Category: News

— Leading research journal, Molecular Therapy, published a manuscript that explores the improvement and expansive use of AAV vectors in gene therapy —  Research Triangle Park, N.C.– January 26, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is pleased to announce the recent publication of “AAV vectors: The Rubik’s cube of human gene therapy” in Molecular Therapy. The manuscript, which reviews the different approaches to improve adeno-associated virus (AAV) vectors for gene therapy, was authored by a team of AskBio scientists; a scientist from AskBio subsidiary Viralgen Vector Core; and AskBio Co-Founder, President and Chief Scientific Officer, R. Jude Samulski, PhD, a world-renowned gene therapy pioneer.  The article focuses on AAV capsid selection and engineering, strategies to overcome anti-AAV immune response and the design of vector genomes. Also included is a look at the expanded use of AAV vectors, beyond treating monogenic genetic diseases, to enable gene transfer approaches for a wide range of neurodegenerative, heart, blood, muscular and retinal disorders, as well as complex cancers, such as glioblastoma. The manuscript ends with a glimpse at vector production methods and the current state of recombinant AAV (rAAV) at the clinical level. “As we see the potential efficacy of rAAV and the assembly and packaging of high yielding vectors for gene transfer, we have opened the door to changing the course of a broader range of monogenic and pathway diseases,” remarked Dr. Samulski. “This article further reveals the possibilities of rAAV and how we can improve current applications of AAV vectors used in gene therapy today.” The manuscript’s lead author was AskBio scientist Amaury Pupo, PhD. Other AskBio contributing authors were Audry Fernández, PhD, SiewHui Low, PhD, and Lester Suárez, DVM, PhD, along with Viralgen Vector Core Director of Molecular Development, Achille François, PhD. This work was conducted under the guidance of Dr. Samulski, who is also a professor of Pharmacology at The University of North Carolina at Chapel Hill, and the manuscript was featured on Molecular Therapy’s December cover. Dr. Samulski was recently awarded the inaugural European Society of Gene and Cell Therapy (ESGCT) “Founders Award” in Edinburgh, Scotland, during the ESGCT 29th Congress. Dr. Samulski developed the foundational technology for making AAV-based gene therapy vectors and self-complementary AAV vectors over 40 years ago. These contributions are believed to be key components of every FDA approved AAV therapeutic today and will remain central to future advances in gene therapy. 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 Pompe disease, Parkinson’s disease, and congestive heart failure. 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

Research Triangle Park, N.C. (January 9, 2023) – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that it has signed a multi-year research collaboration and option agreement with ReCode Therapeutics. Under this agreement, the companies will work together to potentially discover precision genetic medicines through the development of a novel platform for full gene insertion by single vector delivery of gene editing and DNA cargoes. This represents another step forward for AskBio in developing its gene editing and other nonviral delivery technologies and expertise.  “The collaboration pairs AskBio’s expertise in synthetic DNA and CRISPR applications with ReCode’s novel SORT LNP delivery technology to potentially discover new genetic medicines,” said AskBio’s Chief Executive Officer Sheila Mikhail. “Through this collaboration, we have an opportunity to advance AskBio’s gene editing and nonviral delivery efforts, which support and align with our commitment to serve as an industry-leading engine of gene therapy innovation.” Under the agreement, AskBio will combine its synthetic DNA and gene editing nucleases with ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) technology to potentially create an all-in-one solution that enables full gene insertion by delivering with precision both the gene editing tool and DNA as mixed cargo in a single LNP to desired targets. This collaboration could extend the reach of gene editing for liver and lung disease targets.  “AskBio’s pioneering gene editing technology is a powerful complement to our modular SORT LNP genetic medicines platform, which enables the precise delivery of a wide variety of genetic cargoes to specific organs and cells, including large, complex genetic cargoes and mixed payloads,” said Shehnaaz Suliman, M.D., MBA, M.Phil., Chief Executive Officer of ReCode Therapeutics. “We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases.” In June 2022, ReCode announced the closing of a $200 million Series B extension financing round, co-led by Leaps by Bayer, the impact investment unit of Bayer AG, to further strengthen Leaps by Bayer’s innovation portfolio in gene therapies. The funds raised are being used to advance the development of ReCode’s platform and pipeline to selectively deliver genetic medicines to target organ and cell types in a predictable and programmable fashion.  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 Pompe disease, Parkinson’s disease and congestive heart failure. 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, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in

Research Triangle Park, N.C. (December X, 2022) – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly-owned and independently-operated subsidiary of Bayer AG, today announced that the FDA’s approval of a new adeno-associated virus (AAV) gene therapy to treat adults with hemophilia B marks another milestone for the advancement of AAV therapeutics and highlights the important research contributions made by AskBio Co-Founder, President and Chief Scientific Officer, R. Jude Samulski, PhD. The significance of Dr. Samulski’s groundbreaking AAV work is validated by the role his pioneering research and discoveries have played in the development of all currently approved AAV gene therapies and most that are in the clinic. Dr. Samulski’s early discovery of how to clone AAV led to its widespread use as a mechanism to deliver healthy genes to cells, and we believe this is the foundation for AAV genetic medicine as it stands today. “The progress made in gene therapy to date is remarkable,” said Jude Samulski, AskBio Co-Founder, President and Chief Scientific Officer. “It is humbling to see the impact of the research I completed over 40 years ago and how this is accelerating AAV therapeutic development. My ultimate vision, shared by all of us at AskBio, is to make gene therapy more accessible for everyone in need around the world. I believe we will be there soon.” Dr. Samulski led the UNC Gene Therapy Center from 1993 to 2016 and, in 2001, co-founded AskBio, where many advances in recombinant AAV (rAAV) and underlying AAV vector technology were, and are being, made. Since 2017, three AAV gene therapies have been approved for use by the FDA. Research performed at the UNC Gene Therapy Center, and advanced at AskBio, helped pave the way for the development of these therapeutics. Hemgenix®1(etranacogene dezaparvovec-drlb), for hemophilia B, uses the AAV vector with Padua variant R338L; Zolgensma®2(onasemnogene abeparvovec-xioi), for spinal muscular atrophy, uses self-complementary AAV vectors; and Luxturna®3(voretigene neparvovec-rzyl), for inherited retinal disease, uses the original AAV2 capsid. All are based on Dr. Samulski’s pioneering research or on technology that he developed in collaboration with others in the field, as are Duchenne muscular dystrophy AAV therapeutics in clinical development.  “We are incredibly fortunate for Jude’s scientific leadership at AskBio and for his continued contributions to genetic medicine,” added AskBio’s CEO, Sheila Mikhail.  “Every day, we see the faces of the people who are running out of time and can’t wait for answers. It is our responsibility to find solutions. I believe that Jude, our AskBio colleagues and others throughout the industry are providing hope for people with devastating diseases.” AskBio, co-founded by Dr. Jude Samulski and Sheila Mikhail, is a global leader in molecular medicine and serves as a key driver of Bayer’s gene therapy research, manufacturing and clinical advancements. _____________________________________________________ 1Hemgenix is a registered trademark of CSL Behring LLC. 2Zolgensma® is a registered trademark of Novartis AG. 3Luxturna® is a registered trademark of Spark Therapeutics, Inc. About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated

Research Triangle Park, N.C.– November 17, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly-owned and independently-operated subsidiary of Bayer AG, today announced that Nathalie Cartier, MD, AskBio’s Vice President, Sector Lead for Neurobiology, was recently honored with the Ann B. Moser Award by ALD Connect. The award recognizes unusually impactful contributions to the adrenoleukodystrophy (ALD) community at large and major milestones in the history of the disease. “Dr. Cartier’s work has far-reaching impact for advancing ALD treatments,” said Dr. Florian Eichler President of ALD Connect. “Her accomplishments, as a researcher working to develop groundbreaking and life-extending treatments for diseases such as ALD, reflect the spirit of Ann Moser’s work over many decades to advance leukodystrophy research and advocacy.” ALD Connect was founded in January 2013 by a dedicated group of clinicians, scientists, patients and patient advocates focused on improving the quality of life for those living with ALD through advocacy, education, research and support. This award was established in recognition of Ann B. Moser’s dedication to leukodystrophy research, education and newborn screening advocacy. Dr. Cartier was presented the award for her pioneering work on hematopoietic cell gene therapy for ALD that led to marker authorization in Europe and more recently in the United States. “ALD is a devastating disease that affects children, particularly boys ages 4 to 10. Knowing my work may have contributed to a better life for these young people is humbling,” stated Dr. Cartier. “I want to thank those at ALD Connect for this meaningful recognition.” About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, 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 Pompe disease, Parkinson’s disease, and congestive heart failure. 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, UK, the company has generated hundreds of proprietary 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 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.askbio.com or follow us on LinkedIn.  Media Contact: Phil McNamaraVice President, Corporate Communications, AskBioE: pmcnamara@askbio.comT: +1 984.389.1797

Research Triangle Park, N.C.– November 14, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly-owned and independently-operated subsidiary of Bayer AG, today announced that Sheila Mikhail, the company’s Co-Founder and CEO, was named one of the “Fiercest Women in Life Sciences 2022” by industry-leading publication Fierce Pharma. Mikhail is one of 20 female leaders in life sciences to receive this honor. Recipients were selected following a rigorous review process that identified women who are leading the way and making a lasting impact on the industry. The inspirational women being recognized are leaving an indelible footprint across biotech, pharma and medtech. Mikhail, who is at the forefront of life science innovation and business leadership, has left an unparalleled mark on the industry as a serial entrepreneur, company builder and one of its few Latina CEOs. “Sheila is an exceptional businesswoman. Her command of the science of gene therapy is only rivaled by her incredible business acumen,” said Jude Samulski, Ph.D., AskBio Co-Founder and Chief Scientific Officer. “What makes her contribution to our industry most remarkable is her ability to create environments for cutting-edge science to thrive. Sheila’s work has directly resulted in breakthrough gene therapies reaching patients and in the development of others that we hope will do the same in the future. This honor is well deserved.” Mikhail co-founded AskBio over 20 years ago with the unwavering belief that gene therapies could cure disease and change the world. During her tenure at the company, she also led several spinoffs where she realized various strategic partnerships and acquisitions. These included Pfizer’s acquisition of Bamboo Therapeutics in 2016 and the acquisition of Chatham Therapeutics by Baxter (now Takeda) in 2014. More recently, Mikhail negotiated the $4 billion acquisition of AskBio and its cGMP manufacturing subsidiaries by Bayer AG in 2020, a reflection of the tremendous value she and co-founder Dr. Samulski have and continue to build with the company. “I’m honored to be recognized by Fierce for my contribution to our industry,” said Mikhail. “I am fortunate to have been part of the gene therapy field over a period where it matured from concepts to real life examples of transformative benefits for patients. In addition to bringing treatments to patients, my priorities have also included ensuring equitable access and creating opportunities to advance women and underrepresented minorities in biotech. As a head of an organization represented by over 50 percent women, with women holding key leadership positions, I am proud that together at AskBio we have created an inclusive environment where women feel they can thrive.”  Today, AskBio is a global leader in gene therapy and works to bring innovative technology to the market and potentially life-changing therapeutics into the clinic. As CEO, Mikhail has helped grow the company to over 700 employees operating in five countries. She is also Co-Founder and Board Member of Columbus Children’s Foundation (CCF), which develops gene therapies for children with ultra-rare diseases and treats them free of charge. Mikhail was named the Overall US Entrepreneur of the Year

— Award recognizes 40th anniversary of groundbreaking first cloning of AAV plasmid —  Research Triangle Park, N.C.– OCTOBER 11, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that R. Jude Samulski, Ph.D., AskBio President, Co-Founder and Chief Scientific Officer, received the inaugural European Society of Gene and Cell Therapy (ESGCT) Founders Award in Edinburgh, Scotland, during the recent ESGCT 29th Congress.  The Founders Award honors Dr. Samulski’s contribution to the development of engineered recombinant adeno-associated virus (rAAV) vectors, which began 40 years ago with the cloning and rescue of infectious AAV from a recombinant plasmid.1 This work laid the foundation for subsequent developments of rAAV vectors capable of expressing foreign sequences, thereby enabling the use of AAV-based vectors for the delivery of various therapeutic genes into mammalian cells. The AAV inverted terminal repeat (ITR) sequences are an essential component of every AAV therapeutic drug. This and other breakthroughs made by Dr. Samulski have since contributed to three rAAV-based gene therapies becoming available for patients and many more in various stages of development.   “ESGCT created the Founders Award this year to mark the Society’s 30th anniversary,” said Professor Hildegard Büning, ESGCT Board President. “The award highlights benchmark milestones in gene and cell therapy. The Founders Award 2022 recognizes the first cloning of an AAV plasmid, which Dr. Samulski and his team accomplished in 1982. That tremendous breakthrough helped make gene therapy a reality and remains at the core of the field today, making this milestone a natural choice for this inaugural honor.”  Dr. Samulski has worked with AAV for 40 years, and for 25 years he was director of the University of North Carolina Gene Therapy Center. Dr. Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with gene therapy clinical trial approvals in the U.S. He also frequently serves as a gene therapy consultant to the FDA. In 2008, Dr. Samulski was recognized by the American Society of Gene & Cell Therapy (ASGCT) with the inaugural Outstanding Achievement Award. He was also invited by Pope Francis to the Vatican in recognition of his work in the treatment of Canavan disease. Dr. Samulski has played an integral role in advancing gene therapies into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease, congestive heart failure and others. He is the inventor of nearly 500 patented technologies related to AAV technology. “I’m overwhelmed with this acknowledgment and humbled by the thought that ESGCT has considered my early research to represent such a prestigious milestone for the society,” said Dr. Samulski. “I’m grateful to receive the ESGCT Founders Award during these exciting times for gene therapy and honored to be the first scientist to receive this recognition.” “Jude developed the foundational technology for making AAV-based gene therapy vectors and self-complementary AAV vectors,” said Sheila Mikhail, CEO and Co-Founder of AskBio. “His groundbreaking work 40 years ago remains central to our industry’s advances in

Triangle Business Journal Selection Based on Employee-Provided Rankings in Six Categories Research Triangle Park, NC – August 31, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, has been named, by the Triangle Business Journal (TBJ), as one of the Triangle’s Best Places to Work. This award honors companies for creating employee-valued policies and work environments. “At AskBio, we are passionate about leading innovative science and delivering outcomes that transform people’s lives,” said Sheila Mikhail, JD, MBA, CEO and Co-Founder of AskBio. “As gene therapy pioneers, we play an important role in society. To be successful, our workplace must be as exceptional as our mission. Being added to this list is a special honor and one that highlights our focus on this significant aspect of our current and future success.”  The 50 companies named to the 2022 Triangle’s Best Places to Work list represent a cross section of industries in North Carolina’s Raleigh-Durham-Chapel Hill area. Selections were the result of employee feedback on how well each company performs in six business-critical categories: Communication and Resources, Individual Needs, Manager Effectiveness, Personal Engagement, Team Dynamics, and Trust in Leadership.  “We believe this recognition shows that AskBio is succeeding in its ongoing effort to provide the most attractive environment for employees,” added Philip Dana, Chief Human Resources Officer, AskBio. “Our company is committed to investing in the support, development and success of our nearly 800 team members across the US and Europe. We are implementing leading HR technologies, launching employee resource groups, working to deliver compensation and benefit programs similar to those of larger biotech companies and much more.”  About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, 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 Pompe disease, Parkinson’s disease, and congestive heart failure. 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, UK, the company has generated hundreds of proprietary 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 750 patents in areas such as AAV production and chimeric and self-complementary 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. Its 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