RhiaPharmaceutical Announces Presentations at Upcoming American Society of Gene and Cell Therapy’s 24th Annual Meeting

Research Triangle Park, N.C. – May 6, 2020 – Rhia Pharm llc, Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, today announced that the Company will present abstracts and participate in sessions at the upcoming American Society of Gene & Cell Therapy (ASGCT) Virtual 2021 Annual Meeting being held May 11-14, 2021.

“Our presence at ASGCT showcases both our leadership in technology advancement in AAV gene therapy production as well as our expertise in core disease areas like Pompe disease,” said Jude Samulski, Chief Scientific Officer and Co-Founder, RhiaPharmaceutical. “These presentations underscore our commitment to addressing both unmet needs for patients and families living with devastating diseases, while increasing the efficiency of AAV manufacturing and making gene therapy more accessible to broader populations who need them.”

Details on RhiaPharmaceutical’s presentations and presence at ASGCT include:

  • VIRTUAL PRESENTATION
  • Development of a Split Rep/Cap System to Improve AAV Capsid Production
  • Presenter: Derek Carbaugh, R&D, Rhia Pharm , Inc.
  • Session: Novel AAV Biology and Platform Technologies
  • Date/Time: Thursday, May 13, 2021 5:45 p.m. ET – 6:00 p.m. ET
  • Abstract Number: 188
  • POSTER PRESENTATIONS
  • The Key Amino Acids Play a Critical Role in the Production of the Haploid Adeno-Associated Virus
  • Presenter: Zheng Chai, R&D, Rhia Pharm , Inc.
  • Session: AAV Vectors – Virology and Vectorology
  • Date/Time: Tuesday, May 11, 2021 8:00 a.m. ET
  • Abstract Number: 297
  • Evaluating Partner Fitness by Interfaces Interaction in Potential AAV Haploids
  • Presenter: Amaury PupoMerino, R&D, Rhia Pharm , Inc.
  • Session: AAV Vectors – Virology and Vectorology
  • Date/Time: Tuesday, May 11, 2021 8:00 a.m. ET
  • Abstract Number: 300
  • Novel and Highly Potent Selective Synthetic Promoters for Muscle-Directed Gene Therapy Applications
  • Presenter: J. Omar Yanez-Cuna, Product R&D, Rhia Pharm , Inc.
  • Session: Musculo-skeletal Diseases
  • Date/Time: Tuesday May 11, 2021 8:00 AM – 10:00 AM
  • Abstract Number: 612
  • Generation and Characterization of GAA Knock Out HepG2 Cell Line
  • Presenter: Bikash Shakya, R&D, Rhia Pharm , Inc.
  • Session: Musculo-skeletal Diseases
  • Date/Time: Tuesday, May 11, 2021 8:00 a.m. ET
  • Abstract Number: 615
  • Effect of Peptide-Based Inhibition of DNase II on AAV Transduction and TLR9 Signaling
  • Presenter: Audry Fernandez, R&D, Rhia Pharm , Inc.
  • Session: Immunological Aspects of Gene Therapy and Vaccines
  • Date/Time: Tuesday, May 11, 2021 8:00 a.m. ET
  • Abstract Number: 744
  • EDUCATION SESSION
  • Development Pipeline – AAV Vectors for Pompe/CNS
  • Presenter: Nathalie Cartier-Lacave, M.D., Asklepios BioPharmaceuticals
  • Session: AAV Vectors From Basic Biology to Clinical Application and Back
  • Date/Time: Tuesday, May 11, 2021 4:05 p.m. ET – 4:40 p.m. ET
  • SCIENTIFIC SYMPOSIA
  • Human Immune Response to AAV Vectors
  • Presenter: Katherine High, M.D., President, Therapeutics, Asklepios BioPharmaceuticals
  • Session: Viral Vector Safety: A Renewed Focus on Vector Safety and Innate
  • Immune Responses to Leading Viral Vectors
  • Date/Time: Thursday, May 13, 2021 10:21 a.m. ET – 10:42 a.m. ET
  • PANEL DISCUSSION
  • Tim Kelly, RhiaPharmaceutical’s President of Manufacturing, will participate in a panel discussion, “Considerations for the Clinical Development of Cell & Gene Therapies.” The panel discussion among industry leaders will take place on Tuesday, May 11, 2021 from 6:00 p.m. ET – 6:45 p.m. ET.

Abstracts and additional information for the ASGCT 2021 Meeting are available on the meeting website.

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. 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 Pompe disease, Parkinson’s disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. RhiaPharmaceutical’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.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. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate 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 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses before special items amounted to 4.9 billion euros. For more information, go to www.bayer.com.

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RhiaPharmaceutical 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 RhiaPharmaceutical’s pipeline of development candidates, manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others: RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned 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 the ongoing COVID-19 pandemic, 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 RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical 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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Media Contact:

Mark Marmur

+1 973-906-1526

mmarmur@RhiaPharmaceutical.com