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Medicilon Passes FDA Inspection, Reinforcing Global R&D Excellence

BOSTON, May 29, 2025 /PRNewswire/ — On May 20, Medicilon Preclinical Research (Shanghai) LLC (“Medicilon”) announces that it has successfully passed an on-site inspection by the FDA. The company receives an official Establishment Inspection Report (EIR) reaffirming its position as a trusted leader in preclinical R&D services .

A Milestone of Compliance and Capability 

The FDA inspection, conducted with meticulous detail, evaluated Medicilon’s organizational structure, personnel qualifications, SOPs, facilities integrity, documentation processes, experimental operations, quality assurance systems, and computerized infrastructures. Inspectors also reviewed several research projects submitted to the FDA.

FDA officials praised Medicilon for its scientific discipline and operational excellence, expressing anticipation for continued high-quality data submissions. This endorsement validated Medicilon’s enduring commitment to its guiding philosophy of “Quality First.”

Strategic Validation of Global R&D Impact

This is Medicilon’s first on-site FDA inspection in seven years, and the successful outcome significantly enhances its regulatory credibility. The milestone is a strong endorsement of its strategic vision, rooted in being “Innovation Driven, Quality Focused.”

By the end of 2024, Medicilon has helped 520 INDs enter clinical trials, through the approval of China’s NMPA, the US FDA, the EU EMA, the Australian TGA and the South Korean’s KFDA.  These INDs include innovative drugs in various fields, such as 34 antibodies, 28 ADC drugs, 8 GLP-1 drugs, 6 PROTAC drugs, 3 botanical drugs, etc. Looking ahead, Medicilon will remain committed to increasing investment in technological innovation and R&D, and is committed to launching more innovative and more high-quality technology and service solutions, embracing the broad opportunities and challenges of the global pharmaceutical market, and promoting the development of the global pharmaceutical industry!

About Medicilon

From its inception in 2004, Shanghai Medicilon Inc. (stock code: 688202.SH) has been committed to providing comprehensive R&D services to pharmaceutical companies, research institutions, and any organizations working in the preclinical space. By the end of 2024, Medicilon has provided drug development services to over 2,000 clients worldwide, and has been involved in the research and development of 520 new drugs and generic drug projects that have been approved for clinical trials with IND applications.

Grit Biotechnology Presents Clinical Breakthroughs at 2025 ASCO Annual Meeting

SHANGHAI, May 29, 2025 /PRNewswire/ — Grit Biotechnology Co., Ltd. (“Grit Bio“), a clinical-stage biotech pioneering novel immunotherapies announced today that three abstracts featuring new clinical data from tumor-infiltrating lymphocytes (TIL) therapies will be presented at the American Society of Clinical Oncology (ASCO) 42nd annual meeting, which is taking place in Chicago, IL from May 30th to June 3rd.

Grit Bio will provide the clinical updates of its leading TIL programs, including GT101 – an unmodified TIL therapy in treating recurrent and metastatic cervical cancer, GT201 – an armored TIL product expressing membrane bond IL-15 complex in treating various solid tumors, and GT300 – a CRISPR/Cas-edited dual KO TIL product in treating advanced solid tumors. The details of Grit Bio’s poster presentations are included below. The abstracts were published via the ASCO website on May 22, 2025.

  1. Title: GT101 autologous TIL therapy in patients with recurrent and metastatic cervical cancer: A phase 1 study.
    Presentation Type: Poster
    Time: 9:00 AM-12:00 PM CT, Sunday, June 1st
    Poster # 431
    Abstract #: 5533
  2. Title: Assessing the Safety and Efficacy of GT201: A First-in-Class Autologous Tumor-Infiltrating Lymphocyte Monotherapy in Advanced Solid Tumors
    Presentation Type: Poster
    Time: 9:00 AM-12:00 PM CT, Monday, June 2nd
    Poster #: 446
    Abstract #: 6038
  3. Title: Evaluating the Safety and Efficacy of CRISPR/Cas-Modified Tumor Infiltrating Lymphocytes (GT300) as Monotherapy in Advanced Solid Tumors
    Presentation Type: Poster
    Time: 9:00 AM-12:00 PM CT, Sunday, June 1st
    Poster #: 511
    Abstract #: 5613

“We have harnessed cutting-edge technologies, including advanced gene editing tools, to enhance the potency and persistence of our TIL products, enabling the development of multiple differentiated TIL pipelines.” said Dr. Yarong Liu, Founder and Chief Executive Officer of Grit Biotechnology. “These therapies have demonstrated promising clinical potential across a wide range of indications. Looking forward, we remain committed to accelerating their clinical advancement and actively seeking global collaborations to bring these transformative therapies to patients worldwide.”

About Grit Bio
GRIT Bio was founded in 2019 as an innovative biopharmaceutical company, focused on immune cell treatments for oncology and characterized by a R&D pipeline in TIL therapies. GRIT Bio has completed multiple rounds of equity financing and is backed by renowned venture capital funds. GT101, a proprietary injectable developed by GRIT Bio, is the first TIL therapy that entered registrational clinical trial. GT 101 is currently in pivotal Phase II clinical study. GRIT Bio’s GT201 injectable, the first TIL therapy with membrane-bound IL-15 complex, has cleared IND in both US and China. Core R&D platforms of the company include StemTexp® stemness TIL expansion platform, StaViral® stably virus transfected cell lines, ImmuT Finder® immune modulator target discovery platform, and KOReTIL® high-efficiency gene knock-out system. Based on the platforms, GRIT Bio generated a series of next-generation gene-edited TIL therapies. With internationally advanced technology reserve and industry resources, GRIT Bio aims to develop breakthrough therapies for solid tumors and bring new hope to cancer patients. For further information, please visit: www.grit-bio.com

Forward-Looking Statements
This press release contains forward-looking statements about Grit Biotechnology based on management’s current expectations, which are subject to known and unknown uncertainties and risks. Words such as “anticipated,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” “may,” “will,” and variations of these words or similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding our expectations for our technology platforms, gene therapy candidates in development, collaborations, and plans for commercialization. Our actual results could differ materially from those discussed due to a number of factors, including, but not limited to, the risk inherent in developing pharmaceutical product candidates, conducting successful clinical trials, and obtaining regulatory approvals. Except as required by law, Grit Biotechnology assumes no obligation to update any forward-looking statements contained herein as a result of new information, future events, or otherwise.

Investor Contact: +86-21- 50828029, BD@grit-bio.com; Media Contact: +86-21- 50828029, PR@grit-bio.com

O-RAN ALLIANCE Global PlugFest Spring 2025 Demonstrated Steady Evolution of the O-RAN Ecosystem

  • O-RAN ALLIANCE Global PlugFests focus on testing and integration to accelerate the development of O-RAN based products and solutions
  • The O-RAN Global PlugFest Spring 2025 advanced O-RAN technology and procedures for testing, deployment and operation of carrier-grade networks
  • The PlugFest was co-hosted by 22 operators, OTICs, and academic and research institutions in 19 labs around the globe
  • A total of 69 companies and institutions participated, with some involved in multiple venues

BONN, Germany, May 29, 2025 /PRNewswire/ — The O-RAN ALLIANCE (O-RAN) today announced successful completion of the O-RAN ALLIANCE Global PlugFest Spring 2025.

O-RAN PlugFests are designed to accelerate the development of products and solutions based on O-RAN specifications by offering structured testing and integration opportunities in a neutral and collaborative environment.

At each O-RAN PlugFest, participants advance O-RAN-based technology and procedures for testing, deployment and operation of carrier-grade open, intelligent and virtualized Radio Access Networks (RAN).

Participants at the O-RAN Global PlugFest Spring 2025 demonstrated progress in diverse areas, including:

  • Automated, cloud-native RAN deployment & testing
  • AI-powered test automation with intelligent report analysis and automated test generation, enabled by Large Language Models (LLM)
  • Intelligent RAN energy savings of 25 to 30%, enabled by rApps on Non-Real-Time RAN Intelligent Controller (Non-RT RIC)
  • Intelligent network efficiency and service assurance, enabled by cell on-off switching rApp and xApp combined with traffic steering xApp
  • Integration of open-source O-RAN components from multiple open-source communities in O-RAN networks
  • Validation of transport network behavior under congestion and impairments for different deployment options
  • E2E network performance testing in various scenarios
  • Efficient cooperation enabling consistent and repeatable testing across labs and regions

O-RAN Global PlugFest Spring 2025 was hosted by 22 operators, Open Testing and Integration Centers (OTICs) and vendor-independent institutions. It took place in 8 venues and 19 labs around the world. It was conducted from February to May 2025 with a total of 69 participants, some of which participated at more than one venue.

“O-RAN Global PlugFests play a vital role in accelerating innovation built on O-RAN ALLIANCE specifications,” said Chih-Lin I, Co-chair of O-RAN ALLIANCE’s Technical Steering Committee and China Mobile Chief Scientist, Wireless Technologies, China Mobile Research Institute. “Each PlugFest showcases the steady evolution of the O-RAN ecosystem, highlighting progress across a wide range of technologies, solutions, and services that enhance RAN deployment and operations. It’s particularly encouraging to see that the progress of O-RAN intelligence and automation capabilities has been gaining momentum. A big thank you to all hosts, labs and participants for their great efforts.”

Participants in O-RAN ALLIANCE Global PlugFest Spring 2025

O-RAN ALLIANCE appreciates high interest from the community in testing and integration at the O-RAN Global PlugFest Spring 2025 and would like to recognize hosts, labs and participants, including:

Acentury, Airspan Networks, AT&T, Auray Technology, Battelle, CableLabs, Calnex Solutions, Capgemini Engineering, Deutsche Telekom, Digital Catapult, EANTC, Ericsson, EURECOM, Fujitsu, highstreet technologies, i14y Lab, Iowa State University, Juniper Networks, Keysight Technologies, Lenovo, LIONS Technology, LITE-ON Technology, Metanoia Communications, MTI, Nokia, North Carolina State University, Northeastern University, Nvidia, Pegatron, Quanta Cloud Technology (QCT), Rakuten Mobile, Rakuten Symphony, Rimedo Labs, Rohde & Schwarz, Rutgers University, Sera Network, Singapore University of Technology and Design (SUTD), Software Radio Systems, Supermicro, Telefonica, University of Utah, University of New Hampshire InterOperability Laboratory, University of Tokyo, Verizon, VIAVI Solutions, Virginia Polytechnical Institute and State University, Vodafone, VVDN Technologies, Wind River Systems, Xelera Technologies, YRP R&D Promotion Committee.

About O-RAN ALLIANCE
The O-RAN ALLIANCE is a world-wide community of mobile operators, vendors, and research & academic institutions operating in the Radio Access Network (RAN) industry. As the RAN is an essential part of any mobile network, the O-RAN ALLIANCE’s mission is to re-shape the industry towards more intelligent, open, virtualized and fully interoperable mobile networks. The new O-RAN specifications enable a more competitive and vibrant RAN supplier ecosystem with faster innovation to improve user experience. O-RAN based mobile networks at the same time improve the efficiency of RAN deployments as well as operations by mobile operators. To achieve this, the O-RAN ALLIANCE publishes new RAN specifications, releases open software for the RAN, and supports its members in integration and testing of their implementations.
For more information, please visit www.o-ran.org.

O-RAN ALLIANCE PlugFest
O-RAN ALLIANCE PlugFest

 

ETFs Unlock Growth in China’s Booming Tech Landscape

GUANGZHOU, China, May 29, 2025 /PRNewswire/ — In 2025, A-share tech-focused ETFs have continued to attract significant investor interests – the top five industry/thematic ETFs by net inflows, as of May 21, were all technology-related, collectively drawing in US$ 7.87 billion, including the E Fund CSI Artificial Intelligence ETF (159819), which saw a net inflow of US$ 1.17 billion. Meanwhile, leading asset managers in China are observed to actively positioning themselves in tech-focused ETFs, such as AI ETFs, robotics ETFs, and aviation ETFs. Notably, E Fund Management (“E Fund”), the largest mutual fund manager in China, has highlighted six cutting-edge sectors, spanning artificial intelligence, robotics & smart devices, computing technology, healthcare technology, energy technology and space technology and has established a complete range of ETF products to capture growth opportunities.

Six Cutting-edge Sectors Shaping the Future
Six Cutting-edge Sectors Shaping the Future

  1. Artificial Intelligence
    AI serves as the foundational infrastructure for emerging technologies. The E Fund CSI Artificial Intelligence ETF (159819) manages US$ 2.23 billion in assets, ranking first among its peers. Its underlying index, the CSI Artificial Intelligence Index, tracks 50 leading AI firms and emphasizes core technologies, with semiconductor companies, software developers, and computer equipment providers accounting for nearly 70% of its holdings.
  2. Robotics & Smart Devices
    Breakthroughs in humanoid robotics, such as their performances in world’s first humanoid robot half marathon and the RoboCup this year, have spurred market enthusiasm. The E Fund CNI Robot Industry ETF (159530) allocates over 50% of its weighting to humanoid robots, the highest proportion among all robotics-focused ETFs, after changes on index methodology taking effective on April 10.
  3. Computing Technology
    While robust computational power is critical for AI development, cloud computing emerged as a mainstream solution. The E Fund CSI Cloud Computing & Big Data ETF (516510), the largest ETF in its category tracking the same index, holds US$ 489 million in assets. It covers computing equipment, data centers, and cloud services, aligning with China’s push for digital infrastructure.
  4. Healthcare Technology
    Gene-editing technologies are poised to revolutionize treatments for genetic disorders and cancer. The E Fund CSI Biotechnology ETF (159837) invests in companies specializing in gene diagnostics, biopharmaceuticals, and advanced biomedical research, positioning it at the forefront of healthcare innovation.
  5. Energy Technology
    China leads global renewable energy innovation, particularly in photovoltaic installations, nuclear power, and solid-state battery storage. The E Fund CSI New Energy ETF (516090) provides exposure to lithium batteries, solar, wind, hydro, and nuclear energy, supporting the transition to clean power.
  6. Space Technology
    The successful launch of “Three-Body Computing Constellation” on May 15, which enables real-time data processing in orbit, marked a major step in China’s space technology. E Fund is set to launch a new ETF linked to the CNI General Aviation Index, tracking companies involved in aerospace materials, aircraft manufacturing, and flight operations.

Among them, the E Fund CSI Artificial Intelligence ETF (159819), the E Fund CSI Cloud Computing & Big Data ETF (516510), the E Fund CSI Biotechnology ETF (159837), and the E Fund CSI New Energy ETF (516090) are included in ETF Connect, empowering global investors to capitalize on China’s technology trends.

About E Fund

Established in 2001, E Fund Management Co., Ltd. (“E Fund”) is a leading comprehensive mutual fund manager in China with over RMB 3.5 trillion (USD 497 billion) under management.* It offers investment solutions to onshore and offshore clients, helping clients achieve long-term sustainable investment performances. E Fund’s clients include both individuals and institutions, ranging from central banks, sovereign wealth funds, social security funds, pension funds, insurance and reinsurance companies, to corporates and banks. Long-term oriented, it has been focusing on the investment management business since inception and believes in the power of in-depth research and time in investing. It is a pioneer and leading practitioner in responsible investments in China and is widely recognized as one of the most trusted and outstanding Chinese asset managers.

*Source: E Fund. AuM includes subsidiaries. Data as of March 31, 2025. FX rate is sourced from PBoC.

China, ASEAN and GCC Advance Cooperative Frameworks to Strengthen Regional Supply Chain Resilience

KUALA LUMPUR, Malaysia, May 29, 2025 /PRNewswire/ — China, the Association of Southeast Asian Nations (ASEAN) and the Gulf Cooperation Council (GCC) are ramping up efforts to enhance regional economic cooperation and stabilize industrial and supply chains. From the China-ASEAN Economic Forum to the ASEAN-China-GCC Economic Forum and the upcoming 3rd China International Supply Chain Expo (CISCE), these multilateral initiatives are injecting fresh momentum into the global economic recovery. 

China, ASEAN and GCC Advance Cooperative Frameworks to Strengthen Regional Supply Chain Resilience.
China, ASEAN and GCC Advance Cooperative Frameworks to Strengthen Regional Supply Chain Resilience.

On May 26, the ASEAN-China Economic Forum convened nearly 200 representatives from the public and private sectors. CCPIT Chairman Ren Hongbin stressed the importance of recent agreements between China and ASEAN, especially with Malaysia, to deepen cooperation through the Belt and Road Initiative (BRI) and the Regional Comprehensive Economic Partnership (RCEP).

ASEAN representatives widely recognized the significant potential for China-ASEAN cooperation in technology innovation and industrial collaboration. Malaysia and other ASEAN countries expressed their intention to participate in the upcoming 3rd CISCE, working together to build an open, stable regional supply chain network.

With 50 days remaining until its opening, the 3rd CISCE has attracted global attention as the world’s first national-level supply chain expo. Previous editions generated over RMB 300 billion in commercial transactions, with this year’s expo expected to draw hundreds of global companies. A newly established “Innovation Chain Zone” will feature high-value intellectual property and emerging technologies.

Michael Hart, president of the American Chamber of Commerce in China, recently urged U.S. companies to take part, underscoring CISCE’s unique value in a fragmented global trade landscape. Chairman Ren described the expo as “an oasis of cooperation in an uncertain world,” designed to connect global businesses through integrated, sector-specific solutions. 

The 2025 edition will retain its six principal focus areas while introducing new models to support global supply chain resilience. 

The first ASEAN-China-GCC Economic Forum, held from May 27 to 28 in Kuala Lumpur, advanced regional collaboration. Ren led a delegation of over 30 executives from China’s energy, artificial intelligence, and infrastructure sectors, holding talks with ASEAN and GCC officials, resulting in multiple cross-border cooperation memoranda of understanding. 

From low-carbon ice cream to pioneering compressed air energy storage technology, CISCE has established itself as a global launchpad for commercial innovations. At a time of growing trade protectionism, CISCE stands out as a venue for transparent, rules-based cooperation, and is positioned to play a key role in reshaping interregional supply chain dynamics. 

CODIT Launches ‘APEC 2025’ Policy Monitoring Platform

  • AI-Powered Legislative Insights: CODIT’s Global Evidence-Based Policy Institute strengthens policy research and strategy

SEOUL, South Korea, May 29, 2025 /PRNewswire/ — CODIT, an AI-powered leading GovTech firm, has launched a policy monitoring platform developed to support the upcoming 2025 Asia-Pacific Economic Cooperation (APEC) Summit. The platform is intended to assist a wide range of stakeholders by delivering real-time updates on key policy issues in the lead-up to the October summit in Gyeongju, following the Second Senior Officials’ Meeting (SOM2) held this May in Jeju.

CODIT’s ‘APEC 2025’ platform features a real-time policy dashboard that categorizes and visualizes major APEC-related developments. Powered by CODIT’s proprietary AI engine, the platform tracks and analyzes activity across APEC’s core agenda areas, including AI and digital cooperation, ESG, and trade and investment liberalization.

With this year’s APEC Summit expected to bring intensive multilateral dialogue amid a rapidly evolving geopolitical environment, access to timely and reliable policy insights is more important than ever. CODIT’s platform is designed to help summit participants, policy professionals, and media stay informed and strategically prepared.

“GovTech is no longer a future concept—it’s a present-day force transforming how we engage with policy. By harnessing AI to track and interpret complex developments in real time, we’re helping global stakeholders navigate the road to APEC 2025 with clarity, speed, and purpose.”

Ji Eun Chung, CEO of CODIT

As part of its summit-related efforts, CODIT will participate in Session 1: “Advancing Strategic Orientation for Successful AI Transformation within APEC” at the Public-Private Dialogue on AI Cooperation (Day 2), to be held on August 12 in Incheon, during the Third Senior Officials’ Meeting (SOM3). The session will explore AI’s impact across industries and discuss policy directions for sustainable innovation. CODIT will share insights from its AI-powered legal and policy platform, including the new APEC 2025 monitoring service, and highlight the role of GovTech and startup-driven innovation in enabling effective public-private collaboration.

CODIT currently provides tailored legislation, regulation, and policy intelligence services to FORTUNE 500 companies, large enterprises, and unicorns. Since last year, the company has expanded into the U.S. market. Its Global Evidence-Based Policy Institute regularly hosts seminars featuring members of Korea’s National Assembly and government agencies, helping multinational clients anticipate regulatory shifts and adapt their strategies accordingly.

For media inquiries, please contact: contact@thecodit.com 

CODIT Launches 'APEC 2025' Policy Monitoring Platform
CODIT Launches ‘APEC 2025’ Policy Monitoring Platform

 

Neuronata-R® Stem Cell Therapy Shows Promise in ALS Phase 3 Subgroup Analysis, Moves Toward FDA Accelerated Approval

Meaningful efficacy observed in slow-progressor subgroup; NfL biomarker improvements support potential for accelerated regulatory pathway

SEOUL, South Korea, May 29, 2025 /PRNewswire/ — Neuronata-R®, an autologous bone marrow-derived mesenchymal stem cell (MSC) therapy for amyotrophic lateral sclerosis (ALS), has demonstrated meaningful efficacy signals in a subgroup of participants with slow disease progression in its recently completed Phase 3 trial. The therapy also showed consistent reductions in neurofilament light chain (NfL) levels — a biomarker that served as the basis for FDA accelerated approval in ALS, including the recent decision on Tofersen — supporting its potential to pursue a similar regulatory pathway.

Developed by South Korean biotech company CorestemChemon (KOSDAQ: 166480), Neuronata-R® utilizes MSCs derived from a participant’s own bone marrow to modulate inflammation, protect motor neurons, and alter the neurodegenerative microenvironment through paracrine signaling. ALS, a rare and fatal disease with no cure, remains one of the most urgent areas of unmet medical need. Neuronata-R® aims to address the disease’s complex pathology by leveraging the therapeutic potential of autologous MSCs.

In December 2024, CorestemChemon announced topline results from the ALSummit trial (NCT04745299), noting that the study did not meet its primary endpoint — a combined assessment of function and survival known as CAFS — in the overall patient population. However, a post hoc analysis conducted after the completion of the trial and full data collection revealed significant clinical improvements in a subgroup of patients with slower disease progression. To discuss these findings, the company plans to meet with the U.S. Food and Drug Administration (FDA) later this year to discuss these findings, with the aim of submitting a biologics license application by the end of 2025 and potentially securing accelerated approval by mid-2026.

Subgroup Analysis Supports Targeted Efficacy

The final Clinical Study Report (CSR) confirmed that Neuronata-R® demonstrated statistically significant improvements in key efficacy endpoints among participants with slow disease progression. Recognizing the potential efficacy of Neuronata-R® in early-stage ALS, CorestemChemon stratified participants into slow and fast progressors.

Among slow progressors, Neuronata-R® showed statistically significant improvements across multiple measures — including the primary composite endpoint, CAFS (Combined Assessment of Function and Survival), functional outcomes assessed by ALSFRS-R scores, and respiratory function measured by slow vital capacity (SVC).

Notably, the five-dose treatment arm (Group 2) demonstrated statistically significant improvement in ALSFRS-R scores beginning at Month 9 post-treatment — one month earlier than the two-dose arm (Group 1), which reached significance at Month 10.

This final CSR also included full analyses of CAFS and SVC, which had not been previously disclosed. While CAFS served as the trial’s primary efficacy endpoint, SVC is recognized as a clinically meaningful measure of respiratory decline in ALS.

At Month 6, participants in Group 1 receiving Neuronata-R® showed a statistically significant improvement in CAFS (LS Mean 20.95 vs 13.66; 95% CI; p<0.024) compared with placebo. Group 2 also showed improvement (LS Mean 24.78 vs 17.92; 95% CI; p<0.041). In Group 2, significant divergence in SVC compared to the control group emerged from Month 8 post-treatment, suggesting a potential effect in delaying disease progression.

NfL Biomarker Reduction Mirrors Regulatory Precedent

CorestemChemon also highlighted the reduction in NfL levels — a biomarker that served as the basis for FDA accelerated approval in ALS, including the recent decision on Tofersen. In both the two-dose (Group 1) and five-dose (Group 2) arms, NfL levels consistently declined over time. Notably, in Group 2, NfL levels were significantly lower than placebo at both Month 4 and Month 10, suggesting a potential dose-dependent effect.

The consistency between the company’s internal analysis and validation by an independent CRO strengthens the reliability of the dataset, which has been incorporated into the final CSR.


“This subgroup analysis lends strong support to a biomarker-driven approval strategy,” a company official said. “The consistency of our internal analysis with external CRO validation adds credibility to the dataset and provides a concrete basis for regulatory discussions with the MFDS.”

Regulatory Pathway Toward Accelerated Approval

The company views these findings as strategically significant, particularly in light of their alignment with the precedent set by Tofersen, where the FDA granted accelerated approval based on NfL reduction rather than survival benefit — a pathway CorestemChemon now seeks to pursue.

The company plans to request a Pre-BLA or Type C meeting with the FDA in Q3 2025, with the goal of submitting a biologics license application by Q4 2025 and targeting regulatory approval by mid-2026. CorestemChemon will finalize its submission package in collaboration with a global CRO and actively engage with regulatory agencies to pursue worldwide market entry for Neuronata-R®.

Innovative Stem Cell Therapy

Neuronata-R® uniquely addresses the complex mechanisms of ALS by leveraging MSCs derived from the patient’s own bone marrow. These cells exert anti-inflammatory and immunomodulatory effects, protect motor neurons, and, through paracrine signaling, secrete trophic factors, cytokines, and extracellular vesicles that modulate the microenvironment and reduce neuroinflammation. By targeting these underlying pathological processes, Neuronata-R® is designed to interrupt the neurodegenerative cascade.

About Neuronata-R®

Neuronata-R® (Lenzumestrocel), developed and commercialized by CorestemChemon Inc. (KOSDAQ: 166480), is an autologous MSC therapy for ALS patients. The company began ALS research in 2002 and obtained MFDS approval for Neuronata-R® in 2014. To date, it has been administered to more than 400 commercial patients and 190 clinical trial participants with no treatment-related serious adverse events reported.

Neuronata-R® holds Orphan Drug Designation from both the U.S. FDA (2018) and the EMA (2019). CorestemChemon completed a Phase 2 trial (NCT01363401) in 2014 and its Phase 3 trial (NCT04745299) in 2024; the final CSR has been submitted to the MFDS.

AccurEdit Therapeutics’ ART001 Becomes China’s First Gene Editing Therapy to Receive FDA Regenerative Medicine Advanced Therapy (RMAT) Designation

RMAT designation expected to expedite development; 72 weeks interim clinical trial data showing sustainable reduction of TTR over 90% with no significant adverse events.

SUZHOU, China, May 29, 2025 /PRNewswire/ — AccurEdit Therapeutics today announced that its internally developed in vivo gene editing therapy ART001 has been granted a Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA). Public records indicate that ART001 is the first gene editing product from China to receive an FDA RMAT designation, highlighting the FDA’s recognition of its innovative nature and clinical potential. This designation is expected to help expedite the clinical development of ART001 for patients with ATTR amyloidosis.

Based on the existing clinical data, the FDA confirmed that ART001 meets the criteria for RMAT designation. Clinical results have shown that four weeks after administration, subjects in the high-dose cohort experienced an average >90% reduction in peripheral TTR protein levels from baseline, with this reduction sustained for 72 weeks to date.

ART001 has also exhibited a strong safety profile in clinical studies. In an investigator-initiated trial that has been followed up for 72 weeks by far, no infusion-related reactions or AST (aspartate aminotransferase) elevations—side effects commonly observed with similar therapies—were reported. Importantly, no dose-limiting toxicities (DLTs) or serious adverse events (SAEs) occurred during the study. ART001 demonstrated exceptional precision in pre-clinical studies, with no off-target gene edits detected even at dose levels many times higher than the therapeutic dose. These safety and efficacy findings support ART001’s potential as a globally leading, best-in-class treatment for ATTR.

ART001 has achieved several milestones in its development. In August 2023, ART001 became the first lipid nanoparticle (LNP)-delivered in vivo gene editing drug to enter a human clinical trial in China. It later received clearance to conduct clinical trials in both China and the United States (China in July 2024 and U.S. in August 2024), making it the only therapy of its class with regulatory approval to initiate trials in both countries. ART001 has since advanced into a Phase IIa study. In March 2025, the FDA also granted ART001 Orphan Drug designation for the treatment of ATTR amyloidosis, further supporting its potential to address this life-threatening disease.

About Regenerative Medicine Advanced Therapy (RMAT) Designation

The RMAT designation was established under the U.S. 21st Century Cures Act of 2016 as a program to expedite the development and review of innovative regenerative medicine products, including cell therapies, gene therapies, tissue-engineered products, and combination products. An investigational therapy may receive RMAT designation if it is intended to treat a serious or life-threatening condition and preliminary clinical evidence indicates the therapy has the potential to address unmet medical needs by demonstrating a meaningful therapeutic effect on the disease.

About AccurEdit Therapeutics

AccurEdit Therapeutics, founded in 2021, is a biotechnology company focused on developing in vivo gene editing technologies and therapies based on lipid nanoparticle (LNP) and other non-viral delivery systems. The company is dedicated to providing one-time, cost-effective, innovative treatment solutions to patients worldwide. It has assembled a specialized team with successful experience across the biologic drug development lifecycle and has established the world’s first clinically validated, industrial-scale, end-to-end in vivo gene editing platform. AccurEdit Therapeutics has built a broad intellectual property portfolio covering its technologies and products, including a novel base editor ARTbase-A1™, which has been granted a U.S. patent.

AccurEdit Therapeutics is advancing a pipeline targeting both rare genetic diseases and difficult-to-treat common diseases. In August 2023, its lead product ART001 (for ATTR amyloidosis) became the first LNP-delivered in vivo gene editing therapy to enter a human clinical trial in China. In August 2024, ART001 also became the first – and currently only – such product to receive an Investigational New Drug (IND) approval from the U.S. FDA. In February 2025, the company’s second product ART002 (targeting heterozygous familial hypercholesterolemia [HeFH] via the PCSK9 gene) became the world’s first in vivo gene editing product of its class to achieve pharmacodynamic saturation in humans and effectively reduce LDL-C levels in patients with extremely high baseline cholesterol. The safety and efficacy data from both ART001 and ART002 suggest these candidates have the potential to become best-in-class gene editing therapies globally.