Cell and Gene Therapy in Parkinson's Disease Clinical Trials Analysis 2025: Disease-Modifying Strategies and Circuit-Level Restorations Aim to Slow Progression and Improve Motor/Non-motor Outcomes | DelveInsight

DelveInsight's "Cell and Gene Therapy in Parkinson's Disease - Clinical Trials Analysis, 2025" highlights a diversified development landscape pursuing symptomatic relief and disease modification. Programs include dopamine-restorative cell implants, neuroprotective gene therapies targeting α-synuclein aggregation or neuroinflammation, and circuit-targeted gene delivery to rebalance basal ganglia networks. Trials incorporate advanced delivery (stereotactic infusion), standardized imaging and molecular biomarkers, and combination strategies pairing cell/gene therapy with neuromodulation.

Pivotal and late-phase trials focus on motor scales (MDS-UPDRS), OFF-time reduction, levodopa-sparing effects, and non-motor domains such as cognition and autonomic function. Durability of effect, graft survival/integration, and long-term safety (including dyskinesia and immunologic response) are core endpoints. Regulatory interactions emphasize appropriate comparators, long-term follow-up plans, and robust biomarker packages (PET imaging, CSF α-synuclein species) to support claims of meaningful clinical benefit and potential disease modification.

Interested in learning more about the current treatment landscape and the key drivers shaping the Cell and Gene Therapy in Parkinson's Disease pipeline? Click here: https://www.delveinsight.com/report-store/cell-and-gene-therapy-in-parkinsons-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

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 analysis depicts a robust space with 18+ active players working to develop 20+ pipeline drugs for cell and gene therapy in Parkinson's disease treatment.
• The leading cell and gene therapy in Parkinson's disease companies include Sumitomo Dainippon Pharma, BlueRock Therapeutics, Aspen Neuroscience, Prevail Therapeutics, S.Biomedics, MeiraGTx, Asklepios BioPharmaceutical, Hope Biosciences, and others are evaluating their lead assets to improve the cell and gene therapy in Parkinson's disease treatment landscape.
• Key cell and gene therapy in Parkinson's disease pipeline therapies in various stages of development include CT1 DAP001/DSP 1083, BRT-DA01, ANPD 001, LY3884961, A9-DPC, NLX P101, AB-1005, HB-adMSCs, and others.
• In September 2025, Ori Biotech announced that its IRO® platform received Advanced Manufacturing Technology (AMT) designation from the FDA, recognizing it as a next-gen solution for tackling key challenges in cell and gene therapy manufacturing.
• In March 2025, MedRhythms announced that MR-005, its neurorehabilitation system designed to support gait rehabilitation and motor function in adults with Parkinson's disease (PD), has been classified as a Class II, Rx-only medical device by the U.S. Food and Drug Administration (FDA). The system will be marketed under the brand name Movive.
• In March 2025, Medtronic plc, a global leader in healthcare technology, announced the FDA approval of BrainSenseTM Adaptive deep brain stimulation (aDBS) and BrainSenseTM Electrode Identifier (EI). While there is no cure for debilitating neurological conditions like Parkinson's, deep brain stimulation (DBS) has been transforming the lives of patients with Parkinson's and other neurological disorders for over 30 years.
• In Feb 2025, Medtronic received FDA approval for its BrainSenseTM Adaptive deep brain stimulation (aDBS) for Parkinson's disease, marking a significant advancement in neuromodulation therapy.
• In February 2025, AB-1005, AskBio's investigational gene therapy for Parkinson's disease, received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA to accelerate its development and review for treating serious or life-threatening conditions.
• In February 2025, the FDA approved CTEXLI (chenodiol) for treating cerebrotendinous xanthomatosis (CTX) in adults, making it the first FDA-approved drug for this rare lipid storage disease.
• In February 2025, Newronika received FDA Investigational Device Exemption (IDE) clearance to begin a pivotal U.S. clinical trial. The trial will assess the safety and efficacy of its adaptive deep brain stimulation system for patients with movement disorders, including Parkinson's disease.
• In February 2025, Supernus Pharmaceuticals, Inc. announced that the FDA approved ONAPGO (apomorphine hydrochloride) injection, formerly known as SPN-830, as the first and only subcutaneous apomorphine infusion device for treating motor fluctuations in adults with advanced Parkinson's disease.

Request a sample and discover the recent breakthroughs happening in the cell and gene therapy in Parkinson's disease pipeline landscape @ https://www.delveinsight.com/report-store/cell-and-gene-therapy-in-parkinsons-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cell and Gene Therapy in Parkinson's Disease Overview
Cell and gene therapies are emerging as promising treatments for Parkinson's disease (PD), a progressive disorder caused by the loss of dopamine-producing neurons. Cell therapies aim to replace these neurons using stem cell-derived or patient-specific iPSC-derived dopaminergic cells, with companies like BlueRock Therapeutics and Aspen Neuroscience leading clinical efforts. Gene therapies, meanwhile, target the molecular causes of PD by delivering therapeutic genes to the brain to enhance dopamine production, improve enzyme function, or protect neurons, with approaches such as AADC gene transfer showing potential for long-term, disease-modifying benefits.

Find out more about cell and gene therapy in Parkinson's disease medication @ https://www.delveinsight.com/report-store/cell-and-gene-therapy-in-parkinsons-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cell and Gene Therapy in Parkinson's Disease Treatment Analysis: Drug Profile
AB-1005: Asklepios BioPharmaceutical
AB-1005 is an investigational gene therapy utilizing an adeno-associated viral vector serotype 2 (AAV2) to deliver the human glial cell line-derived neurotrophic factor (GDNF) transgene. This allows for stable, localized expression of GDNF in the brain following direct neurosurgical injection with MRI-monitored convection-enhanced delivery. GDNF is a member of the transforming growth factor-β superfamily and has shown promise in promoting the survival and differentiation of dopaminergic neurons in midbrain cultures. It has been investigated as a potential neurorestorative treatment for diseases like Parkinson's, characterized by the degeneration of dopaminergic neurons. AB-1005 is currently in Phase II clinical trials for the treatment of Parkinson's disease.

HB-adMSCs: Hope Biosciences
Hope Biosciences' Adipose-Derived Mesenchymal Stem Cells (HB-adMSCs) are in clinical development for treating Parkinson's disease (PD). While the precise mechanism of action remains unclear, mesenchymal stem cells are generally believed to exert their therapeutic effects through a variety of mechanisms, including tissue repair and modulation of inflammation. Currently, HB-adMSCs are undergoing Phase II clinical trials for the treatment of PD.

Learn more about the novel and emerging cell and gene therapy in Parkinson's disease pipeline therapies @ https://www.delveinsight.com/report-store/cell-and-gene-therapy-in-parkinsons-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cell and Gene Therapy in Parkinson's Disease Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• 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

By Route of Administration
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

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

Scope of the Cell and Gene Therapy in Parkinson's Disease Pipeline Report
• Coverage: Global
• Key Cell and Gene Therapy in Parkinson's Disease Companies: Sumitomo Dainippon Pharma, BlueRock Therapeutics, Aspen Neuroscience, Prevail Therapeutics, S.Biomedics, MeiraGTx, Asklepios BioPharmaceutical, Hope Biosciences, and others.
• Key Cell and Gene Therapy in Parkinson's Disease Pipeline Therapies: CT1 DAP001/DSP 1083, BRT-DA01, ANPD 001, LY3884961, A9-DPC, NLX P101, AB-1005, HB-adMSCs, and others.

Dive deep into rich insights for drugs used for cell and gene therapy in Parkinson's disease treatment; visit @ https://www.delveinsight.com/report-store/cell-and-gene-therapy-in-parkinsons-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Cell and Gene Therapy in Parkinson's Disease Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Cell and Gene Therapy in Parkinson's Disease Pipeline Therapeutics
6. Cell and Gene Therapy in Parkinson's Disease Pipeline: Late-Stage Products (Phase III)
7. Cell and Gene Therapy in Parkinson's Disease Pipeline: Mid-Stage Products (Phase II)
8. Cell and Gene Therapy in Parkinson's Disease Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

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.

This release was published on openPR.