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 Artiva Biotherapeutics, Inc. (Nasdaq: ARTV) (Artiva), a clinical-stage biotechnology company whose mission is to develop effective, safe and accessible cell therapies for patients with debilitating autoimmune diseases, reported financial results for the first quarter ended March 31, 2026, and highlighted recent progress.

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"Artiva has reached an important inflection point, with positive initial clinical data across multiple autoimmune diseases and FDA alignment on a single Phase 3 registrational trial design in refractory RA," said Fred Aslan, M.D., president and chief executive officer of Artiva Biotherapeutics. "The initial RA data demonstrated meaningful responses in highly refractory patients, alongside a tolerability profile supportive of outpatient administration in community rheumatology settings. Together, these data support AlloNK’s potential to become the first deep B-cell depleting therapy to advance into a Phase 3 trial in refractory RA, the autoimmune indication with the largest number of refractory patients."

Dr. Aslan continued, "By combining deep B-cell depletion, meaningful clinical responses and an outpatient profile suited to community rheumatology practices, AlloNK has the potential to redefine the treatment paradigm for patients with refractory autoimmune disease."

Recent Business Highlights

Reported positive initial clinical data from ongoing clinical trials evaluating AlloNK in combination with rituximab across multiple autoimmune diseases

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As of the April 3, 2026 data cutoff, the initial clinical dataset included 21 refractory RA patients with at least 12 weeks of follow-up, including 13 patients with at least six months of follow-up from Artiva’s company-sponsored Phase 2a basket trial and an investigator-initiated basket trial evaluating AlloNK in B-cell driven autoimmune disease. The broader autoimmune dataset also included 11 SjD patients and five SSc patients, including seven SjD patients and four SSc patients with at least six months of follow-up.

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In refractory RA, clinically meaningful improvements were observed across multiple measures of disease activity, including ACR responses, CDAI and DAS28-ESR. Five of seven patients (71%) with six months of follow-up in the company-sponsored Phase 2a basket trial achieved an ACR50 response. Nineteen of 21 RA patients demonstrated clinically meaningful reductions from baseline in both CDAI and DAS28-ESR.

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The AlloNK treatment regimen demonstrated tolerability results supportive of outpatient administration in community rheumatology settings, with no CRS, ICANS or treatment discontinuations related to AlloNK reported as of the data cutoff.

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Deep B-cell depletion was observed across evaluable patients, including complete B-cell depletion using a high-sensitivity assay in all 28 RA patients evaluated as of the data cutoff, supporting AlloNK’s proposed mechanism of action.

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Clinical responses in SjD and SSc were consistent with the RA data and support the potential of AlloNK across B-cell-driven autoimmune diseases.

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More than 70 autoimmune patients have been treated with AlloNK across more than 40 active clinical sites, mostly in community rheumatology settings, providing a strong foundation for planned registrational trial initiation.

Achieved FDA alignment on Phase 3 registrational trial design in refractory RA

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Artiva announced alignment with the FDA on a single Phase 3 registrational randomized controlled trial evaluating AlloNK plus rituximab versus rituximab alone in approximately 150 refractory RA patients, with ACR50 response at six months as the primary endpoint.

Upcoming Milestones

Present AlloNK clinical data at EULAR 2026

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Multiple abstracts accepted for presentation at EULAR 2026, expected to further characterize AlloNK’s mechanism of action, clinical activity and outpatient feasibility, including:

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Late Breaking Oral Abstract Presentation – LB0003: AB-101, an Outpatient-Administered Allogeneic NK Cell Therapy Combined with Rituximab, Generates Robust Clinical Efficacy Responses Comparable with Autologous CAR T in 31 Patients with Rheumatologic Diseases

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Oral Abstract Presentation – OP0129: AB-101, an Allogeneic NK Cell Therapy, Combined with Rituximab was Highly Effective in Severe Sjögren Disease: Experience in First Patient Treated

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Poster View Presentation – POS1177: Robust and Durable Clinical Responses Observed Following Treatment with AB-101, an Allogeneic NK Cell Therapy, Combined with Rituximab in Patients with Severe Rheumatoid Arthritis and Inadequate Response to Multiple Prior Targeted Therapies

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Poster Tour – POS0355: AB-101, an Allogeneic NK Cell Therapy, in Combination with Anti-CD20 Monoclonal Antibodies, Consistently Achieves Deep B-cell Depletion Comparable with CAR T Cell Therapies in Patients with Rheumatologic Diseases

Initiate Phase 3 registrational trial in refractory RA

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In the second half of 2026, Artiva plans to initiate a Phase 3 randomized controlled trial evaluating AlloNK plus rituximab versus rituximab alone in approximately 150 RA patients who have had an inadequate response to two or more biologic or targeted synthetic disease-modifying anti-rheumatic drugs (b/tsDMARDs) of distinct classes, with ACR50 response at six months as the primary efficacy endpoint.

First Quarter 2026 Financial Results

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Cash, Cash Equivalents and Investments. As of March 31, 2026, Artiva had cash, cash equivalents and investments of $86.8 million, which is expected to fund operations into Q2 2027.

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Research and Development Expenses. Research and development expenses were $19.3 million for the three months ended March 31, 2026, compared to $17.1 million for the three months ended March 31, 2025.

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General and Administrative Expenses. General and administrative expenses were $5.1 million for each of the three months ended March 31, 2026 and 2025.

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Other Income, net. Other income, net, was $0.9 million for the three months ended March 31, 2026, compared to other income, net, of $1.9 million for the three months ended March 31, 2025.

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Net Loss. Net loss totaled $23.5 million for the three months ended March 31, 2026, as compared to net loss of $20.3 million for the three months ended March 31, 2025, with non-cash stock-based compensation expense of $1.6 million and $2.1 million for the three months ended March 31, 2026 and 2025, respectively.

(Press release, Artiva Biotherapeutics, MAY 8, 2026, View Source [SID1234665382])

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 bioAffinity Technologies, Inc. (Nasdaq: BIAF; BIAFW), a biotechnology company focused on the need for noninvasive, accurate tests for the detection of early-stage lung cancer and other lung diseases, reported financial results for the first quarter ended March 31, 2026.

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Q1 2026 Highlights

● CyPath Lung unit sales increased 146% year-over-year in the first quarter of 2026, reflecting accelerating physician adoption and expanding clinical use of the Company’s noninvasive lung cancer diagnostic.
● CyPath Lung testing revenue increased approximately 114% to $361,000, compared to $169,000 in the first quarter of 2025.
● Total consolidated revenue decreased approximately 27% to $1.4 million, compared to $1.9 million for the first quarter of 2025, resulting from the discontinuation of certain unprofitable pathology services in March 2025 to focus on higher margin services, including CyPath Lung testing.
● The number of physician offices and clinics ordering CyPath Lung increased 69% from first quarter 2025 to the same period in 2026, reflecting continued productivity and expansion of the Company’ sales force and focus on additional strategic markets. The Company expects growth to accelerate throughout 2026, as a result of these commercial initiatives and increasing awareness of the benefits of CyPath Lung.
● The Company launched a large-scale longitudinal clinical trial partially funded by the U.S. Department of Defense to further validate CyPath Lung performance and support establishing the noninvasive test as a standard of care for military and Veterans Administration (VA) medical centers. The trial is expected to include up to 20 clinical sites, including multiple Department of Veterans Affairs medical centers and leading U.S. military hospitals. The John P. Murtha Cancer Center Research Program is providing support and funding for the study at several federal facilities.

● As part of the Company’s commercial strategy to develop companion tests using its diagnostic platform to more precisely target drugs that treat asthma and COPD, bioAffinity Technologies announced that Brooke Army Medical Center, the military’s largest healthcare institution, was conducting a 40-patient collaborative study. The study will use bioAffinity Technologies’ flow cytometry+AI technology to evaluate whether its proprietary platform can detect biologic drug receptors in sputum, including those for dupilumab and benralizumab, to guide personalized therapy selection and monitor patient response over time. The Company presented findings from its early pipeline development activities at the American Academy of Allergy, Asthma and Immunology (AAAAI) 2026 annual meeting.
● Nationally recognized pulmonary and lung cancer experts joined the Company’s Medical and Scientific Advisory Board (MSAB) to provide independent guidance on strategic priorities, including clinical implementation and broader adoption of CyPath Lung. David Ost, MD, MPH, University of Texas MD Anderson Cancer Center, Daniel Sterman, MD, New York University Langone Medical Center, and J. Scott Ferguson, MD, University of Wisconsin School of Medicine and Public Health, were named to the Company’s panel of experts.
● The Company released three additional patient case studies in first quarter 2026 in which CyPath Lung results of "Unlikely Malignancy" helped avoid unnecessary invasive and costly biopsies when other tests suggested the presence of lung cancer in patients at high risk. CyPath Lung test results were confirmed by follow-up imaging that showed stable or resolved lung nodules.

Management Commentary

"Our first quarter results demonstrate continued momentum for CyPath Lung in the marketplace. As more and more physicians adopt CyPath Lung and share their experiences with peers, we see the opportunity to expand our commercial reach and bridge the diagnostic gap between imaging and invasive procedures, especially when dealing with indeterminate nodules in high-risk patients," said Maria Zannes, President and CEO of bioAffinity Technologies. "We are accelerating our marketing strategy to expand access to CyPath Lung and educate healthcare practitioners and patients alike about the need for accurate, objective information to better stratify risk and improve patient outcomes. On April 8, we hosted our first webinar featuring a panel of pulmonologists who shared how they use CyPath Lung in their diverse practices."

Ms. Zannes continued, "Physicians continue to share their case studies in which CyPath Lung has identified lung cancer as early as Stage 1A when it is most treatable and conversely in which a negative CyPath Lung result helped avoid unnecessary invasive procedures. We believe the growing number of case studies and our longitudinal clinical trial, supported by leading military and VA institutions, will lead to broader adoption of CyPath Lung as part of the standard of care."

Ms. Zannes concluded, "We are uniquely positioned to fulfill the need for an accurate, noninvasive diagnostic for lung cancer, particularly when imaging and risk models are inconclusive or turn out to be wrong. The remainder of 2026 will be focused on scaling commercial execution, expanding into new geographic markets, and driving increased utilization of CyPath Lung through continued physician engagement while also leveraging our flow cytometry and AI platform to advance our pipeline of diagnostics for serious or life-threatening lung diseases."

First Quarter 2026 Financial Results

Revenue for the quarter ended March 31, 2026, was $1.4 million. Revenue was primarily generated from patient service fees, histology services, and medical director fees.

Operating expenses for the first quarter of 2026 were $5.0 million, compared with $4.5 million in the first quarter of 2025.

Direct costs and expenses for the first quarter of 2026 were $0.9 million, down 32% from $1.4 million in the prior-year period, primarily due to targeted strategic actions implemented in March 2025. Research and development expenses decreased 5% year-over-year to $350,000, reflecting lower employee compensation and lab supply costs. Clinical development expenses rose to $334,000, driven by higher professional fees supporting the Company’s longitudinal clinical trial strategy.

Selling, general and administrative expenses were $3.2 million for the first quarter of 2026, up from $2.5 million in the same period last year. The increase was primarily driven by higher employee compensation related to administrative and sales functions, reflecting the addition of personnel and support services to scale the commercialization of CyPath Lung.

Net loss for the quarter ended March 31, 2026, was $3.6 million, or $(0.81) per share, compared with a net loss of $2.7 million, or $(4.80) per share, for the first quarter of 202

Cash and cash equivalents as of March 31, 2026, were $3.1 million, compared with $6.4 million as of December 31, 2025.

About CyPath Lung

CyPath Lung by bioAffinity Technologies is a noninvasive test designed to improve the early detection of lung cancer in patients at high risk for the disease. CyPath Lung uses advanced flow cytometry and proprietary artificial intelligence (AI) to identify cell populations in patient sputum that indicate malignancy. CyPath Lung incorporates a fluorescent porphyrin that is preferentially taken up by cancer and cancer-related cells. In a clinical trial of high-risk patients, CyPath Lung demonstrated 92% sensitivity, 87% specificity, 88% accuracy and 99% negative predictive value (NPV) in detecting lung cancer in patients at high risk for the disease who had small indeterminate lung nodules less than 20 millimeters. The high NPV gives physicians greater confidence that a negative result is truly negative, potentially sparing patients from unnecessary invasive and costly procedures. CyPath Lung is marketed as a Laboratory Developed Test (LDT) and is not intended for use as a sole diagnostic tool and should be considered alongside other clinical findings.

(Press release, BioAffinity Technologies, MAY 8, 2026, View Source [SID1234665383])

On May 8, 2026 Alpha Tau Medical Ltd. (Nasdaq: DRTS, DRTSW) ("Alpha Tau", or the "Company"), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT reported that it will host a conference call and webcast on Monday, May 11, 2026 at 8:30am ET to discuss interim clinical data from the first three patients treated in its U.S. trial of Alpha DaRT for patients with recurrent glioblastoma (GBM), also known as the REGAIN (Recurrent Glioblastoma Alpha-DaRT Intratumoral Therapy) trial.

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Conference Call and Webcast Details

Date: Monday, May 11, 2026
Time: 8:30am ET
Web Access: View Source
An archived webcast will be available following the event.

About the REGAIN Study

The clinical trial is expected to enroll up to ten U.S. patients with recurrent glioblastoma not amenable for surgical resection who have undergone a prior course of central nervous system radiation. The primary objective of the study is to evaluate the feasibility and safety of the treatment, following the Company’s promising results from pre-clinical studies. Additional information about the trial can be found at View Source

(Press release, Alpha Tau Medical, MAY 8, 2026, https://www.globenewswire.com/news-release/2026/05/08/3291131/0/en/alpha-tau-to-host-conference-call-to-discuss-interim-results-from-first-three-patients-treated-for-recurrent-glioblastoma.html [SID1234665399])