On April 21, 2025 BostonGene, a leader in AI-driven molecular and immune profiling that accelerates drug development and personalizes patient care, reported that four abstracts have been selected for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, held from April 25 – 30, at McCormick Place Convention Center in Chicago, IL (Press release, BostonGene, APR 21, 2025, View Source [SID1234652005]). BostonGene will be exhibiting at booth #2452.

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Ferran Prat, PhD, JD, BostonGene’s Chief Commercial Officer, will also speak at the AACR (Free AACR Whitepaper) educational session, Academic Entrepreneurship: Getting Your Discovery to Patients on April 26 from 8:00 AM – 9:30 AM. The session will address key topics such as understanding intellectual property rights and patents, raising capital and developing commercialization pathways. By bridging these knowledge gaps, the session seeks to empower researchers to move innovations from the laboratory to patient care.

BostonGene is also proud to partner with the American Cancer Society to host the BrightEdge Entrepreneurs (BEE) Dinner at AACR (Free AACR Whitepaper) 2025, held on April 28 in Chicago. The event will spotlight eight innovative cancer-focused startups from the BEE Program, which empowers underrepresented scientific entrepreneurs with mentorship, investor access and early-stage funding.

In collaboration with leading academic institutions, BostonGene will present new research that highlights the power of its AI-enabled, integrated multiomic platform to drive precision oncology. By leveraging comprehensive genomic, transcriptomic and immune system profiling, the platform enables high-resolution tumor characterization, molecular subtyping, and immune landscape reconstruction—delivering clinical and mechanistic insights to optimize drug trials and clinical care. From refining transcriptional subtypes in small cell lung cancer and predicting treatment response in triple-negative breast cancer to improving germline variant interpretation and advancing biomarker detection in pan-cancer analyses, these studies demonstrate the platform’s utility in both clinical care and translational research. In addition, the studies also highlight its role in accelerating biomarker-informed drug development.

Details of BostonGene’s poster presentations at the AACR (Free AACR Whitepaper) Annual Meeting are below:

Abstract number: 1101
Title: A novel machine learning classifier for SCLC transcriptional subtypes
Date and time: Sunday, April 27 | 2:00 PM – 5:00 PM
Presenter: Carl Gay, MD, PhD, MD Anderson Cancer Center

In this study, BostonGene applied gradient-boosting machine learning models to refine the small cell lung cancer (SCLC) subtypes developed by Gay et al. (The University of Texas MD Anderson Cancer Center). The model demonstrated high performance metrics during validation, reinforcing its potential clinical impact.

Research done in collaboration with MD Anderson Cancer Center

Abstract number: 5308
Title: Pan-cancer analysis of TRIM37 copy-number and development of fit-for-screening in situ hybridization tools
Date and time: Sunday, April 27 | 2:00 PM – 5:00 PM
Presenter: J.D. Schonhoft, PhD, Repare Therapeutics

BostonGene’s WES platform was used to examine the copy numbers and RNA levels of TRIM37 across over 12,000 adult solid tumor samples. Newly developed DNA- and RNA-based in situ hybridization (ISH) approaches uncovered high TRIM37 expression in many patients, underscoring the need for sensitive ISH methods for examining clinical biomarkers.

Research done in collaboration with Repare Therapeutics

Abstract number: 2036
Title: Transcriptomic immune signatures and reconstructed immune cell types associated with pathological complete response to neoadjuvant chemotherapy in triple-negative breast cancer
Date and time: Monday, April 28 | 9:00 AM – 12:00 PM
Presenter: Tomohiro Oshino, MD, Hokkaido University Hospital

BostonGene’s tumor microenvironment (TME) subtyping and cell deconvolution were used for TME reconstruction in triple-negative breast cancer (TNBC). The findings revealed patients with immune-enriched TME were more likely to achieve a pathological complete response following neoadjuvant chemotherapy, underscoring the potential of TME-based analytical tools to predict treatment response in TNBC.

Research done in collaboration with Hokkaido University Hospital

Abstract number: 5047
Title: Enhancing germline variant calling: Adjustment with tumor samples to filter low-confidence variants from clonal hematopoiesis
Date and time: Tuesday, April 29 | 9:00 AM – 12:00 PM
Presenter: Artur Baisangurov, MS, BostonGene

Leveraging whole-exome sequencing (WES) analysis for 2,078 cancer patients, BostonGene developed a filtering method to improve the accuracy of germline reports. This novel approach identified artifacts caused by molecular and biological variations to streamline quality control of genetic reports for cancer patients.

The abstracts will be published in an online-only Proceedings supplement to the AACR (Free AACR Whitepaper) journal Cancer Research.

On April 21, 2025 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono") reported that it has entered into a research collaboration agreement with Jorna Therapeutics (Headquarters: Cambridge, Massachusetts, USA; CEO: Chengwei Luo; "Jorna") in December 2024 aimed at drug discovery using Jorna’s proprietary ribonucleic acid (RNA) editing platform (Press release, Ono, APR 21, 2025, View Source [SID1234652004]). Having completed the validation of this platform, Ono has begun the search for nucleic acid sequences that could serve as drug candidates.

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Under the agreement, Jorna will design RNA editing drug sequences using its proprietary protein and RNA generative AI model, which is based on the quantum mechanics-based AI called SkyEngine. Ono will have an exclusive option right to discover, develop, and commercialize drug candidates generated by the sequences designed by Jorna worldwide. In addition to the upfront payment already made to Jorna, Ono will also pay for research funding and milestone payments based on the progress of the research.

"We highly value Jorna’s unique generative AI technologies, which combine large-scale amino acid sequence information and language models to design desired proteins," said Seishi Katsumata, Corporate Officer / Executive Director, Discovery & Research of Ono. "Through this partnership, we aim to accelerate drug development using RNA editing technology and provide new treatment options to patients around the world with unmet medical needs."

"We are delighted to collaborate with Ono to advance our shared vision of delivering groundbreaking therapies to patients. This partnership is expected to accelerate efforts to bring RNA editing-based therapies to market by leveraging Ono’s global resources and expertise," said Chengwei Luo, Founder and CEO of Jorna.

On April 21, 2025 Ferring Pharmaceuticals reported the first results from an ongoing Phase 3 trial in Japan assessing the efficacy and safety of nadofaragene firadenovec in patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1) (Press release, Ferring Pharmaceuticals, APR 21, 2025, View Source [SID1234652003]). Nadofaragene firadenovec is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) in this patient population.2 The product is given once every three months.

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The data – presented at the 112th Annual Meeting of the Japanese Urological Association (JUA 2025) held April 17-19 in Fukuoka, Japan – showed a complete response (CR) rate at three months of 75% (15/20) among Japanese patients with CIS ± high-grade Ta/T1.1 Importantly, these results were achieved following a single quarterly administration, representing a significant clinical advantage. The efficacy appears higher than previously reported in the US Phase 3 trial3,4 and adds to a growing body of evidence. Recent real-world data was presented by the Mayo Clinic and showed 79% CR rate,2 demonstrating consistent efficacy and safety when patients are treated with nadofaragene firadenovec. Patients who responded at the three-month assessment received continued every three months doses until disease recurrence. These data will be presented at a future congress. Within this conservatively designed protocol, a replication of CS-003 US Phase 3 trial, patients who did not respond at three months were not offered a re-induction dose. This contrasts with a growing trend in other NMIBC trials, where re-induction is included in the study protocols for patients who do not achieve a response at initial assessment. Re-induction is now being explored with nadofaragene firadenovec in other trials.

"When BCG therapy is ineffective, patients are forced to choose invasive surgery, i.e., total bladder removal, but nadofaragene firadenovec may provide a new treatment option," noted Professor Keiji Inoue, M.D., Ph.D., Department of Urology, Kochi Medical School. "These findings are particularly significant for Japanese patients, as our treatment options have been more limited compared to other regions. The ability to achieve such promising results represents an important advancement for our clinical practice."

Overall, 80% of participants (16 patients) experienced drug-related adverse events (AEs), with 76 total AEs recorded. All reported AEs were either Grade 1 (64 AEs in 15 participants, 84.2%) or Grade 2 (12 AEs in 5 participants, 15.8%). No Grade 3, 4, or 5 AEs were reported, demonstrating a clinically manageable and tolerable safety profile. The data from Japan add to the growing body of evidence, including recent real-world data presented by the Mayo Clinic,2 demonstrating favorable efficacy and safety when patients are treated with nadofaragene firadenovec.

"At Ferring, we are committed to meeting the unmet needs in bladder cancer care and equipping uro-oncologists with critical evidence they need to deliver effective, and life-changing treatment," said Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals. "These new Phase 3 findings affirm the safety profile of nadofaragene firadenovec, demonstrating a three-month efficacy that is higher than previously reported in our Phase 3 clinical trial, and complements results from an ongoing independent real-world study presented earlier this year. Collectively, the data are broadening our understanding of the value that nadofaragene firadenovec offers, furthering our journey to establish nadofaragene firadenovec as the new standard of care and backbone therapy across the urothelial cancer disease spectrum."

Bladder cancer is the 9th most common cancer worldwide by incidence, with approximately 614,000 new cases diagnosed each year.5 Non-muscle invasive bladder cancer (NMIBC) accounts for about 75% of all newly diagnosed bladder cancers.6 For patients who do not respond to BCG treatment, current options are limited, often leading to invasive surgery involving complete bladder removal. The strong efficacy demonstrated in this Japanese patient population suggests nadofaragene firadenovec could address a critical unmet need in the bladder cancer treatment landscape, delaying radical cystectomy and preserving quality of life for thousands of patients annually.

The ongoing Phase 3 Japanese trial is evaluating the efficacy and safety of nadofaragene firadenovec in two cohorts of high-risk BCG-unresponsive NMIBC patients: those with CIS ± HG Ta/T1, and patients with papillary tumors (Ta/T1) only. Results from the cohort with CIS ± HG Ta/T1 were presented on April 19 at JUA 2025.1

About nadofaragene firadenovec

Nadofaragene firadenovec (marketed as ADSTILADRIN in the US) is the first and only FDA-approved intravesical non-replicating gene therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against cancer.

Nadofaragene firadenovec has been studied in a clinical trial programme that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).3

Final 60-month follow-up data demonstrated an 80% overall survival rate and 49% cystectomy-free survival rate in adult patients with high-risk BCG-unresponsive NMIBC with CIS with or without papillary tumors (±Ta/T1), and in patients with high-grade Ta/T1 without CIS5. Most treatment emergent AEs (AEs) at five years were transient Grade 1 or 2 (66% of all patients studied), and <4% of patients experienced Grade 3 AEs with the most common being discharge around the catheter during instillation, fatigue, bladder spasm, urgency to urinate, chills, dysuria, pyrexia, and urinary incontinence. There were no Grade 4 or 5 AEs, no treatment-related deaths, and no new safety signals reported with long-term follow-up.

Ferring is leading the future in uro-oncology treatment with nadofaragene firadenovec at the centre, while expanding access with the support of new, state-of-the-art manufacturing facilities.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body. Bladder cancer is the ninth most commonly diagnosed cancer globally. 75% of bladder cancers present as NMIBC.7 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard of care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.7 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).

On April 21, 2025 DAAN Biotherapeutics, a leading innovative drug development company specializing in T-Cell receptor (TCR)-based therapies, reported that it has signed an exclusive licensing agreement with GC Cell, a gene and cell therapy firm, for the tumor antigen-specific antibody sequence (Press release, DAAN Bio Therapeutics, APR 21, 2025, View Source [SID1234652002]). The agreement grants GC Cell exclusive rights to utilize DAAN Biotherapeutics’ antibody sequence in the research and development of CAR-T (chimeric antigen receptor T cell) and CAR-NK (chimeric antigen receptor natural killer cell) therapies.

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The antibody at the center of the agreement targets a tumor antigen that is highly overexpressed in major solid cancers, including lung and colorectal cancers, and this antigen has continually been used by several pharmaceutical companies in the development of next-generation cancer therapies. The antibody provided by DAAN Biotherapeutics offers enhanced specificity compared to existing antibodies, allowing itself to be positioned as a promising candidate for cell therapies. This increased precision is expected to significantly reduce side effects while improving therapeutic outcomes, making it a more effective option than existing treatments. The combination of GC Cell’s advanced CAR cell-therapy technology and DAAN Biotherapeutics’ antibody technology is expected to significantly enhance the effectiveness of CAR cell therapies.

The licensing deal includes an upfront payment, milestone payments tied to development and commercialization stages, and royalties based on future sales. Specific financial and contractual details remain confidential to safeguard proprietary technologies and business strategies. Both companies view this collaboration as a pivotal step toward the commercialization of cell therapies for the treatment of solid tumors.

Byoung-Chul Cho, MD, the CEO of DAAN Biotherapeutics, stated, "We will continue to innovate our technologies to develop cancer treatments that will change the lives of patients."

On April 21, 2025 Acepodia (6976: TT) reported the presentation of new preclinical data highlighting its proprietary dual-payload antibody-drug conjugate (AD2C) for hepatocellular carcinoma (HCC) (Press release, Acepodia, APR 21, 2025, View Source [SID1234652001]). The data will be featured at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, which will take place from April 25 to April 30, 2025 in Chicago, Illinois.

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The poster presentation, titled "Development of dual-payload anti-GPC3 antibody-drug conjugate by dual-payload antibody conjugation (AD2C) platform for hepatocellular carcinoma treatment," showcases the company’s next-generation ADC generated using Acepodia’s AD2C platform. This platform allows for the conjugation of two distinct payloads to a single antibody without the need of antibody engineering or enzymatic conjugation. AD2C, carrying payloads with different mechanisms of action, offer enhanced therapeutic potency by targeting diverse cell populations in heterogeneous tumors or overcoming resistance to single-payload therapy.

Presentation Details:

Session Title: Novel Drug Delivery Technologies
Session Category: Experimental and Molecular Therapeutics
Date and Time: April 28, 2025 | 9:00 AM – 12:00 PM CDT
Location: McCormick Place Convention Center, Chicago, IL
Poster Board Number: 4 | Poster Section: 23
Abstract Number: 1785

"Acepodia’s dual-payload ADC approach is designed to address key limitations of current single-payload therapies," said Dr. Sonny Hsiao, Chairman and CEO of Acepodia. "By combining targeted delivery with enhanced cytotoxicity, this platform holds promise for more effective treatment of liver cancer and other solid tumors." AD2C platform provides a novel, antibody-engineering-free approach to generate dual-payload ADCs, offering a potential solution to tumor heterogeneity and drug resistance in cancer treatment.

The company’s AD2C pipeline is part of its broader portfolio of conjugation technologies derived from the laboratory of Nobel Laureate Dr. Carolyn Bertozzi, enabling the site-specific delivery of therapeutic payloads using bioorthogonal click chemistry.