On April 27, 2026 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported that two abstracts highlighting preclinical data from its proprietary RSwitch technology, which enables the fine-tuning of transgene levels in gene and cell therapy applications, have been accepted for oral presentation at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting taking place May 11-15, 2026 in Boston, MA.

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Presentation details:

Title: Small Molecule–regulated RNA switch achieves therapeutically safe levels of Frataxin in mouse models of Friedreich’s Ataxia.
Abstract #: 69
Session Title: Neurologic diseases I
Date and Time: May 12, 2026; 10:45 – 11:00 a.m. ET
Presenter: Jon Dempersmier, Ph.D., Senior Scientist, Rgenta Therapeutics

Title: Deep-learning guided optimization of a small molecule-regulated RNA switch for titratable AAV transgene expression.
Abstract #: 1908
Session Title: Engineering programmable gene therapy systems
Date and Time: May 13, 2026; 11:15 a.m. ET
Presenter:
Ian McLachlan, Ph.D., Senior Scientist, Rgenta Therapeutics

About RSwitch
RSwitch is a proprietary regulatable gene therapy system that enables oral, small molecule drug control of transgene levels in gene and cell therapy applications. RSwitch encodes a "dimmer" switch that makes the expression of transgene dependent on the administration of an oral small molecule drug that controls the system. Only when the drug is administered is the system activated. Furthermore, the level of gene expression is dependent on how much drug is administered. This precise gene control has the potential to enable fine control of the expression of a therapeutic protein. Rgenta has demonstrated the RSwitch system’s feasibility in vitro and in vivo, achieving dose-dependent expression of transgenes following small molecule administration. RSwitch technology offers versatile control across multiple gene and cell therapy applications, and the company is actively exploring strategic partnerships.

(Press release, Rgenta Therapeutics, APR 27, 2026, View Source [SID1234664824])

On April 27, 2026 Asgard Therapeutics, a privately held biotech company pioneering in vivo direct cell reprogramming for cancer immunotherapy, reported it will present advanced preclinical data on its lead asset AT-108, a first-in-class, off-the-shelf cancer immunotherapy, in an oral presentation at the ASGCT (Free ASGCT Whitepaper) Annual Meeting, held from 11-15 May, 2026, in Boston, Massachusetts, US.

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Fábio Rosa, Co-founder and VP and Head of Research at Asgard Therapeutics, who will give the oral presentation, said: "These data demonstrate the ability of AT-108 to reprogram tumor cells in situ into antigen-presenting cells with high efficiency, systematically driving activation of tumor-specific immune responses within the tumor microenvironment. This work provides clear preclinical evidence supporting our in vivo cell reprogramming platform and represents another milestone for Asgard as we advance AT-108 toward the clinic."

Shane Olwill, Chief Development Officer at Asgard Therapeutics, said: "We are delighted to have been selected for an oral presentation at ASGCT (Free ASGCT Whitepaper), which will highlight the progress we have made advancing AT-108 – our first-in-class, off-the-shelf gene-based immunotherapy designed to trigger powerful, personalized anti-cancer responses. Our pioneering approach to tackling cancer is potentially transformative and we are excited by AT-108’s broad potential across multiple cancer types."

Asgard had previously demonstrated that intratumoral delivery of AT-018 reprograms tumor cells into cDC1-like antigen-presenting cells and works synergistically with immune checkpoint blockade (ICB) to elicit powerful anti-tumor activity.

This new study, being presented at ASGCT (Free ASGCT Whitepaper) 2026, advances AT-108 across four key dimensions. It demonstrates AT-108’s ability to induce systemic anti-tumor immunity in mouse models leading to abscopal effect and regression on non-treated tumors in the same animals; it establishes AT-108 as the optimized clinical candidate following screening of >25 cassette variants; it defines dosing and treatment parameters required for durable responses; and it identifies pharmacodynamic biomarkers associated with reprogramming and immune activation.

Further highlights of the study include that:

In combination with ICB, Asgard’s approach induced abscopal effects and long-term tumor-free survival in the B16 mouse syngeneic model. These results were associated with increased T cells and NK cells, and reduced regulatory T cells, in both injected and non-injected tumors.
When used as a monotherapy, AT-108 doubled median survival in B16 and YUMM1.7 mouse models, and induced regression in ID8 ascites mouse model.
When used in combination with ICB, AT-108 achieved 50% complete response (CR) in B16, and extended survival in the PANC02 immunosuppressed mouse model.
Cell transduction peaked 1-2 days post injection and persisted for up to 9-15 days, supporting re-dosing every two days to sustain transduction. A three-dose lead cycle was required to achieve CRs, with maintenance dosing improving durability.
Regarding biomarkers, tumor and peripheral blood analyses identified pharmacodynamic signatures associated with AT-108 activity, including increased cytotoxic T cells, expansion of follicular helper T cells and enrichment of dendritic cells.

The abstract is available to view via the ASGCT (Free ASGCT Whitepaper) interactive program here.

The positive study results come as Asgard progresses AT-108 toward clinical development with a focus on solid tumors, by advancing IND-enabling studies and CMC development.

Details of the oral presentation are as follows:

Presentation title: Optimized in situ tumor-to-dendritic cell reprogramming by AT-108 adenoviral vector drives local and systemic antitumor immunity
Presenter: Fabio Rosa, Asgard Therapeutics
Presentation session: Cancer vaccines and oncolytic viruses I
Session date and time: 13 May 2026, 03:30 PM – 05:00 PM EDT
Location: MCEC Room 162AB (Level 1)
Presentation Time: 04:30 PM – 04:45 PM EDT

(Press release, Asgard Therapeutics, APR 27, 2026, View Source [SID1234664823])

On April 27, 2026 Umoja Biopharma, Inc. (Umoja), a clinical-stage biotechnology company committed to delivering innovative and potentially curative immunotherapies for patients with cancer and autoimmune diseases, reported the acceptance of a poster presentation for UB-VV500, its dual-targeted BCMA x GPRC5D in vivo CAR T cell program for the treatment of multiple myeloma, at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting. Preclinical data for UB-VV500 will be presented in a scientific poster at the conference.

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"Development in multiple myeloma is moving beyond single-targeted approaches toward dual-targeting and combination strategies, particularly as patients progress through existing BCMA-directed therapies," said Andrew Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja Biopharma. "We believe the future of multiple myeloma immunotherapy will be shaped by combination products that are designed from the ground-up to more effectively address antigen escape and disease heterogeneity while aiming to deliver greater and more durable efficacy. UB-VV500 is designed to realize that vision through our in vivo CAR T cell approach."

Umoja expects to advance UB-VV500 into a Phase 1 clinical trial by the end of 2026. Initial clinical data disclosures across the company’s other clinical programs are expected to be reported at major medical meetings in the second half of 2026.

"We are encouraged by our safety and early clinical results in our three ongoing Phase 1 programs—UB-VV111 for CD19 and UB-VV400/UB-VV410 for CD22—which use the same VivoVecTM in vivo platform that UB-VV500 is built upon," Dr. Scharenberg added. "UB-VV500’s pre-clinical progress is exciting and we look forward to sharing more at ASGCT (Free ASGCT Whitepaper). Dual-targeting is the future of patient care for multiple myeloma, and we expect UB-VV500 to be at the forefront of that movement."

Details of the ASGCT (Free ASGCT Whitepaper) poster presentation can be found below:

Poster Presentation Title: Preclinical development of UB-VV500, an in vivo potency-enhanced CAR T cell product targeting BCMA and GPRC5D for multiple myeloma
Presentation Date/Time: Wednesday, May 13, 2026, 5:00 – 6:30 p.m. ET
Presenting Speaker: Christopher Nicolai, Ph.D.
Publication Number: 2046

(Press release, Umoja Biopharma, APR 27, 2026, View Source [SID1234664822])

On April 27, 2026 MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ: MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), reported that a study conducted by researchers at the Spanish National Cancer Research Centre (CNIO) has identified macrophage migration inhibitory factor (MIF)–mediated reprogramming of CD74‑positive microglia and macrophages as a central vulnerability in brain metastasis. The research, recently published in the peer‑reviewed journal "Cancer Research" (March 2026), demonstrates pharmacological modulation of this pathway using the brain‑penetrant small molecule ibudilast.

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The work, led by Manuel Valiente PhD, head of CNIO Brain Metastasis group, and colleagues at CNIO, shows that tumor‑derived MIF alters the functional state of microglia and infiltrating macrophages in the brain, converting them from a potentially protective role into a pro‑metastatic one. In multiple experimental models and fresh patient‑derived brain metastasis samples, inhibition of the MIF–CD74 signaling axis significantly reduced metastatic progression. Importantly, the investigators also identified secreted MIF as a candidate liquid biopsy biomarker detectable in cerebrospinal fluid, supporting a translational, biomarker‑guided clinical strategy.

The study further demonstrates that ibudilast can effectively block MIF–CD74 signaling, reverse pro‑metastatic immune reprogramming, and suppress brain metastasis growth in preclinical systems. In addition, transcriptomic analyses define predictive biomarker signatures associated with treatment response, reinforcing the potential for patient stratification in future clinical studies. The findings suggest translational potential for MN-166 (ibudilast), the company’s leading product, in future therapeutic strategies for brain metastasis within neuro-oncology.

MediciNova plans to collaborate with Dr. Valiente and CNIO on future clinical research aimed at patients with solid tumors having brain metastases.

Dr. Valiente commented on the findings, "Brain metastases develop in up to 30% of patients with advanced solid tumors, most commonly arising from lung cancer, breast cancer, melanoma, and colorectal cancer, and remain an area of substantial unmet medical need. Despite recent advances in systemic therapies, patients with brain metastases have historically been excluded from many clinical trials, limiting progress in the development of targeted treatments. By focusing on brain‑specific immune–microenvironment interactions rather than tumor‑intrinsic alterations alone, the CNIO findings open a new therapeutic avenue that may be applicable across multiple primary tumor types."

"Brain metastasis represents one of the most urgent and challenging frontiers in oncology," said Dr. Kazuko Matsuda, Chief Medical Officer. "The publication of this work in Cancer Research provides strong mechanistic and translational rationale to pursue biomarker‑driven clinical strategies. We hold granted patents covering MN‑166 for preventing and minimizing cancer metastasis across multiple solid tumor types, including pancreatic, lung, breast, colorectal and ovarian cancers, as well as melanoma. Our focus is now on advancing future clinical investigations and responsibly translating these insights into studies designed for patients with brain metastases."

The full study, "MIF‑Induced CD74+ Microglia and Macrophages Promote Progression of Brain Metastasis and Are Clinically Relevant Across Central Nervous System Disorders," is available online in Cancer Research. (View Source )

(Press release, MediciNova, APR 27, 2026, View Source [SID1234664821])

On April 27, 2026 Janux Therapeutics, Inc. (Nasdaq: JANX) ("Janux"), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies, reported that it will discontinue further clinical development of JANX008, its EGFR-targeted Tumor Activated T Cell Engager (TRACTr) program.

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Following completion of the Phase 1a portion of the study, which included dose escalation and expansion cohorts across multiple solid tumor indications, and an internal review of the data, the Company has determined to prioritize development resources toward other pipeline opportunities. The decision to discontinue JANX008 is program-specific and does not impact the Company’s broader TRACTr platform strategy. As part of its disciplined portfolio prioritization process, the Company evaluated the JANX008 dataset against predefined development criteria. While durable responses were observed in select patients through extended follow-up, the overall magnitude and consistency of activity were not sufficient to support continued development relative to other pipeline programs.

The study also generated insights relevant to the broader TRACTr platform. The occurrence of cytokine release syndrome (CRS) was infrequent and primarily limited to Grade 1, enabling Safety Review Committee approval for outpatient dosing. In addition, JANX008 demonstrated a differentiated tolerability profile relative to conventional EGFR-targeted therapies, with minimal gastrointestinal, dermatologic, and subcutaneous adverse events typically associated with EGFR antibodies and tyrosine kinase inhibitors. These findings, together with the ability to dose beyond the limitations of conventional T cell engagers, support the potential of the TRACTr platform to improve safety. While musculoskeletal adverse events were dose-limiting, reflecting constraints associated with the EGFR target, the TRACTr format enabled a sufficient therapeutic window to assess clinical activity across a range of doses.

"Our decision to discontinue JANX008 reflects the disciplined approach we take to advancing our pipeline," said David Campbell, Ph.D., President and Chief Executive Officer of Janux. "We evaluate each program against a high bar for safety, activity, and differentiated profile. We prioritize resources toward programs that meet these criteria and offer opportunities to deliver best-in-class outcomes."

"The JANX008 study enabled a rigorous evaluation of activity for this EGFR-targeted TRACTr construct," said William Go, M.D., Ph.D., Chief Medical Officer of Janux. "We observed objective responses and disease control across treated patients. These findings provide important insight into how target biology and masking strategy define the therapeutic window and inform the continued advancement of our pipeline."

(Press release, Janux Therapeutics, APR 27, 2026, View Source [SID1234664820])