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
Category: News
AskBio Announces Publication in Nature Medicine of 12-month data from Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure
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
AskBio Presents Interim Safety Results from Phase 1/Phase 2 LION-CS101 Clinical Trial of AB-1003 in Participants with Limb-Girdle Muscular Dystrophy 2I/R9
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
AskBio Announces 6 Presentations at the European Society of Gene and Cell Therapy 32nd Annual Meeting
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
First European Participants Randomized in AskBio Phase 2 Gene Therapy Trial of AB-1005 for Parkinson’s Disease
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
AskBio Announces Completion of Enrollment in Phase 1 Clinical Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type (MSA-P)
Research Triangle Park, N.C. – SEPTEMBER 17, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the completion of enrollment for REGENERATE MSA-101, its Phase 1 clinical trial of AB-1005, an investigational gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P).1 The completion of enrollment marks a significant milestone in the development of AB-1005, an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, and it brings this potential treatment option one step closer to reaching the many patients affected by MSA-P. MSA-P can initially be difficult to distinguish from Parkinson’s disease and is marked by slow movement, lack of coordination, imbalance, dizziness, and fainting, among other symptoms.2 Individuals experience increasing difficulty with movement and autonomic dysfunction.2 This is a result of progressive loss of nerve cells in the brain and spinal cord.2 Affecting approximately 400,000 people worldwide, MSA is a rare disease that in most cases seems to occur for unknown reasons.2,3,4 Symptoms tend to develop in people over 50 years of age, followed by rapid progression within 5–10 years.2 “There is currently no disease-modifying treatment to stop or slow the progression of MSA, which makes the completion of enrollment in REGENERATE MSA-101 an important step in AskBio’s efforts to advance this clinical program and potentially bring a safe treatment to patients” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA at AskBio. “This latest milestone follows recent additional AB-1005 announcements highlighting meaningful progress in our Parkinson’s disease program, including Phase 1b favorable safety outcomes.” AskBio is also exploring AB-1005 beyond MSA-P in participants living with Parkinson’s disease in REGENERATE-PD, the company’s randomized, double-blind, surgically controlled Phase 2 clinical trial evaluating the safety and efficacy of AB-1005 delivered to the putamen in the brain of adult participants aged 45–75 years with moderate-stage Parkinson’s.5 In January, AskBio announced that the first participants had been randomized in REGENERATE-PD.6 In February, AskBio received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AB-1005 for Parkinson’s disease, following the presentation of Phase 1b 36-month data that demonstrated a favorable safety profile and continued positive trends in assessed clinical outcome measures with no product-related serious adverse events.7,8 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 the Phase 1 REGENERATE MSA-101 Trial REGENERATE MSA-101 is a randomized, double-blind, placebo-controlled Phase 1 trial designed to determine the safety of AB-1005, an investigational gene therapy delivered to the putamen in patients with multiple system atrophy-parkinsonian type (MSA-P).1 The trial will include adults aged 35 to 75 years, with a clinical diagnosis of MSA-P (as defined by current consensus criteria), who are suitable surgical candidates.1 The trial enrolled 11 participants who were randomized on the day of surgery to receive either AB-1005 or a non-invasive control surgery without any
AskBio Announces Publication of Complete Results of Phase 1b Trial of AB-1005 Gene Therapy in Participants with Parkinson’s Disease in Movement Disorders
Research Triangle Park, N.C.– MAY 27, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the recent publication of the complete results of its Phase 1b trial of AB-1005, a glial cell line-derived neurotrophic factor (GDNF) investigational gene therapy for the treatment of Parkinson’s disease (PD), in Movement Disorders, an official peer-reviewed journal of the International Parkinson and Movement Disorder Society.1 “The exploration of the neurorestorative and neuroprotective potential of GDNF gene transfer is critical to advancing our understanding of AB-1005, which may one day be a groundbreaking treatment to slow the progression of Parkinson’s disease,” said Chad Christine, MD, Professor of Neurology at the University of California, San Francisco, and publication author. “These encouraging results support further evaluation of this therapy in a randomized trial.” The publication, which is available online, states that treatment with AB-1005 was well tolerated with no serious adverse events related to GDNF gene therapy in all 11 participants and is associated with numerical stability in clinical assessment readouts (mild cohort) and improvement (moderate cohort) in clinical assessments of motor function at 18 months post-gene transfer. In addition to these data, which were first presented in April 2024 at the American Academy of Neurology Annual Meeting, the publication notes that non-motor assessments remained numerically stable throughout the 18-month follow-up in the mild and moderate cohorts. Stability findings may be an indication of the potential for AB-1005 to positively affect disease progression.1,2 “These results support the continued study of AB-1005 as a potential gene therapy to slow the progression of Parkinson’s disease,” said Lila Collins, PhD, Associate Director of Portfolio Development & Review at the California Institute for Regenerative Medicine. “We are pleased to have supported this important trial and look forward to AskBio’s Phase 2 REGENERATE-PD results, which will provide additional insights into the durability of the clinical response and potential disease-modifying effect of AB-1005.” In February 2025, AB-1005 was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration.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 fully established or evaluated. About Parkinson’s disease Parkinson’s disease (PD) is a progressive neurodegenerative disease.4 It has a significant impact on a person’s daily life.4 In PD, the death of dopamine-producing nerve cells in the brain leads to the continuous loss of motor function.5 Symptoms include tremors, muscle rigidity, and slowness of movement.6 Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression.6 Symptoms typically intensify over time and make everyday tasks increasingly demanding.6 The prevalence of PD has doubled over the past 25 years.4 Today, more than 10 million people worldwide are estimated to be living with PD.7 This makes it the world’s second most prevalent neurodegenerative disease.8 It is also the most frequent movement disorder.4,9 At present there is no cure, and current treatment options lack the holistic management of symptoms,
AskBio Presents Complete Results of Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure at European Society of Cardiology Heart Failure Meeting
Research Triangle Park, N.C. – MAY 19, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, presented—during a dedicated late-breaking science session at this year’s European Society of Cardiology Heart Failure meeting—the complete dataset from a Phase 1 trial investigating AB-1002 for the treatment of congestive heart failure (CHF). This year’s meeting is being held in Belgrade, Serbia, from May 17 to 20, 2025. This non-randomized, sequential dose escalation trial includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with 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 in heart failure remain very high.2,3 The late-breaking oral presentation highlights that single-dose administration of AB-1002 at 12 months post-dose resulted in the following outcomes:4 Meaningful changes from baseline were considered to be: NYHA FC, ≥1 point; LVEF, ≥5%; MLHFQ, ≥10 points; VO2 max, ≥1.5 mL/kg/min; 6MWT, 30 meters. “No treatment-emergent or serious adverse events were deemed related to AB-1002 in this trial, and we saw clinically meaningful improvement of several efficacy assessments in some participants with non-ischemic CHF,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “These results support our belief that the AB-1002 chimeric capsid may be highly cardiotropic when administered as a single intracoronary injection at relatively low doses, and we’re excited to further evaluate the safety and efficacy of AB-1002 in GenePHIT, our currently recruiting Phase 2 trial.” Innovative therapies are urgently needed to address the underlying mechanisms associated with CHF, including improving pump function in participants with HFrEF.5 AB-1002 is a rationally designed cardiotropic adeno-associated virus (AAV) vector delivering a transgene for continuous onsite expression of the inhibitor 1 (I-1c), inhibitor of protein phosphatase 1, a protein that has been linked to heart failure. This Phase 1 trial is ongoing, and participants will be followed for 36 months post-intervention. Preliminary results of this trial, which did not include the additional three participants, were presented at the American Heart Association Scientific Sessions in November of 2023.6 AB-1002 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. About AB-1002 AB-1002 is a one-time gene therapy administered to the heart to help promote increased 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.6,7 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.8 Congestive heart failure results in the slowing of the blood flow out of the heart,
AskBio Announces 15 Presentations at American Society of Gene and Cell Therapy 28th Annual Meeting
Research Triangle Park, N.C.– MAY 8, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver 15 presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which takes place May 13–17, 2025, in New Orleans, Louisiana, USA. The company’s oral and poster presentations will provide updates on key research and development activities in gene therapy and gene editing, as well as advancements in manufacturing capabilities. Featured oral presentations will include “AI Use for Promoter and Transgene Design.” This will be delivered by Mansuo Shannon, Chief Scientific Officer at AskBio, as part of the Novel Approaches to Overcome Limits of Therapeutic Transgene Delivery and Durability scientific session. The presentation will explore how AI/ML (Artificial Intelligence/Machine Learning) may enhance efficacy, safety, and specificity of gene therapies by potentially predicting the best possible genetic sequences for the intended therapeutic outcome. In addition, AskBio’s Srinethe Saravanakumar will present “Vector Assembly Factories in Recombinant Adeno-Associated Virus Type 2 Producing Cells” as part of the AAV Biology and Mechanism session. The presentation will provide an in-depth visualization of rAAV2 (recombinant adeno-associated virus type 2) capsid assembly using electron tomography. It will also explore the potential effects of subcellular retargeting/expansion of capsid assembly through selected AAP (assembly-activating proteins) in vector production. The company’s third oral, “A Rock in a Storm: Using Data as Your Foundation to Speed and De-risk AAV Manufacturing,” will be presented by Viralgen’s María Iglesias González as part of the Tools and Technology session. This will explore how a data-led approach has the potential to significantly reduce variability, prevent costly failures, and shorten timelines from R&D to commercial production by enabling better process understanding, predictive modeling, real-time monitoring, and quality control across the development lifecycle. “Our scientific contribution to ASGCT this year underscores our long-standing commitment to advancing gene therapy research and providing leadership within the scientific community,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “We are sharing updates from our early-stage programs and highlighting how we are adopting innovative approaches in our development work with the use of digitalization and AI. Equally important, we are also showcasing the work being done to advance our manufacturing processes as we continue to strengthen our end-to-end platform capabilities.” AskBio continues to develop an ambitious portfolio of investigational AAV-based gene therapies to treat some of the world’s most serious diseases, including congestive heart failure, 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 tens of millions of patients worldwide.1–6 AskBio’s presentations at ASGCT include (all times CDT): Orals Posters About AskBio AskBio Inc., 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
AskBio Elects Dr. Laura Sepp-Lorenzino Independent Member of Board of Directors
Research Triangle Park, N.C. April 8, 2025 – AskBio Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that its Board of Directors has elected Independent Director Laura Sepp-Lorenzino, PhD, to the Board, effective April 4, 2025. “We are pleased to welcome Dr. Sepp-Lorenzino to our Board of Directors,” said Gustavo Pesquin, CEO, AskBio. “Laura’s extensive experience as a scientist and adviser will support AskBio’s efforts to stay at the cutting-edge of innovation. She brings to our Board broad expertise across modalities and therapeutic areas, including gene therapy and editing, from discovery through late-stage development. Her contributions will be invaluable as we move our clinical programs through and beyond Phase 2 and advance our next pre-clinical candidates.” Dr. Sepp-Lorenzino brings to AskBio’s Board over 30 years of executive and academic experience and leadership skills. She currently serves as Scientific Advisor, Executive Vice President, at Intellia Therapeutics, where she is helping develop and execute Intellia’s strategic vision and plan, positioning the company as the leader in systemic CRISPR medicines. She is also the company’s former Chief Scientific Officer. Prior to her work at Intellia, Dr. Sepp-Lorenzino served as Vice President, Research and External Innovation, Head Nucleic Acid Therapies, at Vertex Pharmaceuticals; Vice President, Entrepreneur-in-Residence, at Alnylam Pharmaceuticals; and held roles of increasing responsibility at Merck & Co., Inc. In addition, she serves on the Boards of Taysha Gene Therapies and Alliance for Regenerative Medicine (ARM), as well as the Scientific Advisory Boards of Thermo Fisher Scientific; Arsenal Capital Partners; and UK Medical Research Council, Nucleic Acid Therapy Accelerator. Dr. Sepp-Lorenzino began her career as Assistant Attending Molecular Biologist at Memorial Sloan Kettering Cancer Institute. She received her undergraduate degree in Biochemistry and Pharmacy from the University of Buenos Aires and her PhD in Biochemistry from New York University. Her recent awards include 2023 Rosalind Franklin Award in Science, 2022 Women in Biopharma, and 2019 Fiercest Women in Life Sciences. Further, Dr. Sepp-Lorenzino currently sits on the editorial boards of the following scientific journals: Nucleic Acid Therapeutics, Molecular Therapy Nucleic Acids, and Cell & Gene Therapy Insights. “Laura is a trail-blazing leader in the industry whose transformational impact on companies and their programs has been widely acknowledged,” said Mansuo Shannon, PhD, Chief Scientific Officer and Member of the Board of Directors, AskBio. “Her decades of innovative leadership in drug discovery and the development of small molecules, biologics, oligonucleotides, and CRISPR-based medicines have resulted in the successful translation of disruptive, enabling technologies into new therapeutic modalities with differentiated products. Her in-depth knowledge of what it takes to steer therapies from research to commercialization will uniquely position the Board to support AskBio through the next phase of its development. We welcome Laura and look forward to her contributions.” “I am excited to join AskBio’s Board,” said Dr. Sepp-Lorenzino. “This is a special time for the company, which is known and respected for its long history of pioneering science and innovation, as it begins to move its clinical pipeline and
