Research Triangle Park, N.C., October 13, 2023. Asklepios BioPharmaceutical, Inc. (AskBio or the company), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced its updated Executive Leadership Team (ELT) structure, which includes four newly created roles designed to improve the company’s ability to efficiently bring its clinical-stage gene therapies to patients. The addition of these new roles aligns AskBio’s leadership structure with its increased scope and capabilities as an end-to-end gene therapy company active from the earliest stages of discovery through to research; development; and, with the expertise and support of Bayer, eventually delivery. “I am pleased to share that we have added four critical roles to our ELT—Chief Business Officer, Chief Strategy Officer, Chief Product Supply Officer, and Head of People and Culture,” said Gustavo Pesquin, CEO, AskBio. “This move ensures that we have the leadership required to build on our more than 20 years of groundbreaking discovery experience and become a high-performing delivery organization. These new leadership positions increase our focus on all that it takes to meet the needs of the tens of millions of people around the world living with debilitating diseases that we hope to one day treat. Our expanded team will usher in a new era of productivity and can dramatically increase our ability to transform lives.” Tracy Dowling has been named Chief Business Officer and General Counsel. Dowling joined AskBio in 2021 from Spark Therapeutics and now oversees Business Development, Alliance Management, Legal, Compliance, Communications, and a newly created Chief of Staff role. She will further develop key partnerships that will enhance AskBio’s technological and clinical portfolio and advance the field. This will be done by creating in-licensing, out-licensing, and other collaboration opportunities, which will grow the company’s capabilities and share its technologies with others. Ralph Herbst serves as Chief Strategy Officer. Herbst joined the AskBio ELT from Bayer. He leads Corporate Development and Portfolio Management, and he will play a key role in optimizing the AskBio-Bayer partnership. Herbst brings a strong track record of building and expanding cell and gene therapy pipelines and capabilities, which includes his work with Bayer’s Cell & Gene Therapy Platform and his critical involvement in Bayer’s acquisitions of BlueRock Therapeutics LP in 2019 and AskBio in 2020. Jason Krentz takes on the role of Chief Product Supply Officer. Overseeing programs that transition clinical development to commercial operations, he leads Product Supply operations, Facilities, and Information Technology. Krentz joined AskBio in 2022 from Tmunity Therapeutics, where, as Chief Technology Officer, he led the company’s Chemistry, Manufacturing, and Controls (CMC) operations. He brings over 20 years of technical operations and manufacturing experience to AskBio. Melissa Murrell has been appointed Senior Vice President, Head of People and Culture, and oversees all areas of Human Resources with a focus on culture and employee empowerment. Murrell joined AskBio in 2021 from Spark Therapeutics and has played key roles in the company’s efforts to create the structure and systems required to support its rapid growth
Category: News
AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9. Research Triangle Park, N.C.– August 3, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been dosed in the Phase 1 / Phase 2 LION-CS101 clinical trial of patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).[1] AB-1003 (also known as LION-101) is a novel investigational FKRP gene replacement therapy. “Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease,” said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i. “Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy. The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life.” LGMD2I/R9 is a form of LGMD caused by changes in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.[2]Those with LGMD2I/R9 notice symptoms in late childhood, around 11 years of age, which may include loss of mobility and impaired heart and lung function.2,[3] As symptoms worsen, individuals generally require wheelchairs.2 LGMD2I/R9 is a rare disease, estimated to affect more than 5,000 people in the US and EU.[4] “While the inherited nature of limb-girdle muscular dystrophy means those with the FKRP gene mutation can’t produce a normal FKRP protein for physiological muscle function, AB-1003 is designed to introduce the normal FKRP gene into the muscle and express a normal protein, and it has shown promise in restoring normal FKRP protein function in muscle in preclinical studies performed in mouse models of LGMD,” said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. “This trial is the first step toward evaluating the safety of AB-1003 and assessing the potential that AB-1003 has to improve the lives of patients with this serious, inherited ultra rare condition.” “The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is another example of AskBio’s success in taking gene therapy candidates, such as AB-1003, from the bench into clinical development,” said Jude Samulski, PhD, Co-Founder and Chief Scientific Officer of AskBio. “With our knowledge and expertise, we are working to harness the power of gene therapies as potential treatments for many of the world’s most devastating diseases. Our investigational therapy for limb-girdle muscular dystrophy is one of these and an important part of our portfolio, which also includes clinical stage investigational therapies for congestive heart failure, Huntington’s disease, multiple system atrophy, Parkinson’s disease, and Pompe disease.” AB-1003, which is manufactured by AskBio’s wholly owned and independently operated subsidiary Viralgen, was granted fast track designation by the FDA and orphan drug designation by the European Commission.[5],[6] Recruitment for the LION-CS101 clinical
AskBio Announces 11 Presentations at the American Society of Gene and Cell Therapy 26th Annual Meeting in 2023
Research Triangle Park, N.C.– MAY 11, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver 11 presentations offering insights into the research and development of adeno-associated virus (AAV) therapies for a range of diseases at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting taking place May 16–20, 2023, in Los Angeles, USA. AskBio presentations cover pre-clinical research into immune responses to AAV, data on methods to improve the efficacy of AAV-gene therapy and research into improved analytics and bioassays. A symposium on a platform approach to AAV manufacturing will also be presented by AskBio’s subsidiary Viralgen. “Our breadth of scientific presence at ASGCT reflects the type of cutting edge pre-clinical research our teams undertake, which we believe is essential for the development of successful gene therapies in the future,” said Jude Samulski, Chief Scientific Officer and Co-Founder of AskBio. “We look forward to presenting updates on pioneering work that spans the subtlety of AAV vector design, quantifiable transgene expression and preclinical efficacy, with the aim of furthering AAV therapy for additional unmet clinical needs.” With an ambitious portfolio of gene therapies at various stages of research and development, AskBio continues to develop AAV-based therapies to treat some of the world’s most debilitating diseases, including congestive heart failure, 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 35 million patients worldwide.1–7 AskBio’s presentations at ASGCT include: 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 clinical testing. An early innovator in the gene therapy field, with over 800 employees in five countries, 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 Viralgen Viralgen, an independently operated Contract Development and Manufacturing Organization (CDMO), was founded in 2017 as a joint venture between AskBio and Columbus Venture Partners (a venture capital firm based in Spain). As one of the world’s leading manufacturers of cGMP-certified AAV, Viralgen uses the Pro10TM based suspension manufacturing platform, a technology licensed from AskBio and developed by CTO Josh Grieger and co-founder R. Jude Samulski, PhD, at University of North Carolina. It is believed that
AskBio to Present Phase 1b Results Investigating AB-1005 (formerly AAV2-GDNF) as Treatment for Parkinson’s Disease at AD/PDTM 2023 International Conference on Alzheimer’s and Parkinson’s Diseases
—Preliminary data suggest that gene therapy via neurosurgical administration is generally safe, well tolerated and associated with enhanced mean putaminal coverage and clinical improvements— —Study uses one-time MRI-monitored surgery for delivery in patients with mild to moderate Parkinson’s disease— Research Triangle Park, N.C.– MARCH 29, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will present the preliminary results of a clinical Phase 1b study investigating the safety and efficacy of AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of mild to moderate Parkinson’s disease at the AD/PD™ 2023 Advances in Science & Therapy Conference taking place March 28–April 1, 2023, in Gothenburg, Sweden. Initial results from the open-label study investigating AB-1005, administered to the putamen via one-time bilateral convection-enhanced delivery, will be presented during an onsite oral session on April 1 at 5:25 p.m. Central European Time, as part of the “Drug Development in AD, PD, LBD” symposium being held in Hall G3. Preliminary data to be presented suggest that both the gene therapy and the neurosurgical administration are generally safe, well tolerated and associated with enhanced mean putaminal coverage and clinical improvements. The study included 11 participants who had completed 9–18 months of clinical follow-up post treatment, of which 10 had completed more than 12 months. More than 10 million people worldwide are currently living with Parkinson’s disease, which is debilitating and the most common neurodegenerative movement disorder in the world.1,2 Globally, disability and death related to Parkinson’s disease are increasing faster than for any other neurological disorder.3 While GDNF administration has been investigated for Parkinson’s disease for years, results have been mixed. It has been rationalized that by improving GDNF delivery of AAV2 gene therapy with MRI-monitored convection enhanced delivery it may be possible to overcome the limitation of insufficient putaminal coverage to achieve clinical improvements in motor function.4-6 Moreover, clinical trials in Parkinson’s disease over the last decade have shown that AAV is a well-tolerated and suitable vector for gene therapy.7 As leaders in the field of gene therapy, AskBio is committed to investigating the potential that GDNF gene therapy holds for patients with mild to moderate Parkinson’s disease. Although ongoing trials are needed to confirm these findings, the company believes they represent a step forward in advancing therapeutic solutions for those with this debilitating disease. AskBio is also exploring GDNF therapy beyond Parkinson’s and is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase 1 trial to assess the preliminary safety, tolerability and efficacy of GDNF therapy for this rapidly progressing condition. ### Media Contact: Phil McNamara Vice President, Corporate Communications, AskBioE: pmcnamara@askbio.com T: +1 984.520-7211 About Parkinson’s Disease Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons and at diagnosis, it is estimated that patients have
AskBio Announces Gustavo Pesquin as New CEO
Research Triangle Park, N.C.– March 9, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that Gustavo Pesquin will become the company’s next Chief Executive Officer (CEO) as of March 11, 2023. Pesquin joins AskBio from Amneal Pharmaceuticals, Inc., where he served as Chief Commercial Officer. Sheila Mikhail, Co-Founder and the company’s first CEO, will transition to a part-time role as Adviser/Co-Founder, also effective March 11, 2023. Mikhail will remain a member of AskBio’s Board. Under Mikhail’s leadership as CEO since 2017, AskBio has grown to over 800 employees operating in five countries and has advanced six therapeutics through IND, including potential treatments for congestive heart failure, Parkinson’s disease, Huntington’s disease, multiple system atrophy, Pompe disease and limb-girdle muscular dystrophy type 2I/R9. Under her leadership, AskBio built two contract development and manufacturing organization (CDMO) businesses, Viralgen Vector Core SL and TAAV Biomanufacturing Solutions SL. In her new role, Mikhail will continue to support the strategic direction and growth of AskBio. She will also continue to support Columbus Children’s Foundation (CCF) in its development of gene therapies for ultra-rare diseases. “I co-founded AskBio, along with Drs. Richard Jude Samulski and Xiao Xiao, to develop AAV gene therapies for patients suffering from devastating diseases with no treatment options,” said Mikhail. “I’m tremendously proud of the contributions made by our team. They have made critical scientific discoveries and pioneered transformative clinical programs. My focus over the last several years has been to assemble the world’s leading experts in gene therapy to solve technological problems and create new clinical programs. Today we are in the clinic with treatments for multiple diseases, and through our subsidiaries we are the leading manufacturer of synthetic DNA and AAV vectors at commercial scale. I look forward to working with Gustavo as AskBio continues to pursue its strategic vision.” “Sheila’s extraordinary leadership moved AskBio from an idea to a strongly positioned global biotech that is advancing multiple products into clinical studies, with direct paths to pivotal trials, marketing approval and manufacturing,” stated Stefan Oelrich, Member of the Board of Management, Bayer, and President of Bayer’s Pharmaceuticals Division. “We are grateful for her contributions and pleased she will continue to work closely with us as an Adviser and Board member. Under Gustavo’s leadership as the new CEO, I am confident that AskBio will continue to advance its world-class therapeutics and manufacturing operations, further its work as a cornerstone of the Bayer CGT Platform and set itself apart from its peers. We are excited about the future and look forward to working with Gustavo.” In addition to his experience as Chief Commercial Officer at Amneal, Pesquin brings to AskBio his more than 10 years of experience at Sanofi, a leading global pharmaceutical company, where he served in leadership roles of increasing responsibility, including North America Head for General Medicines as well as Global Head of the Diabetes and Cardiovascular Franchise. Prior to Sanofi, he held Regional Head, General Manager, Sales Head and Strategy Head
AskBio Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy (LGMD)
— LGMD is a Rare Form of Muscular Dystrophy with No Approved Therapy — Research Triangle Park, NC – February 16, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101)* for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is a novel investigational recombinant adeno-associated virus (AAV) based gene therapy currently being developed as a one-time intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I/R9), a disease subtype affecting 4.5 people per million worldwide, including more than 5,000 people in the EU and US. The EC decision, dated February 15, 2023, follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) and was received through AskBio’s EU-based subsidiary BrainVectis. AB-1003 is being investigated in the US in a Phase 1/2 multicenter study that will evaluate the safety, tolerability and efficacy of a single IV infusion of gene therapy in adult subjects with genotypically confirmed LGMD2I/R9. “The EC orphan drug designation for AB-1003 is an important recognition of the unmet medical need in LGMD, which has no approved therapy,” said Sheila Mikhail, Co-Founder & CEO, AskBio. “The burden of this rare form of muscular dystrophy on patients and their families is significant, and this decision supports our efforts to potentially bring a new therapeutic option to people in the EU living with the 2I/R9 type of this devastating disease.” The EC grants orphan drug designation for medicinal products intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000 in the EU, provided there is no other satisfactory treatment option or the medicine can be of significant benefit to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees. In addition, if the medicine is approved for marketing, this designation will provide 10 years of marketing exclusivity. About Limb-Girdle Muscular Dystrophy (LGMD) and Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) Limb-girdle muscular dystrophy (LGMD) is a term for a group of diseases that cause progressive weakness and wasting of the muscles in the arms and legs.1 The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs.1 The severity, age of onset, and features of LGMD vary among the many subtypes of the condition and are often inconsistent, even within the same family.1 Signs and symptoms may first appear at any age and generally worsen with time, although in some cases they remain mild.1 Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD and is caused by mutations in the FKRP gene.2 In LGMD2I/R9, signs and symptoms often develop in late childhood and may include difficulty running and walking.2 The symptoms gradually worsen over time toward significant disability, and affected people generally rely
AskBio Co-Founder R. Jude Samulski and Team of AskBio Scientists Author Paper Published in Prestigious Journal
— 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
AskBio Announces Strategic Collaboration with ReCode Therapeutics to Explore Single Vector Gene Editing Platform
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
Recombinant Adeno-Associated Virus (rAAV) Technology
Pioneered by AskBio’s Dr. Jude Samulski is Key Component
of All FDA Approved AAV Gene Therapeutics
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
AskBio Vice President and Neurobiology Sector Lead Nathalie Cartier Honored with Ann B. Moser Award
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
