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In the U.S., the FDA is slated to decide on GSK's Blenrep in second-line multiple myeloma by July 23. GSK has secured the first regulatory approval for Blenrep since the antibody-drug conjugate’s global market withdrawal in 2022.The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has become the first to sign off on Blenrep as part of two different combinations for treating multiple myeloma patients who have received at least one prior therapy, GSK said Thursday.The approval allows the BCMA-targeted therapy to be used with either Takeda’s Velcade (bortezomib) or Bristol Myers Squibb’s Pomalyst (pomalidomide), plus dexamethasone in second-line myeloma treatment.This world-first approval marks a milestone in GSK’s plan for Blenrep to reach peak annual sales of more than 3 billion pounds sterling (about $4 billion). Blenrep’s potential global reentry also gave GSK confidence to boost its 2031 sales target to more than 40 billion pounds despite uncertainties around the company’s flagship vaccines business. A regulatory approval further redeems Blenrep’s reputation following its previous market withdrawal triggered by a phase 3 monotherapy flop in third-line multiple myeloma.“As the only BCMA-targeted ADC therapy, Blenrep has the potential, supported by robust phase III data, to extend survival and remission versus standard of care and redefine treatment at or after first relapse,” Hesham Abdullah, M.D., GSK’s global head of oncology R&D, said in a statement Thursday.Despite its history as a monotherapy, Blenrep proved its worth in two combo trials.In DREAMM-7, Blenrep beat Johnson & Johnson’s Darzalex in their respective pairings with Velcade and dexamethasone (Vd). Blenrep-Vd slashed the risk of progression or death by 59% versus Darzalex-Vd. The GSK regimen also helped patients live longer, lowering the risk of death by 42% versus the Darzalex-based comparator.In DREAMM-8, Blenrep’s combination with Pomalyst and dexamethasone (Pd) has shown a 48% reduction in the risk of progression or death versus Velcade-Pd.Besides the U.K., Blenrep combinations are currently under review in 14 territories, according to GSK. In the U.S., the FDA is slated to deliver a verdict by July 23 this year. Blenrep will need to compete with J&J and Legend Biotech’s powerful CAR-T therapy Carvykti on the global market—although not in the U.K. just yet. Back in 2023, J&J shelved Caryvkti’s rollout in the U.K. as the company struggled to churn out enough supply to meet global demand. As an off-the-shelf therapy, Blenrep does boast a convenience edge over CAR-T therapy, which requires a complex production process using patients’ own T cells.“As patients with multiple myeloma increasingly receive combination therapies at diagnosis, treatment options available in the community setting that use different mechanisms like Blenrep are crucial to extending remission and ultimately survival,” Joseph Mikhael, M.D., from City of Hope and chief medical officer of the International Myeloma Foundation, said in a statement Thursday.

Genetically engineered chimeric antigen receptor immune cell with implanted mrna gene strand - 3d illustration Christoph Burgstedt/Getty Images At the GenScript Biotech Global Forum 2025, industry leaders celebrated CAR T cell therapy achievements while discussing ongoing challenges in manufacturing, distribution, treatment center capacity, and global payment structures for cell and gene therapies. FDA approval for chimeric antigen receptor (CAR) T cell therapy occurred 30 years after the initial research began. Prior to the approval, the first successful treatment using CAR T cells was in 2010 for a retired Marine with chronic leukemia. Two years later, Emily Whitehead became the first child treated and cured. Key industry leaders came together to celebrate these and other CAR T cell achievements as well as discuss the therapy’s future at the 2025 GenScript Biotech Global Forum. As one of the keynote speakers, Carl June, MD , professor and director of the Center for Cellular Immunotherapies at the University of Pennsylvania’s Perelman School of Medicine highlighted, “Since the first approval, approximately 50,000 patients have been treated with CAR T cells worldwide, primarily for blood cancers.” June stated that 2024 was a breakthrough year for cell and gene therapies (CGTs). He emphasized that CAR T cell therapies have made remarkable breakthroughs in glioblastoma treatments and early-stage treatments for autoimmune diseases. Alan Bash , president of Legend Biotech, seconded this opinion in a panel discussion following June’s remarks. Carl June, MD delivers one of the keynote speeches at the GenScript Biotech Global Forum 2025 “The products are delivering, and the products are demonstrating the innovation for patients,” Bash said. CGT Therapy Hurdles Discussions throughout the event focused on manufacturing, distribution and payer challenges. However, before diving into those insights, Michael Vreeland , US site head at ProBio, stressed an important element that is frequently left out, which is talent. “One of the things that’s often overlooked is the training piece, right?” Vreeland said. “How do we ensure consistent and repeatable training and execution?” As this article dives into the other challenges, it is worth remembering that talent and experience are two pieces that will influence how the CGT space overcomes current and future hurdles. Manufacturing and Scalability Bash explained that the innovation continues “despite market and technological challenges.” However, he emphasized that the challenges are significant, noting that while regulatory hurdles have fallen, manufacturing and distribution still remain significant obstacles for the space. Rey Mali , chief business officer at Accellix, agreed that improvement is greatly needed in this area to influence both cost and access of CGTs. “Now, it’s about scale,” she said. “Now, it’s about distribution. That part really needs to improve.” It is not a simple task, as Jonathan Esensten , senior advisor at Multiply Labs and director of the Advanced Biotherapy Center at Sheba Medical Center, pointed out. Panelists discuss the current CGT investment landscape at the GenScript Biotech Forum 2025. “Current manufacturing often relies on ‘classified clean rooms,’ where people with their hands are actually manufacturing these products,” he said. While automation and standardization will improve some tasks, Hari Pujar, operating partner at Flagship Pioneering, pointed out that there are limitations due to patient variability. “No matter how much you automate, how much you standardize, you end up in the ex vivo situation with one variable, which is the patient cell,” he said. Treatment Center Limitations Along these lines, Bash highlighted an increasingly limited factor, noting, “Challenges that we are experiencing are not the capacity constraints for internal manufacturing, but the capacity constraints of the treatment centers.” Investors agreed that the ability to get outside of major academic centers and use CGTs in less intense, medically intensive settings is a major focus for investors as they evaluate potential investment opportunities. Supply Chain Supply chain issues remain constant in the space. One of the main issues in the sector is that for many of the critical materials, reagents and equipment, there is a single supplier. This is especially concerning given these materials can be delayed. Additionally, starting material quality inconsistency is a significant challenge. Mali emphasized, “Your drug is only as good as what you start off with...The patient has been through many different therapies, so what they have left can vary drastically.” Market Challenges Panelists discuss the future of CGTs at the GenScript Biotech Global Forum 2025. The current economic market is an understandable challenge for the CGT space as it is for the entire industry. However, Massimiliano Paganelli , chief executive officer and co-founder at Morphocell Technologies, stated that the environment for CGT is worse off due to the initial excitement over cell and gene therapies. He noted that acquisitions of preclinical cell therapy companies for valuations that touched the billion-dollar mark have compounded the market challenges for CGTs. Deeper, high-value valuations have lended to a slowdown of investment for the last five years. However, Paganelli stressed that because of a better understanding of CGTs, things are getting better from an investment perspective. Global Payer Challenges Amongst the existing challenges for the CGT space, the payer challenge is another hurdle some panelists, as investors, are grappling with currently. The panel highlighted that while the payment structure in the U.S. market is challenging, it is even more challenging abroad, with a focus on moving beyond Europe, the Middle East and Africa to the Asia-Pacific region (APAC). That being said, Louis Breton , chief executive officer and co-founder at Rampart Bioscience, specified, “Patient-reported outcomes are becoming more and more critical to the progression of the way that the payers think about CGTs.” Working with governments is only one part of the puzzle, the second being that research and development costs have to be lower. Panelists noted that CGT developers such as Legend Biotech, which can stand alone from a commercial standpoint, are of keen interest for investors. Even with all of the challenges facing CGTs, venture capital funding remains strong, focusing on fewer but larger deals. In 2024, there were 18 initial public offerings. This is a significant swing upward from the six in 2023. As technology and science continue to blend and evolve, unsurprisingly, all panelists agreed that this evolution is going to make a big impact. Automation and the use of robotic systems were highlighted as being the most impactful. Panelists also highlighted in vivo approaches, which offer significant mitigation for the challenges the sector faces. Pujar poised the question , “What if we could create the cell inside the body…in which case we kind of default all of the challenges that have been mentioned?” As a Field, We Need to Continue to Innovate. Guowei Fang, President, Research and Development at Legend Biotech Global Innovation and Collaboration From a global perspective, a conversation regarding innovation is not complete without mentioning APAC. Caroline Stout , partner at EcoR1 Capital, emphasized, “The pace of innovation coming out of China and Japan is formidable.” Bin Li , founder and chief investment officer at Lake Bleu Capital, agreed, as China is becoming a significant source of innovation. “It’s not first in class, but it’s best in class [for them],” Li said. Cross-border collaboration and deals are of significant value despite geopolitical tensions. Josh Resnick , senior managing director at RA Capital Management, expressed hope that politics will not interfere with innovation. “We view the world as fundamentally an open source to solve really hard problems,” Resnick said. This is especially concerning for the CGTs space, as China has been the main source for many biospecifics and T cell engagers. Conclusions The CGT space is still new. The panelists agreed that much work needs to be done to educate investors and regulators. They emphasized the importance of communicating the value of innovation, highlighting that the industry can make sure to transmit that story to the general population. To watch the panel discussions, read summaries, and view more content, please go to the GenScript Biotech Global Forum 2025. This article was written in collaboration with GenScript Biotech. The insights team analyzes and comments on industry trends and creates thought leadership content for BioSpace and clients. The head of insights, Lori Ellis, can be contacted via lori.ellis@biospace.com . Follow her on LinkedIn .

As a BCMA-targeting CAR T-cell therapy, CARVYKTI addresses a high unmet need in relapsed or refractory multiple myeloma patients. With promising clinical efficacy, durable responses, and growing adoption in advanced treatment lines, CARVYKTI is poised for substantial revenue growth. LAS VEGAS, March 24, 2025 /PRNewswire/ -- DelveInsight's " CARVYKTI Market Size, Forecast, and Market Insight Report" highlights the details around CARVYKTI, a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient's T cells with a transgene encoding a CAR that identifies and eliminates cells that express BCMA. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CARVYKTI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Janssen's CARVYKTI (ciltacabtagene autoleucel) Overview CARVYKTI is an autologous T-cell immunotherapy that targets B-cell maturation antigen (BCMA). It works by genetically modifying a patient's T cells with a transgene that encodes a chimeric antigen receptor (CAR). This CAR helps the T cells recognize and destroy cells expressing BCMA, which is primarily found in malignant multiple myeloma B-lineage cells, late-stage B cells, and plasma cells. The CARVYKTI CAR protein includes two single-domain antibodies designed to bind strongly to human BCMA, enhancing T-cell activation, proliferation, and the destruction of target cells upon binding. Currently, CAR-T cell therapies are only available to UK patients through clinical trials. Janssen recently decided not to proceed with seeking approval for cilta-cel (CARVYKTI) for myeloma patients in the UK through the National Institute for Health and Care Excellence (NICE). This decision means cilta-cel will not be available on the NHS for now, although it remains accessible through clinical trials. Janssen's decision does not impact ongoing trials. The drug is currently being evaluated in Phase III trials: CARTITUDE-6 for frontline multiple myeloma transplant eligible vs ASCT and CARTITUDE-5 for frontline multiple myeloma TNI. In 2024, CARVYKTI generated sales of USD 963 million across the world. CARVYKTI Dosage and Administration CARVYKTI is administered as a single infusion containing a suspension of CAR-positive, viable T cells in one infusion bag. The recommended dosage ranges from 0.5 to 1.0 × 10⁶ CAR-positive viable T cells per kilogram of body weight, with a maximum limit of 1 × 10⁸ CAR-positive viable T cells per infusion. Learn more about CARVYKTI projected market size for multiple myeloma @ CARVYKTI Market Potential CAR-T cell immunotherapy is emerging as a revolutionary treatment for multiple myeloma, offering new hope to patients who have exhausted conventional treatment options. Currently, only two CAR-T cell therapies are approved for multiple myeloma: ABECMA (idecabtagene vicleucel) from Bristol-Myers Squibb and CARVYKTI (ciltacabtagene autoleucel) from Johnson & Johnson Innovative Medicine. Their approval highlights the potential of CAR-T therapy to transform multiple myeloma treatment. A key advantage of CAR-T therapies is their "one-and-done" nature, requiring a single administration, unlike bispecific antibodies such as TECVAYLI and TALVEY, which need ongoing dosing. Present CAR-T therapies use a patient's own T cells (autologous), but research is underway to develop allogeneic CAR-T cells derived from healthy donors. Allogeneic therapies could offer off-the-shelf solutions, enhancing accessibility and lowering costs. The approval of CAR-T therapies has created new opportunities for companies focused on advanced-stage multiple myeloma (fourth line and beyond). Several companies are progressing with their CAR-T candidates at different stages of development. The CAR-T cell therapy market for multiple myeloma is expected to grow significantly between 2024 and 2034, driven by its high efficacy and potential for expanded use. Collaboration between pharmaceutical companies and research institutions, along with ongoing innovation, is expected to accelerate market growth. However, challenges such as manufacturing complexity and pricing will need to be addressed to unlock the full potential of CAR-T therapies. Discover more about the CAR T-cell therapy for multiple myeloma market in detail @ CAR T-cell Therapy for Multiple Myeloma Market Report Emerging Competitors of CARVYKTI Some of the CAR-Ts in the multiple myeloma pipeline that will give fierce competition to CARVYKTI include Anito-cel (Arcellx), PHE885 (Novartis), BMS-986393 (Bristol-Myers Squibb), Zevorcabtagene Autoleucel (CARsgen Therapeutics), and GLPG5301 (Galapagos), among others In December 2024, Arcellx announced encouraging new data from its Phase II pivotal iMMagine-1 trial for anito-cel in multiple myeloma. The results were presented during an oral session at the 66th American Society of Hematology (ASH) Annual Meeting on Monday, December 9, 2024, at 5:30 p.m. PT. Also at ASH 2024, BMS shared the first overall survival (OS) and progression-free survival (PFS) results for arlo-cel in an oral presentation. The Phase I trial involved patients with relapsed or refractory multiple myeloma who had previously undergone at least three treatments, including a proteasome inhibitor, an immunomodulatory drug, and anti-CD38 therapy. Among 79 patients evaluable for efficacy, with a median follow-up of 16.1 months (range: 2.8–25.2), arlo-cel showed an overall response rate (ORR) of 87%, indicating durable responses. Minimal residual disease (MRD) was assessed as an exploratory measure, with 57% (48 of 84) of MRD-evaluable patients included. Of these, 46% (22 of 48) were MRD-negative and achieved a complete response (CR) or stringent CR (sCR). Across all treated patients, 27% (23 of 84) were MRD-negative and achieved a CR. Median PFS was 18.3 months (95% CI: 11.8–21.9), while the median OS had not yet been reached. To know more about the number of competing drugs in development, visit @ CARVYKTI Market Positioning Compared to Other Drugs Key Milestones of CARVYKTI In April 2024, Johnson & Johnson announced that the US FDA approved CARVYKTI (ciltacabtagene autoleucel; cilta-cel) for the treatment of adult patients with RRMM who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. In March 2024, Johnson & Johnson announced that the US FDA Oncologic Drugs Advisory Committee (ODAC) recommends CARVYKTI for the treatment of adult patients with RRMM who have received at least one prior line of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) and who are refractory to lenalidomide. In February 2024, Johnson & Johnson announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA recommended the approval of a Type II variation for CARVYKTI for the earlier treatment of RRMM. In September 2022, Legend Biotech announced that Japan's Ministry of Health, Labour and Welfare (MHLW) had approved CARVYKTI (ciltacabtagene autoleucel) for the treatment of adults with relapsed or refractory multiple myeloma, limited to cases meeting both of the following conditions including patients having no history of CAR-positive T-cell infusion therapy targeting BCMA and patients who have received three or more lines of therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody, and in whom multiple myeloma has not responded to or has relapsed following the most recent therapy. In May 2022, Janssen announced that the European Commission (EC) granted conditional marketing authorization for CARVYKTI for the treatment of adults with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy. In February 2022, Janssen announced that the US FDA had approved CARVYKTI (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. In August 2020, Legend Biotech announced that the China Center for Drug Evaluation, National Medical Products Administration (CDE, NMPA) had recommended BTD for ciltacabtagene autoleucel (cilta-cel; LCAR-B38M CAR-T cells) for the treatment of adults with relapsed or refractory multiple myeloma. In December 2019, Janssen announced receipt of a BTD from the US FDA for cilta-cel. In April 2019, the EMA granted a PRIME (PRIority MEdicines) designation for the company's investigational BCMA CAR-T therapy. In February 2019, the US FDA granted ODD for cilta-cel (JNJ-4528), and in February 2020, the European Commission also granted orphan designation for this drug. In December 2017, Janssen entered a worldwide collaboration and license agreement with Legend Biotech to jointly develop and commercialize LCAR-B38M in multiple myeloma Discover how CARVYKTI is shaping the multiple myeloma treatment landscape @ CARVYKTI CAR-T CARVYKTI Market Dynamics The market for CARVYKTI is driven by the increasing prevalence of multiple myeloma and the growing demand for innovative therapies that can address the limitations of existing treatments, such as proteasome inhibitors and immunomodulatory drugs. The competitive landscape includes other BCMA-targeting therapies, such as Bristol Myers Squibb's ABECMA (idecabtagene vicleucel), which was the first BCMA-directed CAR-T therapy approved for multiple myeloma. CARVYKTI's strong clinical efficacy, coupled with its potential for long-term remission, gives it a competitive edge, but challenges related to manufacturing scalability, logistical complexities, and high treatment costs could impact its market penetration and patient accessibility. Strategic partnerships and ongoing clinical development will play a key role in expanding CARVYKTI's market reach. Janssen and Legend Biotech are actively working to improve manufacturing processes to reduce vein-to-vein time and address supply chain bottlenecks. Furthermore, ongoing trials, such as CARTITUDE-4, are exploring CARVYKTI's use in earlier lines of therapy, which could significantly expand its patient base and market potential. If successful in earlier settings, CARVYKTI could shift the treatment paradigm for multiple myeloma and increase adoption among physicians and healthcare providers. Despite its promising clinical profile, CARVYKTI faces challenges related to reimbursement and healthcare infrastructure. The high cost of CAR-T therapies often limits patient access, particularly in markets with strict reimbursement policies. Moreover, the complex nature of CAR-T administration, which requires specialized centers and trained personnel, adds to the barriers. However, continued clinical success, regulatory support, and improvements in manufacturing and delivery infrastructure could strengthen CARVYKTI's market position and establish it as a cornerstone therapy in the multiple myeloma treatment landscape. Dive deeper to get more insight into CARVYKTI's strengths & weaknesses relative to competitors @ CARVYKTI Market Drug Report Table of Contents Related Reports Relapsing Refractory Multiple Myeloma Market Relapsing Refractory Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key RRMM companies including AbbVie, Genentech, Amgen, Onyx Therapeutics Inc., Bristol-Myers Squibb, MedImmune LLC, Novartis Pharmaceuticals, Incyte Corporation, Takeda, among others. Relapsing Refractory Multiple Myeloma Pipeline Relapsing Refractory Multiple Myeloma Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMM companies, including Bristol-Myers Squibb, I-MAB Biopharma, Pfizer, Arcellx, Gilead Sciences, Novartis, Array Biopharma, Hrain Biotechnology Co., Ltd., Cartesian Therapeutics, Xencor, Takeda, Sorrento Therapeutics, Heidelberg Pharma AG, Ichnos Sciences, Allogene Therapeutics, Harpoon Therapeutics, Cellectis, Poseida Therapeutics, Regeneron Pharmaceuticals, ONK Therapeutics, TeneoOne, iTeos Therapeutics, Oricell Therapeutics, Anaveon AG, Luminary Therapeutics, Seagen Inc., Trillium Therapeutics Inc., Virtuoso BINco, Inc., Seagen Inc., Trillium Therapeutics Inc., among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. Multiple Myeloma Pipeline Multiple Myeloma Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key multiple myeloma companies, including CASI Pharmaceuticals, Carsgen Therapeutics, Cartesian Therapeutics, Gracell Biotechnology Shanghai Co., Ltd., Sorrento Therapeutics, TeneoOne, Karyopharma Therapeutics, Arcellx, Poseida Therapeutics, Ichnos Sciences, Nerviano Medical Sciences, Bristol Myers Squib, Ascentage Pharma, Ionis Pharmaceuticals, Chongqing Precision Biotech Co., Ltd., CRISPR Therapeutics, AstraZeneca, IGM Biosciences, Novartis, GlaxoSmithKline, Innovent Biologics, Keymed Biociences, Starton Therapeutics, Takeda, Fate Therapeutics, Gilead Sciences, Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd., Janssen Pharmaceutical, Nanjing IASO Biotechnology Co., Ltd., GPCR Therapeutics, Chimerix, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED