PALO ALTO, Calif.--(BUSINESS WIRE)--Genascence Corporation ('Genascence'), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to GNSC-001, a potential first-in-class gene therapy blocking interleukin 1 (IL-1) for the treatment of knee osteoarthritis (OA). GNSC-001 is a genetic medicine – a recombinant adeno-associated viral vector expressing an optimized human interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks IL-1 signaling. IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation, joint pain, and cartilage destruction. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single intra-articular injection into the affected joint. 'GNSC-001 is the first IL-1 inhibitor for OA that has been shown to generate IL-1Ra expression levels that maintain therapeutic thresholds long-term following a single administration to inhibit pathogenic IL-1. The FDA RMAT designation for GNSC-001 underscores the strength of the clinical data to date, recognizing its potential to transform the treatment paradigm for OA,' said Thomas Chalberg, Ph.D., founder and CEO of Genascence. 'With the RMAT designation for GNSC-001, we look forward to working closely with the FDA as we seek to accelerate late-stage clinical development of GNSC-001 so we can bring a new treatment option to people suffering from this incapacitating, disabling disease.' To date, GNSC-001 has been studied in two human clinical trials. The first-in-human Phase 1 study (NCT02790723) was a single-center, open-label, dose-escalation design in nine participants with knee OA. The DONATELLO Phase 1b clinical trial (NCT05835895) is a double-blind, placebo-controlled dose-ranging study designed to evaluate the safety, tolerability, and pharmacodynamics of a single intra-articular injection of GNSC-001 in patients with OA of the knee that enrolled 67 participants at 10 centers across the U.S. The RMAT designation is a special FDA program aimed at speeding up the development and review of promising cell and gene therapies under the 21 st Century Cures Act. The FDA grants RMAT designation to drug candidates that show potential to treat, modify, reverse, or cure serious or life-threatening diseases and address unmet medical needs. The FDA's RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs. In addition, RMAT designation provides accelerated development and review pathways for regenerative medicine products, including early and intensive FDA guidance, potential eligibility for accelerated approval and priority review, and the possibility of rolling review. The U.S. Food and Drug Administration (FDA) granted GNSC-001 Fast Track designation in the fourth quarter of 2024. Earlier this year, Genascence completed a successful meeting with the FDA on the design of the Phase 2b/3 clinical trial of GNSC-001 focused on clinical efficacy and plans to initiate the Phase 2b/3 study in 2026. The DONATELLO clinical trial was supported by a $12 million award from the California Institute for Regenerative Medicine (CLIN2-14265). About Osteoarthritis (OA) of the Knee Osteoarthritis (OA) is a progressive joint disease that is a leading cause of disability. It is characterized by destruction of cartilage and structural changes in bone within the joint, which contribute to pain and loss of joint function. Osteoarthritis affects more than 30 million Americans and is increasing as a result of the aging population and increasing prevalence of obesity. Osteoarthritis represents a major economic burden, owing to direct medical costs and loss of productivity. Each year, millions of patients are treated for knee OA with NSAIDs, opioids, and steroid injections into the knee to manage their knee pain. There are no currently available therapies known to alter or slow down OA progression. About Genascence Corporation Genascence, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, is developing life-changing treatments for highly prevalent conditions affecting millions of people. The company was founded in 2017 with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. Headquartered in Palo Alto, California, Genascence's founders and leadership team have deep experience in the design, development, and manufacturing of successful gene therapies and biological medicines. For more information, please visit
Time Business News
27-06-2025
- Business
- Time Business News
Pioneering Technologies in Regenerative Medicine
Regenerative medicine is a branch of medical science that aims to repair, replace, and regenerate damaged tissues or organs to bring them back to normal function. Stem cell research and regenerative technologies are creating new opportunities for treatment and accelerating growth in the regenerative medicine market. The growing prevalence of chronic disease, increasing funding, and technology advances in gene therapy, an increasing aging population, and more favorable regulations are major contributors to the growing share and speed of development of the regenerative medicine. Key Growth Drivers and Opportunities Rising Number of Aging Population: With the global aging phenomenon, there will be increased incidences of chronic and degenerative diseases such as osteoarthritis, cardiovascular ailments, neurodegenerations, and musculo-skeletal problems, very much in demand for advanced treatment options. Regenerative medicine comprising stem cell therapy, gene therapy, and tissue engineering, among others, presents viable and sustainable alternatives in contrast to conventional treatments such as medication and surgery, which provide temporary relief. Given that regenerative medicine addresses the actual cause of tissue and organ damage, it significantly enhances the quality of life and decreases long-term care burdens, serving as the prime growth driver for the regenerative medicine. Challenges The regenerative medicine is limited by high treatment costs, difficult regulatory approvals, not enough long-term clinical data, and problems in big manufacturing and making everything the same which can block easy acceptance and availability. Innovation and Expansion Beam Therapeutics' BEAM-302 has granted RMAT status by the FDA for Alpha-1 Antitrypsin Deficiency In May 2025, The U.S. Food and Drug Administration (FDA) has designated BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor, as Regenerative Medicine Advanced Therapy (RMAT). This designation is intended to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). Beam Therapeutics Inc. is a biotechnology company that develops precision genetic medicines through base editing. There is a substantial unmet need for efficient treatments that may address the whole range of symptoms for AATD, an inherited genetic illness that affects the lungs and/or liver and causes early onset emphysema and liver disease. The FDA Gives BrainChild Bio's CAR T Therapy for Pediatric Brain Tumors RMAT Status In May 2025, The U.S. Food and Drug Administration (FDA) has designated the investigational B7-H3 targeting autologous CAR T-cell therapy as a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. This news was released by BrainChild Bio, Inc., a clinical-stage biotechnology company that is developing CAR T-cell therapies to treat tumors in the central nervous system (CNS). The FDA Designates MeiraGTx's AAV-GAD Gene Therapy as an RMAT for Parkinson's disease In May 2025, AAV-GAD has been designated by the U.S. Food and Drug Administration (FDA) as Regenerative Medicine Advanced Therapy (RMAT) for the treatment of Parkinson's disease that is not sufficiently controlled with anti-Parkinsonian drugs. This announcement was made today by MeiraGTx Holdings plc, a vertically integrated, clinical-stage genetic medicines company. This RMAT was granted after the FDA received favorable results from three clinical trials showing the advantages of AAV-GAD when given as a single stereotactic infusion to the brain's subthalamic nucleus. Inventive Sparks, Expanding Markets Key players in the regenerative medicine market are R&D Systems, Inc., Moderna, Novartis AG and others. These key players are focusing on increasing the research and development of advanced therapies and innovative strategies for 3D bioprinting advancement, and more acquisitions to enhance product offerings through collaborations will be projected to drive the target market. About Author: Prophecy is a specialized market research, analytics, marketing and business strategy, and solutions company that offer strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high value opportunities in the target business area. Also, we help our client to address business challenges and provide best possible solutions to overcome them and transform their business. TIME BUSINESS NEWS
Yahoo
24-06-2025
- Business
- Yahoo
Carisma Therapeutics, OrthoCellix enter definitive merger agreement
Carisma Therapeutics (CARM) and OrthoCellix, a wholly-owned subsidiary of Ocugen (OCGN), announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on the development of OrthoCellix's NeoCart technology for the treatment of knee articular cartilage defects and plans to initiate a U.S. Food and Drug Administration-endorsed Phase 3 clinical trial for NeoCart. OrthoCellix is developing NeoCart as an autologous cartilage implant technology utilizing patient cells to repair articular cartilage defects of the knee. The novel platform merges a fortified 3D scaffold and patented bioprocessing technology to grow chondrocytes-the cells responsible for maintaining cartilage health-to produce adolescent-like cartilage at the time of implant. NeoCart has the potential to accelerate healing and reduce pain by creating a similar, functional joint surface to help patients return to normal activities and prevent complications associated with articular cartilage damage. OrthoCellix anticipates launching its Phase 3 clinical trial by the end of 2025. Previously, NeoCart received Regenerative Medicine Advanced Therapy designation and concurrence from the FDA on a single, confirmatory Phase 3 clinical trial to enable submission of a Biologics License Application. Under the terms of the merger agreement, OrthoCellix will merge with and into a wholly-owned subsidiary of Carisma, with OrthoCellix continuing as a wholly-owned subsidiary of Carisma and the surviving company of the Merger. Carisma will issue to the pre-merger OrthoCellix stockholder shares of Carisma common stock as merger consideration in exchange for the cancellation of shares of capital stock of OrthoCellix. Carisma also expects to enter into subscription agreements for a private financing with Ocugen and other select investors, which is expected to close concurrently with the completion of the merger, to enable the combined company to complete the Phase 3 trial of NeoCart without any additional cost or investment from Ocugen. In connection with the closing of the proposed transactions, Carisma stockholders will be issued contingent value rights representing the right to receive certain payments from proceeds received by the combined company, if any, related to Carisma's pre-transaction legacy assets. Under the terms of the merger agreement, upon the closing of the proposed transactions and after giving effect to the contemplated $25M concurrent financing, OrthoCellix's stockholder and the other participants in the concurrent financing are expected to own approximately 90% of the combined company, and existing Carisma stockholders are expected to own approximately 10% of the combined company, each on a fully diluted basis. The percentage of the combined company that each company's former stockholders will own after completion of the merger is subject to adjustment based on Carisma's net cash at the closing and the proceeds from the concurrent financing, among other adjustments, in each case as described in the merger agreement. Upon the closing of the proposed transactions, 'Carisma Therapeutics Inc.' is expected to be renamed 'OrthoCellix, Inc.' and trade on the Nasdaq Capital Market under the ticker symbol 'OCLX.' The transaction has been unanimously approved by the board of directors of both companies and is expected to close in the second half of 2025, subject to customary closing conditions, including approvals by the stockholders of each company and the effectiveness of a registration statement to be filed with the Securities and Exchange Commission to register the shares of Carisma common stock to be issued in connection with the merger. In connection with the companies' entry into the merger agreement, directors and officers of Carisma and OrthoCellix's stockholder have executed support agreements, pursuant to which they have agreed to vote all of their shares of capital stock in favor of the merger or the issuance of Carisma equity in the merger, as applicable. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Disclaimer & DisclosureReport an Issue Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
17-06-2025
- Business
- Yahoo
Lyell Immunopharma Announces Positive New Clinical Data Demonstrating High Rates of Durable Complete Responses from the Phase 1/2 Trial of LYL314 for the Treatment of Aggressive Large B-cell Lymphoma
LYL314 demonstrated robust clinical responses, with an 88% overall response rate and a 72% complete response rate in patients treated in the third- or later-line setting (N = 25) 71% of patients with complete response remained in complete response at ≥ 6 months Manageable safety profile appropriate for outpatient administration with no Grade ≥ 3 cytokine release syndrome and low rates of Grade ≥ 3 ICANS with rapid resolution Pivotal single-arm PiNACLE trial is underway in CAR T-naïve patients with large B-cell lymphoma treated in the third- or later-line setting Lyell to host an investor webcast at 8:00 AM ET today SOUTH SAN FRANCISCO, Calif., June 17, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage company advancing next-generation CAR T-cell therapies for patients with cancer, today announced positive new clinical data from the multi-center Phase 1/2 trial of LYL314, including data from patients with large B-cell lymphoma (LBCL) treated in the third- or later-line (3L+) setting. LYL314 is an autologous dual-targeting CD19/CD20 chimeric antigen receptor (CAR) T-cell product candidate with Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA that is in development for patients with relapsed and/or refractory (R/R) LBCL. In patients treated in the 3L+ setting (N = 25), LYL314 continued to demonstrate robust clinical responses, with an 88% overall response rate and a 72% complete response rate. Of the 3L+ patients who achieved a complete response, 71% remained in complete response at ≥ 6 months. The single-arm pivotal PiNACLE trial, a seamless expansion of the Phase 1/2 trial of patients with R/R LBCL being treated in the 3L+ setting, is underway. 'Based on the LYL314 data to be presented at the International Conference on Malignant Lymphoma and my personal experience treating patients in the clinical trial, I believe that LYL314 has the potential to provide differentiated benefit for patients with relapsed/refractory large B-cell lymphoma in both the complete response rate and durability of response,' stated Akil Merchant, MD, Associate Professor and Co-Director of the Lymphoma Program at the Samuel Oschin Cancer Center, Cedars-Sinai Medical Center, Los Angeles, CA, and an investigator in the Phase 1/2 clinical trial. 'We look forward to completing enrollment in the ongoing single-arm pivotal trial evaluating LYL314 in patients in the third- or later-line setting.' Fifty-one CAR T-naive patients with R/R LBCL received LYL314 as of April 15, 2025 (the data cutoff date for the presentation). The efficacy evaluable population consisted of 36 patients with Day 84 assessments or prior disease progression or death. Patient demographics and baseline disease characteristics were consistent with high-risk patient populations: median ages of 65 and 69 years in the 3L+ and 2L, respectively, 41% of 3L+ and 65% of 2L patients had Stage IV disease at trial entry, and 47% of 3L+ and 82% of 2L patients had primary refractory disease. There were 49 patients who received the recommended Phase 2 dose of 100 x 106 CAR T cells; two patients received a dose of 300 x 106 CAR T cells. CD19/CD20 screening was not required prior to enrollment. In efficacy-evaluable 3L+ patients, with a median follow up of 9 months (N = 25): The overall response rate was 88% (22/25 patients), with 72% (18/25) of patients achieving a complete response 71% (10/14) of patients with complete response remained in complete response at ≥ 6 months In initial data from efficacy-evaluable 2L patients, with a median follow up of 5 months (N = 11): The overall response rate was 91% (10/11 patients), with 64% (7/11) achieving a complete response 100% (7/7) of patients with complete response were in complete response at last assessment, including 3/3 at ≥ 6 months In patients with primary refractory disease, a difficult to treat population, 70% (7/10) achieved a complete response These patients had high-risk features, including primary refractory disease (91%), stage IV disease (64%), and older age (27% > 75 years; median age 73 years) In 51 patients, including patients from both the 3L+ and the 2L cohorts, a manageable safety profile appropriate for outpatient administration was observed. No Grade ≥ 3 and low rates of Grade 1 (22%) or Grade 2 (35%) cytokine release syndrome (CRS) were reported. Immune effector cell-associated neurotoxicity syndrome (ICANS) was reported in 6% (Grade 1), 2% (Grade 2), and 14% (Grade ≥ 3) of patients. The median time to complete resolution of all reports of ICANS was 5 days, with rapid improvement (median of 2 days) to Grade 2 or lower with standard therapy. No deaths were related to LYL314 administration. LYL314 demonstrated robust expansion with a time to peak of 10 days (N = 51). The final drug product contained the desired CD62L-positive naïve T-cell phenotype (median, 95%). Rapid and durable depletion of B cells was demonstrated through month 6 and up to the month 12 assessment using a highly sensitive and robust method. 'Based on these robust data, and our recent End-of-Phase 1 meeting with the FDA, we have initiated PiNACLE, a single-arm pivotal trial of LYL314 in patients with large B-cell lymphoma in the third- or later-line setting and remain on track to initiate a pivotal trial to evaluate LYL314 in the second-line setting by the beginning of 2026,' said Lynn Seely, MD, Lyell's President and Chief Executive Officer. The data will be presented on Wednesday, June 18, 2025 in an oral session at the International Conference on Malignant Lymphoma in Lugano, Switzerland by Akil Merchant, MD, Associate Professor and Co-Director of the Lymphoma Program at the Samuel Oschin Cancer Center, Cedars-Sinai Medical Center, Los Angeles, CA, and will be available as a presentation in the Investors' section of the Company's website. Conference Call Details Lyell's management will host an investor conference call and webcast beginning at 8:00 AM ET today. The Webcast registration link can be accessed here. A replay of the event and presentation materials will be archived on the Investor page of the Lyell Website following the end of the event. About LYL314 LYL314 (formerly IMPT-314) is a next-generation dual-targeting CD19/CD20 CAR T-cell product candidate designed to increase complete response rates and prolong the duration of the responses as compared to the approved CD19‑targeted CAR T-cell therapies for the treatment of large B-cell lymphoma. LYL314 is designed with an 'OR' logic gate to target B cells that express either CD19, CD20 or both. LYL314 is manufactured to produce a CAR T-cell product with higher proportions of naïve and central memory T cells through a proprietary process that enriches for CD62L-expressing cells. This manufacturing process is designed to generate CAR T cells with enhanced antitumor activity. LYL314 has received Regenerative Medicine Advanced Therapy (RMAT) designation, as well as Fast Track Designation, from the U.S. Food and Drug Administration for the treatment of patients with relapsed and/or refractory aggressive B-cell lymphoma in the third- or later-line setting. About the PiNACLE Trial PiNACLE is a single-arm pivotal trial of LYL314, 100 x 106 CAR T cells, in patients with large B-cell lymphoma treated in the third- or later-line setting. The trial is expected to enroll approximately 120 patients with relapsed and/or refractory diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, high grade B-cell lymphoma, grade 3B follicular lymphoma, or transformed follicular lymphoma who have not previously received CAR T-cell therapy. Patients may be treated with LYL314 in either the inpatient or outpatient setting. The primary endpoint of the trial is the overall response rate. More information about the PiNACLE trial can be found on (NCT05826535) here. About Lyell Lyell is a clinical-stage company advancing a pipeline of next-generation CAR T-cell therapies for patients with hematologic malignancies and solid tumors. To realize the potential of cell therapy for cancer, Lyell utilizes a suite of technologies to endow CAR T cells with attributes needed to drive durable tumor cytotoxicity and achieve consistent and long-lasting clinical responses, including the ability to resist exhaustion, maintain qualities of durable stemness and function in the hostile tumor microenvironment. The Lyell LyFE Manufacturing Center™ has commercial launch capability and can manufacture more than 1,200 CAR T-cell doses at full capacity. To learn more, please visit Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements regarding: the potential clinical benefits and therapeutic potential of LYL314 for patients with R/R LBCL, including its potential to increase complete response rates and prolong the duration of responses as compared to approved CD19-targeted CAR T-cell therapies for the treatment of LBCL; the potential benefits, if any, from the RMAT and Fast Track designations from the FDA for the treatment of patients with R/R LBCL in the 3L+ setting; expectations around enrollment and timing of Lyell's ongoing single-arm pivotal trial evaluating LYL314 in patients in the 3L+setting and the initiation of a pivotal trial to evaluate LYL314 in the 2L setting; the potential of Lyell's manufacturing process to generate CAR T cells with enhanced antitumor activity; the sufficiency of the capacity of LyFE to manufacture drug supply for Lyell's ongoing and planned pivotal trials and through potential commercial launch; Lyell's anticipated progress, business plans, business strategy and clinical trials; Lyell's advancement of its pipeline, technology platform and research, development and clinical capabilities; and other statements that are not historical fact. These statements are based on Lyell's current plans, objectives, estimates, expectations and intentions, are not guarantees of future performance and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, but are not limited to, risks and uncertainties related to: the potential for results from clinical trials to differ from nonclinical, early clinical, preliminary or expected results; Lyell's limited experience as a company in enrolling and conducting clinical trials and lack of experience in completing clinical trials; significant adverse events, toxicities or other undesirable side effects associated with Lyell's product candidates; Lyell's ability to initiate or progress clinical trials on the anticipated timelines, if at all; RMAT and Fast Track designations may not actually lead to faster development, regulatory review or approval process, and does not assure ultimate FDA approval; the significant uncertainty associated with Lyell's product candidates ever receiving any regulatory approvals; Lyell's ability to obtain, maintain or protect intellectual property rights related to its product candidates; the complexity of manufacturing cellular therapies and Lyell's ability to manufacture and supply its product candidates for its clinical trials; implementation of Lyell's strategic plans for its business and product candidates; the sufficiency of Lyell's capital resources and need for additional capital to achieve its goals; the effects of macroeconomic conditions, including the effects of disruption between the U.S. and its trading partners due to tariffs or other policies, and any geopolitical instability; potential changes to U.S. drug pricing, including the potential for 'most-favored nations' pricing limitations; and other risks, including general economic conditions and regulatory developments, not within our control; and other risks described under the heading 'Risk Factors' in Lyell's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the Securities and Exchange Commission on May 13, 2025. Forward-looking statements contained in this press release are made as of this date, and Lyell undertakes no duty to update such information except as required under applicable law. Contact: Ellen RoseSenior Vice President, Communications and Investor Relationserose@ while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
