Asklepios BioPharmaceutical, Inc.

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 . 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Bavarian Nordic said it would work with UNICEF to deliver vaccines upon request. Image credit: Shutterstock / Anas-Mohammed. UNICEF has reached an agreement with mpox vaccine manufacturer Bavarian Nordic to supply one million doses of the shot for the hardest-hit countries in Africa. The supply, which includes a 500,000-dose commitment by the Global Alliance for Vaccines and Immunization (GAVI) announced last week, means 2.5 million doses of the MVA-BN vaccine have been outlaid to Africa, according to Bavarian Nordic’s CEO Paul Chaplin. The company said it would work with UNICEF to “swiftly deliver the vaccines upon request”, in a 26 September statement. Other sources of vaccine supply have come from donations by various governments and institutions, as well as a 40,000-dose donation by Bavarian Nordic itself. The European Health Emergency Preparedness and Response Authority ( Hera ), for example, ordered 175,420 doses for Africa’s Centres for Disease Control and Prevention (CDC). See Also: Boehringer Ingelheim opens $66.8m new cancer research facility AskBio and Belief BioMed partner to advance gene therapies Bavarian Nordic’s MVA-BN vaccine is marketed as Imvanex in Europe and Jynneos in other countries including the US and Singapore. Currently, the vaccine is the only one approved by the US Food and Drug Administration (FDA). Bavarian Nordic said earlier this month it would prioritise the production of MVA-BN over other product orders. The Swiss biotech said it would target the production of two million mpox vaccines by the end of this year, which includes the doses promised to UNICEF. Bavarian Nordic added that it is targeting the manufacturing of 13 million doses by the end of next year. Africa Centers for Disease Control and Prevention (CDC) previously stated that more than ten million doses could be required through 2025 to fight the outbreak in the continent, which is being propelled by the more dangerous variant clade Ib. There have been 24,000 confirmed or suspected mpox cases in Africa this year, including over 600 deaths, as per a European Centre for Disease Prevention and Control epidemiological update . MVA-BN was only approved for adults until the European Medicines Agency (EMA) became the first regulator to approve its use in adolescents earlier this month. A Bavarian Nordic spokesperson confirmed to Pharmaceutical Technology at the time that the company has “plans to expand this approval process to other high-risk countries”. Bavarian Nordic is targeting even further coverage of its vaccine via a Phase II clinical trial (NCT06549530) to assess the immunogenicity and safety of MVA-BN in children aged between two to 12 years.

Cobenfy introduces a new mechanism of action to the schizophrenia treatment landscape and an additional option for patients. Credit: 13_Phunkod via Shutterstock. The US Food and Drug Administration (FDA) has approved Bristol Myers Squibb ’s first-in-class schizophrenia drug Cobenfy (xanomeline-trospium), providing patients with a new treatment option not seen in decades. The novel drug – named Cobenfy – introduces a new mechanism of action to the schizophrenia treatment landscape by targeting cholinergic receptors as opposed to dopamine receptors. The market for schizophrenia drugs has long been dominated by dopamine-blocking antipsychotics, which are associated with significant side effects such as Parkinsonian symptoms, sexual dysfunction, and weight gain. Originally developed by Karuna Therapeutics and named KarXT, BMS inherited the drug as part of a $14bn acquisition, which was finalised in March 2024. The acquisition is set to pay off as Cobenfy is set to pull in $2.99bn in sales in 2030, according to GlobalData forecasts. GlobalData is the parent company of Pharmaceutical Technology . Cobenfy is a twice-daily pill that combines two drugs: one that targets muscarinic receptors M1 and M4, which increase activity in the parasympathetic nervous system, and another that lessens side effects from activating those receptors. Clinical trial data has shown that the drug relieves symptoms such as delusions without some of the drastic side effects caused by currently approved antipsychotics. See Also: Boehringer Ingelheim opens $66.8m new cancer research facility AskBio and Belief BioMed partner to advance gene therapies The excitement surrounding Cobenfy also stems from its ability to address both the positive and negative symptoms of schizophrenia. Unlike dopamine receptors, M1 and M4 receptors work on positive symptoms such as hallucinations and delusions, and negative symptoms that include reduced emotional output, speech, and motivation. Data from the Phase III EMERGENT-3 trial (NCT04738123) showed that the drug demonstrated an 8.4-point reduction in Positive and Negative Syndrome Scale (PANSS) total score compared to placebo at week five.  However, the new drug comes with side effects like any other therapy. The prescribing information includes warnings that Cobenfy can cause urinary retention, increased heart rate, decreased gastric movement, or angioedema (swelling beneath the skin) of the face and lips. In the announcement accompanying the approval, Tiffany Farchione, director of the Division of Psychiatry, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research said: “This drug takes the first new approach to schizophrenia treatment in decades. This approval offers a new alternative to the antipsychotic medications people with schizophrenia have previously been prescribed.” Some experts have expressed concerns about the twice-daily dosing schedule of the drug, noting that treatment effectiveness often depends on consistent medication adherence – something that can be difficult for schizophrenia patients, especially given the side effects of traditional antipsychotics. However, in a Q1 2023 earnings call, Karuna’s former CEO Bill Meury suggested that adherence to Cobenfy could surpass standard schizophrenia treatments. To tackle this issue, Terran Biosciences are developing a prodrug version of xanomeline-trospium, called TerXT. Terran’s candidates keep Cobenfy’s formulation while optimising its dosing schedule to a more convenient once-daily oral regimen, and a multi-month injectable. BMS said patients can enrol in a patient assistance scheme called the Cobenfy Cares programme in late October when the drug is set to be available.