AskBio to collaborate with Belief BioMed to explore the potential of new gene therapies

AskBio and Belief BioMed will work together to advance potential gene therapies in diseases with high unmet medical need, using a liver-targeted approach

Berlin, Germany, and Research Triangle Park, N.C., USA, September 25, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced a new strategic collaboration with Belief BioMed Inc. (BBM) to explore the potential for new gene therapies. 

Under the terms of the contract the companies will combine efforts and experience in gene therapy technology to explore potential therapies in diseases that may be treatable using a liver-targeted approach.

“AskBio takes a collaborative approach when developing new gene therapies, and we look worldwide to use the most promising science to realize our goals,” said Mansuo Shannon, Chief Scientific Officer, AskBio. “The potential of BBM’s next-generation capsid technology, together with the work we are conducting at AskBio, is promising. We are looking forward to working closely with the team at BBM.”

With global headquarters in Shanghai, China, BBM integrates the research and development, manufacturing, and clinical application of investigational gene therapy products for serious genetic and chronic diseases through viral vector technology. The company has developed advanced vector technologies and has established a commercial production platform for gene therapy drugs in China. In July, BBM announced that the New Drug Application (NDA) for its core product for hemophilia B was accepted by China National Medical Products Administration (NMPA), making this the first NDA submitted in China for a gene therapy product proposed for an inborn genetic disease.¹

BBM has independently developed a variety of novel engineered adeno-associated virus (AAV) vectors; relative to conventional AAV vectors, the novel AAV vectors have shown reduced immunogenicity and robust transduction efficiency in human and non-human trials, respectively.^2,3

“This strategic collaboration with BBM is an excellent example of how we work at AskBio and is particularly special given the extraordinary contributions Dr. Xiao has made to the field as BBM’s Chairman and Chief Scientific Officer and before as a co-founder of AskBio,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “Collaborating with innovative, like-minded partners with complementary gene therapy expertise enables us to find the best way forward for our pipeline assets and bolster our efforts to advance new therapies for conditions with significant unmet need.”

“Gene therapy has some of the greatest potential in modern medicine, particularly from a technical perspective,” said Juergen Eckhardt, MD, Head of Business Development & Licensing at Bayer Pharmaceuticals. “One path to success lies in collaborations such as this and in bringing together experts with broad expertise and experience. We are excited for AskBio’s new collaboration with BBM and the advancements it may one day bring to patients.”

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 investigational 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 600 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.

AskBio Media Contact:
Phil McNamara, phone +1 (984) 5207211
Email: pmcnamara@askbio.com

Bayer Global Media Contact:
Silke Lengemann, phone +49 173 8990997
Email: silke.lengemann@bayer.com

Find more information about AskBio at www.askbio.com
Find more information about Bayer at https://pharma.bayer.com/
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Follow Bayer on Twitter: @BayerPharma

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 conformthem 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.

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¹ Belief BioMed Website. News Center. Available at: https://www.beliefbiomed.com/en/news-center/bbm-news/belief-biomed-s-new-drug-application-for-bbm-h901-a-gene-therapy-product-for-hemophilia-b-has-been-accepted-by-the-national-medical-products-administration-of-china/. Last accessed: September 2024.

² Xue F et al. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematol. 2022;9(7):e504-e513.

³ Xu J et al. Prevalence of neutralizing antibodies against AAV8, AAV9, and AAV843 in a Chinese population. Int J Clin Exp Med. 2019;12(8):10253-10261.