A Phase 2, Open-label, Single Group, Fixed-dose Study to Evaluate the Safety and Efficacy of a Single Bilateral Intraputaminal Administration of AB-1005 (AAV2-GDNF Gene Therapy) in Japanese Participants With Moderate Stage Parkinson's Disease

The objective of this Phase 2, open-label, single group, one-time, fixed-dose study is to assess the safety and efficacy of bilateral intraputaminal infusion of AB-1005 (AAV2-GDNF gene therapy) in Japanese patients with moderate Parkinson's Disease (PD).

Start Date25 Jan 2027

Sponsor / Collaborator

Bayer AG

[+1]

NCT07215403

/ Not yet recruitingPhase 2

Open-Label, Multi-Stage Study to Optimize the Intraputaminal Administration of AB-1005 Using a Prescriptive Infusion Algorithm (PIA)

This study is being done to test a new way of delivering AB-1005 into the brain. The goal is to make the procedure easier and quicker to perform, while providing similar amounts of drug to the part of the brain that needs treatment.
Technical (Stage 0) To test if the new delivery method (prefrontal surgical approach) can consistently deliver AB-1005 to the putamen using MRI monitoring.
Technical (Stage 1) To test if the new delivery method (PIA-based infusion) can consistently deliver AB-1005 to the putamen using brain imaging by MRI.
Technical (Stage 2) To confirm the new delivery method works without brain imaging by MRI, using standard operating room tools including brain imaging by CT.
Safety (Stage 1 and 2) To assess the safety and tolerability of the new delivery method for AB-1005 up to 6 months after surgery

Start Date01 Nov 2026

Sponsor / Collaborator

AskBio Inc.

[+1]

NCT06285643

/ RecruitingPhase 2

A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease

The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.

Start Date11 Jun 2024

Sponsor / Collaborator

AskBio Inc.

[+1]

100 Clinical Results associated with Ametefgene parvec

100 Translational Medicine associated with Ametefgene parvec

100 Patents (Medical) associated with Ametefgene parvec

News (Medical) associated with Ametefgene parvec

18 May 2026

·www.biospace.com

4 spaces primed for the next wave of gene therapies

iStock, spukkato The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases. Last month, the FDA approved Regeneron Pharmaceuticals’ Otarmeni for a specific type of hearing loss, validating the concept of genetic medicines. As these medicines continue to advance, countless more conditions stand to benefit from the approach. Researchers are taking note. “The hegemony of all cell and gene therapy has been [oncology] for a long time,” Roger Hajjar, head of Mass General Brigham’s Gene & Cell Therapy Institute and chief medical officer at gene therapy company Medera, told BioSpace . “Now, we’re seeing a switch toward neuro diseases, ophthalmology, cardiac diseases.” The ear is also a common focus, he noted. Otarmeni , the first gene therapy for hearing loss, is intended for individuals with congenital deafness caused by a deficiency of the otoferlin protein, which is critical for the hearing process. Eli Lilly, too, is working on an otoferlin-targeted gene therapy for the same condition. The first wave of gene therapies homed in primarily on cancer because of the otherwise very limited treatment options, according to Hajjar. Over time, however, as scientists and doctors have gained experience with novel approaches, “these experimental vectors have become the mainstay of gene therapy and now are being used for other diseases where they can deliver missing genes or they can correct genes.” Amid that ongoing evolution, as researchers continue to branch out beyond oncology for the next wave of targeted gene therapies, a handful of therapeutic areas stand out as prime targets. Gene therapy Sarepta’s Woes Put the Stalwart AAV Under a Harsh Light In the wake of multiple patient deaths from liver injuries related to Sarepta Therapeutics’ AAV gene therapy platform, some in the sector are looking for ways to improve the current technology, while others are eager to move on. August 11, 2025 · 7 min read · Dan Samorodnitsky Read more Lilly, UniQure, Lexeo, more target the CNS Several therapies are currently in development to treat diseases of the central nervous system (CNS). Lexeo Therapeutics , for example, is studying an adeno-associated virus (AAV)-based gene therapy designed to deliver an APOE2 gene variant to treat early to moderate Alzheimer’s disease. Research shows that those with two copies of APOE2 are significantly less likely to develop Alzheimer’s, suggesting that the gene variant may offer some neuroprotective qualities, per the company’s website . Meanwhile, Eli Lilly and Bayer’s AskBio are among those advancing experimental gene therapies for Parkinson’s disease. AskBio’s candidate, AB-1005, aims to restore continuous production of the GDNF protein, enabling nerve cells to trigger healthy body movement, according to the biotech’s website. Lilly’s asset, currently in a Phase 1/2a trial, is intended for patients with at least one mutation in the GBA1 gene. Harboring mutations in this gene is known to increase risk of Parkinson’s. Taysha Gene Therapies , meanwhile, is targeting the rare neurodevelopmental disorder Rett syndrome with an AAV-based therapy of its own. Rett syndrome is caused by mutations in the MECP2 gene, which aids in regulating brain function; TSHA-102 delivers a form of the gene directly to the cerebrospinal fluid. Passage Bio, meanwhile, is developing PBFT02, which delivers a modified DNA encoding copies of the granulin gene directly to the cerebrospinal fluid for a range of neurodegenerative disorders. The company’s lead program is in frontotemporal dementia, but the asset is also being studied in amyotrophic lateral sclerosis (ALS) and Huntington’s disease. Huntington’s is a common target. Last September, uniQure shared Phase 1/2 data revealing that its gene therapy AMT-130, which aims to silence the mutant huntingtin gene, had slowed disease progression by 75% after three years. Excitement over the results was stymied, however, by the announcement that the FDA will require a sham surgery-controlled Phase 3 trial before it will consider AMT-130 for approval, though uniQure has vowed to continue conversations with the regulator about alternate routes forward. As time goes on, precision approaches like these will be a core aim of gene therapy developers, according to Hajjar. “The next frontier is going to be about improving our delivery systems with novel vector systems,” he said, adding that the genetic material being delivered and editing tools being used will also continue to improve. “All these things are evolving toward genetic therapies becoming much more targeted. And as they become more targeted, they will be safer to use.” Huntington’s disease UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for the therapy, perhaps even based on precedent from the recent drama surrounding Moderna’s mRNA flu vaccine. March 5, 2026 · 7 min read · Heather McKenzie Read more REGENXBIO, AbbVie, 4D target VEGF in eye disease Among the handful of companies developing gene therapies to treat various eye diseases are REGENXBIO and AbbVie, which have teamed up to study a potential treatment for wet age-related macular degeneration (AMD) and diabetic retinopathy—two vascular endothelial growth factor (VEGF)-driven conditions in which patients “face high treatment burden that leads to undertreatment and ultimately vision loss over time,” a REGENXBIO spokesperson told BioSpace via email. VEGF stimulates the growth of new blood vessels and increases the permeability of existing ones, causing fluid and blood to leak into the macula , an area at the center of the retina responsible for sharp, central vision. AbbVie and REGENXBIO’s gene therapy, dubbed sura-vec, is delivered either subretinally or suprachoroidally—injected between the sclera and choroid—to inhibit VEGF and fluid accumulation in the retina, potentially preventing disease progression and preserving vision. Pivotal data in wet AMD are expected to arrive in the fourth quarter of this year, with dosing set to begin in a Phase 2b/3 diabetic retinopathy trial this quarter, the spokesperson said. Also studying a gene therapy approach to wet AMD is 4D Molecular Therapeutics. Its 4D-150 , which is designed to durably suppress four different VEGF family members, is currently being studied in two Phase 3 trials, along with an early-phase study in diabetic macular edema. 4D-150 is delivered intravitreally . Tenaya, Medera target cardiovascular disease Targeted treatments are on the rise in cardiovascular indications. Hajjar noted a “longstanding trend” of using gene therapies to treat more common diseases like congestive heart failure via well-characterized pathways, but he added that companies are increasingly seeking to replace the faulty genes at the root of genetic cardiomyopathies. Straddling that line between rare and prevalent heart conditions are Tenaya Therapeutics and Hajjar’s company, Medera. Tenaya’s clinical-stage pipeline includes a pair of gene therapies being studied in hypertrophic cardiomyopathy (HCM) and arrhythmogenic right ventricular cardiomyopathy. The biotech is also conducting early-stage development of gene therapies in certain types of heart failure. Furthest along is the HCM candidate TN-201, which aims to correct for mutations in the MYBPC3 gene, a key player in modulating the heart’s contractions. Early data have shown that after at least one year of follow-up, “multiple parameters of disease had moved toward normal,” CEO Faraz Ali told BioSpace in an email. “With TN-201, we have a unique opportunity to address the underlying genetic cause of disease and to halt and even reverse disease progression for all forms of the disease,” he said. Meanwhile, Medera’s trio of cardiac gene therapies span two common forms of heart failure as well as the much rarer Duchenne cardiomyopathy. Lead program SRD-002 is being developed for heart failure with preserved ejection fraction (HFpEF). Early data on the candidate have been positive, with Medera CEO Ronald Li pointing to “consistent and durable improvements in cardiac hemodynamics and functional measures.” He also noted in an email to BioSpace that no gene therapy–related serious adverse events or side effects have been observed so far, attributing this at least in part to Medera’s intracoronary delivery, where a catheter provides entry directly into the coronary arteries. This approach, Li said, allows for “up to ~300-fold lower vector doses compared to conventional systemic intravenous approaches,” which can reduce costs in addition to improving safety. The company’s approach is “fundamentally different” from the current standard of care, Li explained, as it seeks to address the root causes of impaired heart function rather than saddling patients with a lifetime of disease management. In HFpEF specifically, which affects approximately half of all heart failure patients and where disease-modifying options remain limited, Medera’s approach could represent “a meaningful step toward redefining the treatment paradigm,” he said. “More broadly, it demonstrates how aligning biology, delivery and human-relevant modeling can enable gene therapy to move beyond rare diseases into large, complex indications.” Sarepta, REGENXBIO tackle muscle deficiency in DMD Gene therapies have made significant inroads in neuromuscular diseases. To date, two gene replacement therapies have been approved by the FDA for spinal muscular atrophy: Novartis’ Zolgensma, cleared in 2019 for children under the age of 2, and the Swiss pharma’s Itvisma, greenlit last fall for patients with SMA aged 2 and older. Also active in the neuromuscular space is Sarepta Therapeutics, though it hasn’t been all smooth sailing for the biotech. After earning FDA approval in 2023 for its first gene therapy, Elevidys—which delivers a gene that instructs the body to make a shortened version of the dystrophin protein to treat Duchenne muscular dystrophy (DMD)—Sarepta was plagued last year by multiple patient deaths linked to its platform. This led to a brief pause in Elevidys sales, ultimately followed by the installation of a narrower label and black box warning. Regulatory FDA Imposes Boxed Warning, Narrows Patient Pool for Sarepta’s Duchenne Gene Therapy Elevidys Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy. November 17, 2025 · 1 min read · Tristan Manalac Read more Meanwhile, REGENXBIO reported positive topline Phase 3 results last week from its own DMD candidate, RGX-202 , showing that 93% of patients achieved microdystrophin expression above 10%. There was also a “statistically significant correlation” between this expression and functional improvement, the company said in its press release, “supporting [the] validity of the surrogate endpoint.” RGX-202 comprises a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT domain). Previously, interim one-year data showed positive effects on functional measures, as well as cardiac stability, with a safety pro indicated no evidence of liver injury and no serious adverse events. All together, the spokesperson said, those findings highlight “the potential of RGX-202 to be a differentiated gene therapy option for Duchenne.”

Phase 3Gene TherapyClinical ResultDrug Approval

13 May 2026

·www.einpresswire.com

The 7th World Parkinson Congress Announces 12 Hot Topics Presentations

Both Rising-Star Researchers and Senior Clinicians, and Advocates from around the World Present New Study Findings in Parkinson’s Disease PHOENIX, AZ, UNITED STATES, May 13, 2026 / EINPresswire.com / -- The 7th World Parkinson Congress (WPC 2026) , offered through the World Parkinson Coalition ® , announces today its 12 Hot Topics presentations, highlighting emerging areas in Parkinson’s research and disease management. Both senior and rising-star researchers, clinicians, and advocates from around the world will present new findings across basic science, clinical science, therapeutics, and care. The World Parkinson Coalition leadership, which includes more than 50 reviewers, assessed over 900 submissions, and whittled them down to the final 12 abstracts for Hot Topics presentations. The list of international authors of the selected abstracts will present their findings orally to the broader audience, just before the opening plenary each morning, four per day. WPC 2026 is being held in Phoenix, Arizona from May 24 – 27, 2026. “We are pleased to announce this year’s most exciting, cutting-edge 12 hot topics by both up-and-coming as well as established senior researchers and clinicians whose work adds great value to the Parkinson’s community,” states Elizabeth (Eli) Pollard, executive director, World Parkinson Coalition, the nonprofit, international organization behind the Congress. “The vast array of research, from genetics to improving day-to-day living with a better understanding of ongoing research, is further evidence how the global Parkinson’s community is working toward the common goal of improving disease management while working to find a cure.” The aim of the WPC 2026 is to unite the worldwide Parkinson community for a high-level scientific and educational program organized into four days of pre-congress courses, plenary sessions, workshops, and roundtables allowing for discussions on the most recent science and clinical research as well as advances in treatments designed to improve care and quality of life for people living with Parkinson’s disease. The 12 Hot Topics presentations at the WPC 2026 will look at: O Assessing the accuracy and timeliness of medication administration for hospitalized patients with Parkinson disease by Jake Plagenz (USA) O Predicting Parkinson's disease: exploring prodromal disease algorithms in PREDICT-PD by Ashvin Kuri (UK) O The syn-sleep study: detection of cutaneous phosphorylated alpha-synuclein in REM sleep behavior disorder by Christopher Gibbons (USA) O Whole brain [11C]-PE2I dopamine transporter PET imaging in persons with Parkinson's with freezing of gait or a history of falls by Nicolaas Bohnen (USA) O Clinical and neuropathological features of amygdala-predominant Lewy body disease: insights from a brain bank cohort by Natasja Deshayes (the Netherlands) O Impact of ultrafine particles from air pollution on Parkinson’s disease: from brain biodistribution to neurotoxicity by Emeline Barbier (France) O Parkinson’s disease genetic risk modulates microglia transcriptional states in α-synucleinopathies by Raphael Kubler (USA) O Association between α-synuclein pathology and brain mitochondrial function in an LBD marmoset model by Tetsuya Hirato (Japan) O Explaining early gene therapy research of AB-1005 (GDNF gene therapy) for Parkinson’s disease (PD): patients’/caregivers’ perspective by Esther Labib-Kiyarash (USA) O Lysosomal pathway expression profile of Parkinson’s disease patients is associated with disease severity by Janna van Wetering (the Netherlands) O Associating resting-state functional connectivity and improvements in reactive stepping in Parkinson’s disease by Emily Tobin (USA) O Robot-assisted transplantation of hypoimmune iPSC derived dopaminergic progenitor cells for idiopathic Parkinson’s disease by Jiong Shi (China) For more information about the abstracts, please visit: www. wpc2026.org/scientificprogram To connect with one of the authors to learn about their research or set up an interview, write to Julie@worldpdcoalition.org. ### About the World Parkinson Coalition ® and WPC 2026 The World Parkinson Coalition Inc provides an international forum for learning about the latest scientific discoveries, medical practices, caregiver initiatives and advocacy work related to Parkinson's disease. The triennial World Parkinson Congresses provide a space for the global Parkinson’s community of researchers, clinicians, health care professionals, people with Parkinson’s and their care partners to meet in person, network, and discuss advances in Parkinson’s research, improve understanding and promote advocacy worldwide, while influencing future research and care options. The WPC 2026 will attract more than 4,000 delegates. About Parkinson’s Disease Affecting more than one million Americans and 11 million people worldwide, Parkinson’s disease is the fastest growing neurodegenerative disease on the planet. While Parkinson’s is characterized by limited functional ability, a variety of nonmotor and motor symptoms significantly impact the quality of life of both people living with Parkinson’s and their care partners. This can include resting tremor, muscle rigidity, paid, fatigue, anxiety and depression. Julie Winn World Parkinson Coalition +1 802-578-1704 email us here Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Clinical Study

20 Apr 2026

·www.globenewswire.com

AskBio's Viralgen to Supply Commercial-Scale Drug Product for REGENERATE-PD Investigational Gene Therapy Phase II Trial for Parkinson’s Disease

Berlin, Germany, and Durham, N.C., USA, April 20, 2026 (GLOBE NEWSWIRE) -- Not intended for UK Media Ametefgene parvec (AB-1005) manufactured at Viralgen commercial manufacturing facility with next-generation process to deliver high-purity product with greater efficiency AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that it has introduced its proprietary, commercially ready manufacturing process to supply ametefgene parvec (AB-1005), an investigational gene therapy for the treatment of Parkinson’s disease (PD) and multiple system atrophy-parkinsonian type (MSA-P), following submission of a United States Food and Drug Administration (FDA) Investigational New Drug Application (IND) amendment. This enables AskBio to now advance its REGENERATE-PD Phase II clinical trial in the United States with the material produced at the commercial manufacturing facility of AskBio’s wholly owned subsidiary, Viralgen, with other trial sites to follow, using an intensified, high-efficiency, next-generation, large-scale suspension manufacturing process that provides a consistently high-purity product. “Today’s news marks another important advancement in our Parkinson’s program,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “We are initiating supply with our manufacturing platform, to deliver our investigational gene therapy to participants in our ametefgene parvec trials, including our REGENERATE-PD trial, which recently saw the randomization of our first participants in Germany. The trial is currently enrolling participants in Poland, the United Kingdom, and the United States.” “Manufacturing efficiency of our AAV gene therapy-based drug product is mission critical for our Phase II Parkinson’s disease trial,” said Christian Rommel, PhD, Global Head of Research and Development for Bayer’s Pharmaceuticals Division. “It underscores the innovation of our production technology and reflects our continued commitment to meet high regulatory standards.” In December 2025, AskBio was granted Pioneering Regenerative Medical Product designation (SAKIGAKE) in Japan for ametefgene parvec, and in February 2025 the company was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.1,2 Prior to that, AskBio received FDA Fast Track designation and the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (UK MHRA) innovative medicine designation.3 All designations were for PD. AskBio is also exploring ametefgene parvec in participants in the United States with the parkinsonian subtype of MSA-P in a fully enrolled Phase I clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing neurodegenerative condition.4 Ametefgene parvec 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 (PD) is a progressive neurodegenerative disease.5  It has a significant impact on a person’s daily life.5  In PD, the progressive death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function.6 Symptoms include tremors, muscle rigidity, and slowness of movement.7 Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression.7 Symptoms typically intensify over time and make everyday tasks increasingly demanding.7 The prevalence of PD has doubled over the past 25 years.5 Today, more than 10 million people worldwide are estimated to be living with PD.8 This makes it the world’s second most prevalent neurodegenerative disease.9 It is also the most frequent movement disorder.5,10 At present there is no cure, and current treatment options lack the holistic management of symptoms, so there is an urgent need for new therapies.11 About Parkinsonian subtype of multiple system atrophy The parkinsonian subtype of multiple system atrophy (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.12 Individuals experience increasing difficulty with movement and autonomic dysfunction as a result of progressive loss of nerve cells in the brain and spinal cord.12 Affecting approximately 400,000 people worldwide, MSA-P is a rare disease, and its exact cause is unknown.12,13,14 Symptoms usually start to develop around the age of 50, followed by rapid progression within 5–10 years.12 At present there is no cure and no approved treatments for MSA-P, so there is an urgent need for new therapies.12 About REGENERATE-PD REGENERATE-PD is a Phase II, randomized, double-blind, surgery controlled trial of the efficacy and safety of intraputaminal ametefgene parvec (AB-1005) in the treatment of adults (45–75 years) with moderate-stage Parkinson’s disease.15 The trial will include an estimated number of 127 participants with trial sites located in Germany, Poland, the United Kingdom, and the United States.15 For more information about the REGENERATE-PD clinical trial, visit clinicaltrials.gov (NCT06285643), or visit askbio.com. About ametefgene parvec (AB-1005) Ametefgene parvec (AB-1005) is an investigational gene therapy intended to slow disease progression and improve motor outcomes in people with Parkinson’s disease (PD). Composed of an adeno-associated viral vector containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, ametefgene parvec allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with convection-enhanced delivery.16,17 In nonclinical studies, GDNF has been shown to promote the survival and morphological differentiation of dopaminergic neurons, which could aid in the preservation and restoration of dopaminergic neuronal circuitries normally lost in the disease.17,18 GDNF has long been evaluated as a potential disease-modifying treatment for diseases, such as PD which are marked by progressive degeneration of midbrain dopaminergic neurons.18 Through a combination of an investigational gene therapy and innovative neurosurgical delivery approach, building on the promising outcomes of the open-label Phase 1b trial, we can now test the GDNF hypothesis by getting this neurotrophic factor to these degenerating nigrostriatal neurons in a well-controlled clinical study to understand the effects of GDNF in a moderate PD population.19 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 and Aava™ manufacturing platform, which make 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 http://www.askbio.com or follow us on LinkedIn. About Viralgen Viralgen is a leading contract development and manufacturing organization (CDMO) specializing exclusively in adeno-associated virus (AAV)–based gene therapies. Founded in 2017 as a subsidiary of AskBio Inc. within the Bayer AG group, Viralgen provides end-to-end support from early development through large-scale commercial production. Leveraging its proprietary Pro10™ suspension cell line and Aava™ manufacturing platform, Viralgen achieves industry-leading, high-yield, scalable manufacturing across all AAV serotypes. Located in San Sebastián, Spain, its state-of-the-art facility includes three cGMP suites with 2,000-liter capacity each, certified by the AEMPS/EMA, and integrates services such as plasmid production, process optimization, fill-finish, and in-house QC testing. With more than 1,200 AAV batches produced, Viralgen delivers reliable, efficient solutions that help bring life-changing gene therapies to patients with greater speed and consistency. For more information, visit https://viralgen.com/. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2025, the Group employed around 88,000 people and had sales of 45.6 billion euros. R&D expenses amounted to 5.8 billion euros. For more information, go to www.bayer.com. ime (2026-0007E) References 1. Askbio.com. AskBio’s AB-1005 and AB-1002 Receive Pioneering Regenerative Medical Product Designation in Japan. Available at: https://www.askbio.com/ab-1005-and-ab-1002-receive-designation-in-japan/. Accessed: April 2026. 2. Askbio.com. AskBio Receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease investigational gene therapy. Available at: https://www.askbio.com/askbio-receives-fda-regenerative-medicine-advanced-therapy-designation-for-parkinsons-disease-investigational-gene-therapy/. Accessed: April 2026. 3. Askbio.com. AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinson’s disease. Available at: https://www.askbio.com/askbio-receives-fda-fast-track-and-mhra-innovation-passport-designations-for-ab-1005-investigational-gdnf-gene-therapy-for-parkinsons-disease/. Accessed: April 2026. 4. ClinicalTrials.gov. GDNF Gene Therapy for Multiple System Atrophy. Available at: https://clinicaltrials.gov/study/NCT04680065. Accessed: April 2026. 5. World Health Organization. Parkinson Disease. Available at: https://www.who.int/news-room/fact-sheets/detail/parkinson-disease. Accessed April 2026. 6. Ramesh S & Arachchige A. Depletion of dopamine in Parkinson’s disease and relevant therapeutic options: A review of the literature. AIMS Neurosci. 2023 Aug 14;10(3):200-231. 7. National Institutes of Health. Parkinson’s Disease. Available at: https://www.ninds.nih.gov/health-information/disorders/parkinsons-disease. Accessed: April 2026. 8. Maserejian N, et al. Estimation of the 2020 Global Population of Parkinson’s Disease (PD). MDS Virtual Congress 2020. Abstract number 198. Available at: https://www.mdsabstracts.org/abstract/estimation-of-the-2020-global-population-of-parkinsons-disease-pd. Accessed: April 2026. 9. National Institute of Environmental Health Sciences. Neurodegenerative Diseases. Available at: https://www.niehs.nih.gov/research/supported/health/neurodegenerative. Accessed: April 2026. 10. Stoker T & Barker R. Recent developments in the treatment of Parkinson’s Disease. F1000Res. 2020 Jul 31;9:F1000 Faculty Rev-862. 11. Mayo Clinic. Parkinson’s disease. Available at: https://www.mayoclinic.org/diseases-conditions/parkinsons-disease/diagnosis-treatment/drc-20376062. Accessed: April 2026. 12. National Institutes of Health. Multiple System Atrophy. Available at: https://www.ninds.nih.gov/health-information/disorders/multiple-system-atrophy#toc-how-is-multiple-system-atrophy-diagnosed-and-treated. Accessed April 2026. 13. Vanacore N, Bonifati V, Fabbrini G, et al. Epidemiology of multiple system atrophy. ESGAP Consortium. European Study Group on Atypical Parkinsonisms. Neurol Sci. 2001;22(1):97-99. 14. Bower JH, Maraganore DM, McDonnell SK, Rocca WA. Incidence of progressive supranuclear palsy and multiple system atrophy in Olmsted County, Minnesota, 1976 to 1990. Neurology. 1997;49(5):1284-1288. 15. ClinicalTrials.gov. A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD). Available at: https://clinicaltrials.gov/study/NCT06285643. Accessed: April 2026 16. Heiss J, et al. Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson’s disease. Mov Disord. 2019 Jul;34(7):1073-1078. 17. Kells A, et al. Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. J Neurosci. 2010 Jul 14;30(28):9567-77. 18. Lin L, et al. GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science. 1993;260(5111):1130-1132. 19. Barker RA, et al. GDNF and Parkinson’s Disease: Where Next? A Summary from a Recent Workshop. J Parkinsons Dis. 2020;10(3):875-891. Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. Bayer AG is a holding company with operating subsidiaries worldwide. References to “Bayer” or “the company” herein may refer to one or more subsidiaries as context requires. Forward-Looking Statements AskBio This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding AskBio’s clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. Phil McNamara AskBio Media Contact +1 (984) 5207211 pmcnamara@askbio.com Dr. Imke Meyer Bayer Global Media Contact +49 (214) 60001275 imke.meyer@bayer.com

100 Deals associated with Ametefgene parvec

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10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Young onset Parkinson disease	Phase 2	United States	AskBio Inc.	01 Nov 2026
Parkinson Disease	Phase 2	United States	Bayer AG	11 Jun 2024
Parkinson Disease	Phase 2	Germany	Bayer AG	11 Jun 2024
Parkinson Disease	Phase 2	Poland	Bayer AG	11 Jun 2024
Parkinson Disease	Phase 2	United Kingdom	Bayer AG	11 Jun 2024
Multiple System Atrophy	Phase 1	United States	AskBio Inc.	30 Nov 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


MDS2024	Phase 1	Parkinson Disease	11	AAV2-GDNF (AB-1005) (Mild PD)	otgpruthjp(rmxuupbfzf) = nwnyrfwkih fgsqtxprmf (fotnfdbxoe, 4.5) View more	Positive	27 Sep 2024
				AAV2-GDNF (AB-1005) (Moderate PD)	otgpruthjp(rmxuupbfzf) = uflwzcuwti fgsqtxprmf (fotnfdbxoe, 4.5) View more
NCT04167540 (N2.001, AAN2024)	Phase 1	Parkinson Disease	11	AAV2-GDNF	peivqoyypx(mhrurdfkyo) = hfkoebrlqq megrhwgpqh (yaxgxfofmw ) View more	Positive	09 Apr 2024
				Placebo	peivqoyypx(mhrurdfkyo) = ilirjhiext megrhwgpqh (yaxgxfofmw )
NCT04167540 (Corporate Publications) Manual	Phase 1	Parkinson Disease	11	AB-1005	ficuxzlpso(mmtheuisqo) = The study met its primary objective, no attributed serious adverse events in all 11 patients at 18 months bwxpfxhdsd (bquxxskxkh ) Met	Positive	04 Jan 2024

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