Cell and Gene Therapy in Parkinson's Disease Clinical Trial Pipeline Analysis Demonstrates 18+ Key Companies at the Horizon Expected to Transform the Treatment Paradigm, Assesses DelveInsight

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Oct 09, 2024, 17:31 ET

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,

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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:

Drugs	Company	Phase	RoA
AB-1005	Asklepios BioPharmaceutical	Phase II	Intracerebral
LY3884961	Prevail Therapeutics	Phase I/II	Intra-cisterna magna
ANPD 001	Aspen Neuroscience	Phase I/II	Parenteral
A9-DPC	S.Biomedics	Phase I/II	Intracerebral
NouvNeu001	iRegene Therapeutics	Phase I/II	Intracerebral
BRT-DA01	BlueRock Therapeutics	Phase I	Intracerebral

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.

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Table of Contents

1.	Cell and Gene Therapy in Parkinson's Disease Pipeline Report Introduction
2.	Cell and Gene Therapy in Parkinson's Disease Pipeline Report Executive Summary
3.	Cell and Gene Therapy in Parkinson's Disease Pipeline: Overview
4.	Cell and Gene Therapy in Parkinson's Disease Clinical Trial Therapeutics
5.	Cell and Gene Therapy in Parkinson's Disease Pipeline: Late-Stage Products (Pre-registration)
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.	Cell and Gene Therapy in Parkinson's Disease Pipeline Therapeutics Assessment
10.	Inactive Products in the Cell and Gene Therapy in Parkinson's Disease Pipeline
11.	Company-University Collaborations (Licensing/Partnering) Analysis
12.	Key Companies
13.	Key Products in the Cell and Gene Therapy in Parkinson's Disease Pipeline
14.	Unmet Needs
15.	Market Drivers and Barriers
16.	Future Perspectives and Conclusion
17.	Analyst Views
18.	Appendix

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SOURCE DelveInsight Business Research, LLP