On April 27, 2026 Siren Biotechnology reported the publication of its first peer-reviewed manuscript in Molecular Therapy Oncology, alongside upcoming presentations at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting.

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The publication reports preclinical data supporting Siren’s Universal AAV Immuno-Gene Therapy platform and its application to high-grade glioma, a setting with significant unmet need and limited therapeutic options.

Publication Highlights Platform Potential and Translational Depth

The study demonstrates that localized delivery of AAV-encoded cytokines enables sustained intratumoral expression and drives robust anti-tumor activity across multiple preclinical models of high-grade glioma, including human organoids and orthotopic in vivo systems.

Across these studies, treatment was associated with tumor regression and prolonged survival, alongside evidence of tumor-localized immune activation and transcriptional reprogramming.

The study includes extensive in vivo validation across multiple orthogonal glioma models, representing one of the most comprehensive preclinical datasets reported to date for AAV-based approaches in oncology.

The publication also establishes a foundation for the continued clinical development of Siren’s platform in high-grade glioma.

"This paper represents the most complete view to date of how our platform performs across systems that matter for translation," said Nicole K. Paulk, PhD, Founder and CEO of Siren Biotechnology. "We were deliberate about building a dataset that goes beyond a single model or readout, and instead shows reproducible consistency across orthogonal approaches. That level of rigor is important as we continue advancing into the clinic."

Access the full open-access manuscript here.

Upcoming ASGCT (Free ASGCT Whitepaper) 2026 Presentations

Siren Biotechnology will present new data spanning preclinical and CMC advances at the ASGCT (Free ASGCT Whitepaper) 2026 Annual Meeting in Boston, MA.

MRI-Guided Convection-Enhanced Delivery of an AAV-hIFNβ Vector Achieves Targeted Brain Retention and Durable Transgene Expression in a Large-Animal Model

Wednesday, May 13, 2026, 5:00 – 6:30 pm EST, Poster Hall, Abstract #2241

End-to-End Device Compatibility and In-Use Stability Assessment of an AAV Gene Therapy Delivered by Convection-Enhanced Delivery

Friday, May 15, 2026, 8:15 – 8:30 am EST, MCEC Room 162AB

"Together, these presentations extend the platform story into delivery, manufacturability, and real-world use considerations," added Dr. Paulk. "We’re excited to share data that we believe helps define what effective AAV-based therapies in oncology can look like."

This research was additionally made possible by funding from the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, gene therapy research, and clinical trials (Grant number: TRAN1-15325).

(Press release, Siren Biotechnology, APR 27, 2026, View Source [SID1234664826])

On April 27, 2026 Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, reported positive preliminary Phase 1 dose escalation data for its potentially best-in-class, pan-RAS molecular glue ERAS-0015 in patients with RAS-mutant solid tumors.

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The preliminary data are from Erasca’s ongoing AURORAS-1 Phase 1 dose escalation trial in the U.S. and the ongoing JYP0015M101 Phase 1 dose escalation trial in China sponsored by Joyo Pharmatech Co., Ltd. (Joyo), both evaluating ERAS-0015 in patients with RAS-mutant solid tumors.

"We are thrilled with the robust efficacy results demonstrated so far by our pan-RAS inhibitor ERAS-0015 in patients with lung and pancreatic cancer," said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. "The magnitude of clinical benefit seen during dose escalation is particularly striking and compares favorably with other pan-RAS, pan-KRAS, or KRAS-mutant selective inhibitors. This efficacy is accompanied by generally well-tolerated safety results, primarily characterized by manageable, low-grade adverse events. Notably, preliminary data support ERAS-0015 may be combined with standard-of-care doses of panitumumab, positioning it as a potential backbone therapy for future combination regimens. Together, we believe these findings support the best-in-class potential of ERAS-0015, and we look forward to continued progress in our Phase 1 monotherapy dose expansion cohorts and combination dose escalation cohorts."

Highlights of Phase 1 Preliminary Results

Pharmacokinetics (PK):
Well-behaved PK, with dose-dependent increase in PK exposure up to the maximum administered dose (MAD) of 40 mg once daily (QD) and no exposure plateau observed
Pharmacologically active dose (PAD) range of 16-32 mg QD defined based on mean steady-state average exposures that exceeded target exposure threshold (based on the insensitive xenograft model)
Pharmacodynamics (PD):
Substantial reductions in KRAS G12X circulating tumor DNA (ctDNA) were observed at the PAD doses (16-32 mg QD), with 100% of patients (14/14) showing at least 75% reduction in KRAS G12X variant allele fraction, including 5 out of 14 patients showing 100% reduction
Efficacy: Robust monotherapy overall response rates (ORR) in patients with KRAS G12X non-small cell lung cancer (NSCLC) and with KRAS G12X pancreatic cancer (PDAC), in each case as of the relevant data cutoff (DCO)1,2:
NSCLC
At PADs of 16-32 mg QD, 62% uORR8wk (N=37) in second line or greater (2L+) KRAS G12X NSCLC, which exceeded comparator by 24 percentage points3,4
At PADs of 16-32 mg QD, 75% uORR8wk (N=16) in post-ICI/platinum (2/3L) KRAS G12X NSCLC, which exceeded comparator by 37 percentage points3,4
At recommended doses for expansion (RDEs) of 24-32 mg QD, 64% uORR8wk (N=25) in 2L+ KRAS G12X NSCLC3
PDAC
At PADs of 16-32 mg QD, 40% uORR14wk (N=20) in 2L KRAS G12X PDAC, which exceeded comparator by 11 percentage points5,6
At RDEs of 24-32 mg QD, 42% uORR14wk (N=12) in 2L KRAS G12X PDAC, which exceeded comparator by 13 percentage points5,6
At RDE of 32 mg QD, 50% uORR14wk (N=2) in 2L KRAS G12X PDAC, which exceeded comparator by 15 percentage points5,7
Multiple ongoing responses: Nearly all responding patients—including all unconfirmed responders—remain on treatment as of the DCO:
NSCLC
23 out of 24 responding patients remain on treatment, including all responders treated at 24-32 mg QD RDEs
PDAC
20 out of 23 responding patients remain on treatment, including all responders treated at 24-32 mg QD RDEs
Safety and Tolerability: Generally well-tolerated with mostly low-grade adverse events (AEs), no dose-limiting toxicities (DLTs), low rate of dose interruptions or reductions due to treatment-related adverse events (TRAEs), and no discontinuations due to TRAEs
Monotherapy RDE: Based on the totality of the preliminary Phase 1 dose escalation data, 24 mg and 32 mg QD were selected as the go-forward monotherapy RDEs
Combinability: ERAS-0015 showed promising clinical potential to combine with panitumumab (anti-EGFR monoclonal antibody)
No DLTs observed through the 31Mar2026 DCO (N=3) with 1 unconfirmed partial response (uPR) in 1 efficacy-evaluable patient with metastatic colorectal cancer (CRC)
1 AURORAS-1 data cutoff (DCO) 4Apr2026; JYP0015M101 data cutoff (DCO) 27Feb2026
2 Pooled data from the Company’s Phase 1 trial (US trial, or AURORAS-1) and Joyo’s Phase 1 trial (China (CN) trial, or JYP0015M101) of ERAS-0015
3 The uORR8wk is the ORR (confirmed and unconfirmed responses) for patients who received first dose of ERAS-0015 at least 8 weeks prior to data cutoff date (US trial) or at least one post-dose tumor assessment (CN trial)
4 Comparator as used in this press release, RMC-6236. Punekar et al. Journal of Thoracic Oncology 2025; data cutoff (DCO) 30Sep2024
5 The uORR14wk is the ORR (confirmed and unconfirmed responses) for patients who received first dose of ERAS-0015 (US, CN) at least 14 weeks prior to data cutoff date
6 Wolpin et al. EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) 2024; DCO 23Jul2024
7 Revolution Medicines Press Release (10Sep2025); data cutoff (DCO) 30Jun2025

Key Upcoming and Completed Milestones
The Company initiated ERAS-0015 monotherapy expansion and combination dose escalation cohorts in the US (in Q2 2026 and Q1 2026, respectively), both ahead of previous guidance.

Upcoming milestones include:

AURORAS-1: Phase 1 trial for ERAS-0015 (pan-RAS molecular glue) in patients with RAS-mutant solid tumors
Monotherapy expansion data and combination dose escalation data narrowed to an expected date of H1 2027
BOREALIS-1: Phase 1 trial for ERAS-4001 (pan-KRAS inhibitor) in patients with KRAS-mutant solid tumors
Preliminary Phase 1 monotherapy data expected in the second half of 2026
Initiation of monotherapy expansion cohorts and combination dose escalation cohorts planned for 2027
Conference Call and Webcast Information
Erasca will hold a conference call and webcast Monday, April 27, 2026, at 4:30 pm ET. The webcast link for the conference call is here. The dial-in number is 1-877-407-3982 (U.S./Canada) or 1-201-493-6780 (international) or click the Call me Link. The live webcast and replay may be accessed by visiting Erasca’s website at Erasca.com/events.

About ERAS-0015
ERAS-0015 is an investigational, oral, highly potent pan-RAS molecular glue designed to inhibit RAS signaling with a potential best-in-class profile. Erasca is evaluating ERAS-0015 in the AURORAS-1 Phase 1 trial in patients with RAS-mutant solid tumors. Early dose escalation data in AURORAS-1 demonstrated favorable safety and tolerability results, well-behaved, linear PK, and confirmed and unconfirmed partial responses in multiple patients across multiple tumor types with different RAS mutations, including confirmed and unconfirmed partial responses at doses as low as 8 mg once daily (QD). ERAS-0015 is also designed to prevent resistance against mutant-selective inhibitors through inhibition of RAS wildtype variants. In addition, ERAS-0015 has demonstrated favorable absorption, distribution, metabolism, and excretion (ADME) and pharmacokinetic (PK) properties in multiple animal species.

About ERAS-4001
ERAS-4001 is an investigational, oral, highly potent, and selective pan-KRAS inhibitor with a potential first-in-class and best-in-class profile. Erasca is evaluating ERAS-4001 in the BOREALIS-1 Phase 1 trial in patients with KRAS-mutant solid tumors. ERAS-4001 demonstrated favorable preclinical in vitro potency against KRAS G12X mutations as well as KRAS wildtype amplifications, which may limit treatment resistance mediated through KRAS wildtype activation. No activity was observed for ERAS-4001 against HRAS or NRAS wildtype proteins in preclinical studies, which may enable a better therapeutic window compared to pan-RAS inhibitors. ERAS-4001 showed potent activity against both GTP-bound (active state) and GDP-bound (inactive state) KRAS with single digit nanomolar IC50s. In vivo, ERAS-4001 induced tumor regression in multiple KRAS-mutant models. In preclinical studies, ERAS-4001 showed encouraging ADME and PK properties.

(Press release, Erasca, APR 27, 2026, View Source [SID1234664825])

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])