On May 8, 2026 Ensoma, an in vivo cellular engineering company with a mission to advance the future of medicine through one-time therapies, reported the presentation of initial clinical data from the first participant dosed in its Phase 1/2 trial of EN-374 for the treatment of X-linked chronic granulomatous disease (X-CGD) at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 29th Annual Meeting, taking place May 11-15 in Boston. The data represent the first reported clinical experience with in vivo hematopoietic stem cell (HSC)-directed therapy, from which the patient has the potential to create a continuous source of therapeutic immune and blood cells to treat disease.

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"We are excited to discuss encouraging initial safety data from the first participant in our Phase 1/2 clinical trial of EN-374 for X-CGD, the first-ever in vivo HSC gene insertion therapy in the clinic. While these are early data from a single participant, they mark an important first step in evaluating a new approach to engineering hematopoietic stem cells directly in vivo, and we look forward to continuing to assess safety and potential markers of clinical activity as the study progresses," said Jim Burns, CEO of Ensoma. "Additional ASGCT (Free ASGCT Whitepaper) presentations include promising developments with both Ensoma’s viral vector technology and our approach to producing cancer-killing immune cells. Together, these data advance our goal of bringing the power of in vivo HSC engineering to patients and treating genetic diseases and cancer with a potentially continuous supply of engineered immune and blood cells."

Oral Presentations:

Title: First in vivo hematopoietic stem cell (HSC) gene addition clinical trial: Initial results from EN-374-101 in X-linked chronic granulomatous disease (X-CGD)
Presentation Date/Time: Friday, May 15, 8:00-9:45 a.m. ET
Location: Westin Seaport Commonwealth Ballroom ABC (Concourse level)
Presenter: Ahmad Rayes, M.D., University of Utah
Key Highlights:

Treatment, including HSC mobilization, gene therapy infusion, short-course immune prophylaxis and three cycles of enrichment, was well tolerated
Adverse events (AEs) were all low-grade. There were no serious AEs or dose-limiting toxicities
Follow-up to assess potential efficacy is ongoing and will be reported at a later date
Title: Discovery and development of engineered neutralizing antibody-evading helper-dependent adenovirus capsids as candidates for in vivo gene therapy
Presentation Date/Time: Wednesday, May 13, 11:15-11:30 a.m. ET
Location: Westin Seaport Commonwealth Ballroom ABC (Concourse Level)
Presenter: Marcin Maziarz, Ph.D., Ensoma
Data Summary:

Engineered series of hexon-modified helper-dependent adenovirus (HDAd) capsids designed to evade pre-existing Ad5 neutralizing antibodies (NAbs), a known barrier to gene delivery
Identified an optimized capsid variant (HDAdGen2) that demonstrated evasion of NAbs in human sera
HDAdGen2 maintained transduction efficiency comparable to the standard HDAd5/35++ vector in vitro and in vivo
Findings suggest potential to improve gene delivery in patients with pre-existing immunity to Ad5
Supports continued advancement of optimized capsids for in vivo gene therapy applications
Poster Presentation:

Title: An in vivo engineered and lineage-restricted multiplexed CAR-M, -NK, and -T cell therapy mounts robust solid tumor control in pre-clinical models
Poster Presentation Date/Time: Wednesday, May 13, 5:00-6:30 p.m. ET
Location: MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit level)
Presenter: Yiwen Zhao, Ph.D., Ensoma
Data Summary:

Designed lineage-restricted regulatory elements to drive CAR expression selectively in myeloid, NK and T cell populations
Observed durable HER2+ tumor control and prolonged survival in treated animals compared to controls in preclinical models
Maintained normal hematopoiesis and immune cell differentiation following HSC engineering
Supports potential of a multi-lineage, in vivo-generated cell therapy approach for solid tumors
Additionally, Drew Dietz, M.D., Vice President and Head of Clinical Research & Development at Ensoma, will speak during a scientific symposia session. Details are as follows:

Title: Adenoviral vectors and in vivo selection: Designing clinical strategies for durable benefit
Session Date/Time: Friday, May 15, 11:07-11:33 a.m. ET

About EN-374

EN-374 is a first-in-class in vivo hematopoietic stem cell (HSC)-directed therapy for X-CGD that employs virus-like particles (VLPs) to deliver payloads having a CYBB transgene to HSCs. Neutrophils arising from the engineered HSC then express the protein product of the CYBB transgene. In this way, EN-374 is designed to restore function of the infection-fighting NADPH oxidase enzyme complex critical for immune defense in humans. In preclinical studies, EN-374 demonstrated therapeutic levels of restoration of CYBB gene expression and NADPH oxidase activity in circulating neutrophils. EN-374 represents the first in vivo HSC-directed therapy for X-CGD, building on a mechanism that has been validated ex vivo. The Phase 1/2 study is an open-label, multicenter clinical trial in the US and UK evaluating the safety, tolerability, pharmacodynamics and efficacy biomarkers of EN-374, with the goal of identifying a dose for further clinical development in X-CGD.

(Press release, Ensoma, MAY 8, 2026, View Source [SID1234665398])

On May 8, 2026 Rznomics a biopharmaceutical company specializing in RNA-based gene therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation to RZ-001, its lead investigational candidate for the treatment of hepatocellular carcinoma (HCC).

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RMAT designation is a specialized FDA program created to accelerate the development and review of promising new therapies, including gene therapies, intended to treat serious or life-threatening conditions. Applicant is required to submit preliminary clinical evidence suggesting the potential to address unmet medical needs. This designation provides important opportunities during the drug development process, including increased FDA guidance and eligibility for priority and rolling reviews, as well as accelerated approval pathways. By streamlining these regulatory milestones, the program aims to bring transformative innovations to patients more quickly.

RZ-001 is the next-generation oncology therapeutics based on Rznomics’ proprietary trans-splicing ribozyme technology platform. By replacing cancer-specific RNA with therapeutic RNA, RZ-001 offers a novel mechanism of action designed to overcome the limitations of conventional therapies. The platform’s dual-action approach—enhancing both tumor selectivity and safety—presents a promising new option for HCC patients with limited treatment alternatives. RZ-001 previously received Orphan Drug Designation (ODD) in 2024 and Fast Track Designation (FTD) in 2025 for the treatment of HCC.

The FDA’s decision to grant RMAT status highlights the clinical potential and innovativeness of RZ-001, particularly following the meaningful interim signals from the Phase 1b/2a clinical trial presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2026.

"With the RMAT designation, we plan to accelerate our U.S. development and partnership initiatives by initiating formal discussions with the FDA regarding clinical trial design, Chemistry, Manufacturing, and Controls (CMC), and commercialization strategies," said Sung-woo Hong, Vice President of Rznomics.

Seong-Wook Lee, CEO of Rznomics, added, "Receiving RMAT designation for RZ-001 is a profound validation of the innovation and competitiveness of our RNA editing platform by the FDA. We will concentrate our resources on global development and commercialization to provide a breakthrough therapeutic option in the field of HCC, where unmet medical needs remain exceptionally high."

About RMAT Designation

Introduced under the 21st Century Cures Act in 2016, the RMAT designation was established to foster the development of innovative regenerative medicine therapies and expand patient access. The program encompasses cell and gene therapies, therapeutic tissue engineering products, and combination products. To be eligible, a drug must be a regenerative medicine therapy intended to treat serious conditions, with preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such a condition.

(Press release, Rznomics, MAY 8, 2026, View Source [SID1234665397])

On May 8, 2026 Harbour BioMed (the "Company"; HKEX: 02142), a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics in immunology, oncology and other disease areas, reported that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for HBM7004, enabling the initiation of a first-in-human (FIH) Phase I clinical trial. The study will evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of HBM7004 in subjects with advanced solid tumors.

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HBM7004 is a novel B7H4xCD3 bispecific antibody developed using the Company’s HBICE platform. This bispecific antibody is designed to provide a differentiated approach to cancer immunotherapy with the potential to enhance both efficacy and safety. The development of HBM7004 further demonstrated the HBICE platform’s versatility and plug-and-play advantages. In preclinical studies, HBM7004 demonstrated an intratumor B7H4-dependent T cell activation manner. In multiple animal models, HBM7004 showed strong anti-tumor efficacy, remarkable in vivo stability, and reduced systemic toxicity. Additionally, in preclinical models, HBM7004 exhibited a strong synergistic effect when combined with a B7H4x4-1BB bispecific antibody at a low effector-to-target cell ratio, indicating an encouraging therapeutic window.

"The FDA’s IND clearance for our B7H4xCD3 bispecific antibody HBM7004 marks an important step in advancing our innovative pipeline for patients with advanced solid tumors," said Dr. Jingsong Wang, Founder, Chairman and Chief Executive Officer of Harbour BioMed. "This program reflects our continued focus on developing differentiated biotherapeutics leveraging our industry-leading proprietary platforms to address significant unmet needs in oncology. We are confident in the potential of HBM7004 and look forward to evaluating its clinical benefit in patients with advanced solid tumors."

(Press release, Harbour BioMed, MAY 8, 2026, View Source [SID1234665396])

On May 8, 2026 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. ("Kelun-Biotech" or the "Company") reported that the sNDA (the "Application") for the Company’s TROP2 ADC sacituzumab tirumotecan (sac-TMT, also known as SKB264/MK-2870) (佳泰莱) was accepted for review by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. The application is for sac-TMT in combination with MSD’s[1] anti-PD-1 monoclonal antibody pembrolizumab (KEYTRUDA[2]) as first‑line treatment for adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have PD-L1 tumor proportion score (TPS) ≥1% and are EGFR-negative and ALK-negative. This acceptance is based on the positive results from the OptiTROP-Lung05 registrational Phase III study, and the application is the fifth indication application for sac-TMT that has been accepted by the NMPA.

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The OptiTROP-Lung05 is a randomized, open-label, multicenter, Phase III clinical study that evaluates the efficacy and safety profile of sac-TMT in combination with pembrolizumab versus pembrolizumab monotherapy as first-line treatment for PD-L1-positive locally advanced or metastatic NSCLC. At a pre-specified interim analysis, the study has met its primary endpoint of progression-free survival (PFS) and demonstrated a statistically significant and clinically meaningful improvement as concluded by the Independent Data Monitoring Committee (IDMC). A positive trend in overall survival (OS) was also observed. Notably, the OptiTROP-Lung05 study is the first Phase III study of an immunotherapy and ADC combination to meet its primary endpoint in first-line NSCLC treatment. The study has been selected for an oral presentation at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Abstract number #8506, Lung Cancer –Non-Small Cell Metastatic).

Previously, sac-TMT in combination with intravenous and subcutaneous pembrolizumab for the first-line treatment of patients with locally advanced or metastatic NSCLC who have PD-L1 TPS≥1% and are EGFR-negative and ALK-negative was granted Breakthrough Therapy Designation (BTD) by the NMPA. On April 9, 2026, the CDE’s official website announced that the Application has entered the priority review and approval process. This marks the fifth sac-TMT indication to enter the CDE’s priority review and approval process. Through this process, the review time will be significantly shortened, potentially expediting its approval pathways.

Dr. Michael Ge, CEO of Kelun-Biotech said, "We are delighted to see the acceptance of the fifth indication application for sac-TMT. Compared to immunotherapy alone, the ADC combination with KEYTRUDA as first-line treatment for PD-L1-positive NSCLC has achieved not only positive results in PFS, but also a trend toward benefit in OS. This achievement holds significant importance for improving current treatment regimens for lung cancer. We will continue to collaborate with our partners to advance the clinical development and commercialization of sac-TMT, to help more patients with lung cancer and enhance their survival outcomes."

About sac-TMT

Sac-TMT, a core product of the Company, is a novel human TROP2 ADC in which the Company has proprietary intellectual property rights, targeting advanced solid tumors such as NSCLC, breast cancer (BC), gastric cancer (GC), gynecological tumors and genitourinary tumors, among others. Sac-TMT is developed with a unique, bifunctional linker that maximizes payload delivery to tumor cells both through its irreversible connection with the anti-TROP2 monoclonal antibody sacituzumab and its pH-sensitive cleavage from a belotecan-derivative topoisomerase I inhibitor payload in the lysosome, with a drug-to-antibody-ratio (DAR) of 7.4. Sac-TMT specifically recognizes TROP2 on the surface of tumor cells by recombinant anti-TROP2 humanized monoclonal antibodies, which is then endocytosed by tumor cells and releases the payload KL610023 intracellularly. KL610023, as a topoisomerase I inhibitor, induces DNA damage to tumor cells, which in turn leads to cell-cycle arrest and apoptosis. In addition, it also releases KL610023 in the tumor microenvironment. Given that KL610023 is membrane permeable, it can enable a bystander effect, or in other words kill adjacent tumor cells.

In May 2022, the Company licensed the exclusive rights to MSD (the tradename of Merck & Co., Inc, Rahway, NJ, USA) to develop, use, manufacture and commercialize sac-TMT in all territories outside of Greater China (which includes Mainland China, Hong Kong, Macao and Taiwan).

To date, four indications for sac-TMT have been approved and marketed in China for: 1) unresectable locally advanced or metastatic TNBC who have received at least two prior systemic therapies (at least one of them for advanced or metastatic setting); 2) EGFR mutant-positive locally advanced or metastatic non-squamous NSCLC following progression on EGFR-TKI therapy and platinum-based chemotherapy; 3) EGFR mutant-positive locally advanced or metastatic non-squamous NSCLC who progressed after treatment with EGFR-TKI therapy; 4) unresectable or metastatic HR+/HER2- (IHC 0, IHC 1+ or IHC 2+/ISH-) BC who have received prior ET and at least one line of chemotherapy in advanced setting. The first two indications above have been included in China’s National Reimbursement Drug List (NRDL). This inclusion is expected to bring clinically meaningful benefits to a greater number of patients with BC and NSCLC. Additionally, sac-TMT has been granted six Breakthrough Therapy Designations (BTDs) by the NMPA.

Sac-TMT is the world’s first TROP2 ADC drug approved for marketing in lung cancer. A new indication application for sac-TMT in combination with pembrolizumab (KEYTRUDA) as first‑line treatment for locally advanced or metastatic NSCLC who have PD-L1 TPS≥1% and are EGFR-negative and ALK-negative has been accepted for review by the NMPA, and has entered the priority review and approval process. As of today, Kelun-Biotech has initiated 9 registrational clinical studies in China. MSD has initiated 17 ongoing global Phase III clinical studies of sac-TMT as a monotherapy or in combination with pembrolizumab or other anti-cancer agents for several types of cancer. These studies are sponsored and led by MSD.

(Press release, Kelun, MAY 8, 2026, View Source [SID1234665395])

On May 8, 2026 Day One Biopharmaceuticals, Inc., now part of Servier Group, reported that it has completed enrollment in the FIREFLY-2 clinical trial, which is evaluating the efficacy, safety and tolerability of tovorafenib vs standard of care chemotherapy in the front-line setting of therapy for patients aged 6 months to 25 years with low-grade glioma. The trial is being conducted in collaboration with the European Society for Paediatric Oncology (SIOPe) Brain Tumor Group LOGGIC Consortium (LOGGIC). Tovorafenib, known as OJEMDATM, is currently indicated for treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

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"Reaching full enrollment in this trial is a critical step toward our goal of establishing OJEMDA as standard of care across all lines of therapy for individuals with BRAF-altered pLGG. By moving earlier in the treatment journey, we aim to intervene when we can have the greatest impact on the burden of this challenging cancer," said Elly Barry, M.D., Chief Medical Officer at Day One, now part of Servier Group. "Success in this study would not only further validate the efficacy and safety profile of OJEMDA, but also fundamentally evolve the pLGG treatment paradigm, and potentially establish a new standard of care for patients newly diagnosed with pLGG, the most common brain tumor afflicting children."

FIREFLY-2 is a Phase 3, global, randomized, multicenter, open-label study to evaluate the efficacy, safety, and tolerability of monotherapy tovorafenib, an oral, Type II RAF inhibitor, in patients ages 6 months to 25 years with RAF-altered pLGG requiring first-line systemic therapy. It is being conducted at approximately 140 sites across the U.S., Canada, Europe, Australia, South America, Middle East and Asia, and in just over three years has enrolled approximately 400 participants who are receiving either tovorafenib once weekly or one of four standard of care (SoC) chemotherapy regimens.

"Completing enrollment in FIREFLY-2 is a powerful early signal of momentum. It reflects what’s possible when we bring together deep scientific expertise, a patient-first culture, and the scale to execute globally," said David K. Lee, CEO of Servier Pharmaceuticals. "So soon after Servier’s acquisition of Day One, this milestone reinforces our conviction that joining forces was the right decision for patients and for our oncology strategy. As we aim to accelerate the development of targeted medicines and advance them with the rigor, speed and reach needed to make a meaningful difference, we hope this study helps unlock the potential to improve outcomes for children living with pediatric low-grade glioma."

The primary endpoint of the trial is overall response rate (ORR), including duration of response (DOR), based upon Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO-LGG) criteria, with key secondary endpoints including progression-free survival (PFS) and event-free survival (EFS) per RAPNO-LGG, time to next treatment (TTNT), overall survival (OS) and other measures, including patient-reported outcomes. The primary analysis is expected to occur approximately 12 months after the last patient is enrolled; preliminary insights are expected to be available in 2027. More information on the trial can be found at The FIREFLY-2 Trial – Day One Clinical Trials or View Source

About tovorafenib
Tovorafenib (known as OJEMDATM in the U.S.) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. Tovorafenib is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based, in part, on response rate and duration of response according to multiple response assessment criteria: Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria, Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO LGG) criteria, and Response Assessment for Neuro-Oncology Low-Grade Glioma (RANO LGG) criteria. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.

Please see full prescribing information including important safety information about OJEMDA at www.OJEMDA.com.

About Pediatric Low-Grade Glioma
Pediatric low-grade gliomas (pLGG) are the most common brain tumor with an estimated US incidence of 1,100 and Europe incidence of 700 children per year who are eligible for front-line systemic therapy.i, ii BRAF is the gene most commonly altered in pLGG, which includes two primary types of BRAF alterations – a BRAF gene fusion and BRAF point mutation. These BRAF alterations account for >50% of pLGG cases worldwide and prior to the introduction of OJEMDA, there were no approved treatments for people with pLGG driven by BRAF fusions.i, iii

Pediatric low-grade gliomas can be chronic and relentless, with patients suffering profound side effects from both the tumor and the treatment, which may include chemotherapy and radiation. These side effects can impact their life over the long term, and may include motor deficiencies, vision loss, hormone deficiency and alterations in growth and development. Most children with pLGG will survive their cancer, but for the majority of those in whom a complete surgical resection is not possible, these tumors tend to recur frequently throughout childhood, necessitating multiple treatments. The cumulative toxicity of numerous therapies, along with the damage caused by multiple episodes of tumor progression, take a significant toll on the children and their families.

(Press release, Day One, MAY 8, 2026, View Source [SID1234665394])