Category: News

Research Triangle Park, N.C. – March 7, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort. The recommendation to advance to the second cohort followed the completion of a Data Safety Monitoring Board (DSMB) review of trial recruitment activity, and safety reporting from the first cohort that determined it was safe to proceed to cohort two. DSMBs are independent groups of experts appointed to periodically review information from clinical trials. These reviews typically include risk-benefit assessments and monitoring for serious or unexpected adverse safety events alongside any significant benefit of therapies.1 The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants (18–65 years) who have genetic confirmation of LGMD2I/R9. The trial includes two sequential, dose-level cohorts. Adult participants diagnosed with LGMD2I/R9 will be given a single intravenous infusion of AB-1003 or placebo. The trial was initiated in 2023. It will include up to 14 participants at six sites throughout the United States.2 Participants in the first cohort remain in the study until completion. Enrollment in cohort two is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com. “The burden of LGMD2I/R9 on patients and their families is profound,” said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. “Dosing the first participant in the second cohort of the trial is positive news for people living with LGMD2I/R9. This is a rare and debilitating type of muscular dystrophy, and this advancement brings the LION-CS101 trial another step closer to completion.” AskBio has received rare pediatric disease designation (RPDD), orphan-drug designation (ODD) and fast track designation (FTD) for AB-1003 for the potential treatment of LGMD2I/R9 from the United States Food and Drug Administration (FDA).3-4 These designations serve as clear recognition of the significant unmet medical need in LGMD2I/R9, for which there is no approved therapy.5 “The dosing of the first participant in cohort two marks an important milestone for the trial as enrollment continues for LION-CS101,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We are encouraged by the DSMB’s recommendation to advance our study, following their thorough assessment of AB-1003 in cohort one and are excited to proceed with the second cohort.”   AB-1003 is an investigational recombinant adeno-associative virus (AAV)-based gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. It is designed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9 as a one-time intravenous (IV) infusion.2-4 About Limb-Girdle Muscular Dystrophy Type 2I/R9 LGMD2I/R9 is a rare form of LGMD caused

Not intended for UK Media Berlin, Germany, and Research Triangle Park, N.C., USA, February 19, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA). “The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with Parkinson’s disease and their loved ones,” said Gustavo Pesquin, CEO, AskBio. “This milestone could potentially expedite the development of our important investigational gene therapy program, and it highlights our promising data and the potential of AB-1005 for patients and the medical community. We look forward to working closely with the FDA to accelerate our program.” The FDA determined that AB-1005, an investigational gene therapy intended to slow disease progression and improve motor outcomes in patients with PD, met the criteria for RMAT designation. This decision follows a review of information and data provided by AskBio, including clinical evidence from the open label, uncontrolled study Phase Ib trial of AB-1005. AskBio’s 36-month Phase Ib data showed that the administration of AB-1005 was well tolerated with no product-related serious adverse events.1 Further, the moderate PD cohort showed trends for improvement or stability on several PD-relevant clinical scales at 36 months compared to baseline, including Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and self-reported PD motor diaries, together with trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).1 Most participants in the mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.1 RMAT is a designation granted by the FDA to regenerative therapies, including gene therapies, being developed to treat, modify, reverse, or cure serious or life-threatening diseases or conditions.2 Investigational products receiving this designation must have produced preliminary clinical evidence indicating that they may have the potential to address unmet medical needs for such diseases or conditions.2 RMAT provides recipients with enhanced access to the FDA, which could include intensive guidance on efficient drug development, rolling Biologics License Application (BLA) review, and other actions to expedite review.2 “The RMAT designation for AB-1005 underscores the high unmet medical need and the potential of this investigational gene therapy to make a difference for patients with Parkinson’s disease,” said Christian Rommel, Executive Vice President, Global Head of Research and Development and Member of the Pharmaceuticals Leadership Team at Bayer. “This is the latest example of what can be achieved through the joint commitment of AskBio and Bayer to deliver breakthrough innovation for patients.” The first participants in the AB-1005 Phase II REGENERATE-PD clinical trial have been randomized in the United States, and the trial is currently recruiting.3 Additional study sites in the United States, Germany, Poland, and the United Kingdom are expected to be opened for enrollment in first half of 2025.3 AB-1005 has not been approved by any

Berlin, Germany, and Research Triangle Park, N.C., USA, January 14, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first participants have been randomized in its Phase II clinical trial in patients with Parkinson’s disease (PD). “The randomization of the first participants in REGENERATE-PD is positive news for people living with Parkinson’s disease and the physicians treating them,” said Dr Rajesh Pahwa, MD, Director, Parkinson’s Disease and Movement Disorder Center, University of Kansas Medical Center, USA, and REGENERATE-PD Principal Investigator. “There is a significant need for neurorestorative therapies in Parkinson’s and seeing the advancement of an important investigational gene therapy in a Phase II clinical trial gives hope to patients and the medical community.” In 2024, AskBio initiated recruitment in the United States arm of REGENERATE-PD.1 The objective of this randomized, double-blind, Phase II clinical trial is to evaluate the safety and efficacy of AB-1005 delivered to the putamen in adult participants aged 45–75 years with moderate-stage PD.2 REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers that are being opened in the United States, Germany, Poland, and the United Kingdom. AskBio also presented 36-month Phase Ib data at the International Congress of Parkinson’s Disease and Movement Disorders, which was held in Philadelphia, Pennsylvania, from September 27 to October 1, 2024.3 The data demonstrated that administration of AB-1005 was well tolerated with no attributed serious adverse events.3 The Moderate PD cohort showed trends for improvement or stability on several motor scales at 36 months, including Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and motor diaries, and trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).3 Most participants in the Mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.3 “AskBio continues to mark significant milestones in the clinical development of the investigational gene therapy AB-1005 as we strive to bring a safe and effective gene therapy to patients with moderate stage Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “With REGENERATE-PD participants now being randomized, we are excited by our progress with AB-1005 and look forward to sharing further updates at an appropriate scientific forum as the program advances through next year and beyond.” “The advancement of AskBio’s Parkinson’s program is a significant milestone that highlights our commitment to exploring the potential of gene therapy in an area of significant unmet medical need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development. “We believe that therapeutic modalities like gene therapy can change the treatment landscape for people living with the debilitating effects of Parkinson’s disease and look forward to seeing what we will learn in this next phase of research and development for AB-1005.” AskBio is also exploring AB-1005 beyond Parkinson’s disease and is currently enrolling participants in the United States with the

Research Triangle Park, N.C. – November 7, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). FDA grants rare pediatric disease designation to incentivize the development of new treatments for serious and life-threatening diseases that primarily affect children aged 18 years or younger, with fewer than 200,000 people affected in the US. If AB-1003 is approved, AskBio may qualify for a priority review voucher based on receipt of this designation. A priority review voucher can be applied to another therapy in the company’s pipeline, enabling a shorter review timeline during marketing application review or can be sold and transferred to another company.1 Orphan designation provides orphan status to drugs and biologics for rare diseases that meet certain criteria and potentially gives a company exclusive marketing rights for a seven-year period, along with other benefits.2 “These designations for AB-1003 are clear recognition of the significant unmet medical need in LGMD, including type 2I/R9, which is the focus of AskBio’s clinical program and for which there is no approved therapy,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “The burden of this rare form of muscular dystrophy on patients and their families is profound, and these decisions support our efforts to potentially bring a new therapeutic option to people living with the 2I/R9 type of this devastating disease.” 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.3 People living with LGMD2I/R9 may notice symptoms including loss of mobility, impaired heart or lung function. These symptoms can occur in school age and younger children.3 As symptoms worsen, individuals generally require wheelchairs.3 LGMD2I/R9 is a rare disease, estimated to affect fewer than 5,000 people in the US.3 Currently, there is no treatment that modifies disease progression, and management is based on the signs and symptoms present in each individual.3  With a broad portfolio of investigational gene therapies at various stages of research and development, AskBio continues to develop adeno-associated virus (AAV)-based therapies to treat some of the world’s most debilitating diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications and aims to deliver breakthrough treatments that could potentially benefit tens of millions of patients worldwide.4-10 About Limb-Girdle Muscular Dystrophy (LGMD) 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.9 The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs.9 The severity, age of onset, and features of LGMD vary among the many subtypes of the condition and are

Research Triangle Park, N.C.– OCTOBER 17, 2024 – 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 as well as advancements in manufacturing technologies, at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Meeting taking place October 22–25, 2024, in Rome, Italy. “Our presence at ESGCT this year demonstrates our commitment to strengthening our end-to-end capabilities, successfully advancing our early pre-clinical pipeline and continuing to innovate in the field of manufacturing technology”, said Gustavo Pesquin, Chief Executive Officer, AskBio. “This year’s presentations complement notable recent progress in our clinical pipeline, with the first patient randomized in our Phase 2 GenePHIT trial for AB-1002, an investigational gene therapy in congestive heart failure, and the initiation of recruitment for REGENERATE-PD, our Phase 2 Parkinson’s disease trial for investigational gene therapy AB-1005, both announced earlier this year.” AskBio’s presentations include (all times CEST): Orals Posters With an ambitious portfolio of investigational 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. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications and aims to deliver breakthrough treatments that could potentially benefit tens of millions of patients worldwide.1–6 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 stage 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 Viralgen Viralgen is a fully integrated contract development and manufacturing organization (CDMO) founded in 2017 as an independently operated subsidiary of Asklepios Biopharmaceutical, Inc. (AskBio), part of the Bayer AG group. Viralgen was created to support development through large-scale commercial production of certified good manufacturing practices (cGMP) AAV (adeno-associated virus) for cell and gene therapies. Through the AskBio licensed proprietary Pro10™ suspension manufacturing platform, Viralgen delivers industry-leading titers for all AAV serotypes, optimizing speed to market and cost-of-goods to accelerate clinical development

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

Berlin, Germany, and Research Triangle Park, N.C., USA, July 11, 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 that the United States (U.S.) Food and Drug Administration (FDA) has granted Fast Track Designation for AB-1005, which is being developed for moderate Parkinson’s disease. AB-1005 has also been awarded the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (UK MHRA) innovative medicine designation, for the treatment of Parkinson’s disease. AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy being studied for the treatment of moderate Parkinson’s disease. Earlier this year, AskBio presented the 18-month Phase Ib clinical trial results for AB-1005, which met its primary objective of evaluating the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. “These designations clearly underscore the importance of developing innovative therapies for those living with Parkinson’s disease, where a significant unmet need still exists,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “They further highlight the willingness of key regulatory bodies to support the accelerated development of AB-1005 with a focus on the potential benefit to patients.” The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill unmet medical needs.1 The purpose of the Program is to get important new therapeutics to patients earlier.1 Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the clinical development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review. The UK MHRA Innovation Passport is the entry point to the Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market, facilitating patient access. This designation provides Innovation Passport holders with the opportunity to work with the UK MHRA and partners to create product-specific Target Development Profiles (TDP) for new therapies. The TDP will define key regulatory and development features, identify potential pitfalls, offer access to specialist toolkits, and create a roadmap for delivering early patient access.2,3 “The FDA Fast Track and the UK MHRA Innovation Passport designations represent important accomplishments for the clinical development of AB-1005 and receiving these highlights our goal of bringing a safe neurorestorative treatment to patients with moderate Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We look forward to advancing our Phase II REGENERATE-PD clinical trial, which is currently enrolling patients in the U.S. with sites in the European Union and UK planned to open later this year.” “We are excited about the opportunity to potentially accelerate the development of AB-1005, leveraging the frequent interaction with relevant regulatory bodies,” said Christian Rommel, PhD, Global Head of Research & Development at Bayer’s Pharmaceuticals Division. “The granted designations for AB-1005 highlight the demand to advance novel therapeutic modalities, like gene therapy, for

Research Triangle Park, N.C.– JUNE 25, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced initiation of recruitment to REGENERATE-PD, a Phase 2 clinical study assessing efficacy and safety of AB-1005, an investigational adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease. “By enhancing levels of a naturally occurring growth factor, glial cell line-derived neurotrophic factor (GDNF) gene therapy is intended to promote the survival and functioning of vulnerable brain cells that degenerate in Parkinson’s disease”, said Alan Whone, MD, PhD, Consultant Senior Lecturer in Movement Disorder, Bristol Medical School, and Honorary Consultant Neurologist at North Bristol Trust, UK.  “The advancement of AB-1005 is a significant milestone in the development of a gene therapy for Parkinson’s disease and has the potential to bring an effective treatment one step closer to patients”.  Dr Whone will act as European Lead Principal Investigator (PI) on REGENERATE-PD once the program becomes active in the UK. According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease.1 “Following the encouraging results from the Phase 1b study and the presentation of 18-month data at the American Association of Neurology (AAN) meeting in April, we are excited to be progressing AB-1005 to this larger, Phase 2 study,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “This latest advancement highlights our confidence in the potential of AB-1005 to provide a transformative impact for patients with Parkinson’s disease.” In January, AskBio announced that the Phase 1b trial of AB-1005 met its primary endpoint, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. The investigational gene therapy for the treatment of Parkinson’s disease was well tolerated with no serious adverse events that were considered related to AB-1005 in all 11 patients at 18 months.2 AskBio is also exploring GDNF therapy beyond Parkinson’s disease 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.3 AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. About Parkinson’s disease  Parkinson’s disease is a progressive neurodegenerative disorder caused by the death of nerve cells in the brain, leading to decreased dopamine levels.4 At diagnosis, it is estimated that patients have already lost 50-80% of their dopaminergic neurons.5 The loss of these neurons leads to a progressive loss of motor function and symptoms such as tremors, muscle rigidity, and slowness of movement.6 Even with medication, the symptoms of Parkinson’s disease can fluctuate during the course of the day. According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with approximately one million living in the United States.1 There is no