AskBio Highlights Six Presentations at ASGCT 29th Annual Meeting

AskBio to Present New Gene Therapy and AAV Manufacturing Research at ASGCT 2026

AskBio, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has announced that it will present six scientific sessions at the ASGCT 29th Annual Meeting taking place from May 11 through May 15, 2026.

The presentations will highlight advances in investigational gene therapies, cardiovascular research, and adeno-associated virus (AAV) manufacturing science, while also showcasing new analytical and production technologies developed by Viralgen, AskBio’s manufacturing-focused subsidiary.

The company stated that its oral presentations and scientific posters will provide updates on several key research programs spanning heart failure gene therapy, vector manufacturing optimization, genome integrity analysis, and process scalability.

Gene therapy continues to represent one of the fastest-growing areas within biotechnology, with researchers increasingly focusing on the use of viral vectors such as recombinant adeno-associated viruses to deliver therapeutic genes directly into patient cells. However, scaling these therapies from experimental studies into commercially viable products remains a major scientific and manufacturing challenge.

AskBio’s participation at ASGCT 2026 reflects the company’s broader strategy of combining therapeutic innovation with advances in manufacturing science and analytical quality control.

One of the central highlights of the meeting will occur on Friday, May 15, when Mansuo Shannon co-chairs a scientific symposium titled “Gene Therapy for the Heart: Updates on Clinical Trials.” The symposium was organized by the Cardiovascular Gene and Cell Therapy Committee and will focus on recent developments in cardiovascular gene therapy research.

During the session, Shannon will present an update on an investigational protein phosphatase inhibitor-based gene therapy designed for congestive heart failure.

Heart failure remains one of the leading causes of hospitalization and mortality worldwide, affecting millions of patients and creating significant healthcare burdens. Conventional therapies can improve symptoms and slow disease progression for some individuals, but many patients continue to experience worsening cardiac function despite treatment.

Gene therapy approaches are increasingly being explored as potential strategies to address underlying molecular abnormalities associated with heart failure and other cardiovascular diseases. By targeting pathways involved in cardiac muscle function and cellular signaling, researchers hope to develop therapies capable of producing more durable clinical benefits.

AskBio’s cardiovascular research portfolio has become an important focus area for the company as it expands beyond rare neuromuscular and neurodegenerative disorders into broader therapeutic categories.

Another major oral presentation at ASGCT will focus on intracoronary cardiotropic AAV gene therapy for advanced heart failure patients. The session, presented by Luke Roberts, will discuss findings related to safety, efficacy, and immune response profiles observed during a Phase 1 clinical trial.

The presentation is expected to provide additional insights into how AAV-based cardiovascular gene therapies behave in human patients and how immune system responses may influence treatment outcomes.

Beyond therapeutic development, AskBio and Viralgen are also emphasizing advances in manufacturing technologies intended to improve the scalability, consistency, and quality of AAV vector production.

AAV vectors are among the most widely used delivery systems in gene therapy because they can efficiently transport genetic material into cells while generally demonstrating favorable safety profiles. However, producing high-quality viral vectors at commercial scale remains technically demanding.

Variability in manufacturing processes, vector purity, genome integrity, and analytical testing can all influence product performance and regulatory approval pathways. As the gene therapy industry matures, manufacturing optimization is increasingly viewed as essential for enabling broader commercialization and patient access.

One of Viralgen’s featured presentations at ASGCT 2026 will examine a multivariate approach to small-scale model qualification in upstream AAV manufacturing. The presentation, led by Ainara Apezteguia Garcia, will focus on predictive and scalable process design methodologies.

According to the company, the approach may support improved process understanding and facilitate more reliable scaling from laboratory development into larger commercial manufacturing operations.

Another scientific poster will explore advances in genome integrity analysis using long-read sequencing technology. The presentation, delivered by Emilie Lecomte, will evaluate the repeatability and intermediate precision of a genome integrity assay for recombinant AAV products.

Long-read sequencing technologies are becoming increasingly important in gene therapy because they enable more detailed characterization of viral vector genomes and can help identify structural abnormalities or inconsistencies that may impact product quality or safety.

Viralgen will also present research involving a droplet digital polymerase chain reaction, or ddPCR, method designed to quantify residual adenovirus early region 1A (E1A) gene fragments that may remain in recombinant AAV products.

This poster presentation, led by María García, focuses on validating analytical methods and evaluating correlations between residual E1A content and total host cell DNA levels.

Residual impurities in viral vector products represent an important regulatory and manufacturing consideration because maintaining high purity standards is critical for patient safety and product consistency.

AskBio stated that these analytical and process-related advances reflect Viralgen’s commitment to improving the robustness and scientific understanding of AAV manufacturing.

Gustavo Pesquin, Chief Executive Officer of AskBio, stated that advancing gene therapy requires not only therapeutic innovation but also major improvements in manufacturing science and process control.

According to Pesquin, Viralgen continues strengthening the relationship between process development, analytical precision, and product quality within AAV production systems. He emphasized that the work presented at ASGCT 2026 reflects efforts to combine deeper process understanding with advanced analytical tools to improve quality, consistency, and confidence in AAV-based therapies.

Pesquin also noted that these efforts are important for supporting broader clinical development goals and helping unlock the therapeutic potential of gene therapy across multiple disease areas.

In addition to cardiovascular disease, AskBio continues developing a broad portfolio of investigational AAV-based gene therapies targeting several serious and often underserved medical conditions.

The company’s pipeline includes programs focused on limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, Pompe disease, and congestive heart failure.

Many of these disorders currently have limited treatment options, and gene therapy approaches are being investigated as potential strategies to address the underlying genetic or molecular causes of disease.

The ASGCT Annual Meeting is widely regarded as one of the leading global scientific conferences dedicated to gene and cell therapy research. The event brings together biotechnology companies, academic researchers, clinicians, regulators, and manufacturing experts to discuss advances in vector engineering, genome editing, translational medicine, and clinical development.

AskBio’s presence at the conference underscores both the company’s expanding scientific ambitions and the increasing importance of manufacturing innovation within the rapidly evolving gene therapy sector.

As more gene therapies progress through clinical development and toward commercialization, companies are placing greater emphasis on scalable production systems, advanced analytics, and regulatory-ready manufacturing strategies.

By presenting research spanning therapeutic development, analytical science, and process engineering, AskBio and Viralgen are positioning themselves within a growing industry effort focused on improving the reliability, scalability, and long-term viability of gene therapy platforms.

The company believes that continued progress in these areas may ultimately support the development of transformative therapies capable of benefiting millions of patients worldwide living with serious genetic, cardiovascular, neurological, and neuromuscular disorders.

About AskBio

AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to steering gene therapy into a new era where it can transform the lives of a wider range of people living with rare and more common diseases. The company maintains a portfolio of clinical programs across a range of disease indications related to a single gene or multiple factors across cardiovascular, central nervous system, and neuromuscular conditions, with a clinical-stage pipeline that includes investigational therapeutics for heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease.

AskBio’s end-to-end gene therapy platform includes our Pro10™ technology and Aava™ manufacturing platform, which make gene therapies more accessible by making research and commercial grade manufacturing more affordable. With global headquarters in Durham, North Carolina, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field with over 900 employees in five countries, the company holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at http://www.askbio.com/ or follow us on LinkedIn.

About Viralgen

Viralgen is a leading contract development and manufacturing organization (CDMO) specializing exclusively in adeno-associated virus (AAV)–based gene therapies. Founded in 2017 as a subsidiary of AskBio Inc. within the Bayer AG group, Viralgen provides end-to-end support from early development through large-scale commercial production. Leveraging its proprietary Pro10™ suspension cell line and Aava™ manufacturing platform, Viralgen achieves industry-leading, high-yield, scalable manufacturing across all AAV serotypes.

Located in San Sebastián, Spain, its state-of-the-art facility includes three cGMP suites with 2,000-liter capacity each, certified by the AEMPS/EMA, and integrates services such as plasmid production, process optimization, fill-finish, and in-house QC testing. With more than 1,500 AAV batches produced, Viralgen delivers reliable, efficient solutions that help bring life-changing gene therapies to patients with greater speed and consistency. For more information, visit https://viralgen.com/.

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses.

At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2025, the Group employed around 88,000 people and had sales of 45.6 billion euros. R&D expenses amounted to 5.8 billion euros. For more information, go to www.bayer.com.

AskBio Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding AskBio’s clinical trials.

These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines.

Its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

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