Category: News

Durham, N.C.,– APRIL 1, 2026 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the randomization of the last participant was completed earlier this year for GenePHIT, its Phase 2 clinical trial of AB-1002, an investigational gene therapy being developed as a potential treatment for heart failure with reduced ejection fraction (HFrEF). “Heart failure is a major public health challenge and places a massive strain on healthcare systems around the world,” said Timothy D. Henry, MD, MSCAI, GenePHIT Principal Investigator and Steering Committee Member. “Prevalence is increasing, and the need for innovative therapies has never been greater. Completing enrollment in this trial brings us another step closer to evaluating a potential treatment strategy for heart failure with reduced ejection fraction.” GenePHIT includes 173 participants, and the completion of enrollment marks a significant milestone in the development of AB-1002, an investigational gene therapy administered via a single intracoronary infusion on top of the standard of care in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms.1 Initial trial results are expected in the first half of 2027. “We are pleased to have randomized the last participant in our GenePHIT trial,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “AskBio’s Phase 2 heart failure program investigates the efficacy and safety of AB-1002, which is designed to potentially mitigate the symptoms of heart failure with reduced ejection fraction and improve survival rates and quality of life. The data we receive from the participants will help us better understand the potential of our investigational gene therapy in an area of significant medical need.” An estimated 64 million people worldwide are living with heart failure, and despite advances in treatment, mortality and morbidity remain very high.2,3 The completion of enrollment represents a significant step in potentially bringing a new treatment to those who need it most. AskBio explored AB-1002 in a Phase 1 non-randomized, sequential dose escalation trial for participants with NYHA Class III non-ischemic HFrEF.4 Twelve-month data were published online in Nature Medicine in October 2025 and in print in November 2025.5 These data had previously been presented in May 2025 as a late-breaker presentation at the European Society of Cardiology Heart Failure Meeting.6 AB-1002 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 AB-1002 AB-1002 is an investigational one-time gene therapy administered directly to the heart to promote production of a modified version (I-1c) of the naturally occurring protein inhibitor-1, designed to block the action of protein phosphatase 1, which is linked to heart failure.7,8 About Heart Failure Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs.9 This causes congestion in the body’s tissues.10 Symptoms may include shortness of breath, swelling in the legs and ankles

Durham, N.C. – JANUARY 8, 2026 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the United States Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AB-1009, an adeno-associated virus (AAV) gene therapy being developed for the treatment of late-onset Pompe disease (LOPD). With this announcement, the AB-1009 program advances to Phase 1/Phase 2, and AskBio has initiated a clinical trial in the United States to explore the safety of AB-1009. The company anticipates recruiting its first patient in early 2026.  “This investigational gene therapy is being studied for its potential to address the underlying genetic defect and to explore whether it can increase production of the deficient enzyme in patients with Pompe disease,” said Tahseen Mozaffar, MD, Director of the UCI Health ALS & Neuromuscular Center, and Principal Investigator, AB-1009 Clinical Trial Program. “Patients receiving gene therapy may reduce reliance on exogenous enzyme replacement. AskBio’s approach leverages its experience in gene therapy development as it seeks to advance treatment options for Pompe disease.”  In addition to the initiation of the AB-1009 PROGRESS-GT LOPD (NCT07282847) clinical trial program, the therapy was recently granted FDA Fast Track and Orphan Drug designations. The FDA Fast Track process 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 process 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. Orphan Drug 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 advancements in the AB-1009 program, particularly the recently granted regulatory designations, highlight the recognized need for late-onset Pompe treatments,” said Mansuo Shannon, PhD, Chief Scientific Officer, AskBio. “Today’s news demonstrates AskBio’s commitment to progressing early-stage assets into the clinic and adding those to our clinical portfolio.”  Pompe disease is a rare, progressive, debilitating genetic disorder that is estimated to affect at least 5,000 to 10,000 people worldwide.3 Pompe disease ranges in severity from infantile-onset Pompe disease (IOPD) to LOPD.4 LOPD is associated with significant morbidity and is characterized by progressive skeletal muscle weakness and respiratory insufficiency.5 Patients typically present with progressive proximal myopathy; however, respiratory involvement can be the primary presenting clinical feature.5 This causes severe muscle weakness and wasting, leading to the loss of mobility.5 The disease can lead to premature death from respiratory failure.5 Today, there are multiple approved enzyme replacement therapies (ERTs) with recombinant human acid alpha-glucosidase (rhGAA), and these are chronically administered.4,5 These can also be used in combination with a small-molecule pharmacological chaperone treatment.6 There remains an unmet medical need, as some individuals receiving ERT

Not intended for UK Media Berlin, Germany, and Durham, N.C., USA, December 9, 2025 – AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for two of AskBio’s investigational gene therapy programs: AB-1005, for the treatment of Parkinson’s disease (PD), and AB-1002, for the treatment of non-ischemic heart failure with reduced left ventricular ejection fraction and New York Heart Association (NYHA) Class III heart failure despite appropriate medical therapy. The designation reflects Japan’s commitment to expediting the development and review of breakthrough therapies and is awarded to products demonstrating innovativeness (a new mode of action), prominent efficacy or safety data, and the potential to address severe diseases, especially when submitted first or simultaneously with other countries. This recognition offers significant advantages, including priority consultations and accelerated review timelines, thereby facilitating earlier participant access to transformative treatments. “Having AB-1005 and AB-1002 receive the Pioneering Regenerative Medical Product designation in Japan highlights our dedication to advancing innovative gene therapies for participants facing serious diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “This recognition not only accelerates regulatory review but also reaffirms our commitment to delivering advanced treatments to those living with serious chronic diseases that lack therapies targeting root causes.” AB-1005, currently being evaluated in the Phase II REGENERATE-PD trial, is an investigational gene therapy with adeno-associated viral (AAV) vector-mediated delivery of the glial cell line-derived neurotrophic factor (GDNF) gene for participants with moderate-stage PD. The therapy aims to restore neuronal function and potentially slow disease progression for people with limited treatment options. AB-1005 previously received US Food and Drug Administration (FDA) Regenerative Medicine Advanced Therapy (RMAT), FDA Fast Track, and UK Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport designations, underscoring its global significance and potential for participants.1 AB-1002 is an investigational AAV gene therapy being studied for the treatment of adults with NYHA Class III heart failure with non-ischemic etiology. It previously received FDA Fast Track designation, and is designed as a one-time gene therapy targeting protein phosphatase 1 inhibition, with the intention of improving cardiac function and addressing the substantial global burden of congestive heart failure.2 “Bayer and AskBio’s collaboration continues to drive progress in gene therapy with a robust pipeline targeting central nervous system, cardiovascular, and other disease indications,” said Christian Rommel, PhD and Global Head of Research and Development for Bayer’s Pharmaceuticals Division. “Receiving the designation in Japan, which is a first for Bayer, marks an important milestone in expanding global access to pioneering therapies and reinforces our shared commitment to delivering breakthrough science to improve outcomes for patients worldwide.” FDA 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.3 Investigational products receiving this designation must have produced preliminary clinical evidence indicating that they

Research Triangle Park, N.C.– October 21, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the publication in peer-reviewed journal Nature Medicine of 12-month data from its Phase 1 trial of AB-1002 investigational gene therapy in participants with congestive heart failure (CHF).1 This non-randomized, sequential dose-escalation trial (NCT04179643) includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).1 It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity remain very high.2,3 The publication, which is available online, confirms that no adverse events were deemed related to AB-1002 in this trial and that clinically meaningful improvements were recorded across several efficacy assessments in participants with non-ischemic CHF.1 The data further support that the AB-1002 capsid may be highly cardiotrophic when administered as a single intracoronary injection. AskBio thanks the participants who volunteered for this important clinical trial, the sites that made this effort possible, and the skilled investigators who conducted this invaluable research and contributed to the scientific body of knowledge related to AB-1002. “We believe there is a critical need to progress innovative therapies that target the root causes of congestive heart failure, so we’re pleased to see these data for AB-1002 published and shared with the scientific community via Nature Medicine, a high-impact peer-reviewed journal,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio.”We’re eager to further assess the safety and efficacy of AB-1002 in our ongoing Phase 2 trial, GenePHIT, which is currently enrolling in Canada, Europe, the United Kingdom, and the United States, and look forward to sharing those results once available.” GenePHIT is a Phase 2, adaptive, randomized, double-blind, placebo-controlled trial investigating the safety and efficacy of AB-1002 in non-ischemic heart failure. About AB-1002 AB-1002 is an investigational one-time gene therapy administered to the heart to promote production of a modified version of the therapeutic inhibitor 1 (I-1c) protein designed to block the action of protein phosphatase 1, which is linked to CHF.4,5 This investigational gene therapy has not been approved by any regulatory authority, and its efficacy and safety have not yet been established or fully evaluated. About Congestive Heart Failure Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs.6 Congestive heart failure results in the slowing of the blood flow out of the heart, which causes the blood returning to the heart through the veins to back up.7 This causes congestion in the body’s tissues.8 Symptoms may include shortness of breath, swelling in the legs and ankles caused by fluid retention, and fatigue.8 More than 64 million people worldwide are estimated to be living with heart failure.2 About GenePHIT GenePHIT is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center

Research Triangle Park, N.C. – October 10, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced it will present initial safety data from the first cohort of participants from its Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in participants with limb-girdle muscular dystrophy (LGMD) 2I/R9 at the 30th Annual International Congress of the World Muscle Society, taking place October 7–11, 2025, in Vienna, Austria.  The presentation represents interim, blinded Cohort 1 safety data. Participants enrolled in Cohort 1 received a single intravenous infusion of AB-1003 or placebo and were followed for 52 weeks post-treatment during the main trial before entering a planned four-year long-term follow-up period. Safety assessments included adverse event monitoring, laboratory testing, physical exams, vital signs, electrocardiograms, and echocardiograms.1 There were no dose-limiting toxicities or serious adverse events reported up to 52 weeks post-treatment. Commonly reported (>2 participants) treatment-emergent adverse events were mild-to-moderate in severity and included headaches, falls, and nausea. Three participants reported asymptomatic transient transaminase elevations without changes in bilirubin levels, which returned to baseline levels after adjusting corticosteroid treatment.1 The data will be presented by Chris Passalacqua, MD, Vice President of Neuromuscular Medical Affairs, AskBio, at 3:45 p.m. CEST Friday, October 10, 2025. “These initial safety data are encouraging and suggest an acceptable safety profile for AB-1003,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We believe AAV-mediated gene therapy has the potential to restore FKRP function and stabilize disease progression, and we are excited to continue our clinical research efforts with the goal of developing an effective treatment for limb-girdle muscular dystrophy.” The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation 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 receive 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. Five participants were enrolled in the first cohort. All are actively participating and should remain in the trial until completion. Enrollment in Cohort 2 is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com. 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 fukutin-related protein (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 by mutations in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.5 Symptoms may start to appear from childhood to adulthood, and affected individuals may experience difficulty running and walking. The symptoms gradually worsen over time, and

Research Triangle Park, N.C.– OCTOBER 2, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver 6 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) 32nd Annual Meeting taking place October 7–10, 2025, in Seville, Spain. “ESGCT brings together leading researchers, clinicians, and industry experts to drive forward the field of gene and cell therapy and, as a company with a significant presence both in U.S. and Europe, this meeting is a special opportunity for us to interact with these prestigious stakeholders,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “This year, alongside Viralgen, AskBio is contributing six presentations that highlight not only our latest clinical milestones but also our advanced manufacturing capabilities. As we share new data spanning discovery, pre-clinical, and clinical stages, we reaffirm our commitment to delivering therapies at scale, with robust production and translational excellence. It is through this confluence of scientific rigor and operational strength that we aim to translate hope into real-world impact for patients facing serious rare and more common diseases.” AskBio’s presentations include (all times CEST): Oral Posters With an ambitious portfolio of gene therapies at various stages of research and development, including Phase 2, AskBio continues to develop AAV-based therapies to treat some of the world’s most debilitating diseases, including congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. By targeting diseases in several therapeutic areas, AskBio aims to deliver breakthrough treatments that could benefit more than 35 million patients worldwide.1–6 About AskBio AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to steering gene therapy into a new era where it can transform the lives of a wider range of people living with rare and more common diseases. The company maintains a portfolio of clinical programs across a range of disease indications related to a single gene or multiple factors across cardiovascular, central nervous system, and neuromuscular conditions,with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s end-to-end gene therapy platform includes our Pro10™ technology, which makes gene therapies more accessible by making research and commercial grade manufacturing more affordable. With global headquarters in Research Triangle Park, North Carolina, 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, founded in 2017 as a subsidiary of AskBio Inc. within the Bayer AG group, is a contract development and manufacturing organization

Research Triangle Park, N.C. – September 22, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first European participants have been randomized in REGENERATE-PD, a Phase 2 clinical trial in participants with moderate-stage Parkinson’s disease (PD).  “I believe the randomization of the first European participants in REGENERATE-PD, which makes this the first neurosurgical gene therapy program for Parkinson’s to successfully randomize patients from both the United States and Europe into a single Phase 2 trial, is positive news for people living with Parkinson’s disease and the physicians treating them,” said Alan Whone, MD, PhD, REGENERATE-PD Europe Lead and 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 2 clinical trial will give hope to patients and the medical community alike.”  PD is a progressive, neurodegenerative disorder affecting 1.2 million people in Europe, and this number is expected to double by 2030.1 With the worldwide unmet medical need in mind, the objective of REGENERATE-PD, a randomized, double-blind, surgically controlled clinical trial, is to evaluate the safety and efficacy of investigational gene therapy AB-1005 delivered to the putamen in the brain of adult participants aged 45–75 years with moderate-stage PD.2 REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers in Germany, Poland, the United Kingdom, and the United States.2  “Today’s announcement marks an important milestone in the clinical development of our investigational gene therapy AB-1005, as we continue to work hard to deliver a safe and effective treatment option that may be neurorestorative for certain populations and may benefit people living with moderate-stage Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “We are encouraged by the initiation of the REGENERATE-PD program in Europe and look forward to sharing further clinical updates as the program advances over the coming year.”  Earlier this year, AB-1005 was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA).3 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.4 AB-1005 met the RMAT requirements as a gene therapy that is intended to slow disease progression and improve motor outcomes in patients with PD. 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.4  AskBio is also exploring AB-1005 beyond PD, in participants in the United States with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase 1 clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing condition.5  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