Category: News

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

Research Triangle Park, N.C.– MAY 2, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver nine presentations offering insights into its adeno-associated virus (AAV) research and development, a key area of gene therapy focus for the company, at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which takes place May 7–11, 2024, in Baltimore, Maryland, USA. Presentations will focus on AAV immune-mediated responses as well as the results from the ongoing Phase 1 clinical trial of AB-1002 gene therapy in patients with advanced heart failure. Luke Roberts, MBBS, PhD, Medical Director for Clinical Development at AskBio, will deliver an oral presentation sharing new clinical data from the company’s ongoing Phase 1 trial of AB-1002 in patients with advanced heart failure. This follows AskBio’s recent news that AB-1002 was granted FDA Fast Track Designation for the treatment of congestive heart failure (CHF). AB-1002 (also known as NAN-101) is an investigational gene therapy that has not yet received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio previously communicated that the delivery of AB-1002 was well tolerated and resulted in positive preliminary efficacy outcomes in some patients with non-ischemic CHF and may validate that the AAV2i8 vector capsid used is highly cardiotropic when administered as a single intracoronary infusion at relatively low doses. Preliminary data from the Phase 1 trial of AB-1002 were presented at the 2023 American Heart Association Scientific Sessions in November, and AskBio began enrolling patients in its Phase 2 GenePHIT study of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms in January 2024. AskBio’s ASGCT presence will also include key presentations showcasing the company’s continued commitment to optimizing AAV as a gene therapy, with a focus on preventing or reducing AAV immune response-related adverse events, which remains a vital area of investigation across the gene therapy treatment landscape. Attendees can look forward to an ASGCT spotlight speaker presentation on current immune modulation strategies given by Shari Gordon, PhD, Senior Director of Immunology at AskBio, on Day 3. On Day 4, Shari Gordon will deliver on behalf of Audry Fernandez, PhD, Principal Scientist & Group Lead, Immunology R&D at AskBio, an oral presentation on pre-clinical research into AAV-specific immune responses, and Liujiang Song, PhD, Principal Scientist for R&D Capsid and Biology at AskBio, will offer insights into AAV biology and vector intracellular fate during an oral presentation on AAV episome configuration using third generation long-read sequencing technologies and advanced bioinformatics. “Our presence at ASGCT this year highlights our continued commitment to sharing AAV developments with the gene therapy community. Covering clinical and pre-clinical research, our presentations show our robust progress and ongoing ambition to bring to patients transformative therapies that were once unthinkable,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “With our clinical and early-stage programs advancing, these are exciting times at AskBio.” With an ambitious

Berlin, Germany, and Research Triangle Park, NC, USA, April 18, 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 U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1. Inhibiting the function of this protein, which is linked to congestive heart failure (CHF), could potentially lead to a therapeutic effect on the heart.[1],[2] “The FDA Fast Track Designation for AB-1002 is an important accomplishment for the clinical development of this program and highlights our goal of potentially bringing effective treatments to patients with advanced congestive heart failure,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We look forward to completing our Phase II GenePHIT clinical trial, which is currently enrolling patients with severe heart failure, and are committed to exploring the full potential of AB-1002 for the treatment of this devastating disease.” 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.[3] The purpose of the Program is to get important new therapeutics to patients earlier.3 Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review. “The Fast Track Designation for AB-1002 emphasizes the need to rapidly advance new therapeutic modalities such as gene therapy for people living with congestive heart failure,” said Christian Rommel, PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division. “This designation underpins the potential of AB-1002 to address currently high unmet medical need, and we are excited about the opportunity to accelerate its development.” AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio is currently enrolling patients in the Phase II GenePHIT (Gene PHosphatase Inhibition Therapy) trial of AB-1002 (also known as NAN-101) for the treatment of CHF.[4]  About GenePHIT GenePHIT is a Phase II adaptive, double-blinded, placebo-controlled, randomized, multi-center trial to evaluate the safety and efficacy of the one-time administration of AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms.4 Subjects are randomized into one of three treatment groups in a 1:1:1 fashion to either low dose, high dose, or placebo. Primary outcome measures include cardiovascular related death and change from baseline in NYHA classification, left ventricular ejection fraction (LVEF), peak oxygen uptake (pVO2), and Six Minute Walk Test (6MWT).4 For more information, please visit clinicaltrials.gov

Berlin, Germany, and Research Triangle Park, NC, USA, April 16, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, on Sunday April 14 presented results from the 18-month Phase Ib clinical trial for AB-1005, an investigational gene therapy for treating patients with Parkinson’s disease (PD).1,2The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA. The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Eleven patients were enrolled into two cohorts, Mild stage PD (6 patients) and Moderate stage PD (5 patients), based on timing from PD clinical diagnosis and the severity of PD symptoms at trial screening.1 As of November 3, 2023, 57 nonserious adverse events (AEs) and 6 serious adverse events (SAEs) were reported. Most AEs were transient and were expected perioperative events (<1 month from treatment). These included headache, tremor, dyskinesia, arthralgia, musculoskeletal chest pain, fatigue, COVID-19, and magnetic resonance imaging (MRI) abnormalities. The 6 SAEs reported in 3 patients (n = 1 in the Mild Cohort and n = 2 in the Moderate Cohort) were all assessed as unrelated to the treatment by the Investigator and the Sponsor. Bilateral infusions of AB-1005 within the putamen (up to 1.8 mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent. Neurosurgical delivery of AB-1005 resulted in putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005. Scheduled 6-month postoperative MRIs revealed findings of asymptomatic unilateral T1 hypointensity adjacent to 3 of the putaminal infusion trajectories. Clinical follow-up for up to 5 years post administration is ongoing.2 “These early findings are encouraging and show AB-1005 to be well tolerated in this study in patients with mild to moderate Parkinson’s disease,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “Further, they highlight areas of potential future exploration in our upcoming Phase II REGENERATE PD trial, which will look more closely at the potential efficacy of AB-1005 in the treatment of Parkinson’s disease.” Patients also completed 18-month neurological assessments and self-reported questionnaires at regular intervals to evaluate the severity of motor and non-motor symptoms associated with PD.1 Mild Cohort The Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) is an internationally recognized tool used to assess the severity of PD symptoms, including motor symptoms. The Mild Cohort (n = 6) demonstrated relative stability from baseline to 18 months for both MDS-UPDRS Part II patient-reported Activities of Daily Living scores and Part III clinician-rated Motor Examination scores in “ON” and “OFF” medication states. Patient-reported PD Motor Diaries provide a tool for assessing patient motor state over an extended 3-day period and then normalized to a 16-hour waking day. The Mild Cohort (n = 5) showed a -1.3 hour reduction in “Good ON” time, a 0.2 hour increase in “ON” time with troublesome

Not intended for UK Media Berlin, Germany and Research Triangle Park, NC, USA, February 13, 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 first patient has been randomized in GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase II trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). GenePHIT is an adaptive, double-blind, placebo-controlled, randomized, multicenter trial to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms who have been medically stable for at least four weeks. This milestone in the development of AB-1002 for the treatment of CHF potentially brings this investigational therapy one step closer to treating patients with high unmet medical need.[2] GenePHIT will include between 90 and 150 adults with left ventricular ejection fraction between 15 and 35 percent, who continue to suffer from heart failure symptoms despite guideline recommended therapy. The primary efficacy endpoint at 52 weeks is a modified win ratio of several clinically meaningful assessments.1 “The randomization of the first patient as a part of the Phase II GenePHIT trial is an important moment for the heart failure community,” said Timothy D. Henry, MD, MSCAI, Principal Investigator and Steering Committee Member. “GenePHIT will evaluate the safety and efficacy of AB-1002 in the largest number of patients to date and improve our understanding of gene therapy overall for the treatment of congestive heart failure. The initiation of this trial brings us a step closer to potentially changing the course of this deadly and devastating disease.” “Being able to announce this important GenePHIT trial update during Heart Failure Awareness Week adds special significance to this milestone,” said Roger J. Hajjar, MD, Scientific Chair CHF, AskBio. “The enrollment of this first patient in the Phase II trial represents the culmination of many years of dedicated research and development in all aspects of cardiac gene therapy for congestive heart failure. Although there is still much to learn about this early-stage investigational gene therapy, we hope today’s announcement, which highlights AskBio’s ability to advance AB-1002 gene therapy for the treatment of congestive heart failure, is encouraging news for everyone hoping to see new treatment options.” “Heart failure is a devastating disease with increasing unmet medical need, especially in a progressively aging population,” said Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “The potential impact of gene therapy to address this disease at its root cause is immense, and we are thrilled about this step in our path to deliver truly innovative treatment options for patients.” AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AB-1002 is manufactured by Viralgen Vector Core, S.L., a wholly owned and independently operated subsidiary of