RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Priority Review (China), Breakthrough Therapy (China), Conditional marketing approval (China), Orphan Drug (South Korea), Orphan Drug (United Kingdom), Priority Review (United States), Special Review Project (China), Conditional marketing approval (European Union)

Structure/Sequence

Sequence Code 13031374

Source: *****

Clinical Trials associated with Ciltacabtagene autoleucel

NCT07093554

/ Not yet recruitingPhase 1IIT

Cilta-Talq Fusion Study: A Phase 1b Study of Talquetamab Bridging Therapy Followed by Ciltacabtagene Autoleucel in Patients With Relapsed/Refractory Multiple Myeloma

This is a single-arm, open-label, phase 1b study evaluating the safety and feasibility of using talquetamab as bridging therapy prior to cilta-cel in patients with relapsed and refractory multiple myeloma (RRMM).

Start Date01 Dec 2025

Sponsor / Collaborator

Medical College of Wisconsin

NCT07106684

/ RecruitingPhase 2IIT

A Prospective, Single-Arm, Phase II Study of Autologous Stem Cell Transplantation Combined With BCMA CAR-T Therapy Followed by GPRC5D/CD3 Bispecific Antibody Maintenance in Transplant-Eligible Patients With Primary Plasma Cell Leukemia

This is a prospective, single-arm, phase II study to evaluate the efficacy and safety of autologous stem cell transplantation combined with BCMA CAR-T therapy followed by GPRC5D/CD3 bispecific antibody maintenance in transplant-eligible patients with primary plasma cell leukemia.

Start Date10 Aug 2025

Sponsor / Collaborator-

NCT07106710

/ RecruitingPhase 2IIT

Start Date10 Aug 2025

Sponsor / Collaborator-

100 Clinical Results associated with Ciltacabtagene autoleucel

100 Translational Medicine associated with Ciltacabtagene autoleucel

100 Patents (Medical) associated with Ciltacabtagene autoleucel

344

Literatures (Medical) associated with Ciltacabtagene autoleucel

31 Dec 2025·Hematology

BCMA CAR-T therapy as salvage therapy in patients with plasmablastic myeloma

Article

Author: Jin, Chengwei ; Zhu, Jun ; Li, Su ; Deng, Jing ; Kang, Liqing ; Jiang, Ying

OBJECTIVES:

Plasmablastic myeloma (PBM) is a variant of multiple myeloma associated with a poor prognosis. We investigated the efficacy and safety of B-cell maturation antigen (BCMA) chimeric antigen receptor T cell (CAR-T) therapy in patients with PBM.

METHODS:

The study comprised six patients diagnosed with PBM between January 1, 2023 and December 31, 2023. The patients received BCMA single-target CAR-T therapy or BMCA/CD19 dual-target CAR-T therapy, with some patients undergoing hematopoietic stem cell transplantation before treatment. The median patient age was 55.5 years (range, 41-63). Four patients exhibited high-risk cytogenetic abnormalities.

RESULTS:

The objective response rate (ORR) was 83.3%, with four of six patients achieving a complete response or better and three of six achieving a strigent complete response. Two patients exhibited progression-free survival (PFS) of at least 6 months, one of whom succumbed to a pulmonary infection, whereas four patients died of disease progression. Cytokine release syndrome (CRS) was observed in all patients, three of whom experienced grade 3-4 CRS. Two patients experienced grade 1-2 immune effector cell-associated neurotoxicity syndrome. There were no CRS-related deaths.

CONCLUSION:

BCMA CAR-T therapy was safe and effective as a salvage treatment for PBM, and its toxicity was controllable. Future research will examine the use of CAR-T therapy as part of combination regimens.

01 Dec 2025·Human Vaccines & Immunotherapeutics

The hotspots and publication trends in glioblastoma and CAR-T immunotherapy: A bibliometric analysis.

Article

Author: Xie, Yun ; Gu, Jingyu ; Song, Zhaoming ; Nong, Xun ; Chen, Zhouqing ; Guo, Yanao ; Yang, Chen ; Wang, Zhong ; Qu, Ruisi

In recent years, chimeric antigen receptor T cell (CAR-T) immunotherapy has made considerable progress in the treatment of glioblastoma. The aim of this study was to comprehensively explore the prospects and future trends of CAR-T immunotherapy for glioblastoma through systematic bibliometric analysis. Publications pertaining to glioblastoma and CAR-T immunotherapy from 2008 to 2024 were extracted from the Web of Science Core Collection. Utilizing VOSviewer (version 1.6.20), CiteSpace (version 6.3.R1), and R 4.3.3, this study concentrated on evaluating contributions from countries, institutions, authors, and journals, while also identifying research hotspots and emerging trends. A total of 570 publications were identified, demonstrating an annual growth rate of 31.71%. The USA led the field with 269 publications, followed by China (113). The University of Pennsylvania, Harvard University, and the University of California System emerged as the most prolific institutions. Frontiers in Immunology published the most articles (42), while Clinical Cancer Research garnered the highest number of citations (2,867). Recent keyword bursts (2022-2024) underscored an increasing focus on combination therapy approaches and outcomes, particularly emphasizing "radiotherapy" (strength 3.49), "solid tumor" (strength 3.49), and "efficacy" (strength 2.79). In recent years, research on CAR-T immunotherapy for glioblastoma has gradually shifted from the exploration of basic mechanisms to the application of clinical combination therapy, and this shift in research direction indicates that CAR-T immunotherapy has a relatively mature technology and great clinical translation potential. In the coming years, CAR-T immunotherapy is expected to usher in a golden era and benefit more patients suffering from glioblastoma.

01 Sep 2025·CURRENT OPINION IN ONCOLOGY

Chimeric antigen receptor T-cell therapy for multiple myeloma

Review

Author: Liu, Lawrence ; Htut, Myo

Purpose of review:

Chimeric antigen receptor T-cell therapy (CAR T) in relapsed, refractory multiple myeloma (RRMM) has rapidly expanded with two FDA-approved agents and many more in the clinical trial pipeline. As such, we aim to review the standard of care and investigational products.

Recent findings:

Idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel) were initially approved in the fourth-line setting and quickly moved to the second-line and third-line settings due to efficacy. Delayed neurotoxicities remain a challenging entity to consider when deciding on CAR T versus other options. Given the high efficacy of cilta-cel, many trials are evaluating its role as frontline consolidation or even in smoldering myeloma. Many novel CAR T products are being studied and will improve the impressive armamentarium of RRMM therapeutics.

Summary:

This is an exciting area with countless studies investigating novel CAR T constructs and sequencing in hopes of further extending and improving our patients’ lives.

440

News (Medical) associated with Ciltacabtagene autoleucel

11 Aug 2025

·www.globenewswire.com

Legend Biotech Reports Second Quarter 2025 Results and Recent Highlights

CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) net trade sales of approximately $439 millionCARVYKTI® demonstrated positive long-term outcomes in CARTITUDE-1 study with one-third of patients remaining progression-free for ≥5 yearsPresented other important CARVYKTI® and new solid tumor data at ASCOOver 7,500 patients treated to dateCash and cash equivalents, and time deposits of $1.0 billion, as of June 30, 2025 SOMERSET, N.J., Aug. 11, 2025 (GLOBE NEWSWIRE) -- Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, today reported its second quarter 2025 unaudited financial results and key corporate highlights. “The groundbreaking five-year survival data from CARTITUDE-1, with one-third of patients remaining progression-free, reinforces CARVYKTI’s durability and potential to redefine the standard of care when treating relapsed and refractory multiple myeloma patients. These findings mark the latest step forward in ensuring patients in need of long-term relief from disease progression and the burden of continuous treatments can benefit from a one-time infusion of our differentiated therapy,” said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. “Further, our continued commitment to expanding access to CARVYKTI, with recent launches in several new markets, is underscored by a record quarterly performance representing the strongest single period of any CAR-T therapeutic sales to date. As we advance our pipeline of CAR-T programs and work towards profitability in 2026, we remain guided by our mission of delivering innovative cell therapy solutions to patients worldwide.” Regulatory Updates The U.S. Food and Drug Administration (FDA) removed Risk Evaluation and Mitigation Strategies (REMS) for currently approved BCMA- and CD19-directed autologous chimeric antigen receptor (CAR) T cell immunotherapies, including CARVYKTI. In addition, product labeling was updated to include the reduction of certain monitoring requirements for CARVYKTI patients. Key Business Developments Treated over 7,500 clinical and commercial patients to date.At the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting: Announced positive long-term outcomes from the CARTITUDE-1 study, demonstrating one-third of patients with relapsed and refractory multiple myeloma remained progression-free for ≥5 years.Presented Phase 3 CARTITUDE-4 study subgroup analyses at a median follow-up of 33.6 months, which highlighted consistent, durable progression-free and overall survival benefit when compared to standard therapies across cytogenetic risk groups as early as second-line of therapy at ASCO.Presented safety, tolerability, and preliminary efficacy results of a Phase 1 dose-escalating study of LB2102, an autologous DLL3-targeted CAR-T therapy, which demonstrated no dose-limiting toxicities and preliminary efficacy signal was observed up to four dose levels in patients with relapsed or refractory small-cell lung cancer and large cell neuroendocrine carcinoma.Announced preliminary results of a first-in-human Phase 1 study of LB1908, an autologous Claudin 18.2-targeted CAR-T product, which demonstrated encouraging antitumor activity with manageable safety and tolerability in patients with advanced gastric, gastroesophageal, and esophageal adenocarcincoma. At the European Hematology Association (EHA) 2025 Congress, presented an analysis of 355 patients from the CARTITUDE program on the association of key biomarker Absolute Lymphocyte Count (ALC) with select neurocognitive treatment-emergent adverse events post-treatment. The analysis showed that patients with a Movement and Neurocognitive Treatment-emergent event or Cranial Nerve Palsy had significantly higher ALC compared to control, suggesting ALC may help guide closer monitoring and preemptive interventions.Cash and cash equivalents, and time deposits of $1.0 billion, which Legend Biotech believes will provide financial runway into 2026, when Legend Biotech anticipates potentially achieving an operating profit excluding unrealized foreign exchange gains or losses. Second Quarter 2025 Financial Results Cash Position: Cash and cash equivalents, and time deposits were $1.0 billion as of June 30, 2025.License Revenue: License revenue was $35.3 million for the three months ended June 30, 2025, compared to $90.8 million for the three months ended June 30, 2024. The decrease was primarily driven by the timing of $75.1 million of milestones achieved during the three months ended June 30, 2024, under the Janssen Agreement, while we did not achieve any milestones from the Janssen Agreement for the three months ended June 30, 2025.The decrease was offset by an increase in license revenue recognized in the three months ended June 30, 2025, under an exclusive agreement with a related party. For the three months ended June 30, 2025, we recognized $20.0 million in license revenue under this agreement. No license revenue was recognized under this agreement during the three months ended June 30, 2024. Collaboration Revenue: Collaboration revenue was $219.7 million for the three months ended June 30, 2025, compared to $93.3 million for the three months ended June 30, 2024. The increase was due to an increase in revenue generated from sales of CARVYKTI® in connection with the Janssen Agreement.Cost of Collaboration Revenue: Cost of collaboration revenue was $94.9 million for the three months ended June 30, 2025, compared to $45.4 million for the three months ended June 30, 2024. The increase was primarily due to our share of the cost of sales in connection with CARVYKTI® sales under the Janssen Agreement and expenditures to support expansion in manufacturing capacity.Research and Development Expenses: Research and development expenses were $98.3 million for the three months ended June 30, 2025, compared to $112.6 million for the three months ended June 30, 2024. The decrease was due to higher research and development activities in cilta-cel for the three months ended June 30, 2024, driven by start-up costs for clinical production at our two Belgium facilities. With one of those facilities now manufacturing commercial product, clinical production has scaled back, resulting in lower research and development expenses for the current period. Administrative Expenses: Administrative expenses were $32.6 million for the three months ended June 30, 2025, compared to $35.4 million for the three months ended June 30, 2024. Administrative expenses remained relatively flat, with an increase in staffing-related expenses due to higher headcount, offset by lower IT expenses due to the timing of completion of existing projects or the initiation of new projects compared to the same period in the prior year.Selling and Distribution Expenses: Selling and distribution expenses were $48.1 million for the three months ended June 30, 2025, compared to $30.1 million for the three months ended June 30, 2024. The increase was due to increased costs associated with commercial activities, including expansion of the sales force due to growing sales of CARVYKTI®. Adjusted Net Income (Loss): Adjusted net income was $10.1 million for the three months ended June 30, 2025, compared to an adjusted net loss of $2.5 million for the three months ended June 30, 2024. Webcast/Conference Call Details: Legend Biotech will host its quarterly earnings call and webcast today at 8:00 am ET. To access the webcast, please visit this weblink. A replay of the webcast will be available on Legend Biotech’s website at https://investors.legendbiotech.com/events-and-presentations. About Legend Biotech With over 2,800 employees, Legend Biotech is the largest standalone cell therapy company and a pioneer in treatments that change cancer care forever. The company is at the forefront of the CAR-T cell therapy revolution with CARVYKTI®, a one-time treatment for relapsed or refractory multiple myeloma, which it develops and markets with collaborator Johnson & Johnson. Centered in the US, Legend is building an end-to-end cell therapy company by expanding its leadership to maximize CARVYKTI’s patient access and therapeutic potential. From this platform, the company plans to drive future innovation across its pipeline of cutting-edge cell therapy modalities. Learn more at www.legendbiotech.com, and follow us on X (formerly Twitter) and LinkedIn. CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to Legend Biotech’s strategies and objectives; statements relating to CARVYKTI®, including Legend Biotech’s expectations for CARVYKTI® and its therapeutic potential; statements related to Legend Biotech manufacturing expectations for CARVYKTI® and the ability of Legend Biotech’s manufacturing expansion and commercial execution to maintain CARVYKTI’s market leadership position; statements related to Legend Biotech’s ability to fund its operations into 2026 and to achieve profitability in 2026; and the potential benefits of Legend Biotech’s product candidates. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Legend Biotech’s expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products; unexpected clinical trial results, including as a result of additional analysis of existing clinical data or unexpected new clinical data; unexpected regulatory actions or delays, including requests for additional safety and/or efficacy data or analysis of data, or government regulation generally; unexpected delays as a result of actions undertaken, or failures to act, by our third party partners; uncertainties arising from challenges to Legend Biotech’s patent or other proprietary intellectual property protection, including the uncertainties involved in the U.S. litigation process; government, industry, and general product pricing and other political pressures; as well as the other factors discussed in the “Risk Factors” section of Legend Biotech’s Annual Report on Form 20-F for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) on March 11, 2025 and Legend Biotech’s other filings with the SEC. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in this press release as anticipated, believed, estimated or expected. Any forward-looking statements contained in this press release speak only as of the date of this press release. Legend Biotech specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. INVESTOR CONTACT:Jessie YeungTel: (732) 956-8271jessie.yeung@legendbiotech.com PRESS CONTACT:Mary Ann OndishTel: (914) 552-4625media@legendbiotech.com LEGEND BIOTECH CORPORATIONCONDENSED CONSOLIDATED STATEMENTS OF PROFIT OR LOSS(UNAUDITED; DOLLARS IN THOUSANDS, EXCEPT PER SHARE AND SHARES DATA) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024REVENUE License revenue$35,338 $90,846 $44,686 $103,027 Collaboration revenue 219,717 93,254 405,332 171,735 Other revenue 3 2,423 93 5,752 Total revenue 255,058 186,523 450,111 280,514 Cost of collaboration revenue (94,872) (45,355) (164,369) (94,456)Cost of license and other revenue (3,119) (5,096) (4,966) (10,734)Research and development expenses (98,302) (112,626) (200,226) (213,590)Administrative expenses (32,594) (35,353) (64,057) (67,282)Selling and distribution expenses (48,052) (30,063) (89,021) (54,286)Loss on asset impairment — — (970) — Finance costs (5,222) (5,484) (10,283) (10,959)Finance income* 10,433 17,049 22,489 30,919 Other (expense)/income, net* (108,128) 12,435 (162,636) 62,116 Loss before tax (124,798) (17,970) (223,928) (77,758)Income tax expense (582) (226) (2,368) (231)Net loss$(125,380) $(18,196) $(226,296) $(77,989) LOSS PER SHARE Basic$(0.34) $(0.05) $(0.62) $(0.21)Diluted$(0.34) $(0.05) $(0.62) $(0.21) Weighted average shares outstanding Basic 368,271,125 365,204,154 367,900,548 364,610,589 Diluted 368,271,125 365,204,154 367,900,548 364,610,589 *Certain prior year amounts have been reclassified to present finance income as a separate line item and to combine other income/(expense), net for comparative purposes. LEGEND BIOTECH CORPORATIONCONDENSED CONSOLIDATED STATEMENTS OF FINANCIAL POSITION(DOLLARS IN THOUSANDS) June 30, 2025 December 31, 2024NON-CURRENT ASSETS(Unaudited) Property, plant and equipment$106,381 $99,288Right-of-use assets 127,217 101,932Time deposits — 4,362Intangible assets 2,137 2,160Collaboration prepaid leases 198,646 172,064Other non-current assets* 5,659 6,430Total non-current assets$440,040 $386,236CURRENT ASSETS Collaboration inventories, net$35,589 $23,903Trade receivables 27,584 6,287Prepayments, other receivables and other assets 219,076 130,975Pledged deposits 70 70Time deposits 700,969 835,934Cash and cash equivalents 266,586 286,749Total current assets 1,249,874 1,283,918TOTAL ASSETS$1,689,914 $1,670,154 CURRENT LIABILITIES Trade payables$75,361 $38,594Other payables and accruals 138,836 166,180Government grants 651 532Lease liabilities 5,906 4,794Tax payable 11,550 20,671Contract liabilities 33,178 46,874Total current liabilities$265,482 $277,645NON-CURRENT LIABILITIES Collaboration interest-bearing advanced funding$310,264 $301,196Lease liabilities long term 71,742 44,613Government grants 6,887 6,154Total non-current liabilities 388,893 351,963TOTAL LIABILITIES$654,375 $629,608 EQUITY Share capital$37 $37Reserves 1,035,502 1,040,509Total equity 1,035,539 1,040,546TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY$1,689,914 $1,670,154 *Certain prior year amounts have been reclassified to combine advance payments for property, plant, and equipment into other non-current assets for comparative purposes. LEGEND BIOTECH CORPORATIONCONDENSED CONSOLIDATED STATEMENTS OF CASH FLOW(UNAUDITED; DOLLARS IN THOUSANDS) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024 Loss before tax$(124,798) $(17,970) $(223,928) $(77,758)Cash flows (used in) / provided by operating activities (13,042) (1,651) (116,796) 13,867 Cash flows (used in) / provided by investing activities (165,525) (695,631) 91,115 (1,091,779)Cash flows (used in) / provided by financing activities (990) 955 (323) 1,786 Net decrease in cash and cash equivalents (179,557) (696,327) (26,004) (1,076,126)Effect of foreign exchange rate changes, net 4,441 9 5,841 (334)Cash and cash equivalents at beginning of the year$441,702 $897,571 $286,749 $1,277,713 CASH AND CASH EQUIVALENTS AT END OF THE PERIOD$266,586 $201,253 $266,586 $201,253 Analysis of balances of cash and cash equivalents Cash and bank balances$967,625 $1,254,469 $967,625 $1,254,469 Less: Pledged deposits 70 431 70 431 Time deposits 700,969 1,052,785 700,969 1,052,785 Cash and cash equivalents as stated in the statement of financial position$266,586 $201,253 $266,586 $201,253 Cash and cash equivalents as stated in the statement of cash flows$266,586 $201,253 $266,586 $201,253 RECONCILIATION OF IFRS TO NON-IFRS MEASURES We use Adjusted Net Income (Loss) and Adjusted Net Income (Loss) per Share (which we sometimes refer to as “Adjusted EPS” or “ANI per Share”, respectively) as performance metrics. Adjusted Net Income (Loss) and ANI per share are not defined under IFRS, are not a measure of operating income, operating performance, or liquidity presented in accordance with IFRS, and are subject to important limitations. Our use of Adjusted Net Income (Loss) has limitations as an analytical tool, and you should not consider it in isolation or as a substitute for analysis of our results as reported under IFRS. For example: Although depreciation and amortization are non-cash charges, the assets being depreciated and amortized may have to be replaced in the future, and Adjusted Net Income (Loss) does not reflect cash capital expenditure requirements for such replacements or for new capital expenditure requirements.Adjusted Net Income (Loss) excludes unrealized foreign exchange gain or loss, which resulted primarily from changes in the intercompany loan balances and cash balances as a result of exchange rate changes between USD and EUR.Adjusted Net Income (Loss) does not reflect changes in, or cash requirements for, our working capital needs.In addition, Adjusted Net Income (Loss) excludes such as share based compensation expense, which has been, and will continue to be for the foreseeable future, a significant recurring expense for our business and an important part of our compensation strategy. Also, our definition of Adjusted Net Income (Loss) and ANI per Share may not be the same as similarly titled measures used by other companies. However, we believe that providing information concerning Adjusted Net Income (Loss) and ANI per Share enhances an investor’s understanding of our financial performance. We use Adjusted Net Income (Loss) as a performance metric that guides management in its operation of and planning for the future of the business. We believe that Adjusted Net Income (Loss) provides a useful measure of our operating performance from period to period by excluding certain items that we believe are not representative of our core business. We define Adjusted Net Income (Loss) as net income (loss) adjusted for (1) non-cash items such as depreciation and amortization, share based compensation, impairment loss, and (2) unrealized foreign exchange gain or loss mainly related to intercompany loan balances and cash deposit balances as a result of exchange rate changes between USD and EUR. ANI per Share is computed by dividing Adjusted Net Income (Loss) by the weighted average shares outstanding. A reconciliation between Adjusted Net Income (Loss) and Net Loss, the most directly comparable measure under IFRS, has been provided in the table below. LEGEND BIOTECH CORPORATIONRECONCILIATION OF IFRS TO NON-IFRS(UNAUDITED; DOLLARS IN THOUSANDS, EXCEPT PER SHARE AND SHARES DATA) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024Net loss$(125,380) $(18,196) $(226,296) $(77,989)Depreciation and amortization 5,854 5,369 11,053 11,091 Share based compensation 18,697 21,739 34,643 40,442 Impairment loss — — 970 — Unrealized foreign exchange loss/(gain) (included in Other income/(expense), net) 110,920 (11,419) 162,722 (61,308)Adjusted net income/(loss) (ANI)$10,091 $(2,507) $(16,908) $(87,764) ANI per share: ANI per share - basic$0.03 $(0.01) $(0.05) $(0.24)ANI per share - diluted*$0.03 $(0.01) $(0.05) $(0.24) *The diluted weighted average shares outstanding used in the calculation of the diluted ANI per share for the three months ended June 30, 2025, is 377,041,415.

Clinical ResultCell TherapyImmunotherapyFinancial StatementPhase 1

05 Aug 2025

·www.prnewswire.com

Cryoport Reports Second Quarter 2025 Financial Results

Second quarter revenue increased 14% year-over-year to $45.5 million Commercial Cell & Gene Therapy revenue increased 33% year-over-year to $8.7 million Life Sciences Services revenue rose 21% year-over-year, including a 28% increase in BioStorage/BioServices revenue Launched strategic partnership agreement with the DHL Group; closed CRYOPDP divestiture Company reaffirms full year 2025 revenue guidance of $165 to $172 million NASHVILLE, Tenn., Aug. 5, 2025 /PRNewswire/ -- Cryoport, Inc. (NASDAQ: CYRX) ("Cryoport" or the "Company"), a leading global provider of temperature-controlled supply chain solutions for the life sciences, today announced financial results for its second quarter (Q2) and first half (H1) of 2025. Jerrell Shelton, CEO of Cryoport, commented, "Cryoport delivered strong, double-digit growth across all revenue streams within Life Sciences Services in the second quarter, increasing 21% year-over-year and accounting for 54% of total revenue from continuing operations. Notably, revenue from BioLogistics Solutions increased 20% and BioStorage/BioServices revenue rose 28%, underscoring the growing demand for our integrated platform. "Revenue from the support of commercial cell and gene therapies increased 33% year-over-year to $8.7 million. This growth continues to be fueled by the increasing development and adoption of cell & gene therapies, a positive trend we believe will continue for years to come. "Life Sciences Products revenue grew 8% year-over-year. This solid performance was primarily driven by stronger demand, particularly from animal health customers. "The 14% year-over-year increase in total revenue from continuing operations, combined with our planned pathway to profitability, contributed to an increase in gross margin and a meaningful improvement in our adjusted EBITDA. With strong execution across all business units, we are reaffirming our full-year 2025 revenue guidance as we move towards our goal of sustainable, long-term profitability. "A key milestone this quarter was the launch of our strategic partnership with the DHL Group and DHL's acquisition of CRYOPDP. This transaction with DHL delivered both a strong infusion of capital, a substantial return on investment, and strengthened our global biologistics capabilities and effectiveness. By leveraging DHL's competencies, scale, and reach in APAC and EMEA, we believe we will be increasingly well positioned to expand our Life Sciences Services business and deepen our leadership in the developing global regenerative medicine market. "In summary, the second quarter was marked by strong revenue growth, improved profitability, and the execution of a transformative partnership strategy. We are entering the second half of the year with strong momentum and a clear focus on driving long-term shareholder value," concluded Mr. Shelton. In tabular form, Q2 2025 and H1 2025 revenue compared to Q2 2024 and H1 2024, respectively, were as follows: BioStorage/BioServices revenue continued its strong growth trajectory year-over-year, increasing 28% in Q2 2025 as we continue to introduce our capabilities to existing clients, add new clients into our global network, and as more commercial therapies progress in the number of patients treated. Revenue from the support of commercial cell & gene therapies increased 33% year-over-year to $8.7 million and included revenue from BioLogistics Solutions and accessories. As of June 30, 2025, we supported eighteen (18) commercial therapies. As of June 30, 2025, Cryoport supported a total of 728 global clinical trials, a net increase of 44 clinical trials over June 30, 2024, with 82 of these clinical trials in Phase 3. The number of trials by phase and region are as follows: In Q2 2025, one Marketing Authorization Applications (MAA) filing occurred and two Biologics License Applications (BLA) filings have occurred for label/geographic expansions post the quarter end. During the quarter, Cryoport's customer Abeona Therapeutics received U.S. Food and Drug Administration (FDA) approval for their cell therapy ZEVASKYNTM, for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). During the remainder of 2025, we anticipate up to an additional twenty (20) application filings, one (1) new therapy approval and an additional three (3) approvals for label/geographic expansions. Additionally, in late June 2025, the FDA announced it was removing the Risk Evaluation and Mitigation Strategies (REMS) requirements for approved BCMA- and CD19-directed autologous CAR-T cell immunotherapies. This reduces the regulatory burden and can lead to increased patient access and faster commercial scaling for these therapies. Cryoport-supported therapies such as Carvykti®, Yescarta®, Tecartus®, and Breyanzi® are included in this FDA action. Operational milestones Life Sciences Services Continued plans to complete our Global Supply Chain Centers in Paris, France and Santa Ana, California, with Paris expected to begin its launch in late 2025 and Santa Ana in the second half of 2026. CryoGene opened the first southeast regional automated sample storage center in partnership with Texas Children's Hospital. Launched our Cryoshuttle service in Tokyo, Japan, supporting multiple commercial therapies. Life Sciences Products MVE Biological Solutions launched its next generation SC 4/2V and SC 4/3V vapor shippers, offering improved safety and reliability for transporting and preserving sensitive biological materials at cryogenic temperatures. Recorded multiple sales of MVE's cryogenic storage system, the MVE High-Efficiency 800C, which was released earlier this year, meeting the needs of facilities that have limited space for cryostorage yet require high capacity and security. Deployed the highest number of MVE cryogenic dewars to the animal health industry since 2013. Financial Highlights On June 11, 2025, the Company completed its previously announced divestiture of its specialty courier CRYOPDP business to DHL Supply Chain International Holding B.V. ("DHL") and entered into a strategic partnership with DHL. The divestiture and strategic partnership are expected to enhance the Company's ability to develop its business, particularly in the EMEA and APAC regions, and to provide differentiated and high-value services aligned with Cryoport's long-term growth strategy. The results of CRYOPDP, a former business within Cryoport's Life Sciences Services business, are presented as discontinued operations for all periods presented within the Condensed Statements of Operations and Condensed Consolidated Balance Sheets included in this press release and are also not included in the non-GAAP financial measures presented herein. Revenue Total revenue from continuing operations for Q2 2025 was $45.5 million compared to $39.7 million for Q2 2024, a year-over-year increase of 14% or $5.7 million and up $4.4 million or 11% sequentially. Life Sciences Services revenue for Q2 2025 (representing 54% of our total revenue) was $24.4 million compared to $20.2 million for Q2 2024, up 21% year-over-year and 7% sequentially, including BioStorage/BioServices revenue of $4.5 million, up 28% year-over-year and 4% sequentially. Life Sciences Products revenue for Q2 2025 (representing 46% of our total revenue) was $21.1 million compared to $19.6 million for Q2 2024, up 8% year-over-year and 16% sequentially. Total revenue from continuing operations for H1 2025 was $86.5 million compared to $77.0 million for H1 2024. Life Sciences Services revenue for H1 2025 was $47.2 million compared to $39.6 million for H1 2024, including BioStorage/BioServices revenue of $8.8 million for H1 2025 compared to $7.1 million for H1 2024. Life Sciences Products revenue for H1 2025 was $39.3 million compared to $37.4 million for H1 2024. Gross Margin Total gross margin from continuing operations was 47.0% for Q2 2025 compared to 44.5% for Q2 2024. Gross margin for Life Sciences Services was 48.9% for Q2 2025 compared to 46.7% for Q2 2024. Gross margin for Life Sciences Products was 44.9% for Q2 2025 compared to 42.2% for Q2 2024. Total gross margin from continuing operations was 46.3% for H1 2025 compared to 42.5% for H1 2024. Gross margin for Life Sciences Services was 48.4% for H1 2025 compared to 45.1% for H1 2024. Gross margin for Life Sciences Products was 43.7% for H1 2025 compared to 39.7% for H1 2024. Operating Costs and Expenses Operating costs and expenses from continuing operations were $31.2 million for Q2 2025 compared to operating cost and expenses of $95.7 million for Q2 2024. The decrease for Q2 2025 reflects an impairment charge of $63.8 million in Q2 2024, which was primarily related to the write off of remaining goodwill for MVE Biological Solutions. Operating costs and expenses for H1 2025 decreased to $59.3 million compared to $128.3 million for H1 2024, reflecting the impairment charge relating to MVE Biological Solutions. Excluding the impairment charge, adjusted operating costs and expenses for H1 2025 were $59.3 million, compared to $64.5 million for H1 2024. Net Income (Loss) – including Discontinued Operations Net income for Q2 2025 and H1 2025 was $105.2 million and $93.2 million, respectively, compared to a net loss of $78.0 million and $96.9 million for the same periods in 2024, respectively. Net income for Q2 2025 and H1 2025 was primarily driven by the sale of our CRYOPDP specialty courier business during Q2 2025, which contributed $117.4 million and $114.4 million, net of taxes, respectively, to income from discontinued operations. Net income attributable to common stockholders was $103.2 million, or $2.05 per share, and $89.2 million, or $1.78 per share, for Q2 2025 and H1 2025, respectively. This compares to a net loss attributable to common stockholders of $80.0 million, or $1.62 per share, and $100.9 million, or $2.05 per share, for Q2 2024 and H1 2024, respectively. Excluding the gain on sale of CRYOPDP, net loss $12.2 million and $21.2 million for Q2 2025 and H1 2025, respectively, compared to $14.2 million and $28.1 million for Q2 2024 and H1 2024, respectively. Adjusted EBITDA Adjusted EBITDA was a negative $0.9 million for Q2 2025, compared to negative $5.6 million for Q2 2024. Adjusted EBITDA for H1 2025 was a negative $3.7 million compared to negative $12.2 million for H1 2024. Cash, Cash equivalents, and Short-Term Investments Cryoport held $426.0 million in cash, cash equivalents, and short-term investments as of June 30, 2025. Share Repurchase Programs During Q2 2025, the Company purchased 628,217 shares of its common stock under its repurchase programs, at an average price of $6.76 per share, for an aggregate amount of $4.2 million. Subsequent to the end of Q2 2025, the Company purchased an additional 371,783 shares of its common stock under its repurchase programs, at an average price of $7.36 per share, for an aggregate amount of $2.7 million, resulting in a total of 1 million shares repurchased since the beginning of Q2 2025.These shares were returned to the status of authorized but unissued shares of common stock. Following these repurchases, the Company had approximately $66.9 million in total of repurchase authorization available under its two repurchase programs. Guidance for Continuing Operations for Full Year Fiscal 2025 The Company is reiterating its revenue guidance for fiscal year 2025: total revenue from continuing operations is expected to be in the range of $165.0 million to $172.0 million, representing 5% to 10% growth year-over-year. The Company's 2025 guidance is dependent on its current business and expectations, which may be further impacted by, among other things, factors that are outside of our control, such as national economic factors, the global macroeconomic and geopolitical environment, supply chain constraints, inflationary pressures, tariffs and other trade restrictions and/or the effects of foreign currency fluctuations, as well as the other factors described in the Company's filings with the Securities and Exchange Commission ("SEC"), including in the "Risk Factors" section of its most recently filed periodic reports on Form 10-K and Form 10-Q, as well as in its subsequent filings with the SEC. Note: All reconciliations of GAAP to adjusted (non-GAAP) figures above are detailed in the reconciliation tables included later in the press release. Additional Information Further information on Cryoport's financial results is included in the attached condensed consolidated balance sheets and statements of operations, and additional explanations of Cryoport's financial performance are provided in the Company's Quarterly Report on Form 10-Q for the three months ended June 30, 2025, which is expected to be filed with the SEC on August 7, 2025. Additionally, the full report will be available in the SEC Filings section of the Investor Relations section of Cryoport's website at . Earnings Conference Call Information IMPORTANT INFORMATION: In addition to the earnings release, a document titled "Cryoport Second Quarter 2025 in Review", providing a review of Cryoport's business update, will be issued at 4:05 p.m. ET on Tuesday, August 5, 2025. The document is designed to be read in advance of the questions and answers conference call and will be accessible at . Cryoport management will host a conference call at 5:00 p.m. ET on August 5, 2025. The conference call will be in the format of a questions and answers session and will address any queries investors have regarding the Company's reported results. A slide deck will accompany the call. Conference Call Information The questions and answers call will be recorded and available approximately three hours after completion of the live event in the Investor Relations section of the Company's website at for a limited time. To access the replay of the questions and answers click here. A dial-in replay of the call will also be available to those interested, until August 12, 2025. To access the replay, dial 1-844-512-2921 (United States) or 1-412-317-6671 (International) and enter replay entry code: 1197564#. About Cryoport, Inc. Cryoport, Inc. (Nasdaq: CYRX), is a leading global provider of temperature-controlled supply chain solutions for the Life Sciences, with an emphasis on regenerative medicine. We support biopharmaceutical companies, contract manufacturers (CDMOs), contract research organizations (CROs), developers, and researchers with a comprehensive suite of services and products designed to minimize risk and maximize reliability across the temperature-controlled supply chain for the Life Sciences. Our integrated supply chain platform includes the Cryoportal® Logistics Management Platform, advanced temperature-controlled packaging, informatics, specialized biologistics, biostorage, bioservices, and cryogenic systems, which in varying combinations deliver end-to-end solutions that meet the rigorous demands of the life sciences. With innovation, regulatory compliance, and agility at our core, we are "Enabling the Future of Medicine™." Headquartered in Nashville, Tennessee, our company maintains a strong global presence with operations across the Americas, EMEA, and APAC. For more information, visit or follow via LinkedIn at or @cryoport on X, formerly known as Twitter at for live updates. Forward-Looking Statements Statements in this press release which are not purely historical, including statements regarding the Company's intentions, hopes, beliefs, expectations, representations, projections, plans or predictions of the future, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, those related to the Company's industry, business, long-term growth prospects, plans, strategies, acquisitions, future financial results and financial condition, such as the Company's outlook and guidance for full year 2025 revenue and the related assumptions and factors expected to drive revenue, projected growth trends in the markets in which the Company operates, the Company's plans and expectations regarding the launch of new products and services, such as the expected timing and benefits of such products and services launches, the Company's expectations about future benefits of its acquisitions, and anticipated regulatory filings, approvals, label/geographic expansions or moves to earlier lines of treatment approved with respect to the products of the Company's clients. Forward-looking statements also include those related to the Company's expectations about future benefits relating to the CRYOPDP divestiture and strategic partnership with DHL (collectively, the "DHL Transaction") and the Company's plans regarding the completion of its Global Supply Chain Centers, including expected timing. It is important to note that the Company's actual results could differ materially from those in any such forward-looking statements. Factors that could cause actual results to differ materially include, but are not limited to, risks and uncertainties associated with the effect of changing economic and geopolitical conditions, supply chain constraints, inflationary pressures, tariffs and other trade restrictions, the effects of foreign currency fluctuations, trends in the products markets, variations in the Company's cash flow, market acceptance risks, and technical development risks. Additional risks and uncertainties relating to the DHL Transaction include, but are not limited to, the risk that any disruption resulting from the DHL Transaction may adversely affect our businesses and business relationships, including with employees and suppliers. The Company's business could be affected by other factors discussed in the Company's SEC reports, including in the "Risk Factors" section of its most recently filed periodic reports on Form 10-K and Form 10-Q, as well as in its subsequent filings with the SEC. The forward-looking statements contained in this press release speak only as of the date hereof and the Company cautions investors not to place undue reliance on these forward-looking statements. Except as required by law, the Company disclaims any obligation and does not undertake to update or revise any forward-looking statements in this press release. Note Regarding Use of Non-GAAP Financial Measures To supplement our financial statements, which are presented on the basis of U.S. generally accepted accounting principles (GAAP), the following non-GAAP measures of financial performance as defined in Regulation G of the Securities Exchange Act of 1934 are included in this release: revenue at constant currency, revenue growth rate at constant currency, adjusted operating costs and expenses, adjusted net income (loss), and adjusted EBITDA. Non-GAAP financial measures are not calculated in accordance with GAAP, are not based on any comprehensive set of accounting rules or principles and may be different from non-GAAP financial measures presented by other companies. Non-GAAP financial measures, including revenue at constant currency, revenue growth rate at constant currency, adjusted operating costs and expenses, adjusted net income (loss), and adjusted EBITDA, should not be considered as a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We believe that revenue growth is a key indicator of how Cryoport is progressing from period to period and we believe that the non-GAAP financial measures, revenue at constant currency and revenue growth rate at constant currency, are useful to investors in analyzing the underlying trends in revenue. Under GAAP, revenue from continuing operations received in local (non-U.S. dollar) currency is translated into U.S. dollars at the average exchange rate for the period presented. As a result, fluctuations in foreign currency exchange rates affect the results of our operations and the value of our foreign assets and liabilities, which in turn may affect results of operations and cash flows and the comparability of period-to-period results of operations. When we use the term "constant currency," it means that we have translated local currency revenue from continuing operations for the current reporting period into U.S. dollars using the same average foreign currency exchange rates for the conversion of revenue from continuing operations into U.S. dollars that we used to translate local currency revenue for the comparable reporting period of the prior year. Revenue growth rate at constant currency refers to the measure of comparing the current reporting period revenue from continuing operations at constant currency with the reported GAAP revenue from continuing operations for the comparable reporting period of the prior year. However, we also believe that data on constant currency period-over-period changes have limitations, particularly as the currency effects that are eliminated could constitute a significant element of our revenue and could significantly impact our performance. We therefore limit our use of constant currency period-over-period changes to a measure for the impact of currency fluctuations on the translation of local currency revenue into U.S. dollars. We do not evaluate our results and performance without considering both period-over-period changes in non-GAAP constant currency revenue on the one hand and changes in revenue prepared in accordance with GAAP on the other. We caution the readers of this press release to follow a similar approach by considering revenue on constant currency period-over-period changes only in addition to, and not as a substitute for, or superior to, changes in revenue prepared in accordance with GAAP. Adjusted operating costs and expenses is defined as operating costs and expenses, excluding impairment losses, if any. Adjusted net income (loss) is defined as net income (loss), excluding impairment losses, if any. Management believes these measures, when read in conjunction with, and as supplemental to, the corresponding GAAP financial measures, provide useful measures to investors of Cryoport's expenses and operating results, meaningful comparisons with historical results, and insight into Cryoport's operating performance. Adjusted EBITDA is defined as net income (loss) adjusted for loss from discontinued operations, net interest expense, income taxes, depreciation and amortization expense, stock-based compensation expense, acquisition and integration costs, divestiture costs, cost reduction initiatives, investment income, unrealized (gain)/loss on investments, foreign currency (gain)/loss, net gain on extinguishment of debt, impairment loss, changes in fair value of contingent consideration and charges or gains resulting from non-recurring events, as applicable. Management believes that adjusted EBITDA provides a useful measure of Cryoport's operating results, a meaningful comparison with historical results and with the results of other companies, and insight into Cryoport's ongoing operating performance. Further, management and the Company's board of directors utilize adjusted EBITDA to gain a better understanding of Cryoport's comparative operating performance from period to period and as a basis for planning and forecasting future periods. Adjusted EBITDA is also a significant performance measure used by Cryoport in connection with its incentive compensation programs. Management believes adjusted EBITDA, when read in conjunction with Cryoport's GAAP financials, is useful to investors because it provides a basis for meaningful period-to-period comparisons of Cryoport's ongoing operating results, including results of operations, against investor and analyst financial models, helps identify trends in Cryoport's underlying business and in performing related trend analyses, and it provides a better understanding of how management plans and measures Cryoport's underlying business. SOURCE Cryoport, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyAcquisitionFinancial Statement

05 Aug 2025

·www.biospace.com

Cracking the Commercial Code for Cell and Gene Therapies

z_wei/ iStock From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to make CGTs commercially viable. But it is possible, experts agreed at a recent panel. This year has seen two prime exemplars of the challenges that face the cell and gene therapy sector: Sarepta Therapeutics and bluebird bio . Despite having products on the market and once being valued north of $10 billion, both took disastrous turns in recent months, as Sarepta’s market cap plummeted to just over $1 billion while bluebird was scooped up by two private equity firms for just under $50 million. Embroiled in a saga of patient safety , Sarepta’s fate as a company is now uncertain. But so too is the future of bluebird’s products, as the new owners, including the massive Carlyle Group, take over operations. The fates of the two companies highlight the ongoing challenges that face the CGT space more broadly. At a BioSpace -moderated panel at the BIO Convention in Boston this summer, experts mulled over what needs to happen to make this new class of medicines successful in the long term. Major considerations discussed included decentralized manufacturing and its regulation, factors that could lower prices and expand access, and the potential impact of tariffs. As the field advances on these fronts, all eyes will be on the companies trying to bring these therapies to the patients who need them. “I think the Carlyle purchase of bluebird is super interesting,” said panelist Jason Foster , CEO of Oribiotech. “The commercial viability question is still out there, so a very interesting case study for us to watch is: Can we take a failed product and company or set of products and turn it into something that’s commercially viable using this next generation of technologies?” Those next-gen technologies exist largely on the manufacturing side of the CGT ecosystem, both in terms of production itself and how that production is regulated, Foster and his fellow panelists at BIO agreed. And industry is starting to recognize the importance of this part of the equation. “Companies are coming to us, even early companies now, and having that conversation with us from the very beginning,” ElevateBio CEO Mike Paglia said. Foster emphasized that timing is everything. “You have to figure out the economics first,” he said. “That means, before you get into the clinic, fix your manufacturing.” Beyond that, the panelists in Boston said, are bigger conversations about lives and costs saved by CGT over the long run. But the industry must be prepared to scale. “These therapies work,” Foster said. “I think we could make the economics work, but we can’t rely on decade-old technologies and decade-old methodologies.” Advances in Manufacturing and Regulation In late July, the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) launched a new regulatory framework for decentralized manufacturing. In a series of guidelines published in advance of the launch, the regulator discussed how to implement new modular manufacturing and point of care legislation passed in January, including obtaining marketing authorization for the designation and the application of good manufacturing practices in far-flung facilities. Foster applauded the MHRA’s initiative. “Anything that enhances our flexibility is a good thing,” he said at BIO, noting that Ori is agnostic to the location of manufacturing. He explained that MHRA is one of the only regulatory agencies in the world to have established such a framework. The FDA released commentary on decentralized manufacturing in November 2023—“some of it was helpful, some of it wasn’t as helpful,” Foster summarized—but no formal guidelines have been released or implemented in the U.S. The advantages of distributed manufacturing include shortening so-called vein to vein times to get patients, often terminally ill, treated more quickly and without needing to freeze cells for shipping. But this manufacturing model also carries challenges. “I see decentralization as a method to hopefully improve accessibility, but it requires quite a lot of innovation,” Foster said. One challenge is consistency in manufacturing approaches across manufacturing sites. Foster noted that existing challenges in replicating processes within a single site become exponentially harder when multiple facilities are involved. “So you need automation, you need digitization,” he said. “You can’t do this on paper.” Panelists repeatedly spoke of the need to get away from paper batch records, which are still used despite their limitations. “It really comes down to control,” Paglia agreed. “When you move into multiple sites and multiple locations, [it becomes] difficult to standardize those procedures and policies.” Additional challenges come with the regulatory oversight of multiple locations. Foster contended that it’s impractical to expect a regulator to oversee “every single spoke just as if it was its own facility.” Rather, he said, the hub could be responsible for the quality of each spoke. “There’s some regulatory innovation required to really do this in the right way,” he said. Another critical lever in the process is quality control, said Mayo Venture Partner Audrey Greenberg. She pointed to Sarepta and Novartis as two examples of gene therapy makers that have gotten “dinged almost at the finish line” due to evolving FDA requirements for more-rigorous potency assays. Lacking global regulatory harmonization, she said, quality assurance “is one of the most difficult, if not most, expensive [considerations] from both a timeline as well as a regulatory perspective for cell and gene therapy.” Especially in the U.S., she added, “the constant regulatory changes within the FDA in terms of what’s deemed to be acceptable have also caused a lot of uncertainty.” Greenberg continued, “Having harmonization will help the patients, will help the drug manufacturers expedite that path to approval and certainly provide more transparency . . . from an investor perspective, and bringing more dollars into the space.” Past Lessons and Future Conversations The scaling problem is quickly approaching critical mass. Cell therapies in cancer are rapidly moving toward first-line approvals, with J&J and Legend Biotech’s Carvykti getting the greenlight as a second-line therapy last year and Yescarta from Gilead’s Kite Pharma expected to be approved as a first-line therapy by year’s end, according to Foster. Cell therapy developers are also now eyeing much bigger patient populations with autoimmune disorders. “All the big boys—BMS, Novartis, AstraZeneca—they all have plays in autoimmune,” Foster said. “The data looks really good.” While these advancements demand improvements in manufacturing, they simultaneously provide the means to drive that innovation. “What consistently brings down price is competition,” Foster said. “So if we’re able to bring more products to market more quickly in the same or similar indications, you’ll see prices go down.” He predicted that automation and other advances will allow manufacturers to lower the cost of goods for some cell therapies to $30,000–$35,000, cheap enough to “very reliably deliver front line therapy,” he said. “We’ve got line of sight of that.” Courtney Rice, principal at Acadia Strategy Partners, agreed that the drive into bigger patient populations will support CGT’s progress. “Whether it’s MS or lupus, or wherever this expands, I think then we start to get critical mass, and maybe that’s when the infrastructure follows,” she said. “The demand itself will back all the way up that supply chain and make things possible.” The pharmaceutical industry has been here before with the advent of monoclonal antibodies (mAbs). As with CGT now, the explosion of mAbs demanded advancements in manufacturing. “There is a direct parallel with mAbs in that about 10 or 15 years into the journey for biologics . . . the costs were too high,” Foster explained. “Then you saw single-use bioreactors come online. You saw stable cell lines come online. You saw . . . innovation that actually drove the cost down.” For CGT, he continued, “there is a new phase of innovation required and happening to have that same leap occur.” The problem now is framed by the unique challenges associated with developing truly personalized medicines with a so-called circular supply chain that starts and ends with the patient. “How do we mass produce a personalized medicine?” Foster mused. “Just sounds counterintuitive, mass-producing personalized medicine, but we can do it, and we can do it now through innovation.” Paglia agreed, joking that he felt like “the old guy in the room” as he interacted with his younger colleagues who “have grown up in the age of AI.” He said this younger generation is thinking about manufacturing in entirely different ways—not just production processes but regulatory ones. He noted that Elevate now employs someone with a PhD in regulatory science. “[It’s] just unprecedented times on the availability of technology that we have,” he said. Even at higher volumes and lower costs, though, CGT makers have to work harder to communicate the value of their products to payers, the panelists concurred. “We’ve all heard people say the first finish line is approval, but the real finish line is payers,” Rice said. “And I think that is true.” To this point, she continued, “we’ve done such an interestingly bad job at communicating value. We haven’t told a good story.” That story, of course, is that these life-saving treatments save payers money in the long run, with patients needing far less medical intervention after receiving a cell or gene therapy. “We need to do a better job of packaging [the initial high price tag] in a palatable way to payers,” Rice said. Getting the CGT sector on track to commercial viability will also require improvements in the funding environment, Greenberg added. “We need the capital markets to open up a bit more first, and hopefully simultaneously, see the science progress . . . not just therapeutic progression, but also in terms of manufacturing technologies.” Other Macro Uncertainties Facing CGT The panel discussion also touched repeatedly on the potential impact of tariffs. A new trade deal between the U.S. and the European Union will carry 15% taxes for pharmaceutical imports, while President Donald Trump continues to threaten additional pharma-specific levies, most recently of up to 200% . Paglia said it is now a daily conversation with ElevateBio’s clients, regarding the potential effects both on costs and availability of products. “The clients are always worried about supply,” Paglia explained. Foster said that tariffs will likely affect cost, and “we’ll have to deal with it in some way, through price or through cost reduction.” But on a broader scale, “the nature of supply and the global footprint of this industry sort of remains unaffected, in my view, so we just need to plan for it.” Greenberg added that the onshoring of manufacturing in the U.S. will likely also continue. “I don’t see that as cyclical.” Still, she was blunt when it came to the matter of import levies. “Tariffs are geopolitical warfare,” she said, “and it’s a way for the U.S. to gain dominance in whatever particular modality Trump has in mind at the moment.” Looking further into the future, the panelists were optimistic about the industry’s ability to grow CGT’s reach. As the science evolves to better support mass production that is reliable and reproducible, they said, the technologies will find their way to the patients who need them. “The scale problem,” Paglia said, “is starting to get solved.”

100 Deals associated with Ciltacabtagene autoleucel

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Multiple Myeloma	United States	Nanjing Legend Biotech Co., Ltd.	28 Feb 2022
Multiple Myeloma	United States	Janssen Biotech, Inc.	28 Feb 2022

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Relapse multiple myeloma	Phase 3	United States	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Japan	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Australia	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Belgium	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Denmark	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	France	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Germany	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Greece	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Israel	Janssen Research & Development LLC	12 Jun 2020
Relapse multiple myeloma	Phase 3	Italy	Janssen Research & Development LLC	12 Jun 2020

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04181827 (CARTITUDE-4, ASCO2025) Manual	Phase 3	Multiple Myeloma	419	Ciltacabtagene autoleucel (cilta-cel) (pts with extramedullary disease (EMD)	scuqvkkhew(vxbvbjovqu) = vjorvbueok biuieqlrtd (ppqlymgchl ) View more	Positive	30 May 2025
				Standard of Care (SOC) (pts with extramedullary disease (EMD)	scuqvkkhew(vxbvbjovqu) = mulonfqyni biuieqlrtd (ppqlymgchl ) View more
NCT03548207 (CARTITUDE-1, ASCO2025)	Phase 1/2	Relapse multiple myeloma hemoglobin \| platelets \| effector-to-target ratio	97	Ciltacabtagene autoleucel (cilta-cel)	tbgfiatpul(vledeggifx) = vokymcpqpw uyqykbmqnj (addvchtanl, 41.9 - NE) View more	Positive	30 May 2025
Cartitude 1 and 4 (ASCO2025)	Not Applicable	Refractory Multiple Myeloma	140	ciltacabtagene-autoleucel (Patients with CNP)	zmprbnkizn(bdrxymznpu) = pkljeufavw klotqhaglp (tdankdlldc ) View more	Positive	30 May 2025
NCT05201781 (CARTITUDE-1, EHA2025)	Phase 4	Relapse multiple myeloma hemoglobin \| platelets \| effector-to-target ratio	97	Ciltacabtagene autoleucel	rhqynejsoj(njxytvyuft) = vittwmepdm yyeqpqumwg (lgzkewwtml ) View more	Positive	22 May 2025
CARTITUDE-4 (EHA2025)	Not Applicable	Relapse multiple myeloma lenalidomide-refractory	-	Ciltacabtagene autoleucel (cilta-cel)	azbkkrchqg(msdqtizynn) = yzhnkhzjuh cmmdcgqtdu (qkrubghcqo ) View more	Positive	22 May 2025
				Standard of care (PVd or DPd)	azbkkrchqg(msdqtizynn) = jqrcqzeubf cmmdcgqtdu (qkrubghcqo ) View more
NCT04181827 (CARTITUDE-4, CTgov)	Phase 3	Relapse multiple myeloma \| Multiple Myeloma	419	daratumumab+pomalidomide+bortezomib+dexamethasone (DPd) (Arm A: Standard Therapy: PVd or DPd)	aorwkntvvb(xuzsafvwuh) = skvieilhyd zqupfyuyqn (vfsoazzxdn, gktxsqgakt - ilkefcdkgq) View more	-	20 May 2025
				Autoleucel [Cilta-cel]+JNJ-68284528 (Arm B: JNJ-68284528 (Ciltacabtagene Autoleucel [Cilta-cel]))	aorwkntvvb(xuzsafvwuh) = upprskqora zqupfyuyqn (vfsoazzxdn, tchapcbnxj - khomslecai) View more
PS2152 (EHA2025)	Not Applicable	Multiple Myeloma	235	Cilta-cel	qqiqfxxkes(zzkmvluqyk) = caxdsreqza jxprbfsmwf (ejqrfbvlvj ) View more	-	14 May 2025
				Cilta-Cel (Control group)	qqiqfxxkes(zzkmvluqyk) = oythnsancj jxprbfsmwf (ejqrfbvlvj ) View more
PB2891 (EHA2025)	Phase 3	BCMA	-	Belantamab mafodotin plus bortezomib/dexamethasone	oqlldldzuz(lsaumvznnt): HR = 0.67 (95% CI, 0.26 - 1.75)	Positive	14 May 2025
				Ciltacabtagene autoleucel
PB3088 (EHA2025)	Not Applicable	Relapse multiple myeloma	-	BCMA CAR-T (EMD-negative patients)	vfxhndeeqd(jrekiwpvet) = ffiofdtcar qpekmvkywj (ysqlbelghq, 0.42 - 0.59) View more	-	14 May 2025
				BCMA CAR-T (EMD-positive patients)	vfxhndeeqd(jrekiwpvet) = hkccbbaqzk qpekmvkywj (ysqlbelghq, 0.32 - 0.51) View more
CARTITUDE-1 (EHA2025)	Not Applicable	Refractory Multiple Myeloma	105	Ciltacabtagene autoleucel	btqkyfyros(kattuwebsh) = Infections occurred in 49% of patients and were severe in 32%. Earlier infections in the first 30 days were equally bacterial (42%) and viral (42%). Later infections between days 31-100, and after day 100 were mostly viral (59% and 60%), with only 32% and 12% being grade ≥3 at each time period. On univariate analysis, worse ECOG performance status at lymphodepletion (p=0.012), higher maximum grade of CRS (p=0.036), steroid and anakinra use (p=0.042 and p=0.024), and lower IgA levels at day 90 (p=0.014) were associated with severe infections. At the end of follow-up, 16 patients had expired. Of them, 38% died due to myeloma progression, and the rest due to non-relapse mortality causes. The most common cause of non-relapse mortality was infection (31% of all deaths) qnrxosdlxf (cekfpgqmfr )	-	14 May 2025

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Ciltacabtagene autoleucel

Overview

Basic Info

Structure/Sequence

Related

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation