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AskBio Announces Nine Presentations at American Society of Gene and Cell Therapy 27th Annual Meeting 2024

  • Four oral and five poster presentations demonstrate breadth and progress of AskBio’s gene therapy research, development, and clinical activities 

Research Triangle Park, N.C.– MAY 2, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver nine presentations offering insights into its adeno-associated virus (AAV) research and development, a key area of gene therapy focus for the company, at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which takes place May 7–11, 2024, in Baltimore, Maryland, USA. Presentations will focus on AAV immune-mediated responses as well as the results from the ongoing Phase 1 clinical trial of AB-1002 gene therapy in patients with advanced heart failure.

Luke Roberts, MBBS, PhD, Medical Director for Clinical Development at AskBio, will deliver an oral presentation sharing new clinical data from the company’s ongoing Phase 1 trial of AB-1002 in patients with advanced heart failure. This follows AskBio’s recent news that AB-1002 was granted FDA Fast Track Designation for the treatment of congestive heart failure (CHF). AB-1002 (also known as NAN-101) is an investigational gene therapy that has not yet received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio previously communicated that the delivery of AB-1002 was well tolerated and resulted in positive preliminary efficacy outcomes in some patients with non-ischemic CHF and may validate that the AAV2i8 vector capsid used is highly cardiotropic when administered as a single intracoronary infusion at relatively low doses.

Preliminary data from the Phase 1 trial of AB-1002 were presented at the 2023 American Heart Association Scientific Sessions in November, and AskBio began enrolling patients in its Phase 2 GenePHIT study of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms in January 2024.

AskBio’s ASGCT presence will also include key presentations showcasing the company’s continued commitment to optimizing AAV as a gene therapy, with a focus on preventing or reducing AAV immune response-related adverse events, which remains a vital area of investigation across the gene therapy treatment landscape. Attendees can look forward to an ASGCT spotlight speaker presentation on current immune modulation strategies given by Shari Gordon, PhD, Senior Director of Immunology at AskBio, on Day 3. On Day 4, Shari Gordon will deliver on behalf of Audry Fernandez, PhD, Principal Scientist & Group Lead, Immunology R&D at AskBio, an oral presentation on pre-clinical research into AAV-specific immune responses, and Liujiang Song, PhD, Principal Scientist for R&D Capsid and Biology at AskBio, will offer insights into AAV biology and vector intracellular fate during an oral presentation on AAV episome configuration using third generation long-read sequencing technologies and advanced bioinformatics.

“Our presence at ASGCT this year highlights our continued commitment to sharing AAV developments with the gene therapy community. Covering clinical and pre-clinical research, our presentations show our robust progress and ongoing ambition to bring to patients transformative therapies that were once unthinkable,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “With our clinical and early-stage programs advancing, these are exciting times at AskBio.”

With an ambitious portfolio of gene therapies at various stages of research and development, AskBio continues to develop AAV-based therapies to treat some the world’s most debilitating diseases, including CHF, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. By targeting these therapy areas, AskBio aims to deliver breakthrough treatments that could benefit more than an estimated 35 million patients worldwide.1–7

AskBio’s presentations at ASGCT include:

  • Molecular Origami: AAV Genome Recombination Patterns Mediated by ITRs in the Liver. Presented by Liujiang Song, PhD, Principal Scientist, R&D, AskBio. Saturday, May 11, 10:45 a.m. EDT.
  • Seroprevalence of Liver Tropic AAV Capsids and the Evaluation of FcRN Inhibitors to Reduce Circulating AAV Antibodies. Presented by Yash Shah, PhD, Scientist II, R&D Immunology, AskBio. Thursday, May 9, 12:00 – 7:00 p.m. EDT.
  • STING and Type-I Interferon Signaling do not Contribute to AAV Immunogenicity in Mice. Presented by Robert Junkins, PhD, Scientist III, R&D Immunology, AskBio. Wednesday, May 8, 12:00 – 7:00 p.m. EDT.
  • Modulating the AAV-Specific Immune Response: A Comparison of T Cell Co-Stimulation Blockade with Rapamycin plus B Cell Depletion. Presented by Shari Gordon on behalf of Audry Fernandez, PhD, Principal Scientist & Group Lead, R&D Immunology, AskBio. Saturday, May 11, 8.45 a.m. EDT.
  • Preclinical Modeling of Immunity in Murine Liver Following Adeno-Associated Virus Delivery: A Cross-Sectional Comparison of AAV8 and an AAV8/6 Chimeric Capsid. Presented by Alan Curtis, PhD, Scientist II, Cellular Immunology, AskBio. Thursday, May 9, 12:00 – 7:00 p.m. EDT.
  • Development of an Analytical Method for AAV Capsid Proteins for use in Stability Studies. Presented by Aitor Navarro Nieto, PhD, AAV Molecular Development, Senior Scientist, Viralgen Vector Core, Friday, May 10, 12:00 – 7:00 p.m. EDT.
  • Safety and Efficacy of AB-1002 Gene Therapy in Patients with Advanced Heart Failure: Results from an Ongoing Phase 1 Clinical Trial. Presented by Luke Roberts, MBBS, PhD, Medical Director, Clinical Development, AskBio. Wednesday, May 8, 9:15 a.m. EDT.
  • IgG Enzymatic Cleavage by Imlifidase Reduces Uptake of AAV and Activation of Phagocytic Immune Cells from Seropositive Human Donors. Presented by Blake Rust, PhD, Senior Scientist, Clinical Immunology, AskBio. Saturday, May 11, 9:30 a.m. EDT.
  • Quadplex dPCR as a Tool to Analyze Genome Integrity and Heterogeneity for Gene Therapy Drug Products. Presented by Ryan Massopust, PhD, Scientist II, Analytical Development, AskBio. Thursday, May 9, 12:00 – 7:00 p.m. EDT.

About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, 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 800 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at www.askbio.com or follow us on LinkedIn.

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 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.

About Viralgen Vector Core

Viralgen is a Contract Development and Manufacturing Organization (CDMO) founded in 2017 and exists as an independently operated subsidiary of AskBio, which is wholly owned and independently operated as a subsidiary of Bayer AG. As a manufacturer of Current Good Manufacturing Practice (cGMP) certified AAV, Viralgen offers the Pro10™ based suspension manufacturing platform, a technology licensed from AskBio and developed by Chief Technical Officer Josh Grieger, PhD, and Co-Founder R. Jude Samulski, PhD, at University of North Carolina. The Pro10™ platform has been found to increase scalability, performance, and yield of AAV therapies.8 Located in Spain, in the Gipuzkoa Science and Technology Park, Viralgen produces AAV gene therapy treatments for pharmaceutical and biotech companies with the aim of accelerating the delivery of new treatments that may improve patients’ lives.

The company’s clinical facilities have four cGMP manufacturing suites, with 250-liter and 500-liter bioreactors. In 2020, Viralgen expanded within the Scientific Park by constructing a new building with three modules for large-scale commercial manufacturing. Each module of the state-of-the-art space includes three cGMP suites with a manufacturing capacity of >2,000 liters. The first module, which includes a suite dedicated to fully automated fill and finish operations, has received cGMP certification by the Spanish Agency for Medicines and Medical Devices (AEMPS) as part of the EMA network. For more information, visit viralgenvc.com.

Bayer Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

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.

Media Contacts:

Phil McNamara
Vice President, Corporate Communications, AskBio
E: pmcnamara@askbio.com T +1 (984) 5207211

Andrew Thomas
Corporate Communications, AskBio
E: athomas-c@askbio.com T +44 (7803) 585254

References

  1. Balestrino R, Schapira AHV. Parkinson disease. Eur J Neurol. 2020;27(1):27-42.
  2. World Health Organization. Parkinson Disease. Available at: https://www.who.int/news-room/fact-sheets/detail/parkinson-disease. Accessed April 2024.
  3. Medina A, et al. Prevalence and Incidence of Huntington’s Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord. 2022;37(12):2327-2335.
  4. Malik A, et al. Congestive Heart Failure. In: StatPearls. Treasure Island (FL): StatPearls Publishing; November 7, 2022.
  5. MedlinePlus Genetics. NIH. Limb-girdle muscular dystrophy. Available at: https://medlineplus.gov/genetics/condition/limb-girdle-muscular-dystrophy/#frequency. Accessed April 2024.
  6. Goh Y, et al. Multiple system atrophy [published online ahead of print, 2023 Mar 16]. Pract Neurol. 2023; practneurol-2020-002797.
  7. Stevens D, et al. Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview. Curr Treat Options Neurol. 2022;24(11):573-588.
  8. Grieger JC, Soltys SM, Samulski RJ. Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector. Mol Ther. 2016;24(2):287-297.