Cell And Gene Therapy In Parkinson's Disease- Pipeline Insight,2025

This “Cell and Gene Therapy in Parkinson’s Disease - Pipeline Insight, 2025” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in Cell and Gene Therapy in Parkinson’s Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Cell and Gene Therapy in Parkinson’s Disease: Understanding

Cell and Gene Therapy in Parkinson’s Disease: Overview

According to the Parkinson’s Foundation, Parkinson’s disease is a neurodegenerative disorder that affects predominately dopamine-producing (dopaminergic) neurons in a specific area of the brain called the substantia nigra. Because Parkinson’s disease develops over time, various stages help identify how symptoms have progressed and what should be expected next. Generally, there are five stages of Parkinson’s disease: Stage I, Stage II, Stage III, Stage IV, and Stage V.

According to the National Health Service (NHS), there are many different symptoms associated with Parkinson’s disease. The main symptoms referred to by doctors as parkinsonism include tremors, slowness of movement (bradykinesia), and muscle stiffness (rigidity), which are classified as motor symptoms while non-motor symptoms include cognitive changes, autonomic dysfunctioning, mood and sleep disorders, and others.

Several mechanisms have been implicated in Parkinson’s disease pathogenesis, with α-synuclein aggregation central to the development of the disease. Multiple other processes are thought to be involved, with several studies suggesting that abnormal protein clearance, mitochondrial dysfunction, and neuroinflammation play a role in the onset and progression of Parkinson’s disease. However, the relationship between these pathways remains unclear. Native α-synuclein in the brain is mostly unfolded without a defined tertiary structure, although in aqueous solutions, it can be present in stable tetramers that resist aggregation.

At present, there is no definitive test for Parkinson’s disease. However, there are various symptoms and diagnostic tests used in combination. Making an accurate diagnosis of Parkinson’s - particularly in its early stages - is difficult, but a skilled practitioner can conclude it is Parkinson’s disease. There is no one way to diagnose Parkinson’s disease; however, it can often be identified by a general neurologist trained to diagnose and treat neurologic disorders. To avoid misdiagnosis, consultation with a movement disorder specialist (MDS) is recommended. Medications are the most common therapy for Parkinson’s disease. The goal is to correct the shortage of dopamine; this deficiency causes the symptoms. Pharmacological treatment is usually started when symptoms become disabling or disrupt daily activities. Treatments may differ according to the patient’s symptoms, age, and responses to specific drugs. Finding the best combination of drugs for each patient takes time. Levodopa (L-dopa, l-3, 4-dihydroxyphenylalanine), the metabolic precursor of dopamine, is the single most effective agent for treating Parkinson’s disease. Levodopa is largely inert; its therapeutic and adverse effects result from the decarboxylation of levodopa to dopamine. Furthermore, a new area being explored in the treatment of Parkinson’s disease is gene therapy. Both non-disease and disease-modifying transgenes are being researched.

'Cell and Gene Therapy in Parkinson’s Disease- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Cell and Gene Therapy in Parkinson’s Disease pipeline landscape is provided which includes the disease overview and Cell and Gene Therapy in Parkinson’s Disease treatment guidelines. The assessment part of the report embraces, in depth Cell and Gene Therapy in Parkinson’s Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Cell and Gene Therapy in Parkinson’s Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Cell and Gene Therapy in Parkinson’s Disease R&D. The therapies under development are focused on novel approaches to treat/improve Cell and Gene Therapy in Parkinson’s Disease.

Cell and Gene Therapy in Parkinson’s Disease Emerging Drugs Chapters

This segment of the Cell and Gene Therapy in Parkinson’s Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Cell and Gene Therapy in Parkinson’s Disease Emerging Drugs

AB-1005 : Asklepios BioPharmaceutical

AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 containing the human glial cell line-derived neurotrophic factor (AAV2-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. 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 disease modifying neurorestorative treatment for diseases, such as Parkinson’s, marked by progressive degeneration of midbrain dopaminergic neurons. The drug is currently being evaluated in Phase II stage of development for the treatment of patients with Parkinson’s disease.

HB-adMSCs: Hope Biosciences

Hope Biosciences Adipose Derived Mesenchymal Stem Cells (HB-adMSCs) is in clinical development for the treatment of Parkinson's disease (PD). The exact mechanism of action (MOA) by which HB-adMSCs exert their therapeutic effects in PD is not fully elucidated. However, mesenchymal stem cell therapies in general are thought to work through various mechanisms. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Parkinson’s Disease.

ANPD 001: Aspen Neuroscience

ANPD001 is an investigational autologous cell therapy developed by Aspen Neuroscience for the treatment of Parkinson's disease (PD). This therapy aims to replace lost dopamine neurons by using dopamine neuronal precursor cells (DANPCs) derived from patient-specific induced pluripotent stem cells (iPSCs). The iPSCs are generated from the patient's own skin cells, ensuring a personalized treatment approach that minimizes the risk of immune rejection and eliminates the need for immunosuppressive drugs, which can be poorly tolerated in older populations. Currently, the drug is in the Phase I/II stage of development to treat Parkinson’s Disease.

BRT-DA01: BlueRock Therapeutics

Bemdaneprocel (BRT-DA01) is an investigational cell therapy developed for the treatment of Parkinson's disease, designed to restore dopamine-producing neurons that are lost due to the disease. This therapy is derived from pluripotent stem cells and involves the surgical implantation of dopamine neuron precursors into the brain, specifically targeting the putamen, a critical area for motor function. Currently, the drug is in the Phase I stage of development to treat Parkinson’s Disease.

Cell and Gene Therapy in Parkinson’s Disease: Therapeutic Assessment

This segment of the report provides insights about the different Cell and Gene Therapy in Parkinson’s Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Cell and Gene Therapy in Parkinson’s Disease

• There are approx. 18+ key companies which are developing the therapies for Cell and Gene Therapy in Parkinson’s Disease. The companies which have their Cell and Gene Therapy in Parkinson’s Disease drug candidates in the most advanced stage, i.e. Phase II include, Asklepios BioPharmaceutical and Hope Biosciences.

Phases

The report covers around 20+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Cell and Gene Therapy in Parkinson’s Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Cell and Gene Therapy in Parkinson’s Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Cell and Gene Therapy in Parkinson’s Disease therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Cell and Gene Therapy in Parkinson’s Disease drugs.

Cell and Gene Therapy in Parkinson’s Disease Report Insights

• Cell and Gene Therapy in Parkinson’s Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Cell and Gene Therapy in Parkinson’s Disease Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Cell and Gene Therapy in Parkinson’s Disease drugs?
• How many Cell and Gene Therapy in Parkinson’s Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cell and Gene Therapy in Parkinson’s Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Cell and Gene Therapy in Parkinson’s Disease therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Cell and Gene Therapy in Parkinson’s Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Sumitomo Dainippon Pharma
• BlueRock Therapeutics
• Aspen Neuroscience
• Prevail Therapeutics
• S.Biomedics
• MeiraGTx
• Asklepios BioPharmaceutical
• Hope Biosciences

Key Products

• CT1 DAP001/DSP 1083
• BRT-DA01
• ANPD 001
• LY3884961
• A9-DPC
• NLX P101
• AB-1005
• HB-adMSCs

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