GDNF

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AskBio’s investigational Parkinson’s disease (PD) gene therapy. The FDA grants RMAT designation to regenerative therapies being developed for serious or life-threatening diseases and conditions. The Bayer subsidiary’s AB-1005 will now benefit from improved access to the regulator, which may include comprehensive guidance on efficient drug development, rolling Biologics License Application review, and other measures to accelerate both the development and approval process of the therapy. More than ten million people worldwide are estimated to be living with PD, a progressive neurodegenerative disease characterised by symptoms such as tremor, muscle rigidity and slowness of movement. Recombinant glial cell line-derived neurotrophic factor (GDNF), a protein that helps neurons survive and differentiate, has long been evaluated as a potential treatment for diseases marked by progressive degeneration of midbrain dopaminergic neurons, including PD. AB-1005, formerly known as AAV2-GDNF, is designed to allow for stable and continuous expression of GDNF in localised regions of the brain following direct neurosurgical injection. Among the evidence supporting the FDA’s decision were the 36-month results from an open-label phase 1b trial of AB-1005, which demonstrated continued positive trends in assessed clinical outcome measures and showed that the candidate was well tolerated and not associated with any product-related serious adverse events. The first patients in the phase 2 REGENERATE-PD study of AB-1005 have already been randomised in the US, and additional study sites in the US, UK, Germany and Poland are expected to open for enrolment in the first half of 2025. AskBio’s chief executive officer, Gustavo Pesquin, said: “The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with PD and their loved ones. “This milestone could potentially expedite the development of our important investigational gene therapy programme, and it highlights our promising data and the potential of AB-1005 for patients and the medical community. We look forward to working closely with the FDA to accelerate our programme” Christian Rommel, executive vice president, global head of research at Bayer, added: “The RMAT designation for AB-1005 underscores the high unmet medical need and the potential of this investigational gene therapy to make a difference for patients with PD.”

The market for cell and gene therapy in Parkinson's disease is driven by the critical unmet medical need for disease-modifying treatments, advancements in clinical trials showing promising results, and regulatory support for innovative therapies. Additionally, the economic burden of managing PD and the rise in patient advocacy for cutting-edge solutions are further accelerating investment and development in this area. Together, these factors position cell and gene therapies as transformative in addressing the current treatment gaps in Parkinson's disease. LAS VEGAS, Oct. 9, 2024 /PRNewswire/ -- DelveInsight's ' Cell and Gene Therapy in Parkinson's Disease Pipeline Insight – 2024 ' report provides comprehensive global coverage of pipeline cell and gene therapy in Parkinson's disease in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space and future growth potential of the cell and gene therapy in Parkinson's disease pipeline domain. Key Takeaways from the Cell and Gene Therapy in Parkinson's Disease Pipeline Report DelveInsight's cell and gene therapy in Parkinson's disease pipeline report depicts a robust space with 18+ active players working to develop 20+ cell and gene therapies for Parkinson's disease treatment. Key cell and gene therapy in Parkinson's disease companies such as Sumitomo Dainippon Pharma, BlueRock Therapeutics, Aspen Neuroscience, Prevail Therapeutics, S.Biomedics, MeiraGTx, Asklepios BioPharmaceutical, iRegene Therapeutics, Kenai Therapeutics, Tetraneuron, and others are evaluating new cell and gene therapy in Parkinson's disease to improve the treatment landscape. Promising pipeline cell and gene therapy in Parkinson's disease in various stages of development include CT1 DAP001/DSP 1083, BRT-DA01, ANPD 001, LY3884961, A9-DPC, AAV-GAD, AB-1005, NouvNeu 001, RNDP 003, TET AAV E2F4DN, and others. In July 2024, S.BIOMEDICS announced positive one-year assessment data from the first 3 participants (first low dose cohort) of Phase I/IIa clinical trial for Parkinson's disease with TED-A9, hESC (human embryonic stem cell)-derived midbrain dopaminergic progenitors. In July 2024, Bayer AG and Asklepios BioPharmaceutical announced that the United States (U.S.) Food and Drug Administration (FDA) had granted Fast Track Designation for AB-1005, which is being developed for moderate Parkinson's disease. In June 2024, The U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene therapeutics targeting Parkinson's Disease. Previously, the "Combined Phase I/II Clinical Study" of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. In May 2024, Bayer AG and BlueRock Therapeutics announced that BlueRock's investigational cell therapy bemdaneprocel for the treatment of Parkinson's disease had been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). In April 2024, Aspen Neuroscience, Inc. announced that the first patient had been dosed in the ASPIRO trial, a Phase I/IIa open label clinical trial to assess safety and tolerability of ANPD001, an autologous, dopaminergic neuron cell replacement therapy for participants with moderate to severe Parkinson's disease (PD). In March 2024, Bayer AG and BlueRock Therapeutics announced details of 18-month data from the phase I clinical trial for bemdaneprocel (BRT-DA01), an investigational allogeneic stem cell derived cell therapy for treating Parkinson's disease. In January 2024, Bayer AG and Asklepios BioPharmaceutical announced the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson's disease (PD).1, Request a sample and discover the recent advances in cell and gene therapy in Parkinson's disease drug treatment @ Cell and Gene Therapy in Parkinson's Disease Pipeline Report The cell and gene therapy in Parkinson's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage cell and gene therapy in Parkinson's disease, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the cell and gene therapy in Parkinson's disease clinical trial landscape. Cell and Gene Therapy in Parkinson's Disease Overview Cell and gene therapy holds significant promise for treating Parkinson's disease, a neurodegenerative disorder characterized by the progressive loss of dopaminergic neurons in the brain. Cell therapy focuses on the transplantation of stem cells or progenitor cells into the brain to replace the lost neurons or support their survival. These transplanted cells can potentially differentiate into dopaminergic neurons, helping restore the dopamine levels that are crucial for motor control. Recent advances in induced pluripotent stem cells (iPSCs) have enabled the development of patient-specific neurons, which can minimize immune rejection and offer personalized treatment options. Early clinical trials have shown potential, though challenges remain in ensuring long-term survival and functionality of the transplanted cells. Gene therapy, on the other hand, aims to modify the genetic makeup of neurons to slow down or reverse the disease process. Techniques like viral vectors are used to deliver genes that can boost dopamine production, protect neurons from degeneration, or reduce the buildup of harmful proteins like alpha-synuclein, which is implicated in Parkinson's disease pathology. One notable example is the delivery of the GDNF (glial cell line-derived neurotrophic factor) gene, which has been shown to support the survival of dopaminergic neurons. Although gene therapy for Parkinson's disease is still in its experimental stages, it holds potential as a long-lasting and less invasive approach compared to traditional treatments like deep brain stimulation or dopamine replacement therapy. With ongoing advancements, both cell and gene therapies could revolutionize how Parkinson's disease is managed in the future. Find out more about cell and gene therapy in Parkinson's disease @ New Cell and Gene Therapies in Parkinson's Disease A snapshot of the pipeline Cell and Gene Therapy in Parkinson's Disease mentioned in the report: Learn more about the emerging cell and gene therapy in Parkinson's disease @ Cell and Gene Therapy in Parkinson's Disease Clinical Trials Cell and Gene Therapy in Parkinson's Disease Therapeutics Assessment The cell and gene therapy in Parkinson's disease pipeline report proffers an integral view of emerging cell and gene therapy in Parkinson's disease segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Cell and Gene Therapy in Parkinson's Disease Pipeline Report Coverage: Global Cell and Gene Therapy in Parkinson's Disease Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Cell and Gene Therapy in Parkinson's Disease Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Cell and Gene Therapy in Parkinson's Disease Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Cell and Gene Therapy in Parkinson's Disease Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Key Cell and Gene Therapy in Parkinson's Disease Companies: Sumitomo Dainippon Pharma, BlueRock Therapeutics, Aspen Neuroscience, Prevail Therapeutics, S.Biomedics, MeiraGTx, Asklepios BioPharmaceutical, iRegene Therapeutics, Kenai Therapeutics, Tetraneuron, and others. Key Cell and Gene Therapy in Parkinson's Disease Pipeline Therapies: CT1 DAP001/DSP 1083, BRT-DA01, ANPD 001, LY3884961, A9-DPC, AAV-GAD, AB-1005, NouvNeu 001, RNDP 003, TET AAV E2F4DN, and others. Dive deep into rich insights for new cell and gene therapy for Parkinson's disease treatment; visit @ Cell and Gene Therapy for Parkinson's Disease Treatment Table of Contents For further information on the cell and gene therapy in Parkinson's disease pipeline therapeutics, reach out @ Cell and Gene Therapy in Parkinson's Disease Drug Treatment Related Reports Cell and Gene Therapy in Parkinson's Disease Market Cell and Gene Therapy in Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cell and gene therapy in Parkinson's disease companies, including MeiraGTx, Hope Biosciences, Sumitomo Pharma, Prevail Therapeutics, BlueRock Therapeutics, Voyager Therapeutics, among others. Parkinson's Disease Pipeline Parkinson's Disease Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Parkinson's disease companies, including Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, Inc., Modag, Annovis Bio Inc., BioVie Inc., United Neuroscience Ltd., Luye Pharma Group, AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., Nobilis Therapeutics Inc., BlueRock Therapeutics, Taiwan Mitochondrion Applied Technology Co., Ltd., among others. Psychosis in Parkinson's and Alzheimer's Disease Market Psychosis in Parkinson's and Alzheimer's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key psychosis in Parkinson's and Alzheimer's disease companies, including Sunovion Pharmaceuticals, Karuna Therapeutics, Vanda Pharmaceuticals, Suven Life Sciences, Enterin, Intra-Cellular Therapies, Merck Sharp & Dohme, among others. Parkinson's Disease-Related Dementia Market Parkinson's Disease-Related Dementia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease-related dementia companies, including AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

RESEARCH TRIANGLE PARK, NC; USA I June 25, 2024 I 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 cure, and the effectiveness of current treatments frequently decreases over time. 7 About REGENERATE-PD REGENERATE-PD is a Phase 2, randomized, double-blind, surgery-controlled study of the efficacy and safety of intraputaminal AAV2-GDNF in the treatment of adults (45-75 years) with moderate stage Parkinson’s disease.  Subjects will receive either bilateral image-guided infusion of AAV2-GDNF into the putamen, single dose (active treatment arm), or bilateral partial burr/twist holes (control arm). The trial will include an estimated 87 subjects with study sites located in the United States, UK and Europe. For more information about the REGENERATE-PD clinical trial, visit clinicaltrials.gov (NCT06285643), or visit www.askbio.com . About AB-1005 AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery 8,9 GDNF is a homodimer that is a distantly related member of the transforming growth factor-β superfamily. In midbrain neuronal cell cultures, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake. GDNF has long been evaluated as a potential treatment for diseases, such as Parkinson’s, marked by progressive degeneration of midbrain dopaminergic neurons. 10 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 nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes 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 800 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 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 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com . References [1] Parkinson’s Foundation. Who has Parkinson’s? Available at: https://www.parkinson.org/understanding-parkinsons/statistics . Accessed: June 2024. [2] Asklepios BioPharmaceutical, Inc. (January 2024). AskBio Phase Ib trial of AB-1005 gene therapy in patients with Parkinson’s disease meets primary endpoint [Press Release]. https://www.askbio.com/askbio-phase-ib-trial-of-ab-1005-gene-therapy-in-patients-with-parkinsons-disease-meets-primary-endpoint [3] ClinicalTrials.gov. GDNF Gene Therapy for Multiple System Atrophy. Available at: https://clinicaltrials.gov/study/NCT04680065 . Accessed: June 2024. [4] Michael J. Fox Foundation. Parkinson’s 101 – What is Parkinson’s disease? Available at: https://www.michaeljfox.org/parkinsons-101 . Accessed: June 2024. [5] DeMaagd G, Philip A. Parkinson’s Disease and Its Management: Part 1: Disease Entity, Risk Factors, Pathophysiology, Clinical Presentation, and Diagnosis. P T. 2015 Aug;40(8):504-32. PMID: 26236139; PMCID: PMC4517533. [6] Parkinson’s Foundation. Motor-fluctuations. Available at: https://www.parkinson.org/library/fact-sheets/motor-fluctuations . Accessed: June 2024. [7] Mayo Clinic. Parkinson’s disease. Available at: https://www.mayoclinic.org/diseases-conditions/parkinsons-disease/diagnosis-treatment/drc-20376062 . Accessed: June 2024. [8] Heiss JD, Lungu C, Hammoud DA, Herscovitch P, Ehrlich DJ, Argersinger DP, Sinharay S, Scott G, Wu T, Federoff HJ, Zaghloul KA, Hallett M, Lonser RR, Bankiewicz KS. Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson’s disease. Mov Disord. 2019 Jul;34(7):1073-1078. [9] Kells AP, Eberling J, Su X, Pivirotto P, Bringas J, Hadaczek P, Narrow WC, Bowers WJ, Federoff HJ, Forsayeth J, Bankiewicz KS. Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. J Neurosci. 2010 Jul 14;30(28):9567-77. [10] Lin LF, Doherty DH, Lile JD, Bektesh S, Collins F. GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science. 1993;260(5111):1130-1132. SOURCE: Asklepios BioPharmaceutical