Cell and Gene Therapy in Parkinson's Disease Market Accelerates as Regenerative and Disease-Modifying Approaches Move Into Late-Stage Trials

The Cell and Gene Therapy in Parkinson's Disease (PD) market is moving from concept to commercial reality. With pluripotent stem cell-derived dopaminergic neuron grafts now in Phase II/III and multiple AAV gene therapies entering or advancing through clinical trials, this niche is evolving into one of the most closely watched segments of the global neurodegeneration pipeline. Over the next decade, cell and gene therapies are expected to complement (and in some cases challenge) deep brain stimulation and chronic dopaminergic drugs as long-term treatment options for selected PD patients.

Download Full PDF Sample Copy of Market Report @ https://exactitudeconsultancy.com/request-sample/72098

Keyword definition
In Parkinson's disease, cell and gene therapies aim to go beyond symptomatic dopamine replacement by:
• Cell therapy / cell replacement therapy (CRT)
Transplanting dopaminergic neurons or progenitors (often derived from human embryonic stem cells or iPSCs) into the striatum to restore dopaminergic input and improve motor function.
• Gene therapy
Using viral vectors (typically AAV) to deliver genes that:
o Enhance dopamine synthesis (e.g., AADC gene delivery)
o Express neurotrophic factors (e.g., GDNF, neurturin)
o Correct or modulate genetic drivers such as GBA mutations or α-synuclein overexpression (e.g., GBA-targeting vectors, RNAi approaches).
These approaches are intended either to rebuild dopaminergic circuitry or slow/modify disease progression, addressing the limitations of current levodopa-based pharmacotherapy.

Market size, CAGR and outlook
There is not yet a large standalone revenue base for PD-focused cell and gene therapies, but the investment and pipeline activity are substantial:
• The global cell and gene therapy market was about USD 21.9-22.7 billion in 2023-2024 and is projected to reach around USD 119-132 billion by 2034-2035, at a CAGR of ~18-29%.
• Parkinson's disease remains one of the most prominent CNS indications in this broader segment, with multiple cell and gene programs in Phase I-III.

Positioning for client use:
• Today, revenue from PD cell and gene therapies is minimal and largely trial-related, but deal values, infrastructure investments and Phase II/III activity indicate a high double-digit growth trajectory once the first products are approved.
• Given PD's >10 million global patient burden and the strong focus by large pharma (e.g., Bayer/BlueRock, Novartis, AAV/ RNAi deals), PD is expected to represent a multi-billion-dollar addressable opportunity within the broader cell and gene therapy market over the next decade.

Key players and programs
Cell therapy (dopaminergic neuron replacement)
• Bayer / BlueRock Therapeutics - bemdaneprocel (BRT-DA01)
hESC-derived dopaminergic neuron therapy; Phase I data show sustained safety and encouraging motor trends at 36 months, and the program has now advanced into Phase III.
• Academic and biotech consortia in Japan, Europe and the US
Several groups are running Phase I/II trials of hESC- or iPSC-derived dopaminergic progenitors with early safety and feasibility data reported in high-impact journals.

Gene therapy
• AskBio (Bayer) - AB-1005 (GDNF gene therapy)
AAV2-based delivery of GDNF; currently in Phase II (REGENERATE-PD) with sites enrolling in the US and Europe.
• Capsida Biotherapeutics - CAP-003 (GBA-focused gene therapy)
IV-administered AAV gene therapy for PD associated with GBA mutations; received FDA IND clearance in 2025, with a Phase 1/2 trial initiating.
• Novartis / Arrowhead - ARO-SNCA (RNAi against α-synuclein)
Preclinical RNA interference therapy aimed at reducing α-synuclein production, backed by a licensing deal potentially worth over USD 2 billion, highlighting pharma's long-term commitment.
• Multiple earlier-generation programs (e.g., AADC gene therapy)
AADC gene therapy trials have demonstrated multi-year safety and functional benefit but some partnered programs (e.g., Voyager/Neurocrine VY-AADC) were discontinued after portfolio review, informing design of newer approaches.

Market segmentation and growth insights
By modality
• Cell therapy (allogeneic and autologous dopaminergic grafts)
o Targets moderately advanced PD with significant motor fluctuations.
o High upfront procedure cost, but potential for long-lasting benefit and reduced medication burden.
o Likely to launch initially via highly specialized centers of excellence.
• In vivo gene therapy (AAV/viral vectors)
o Includes dopamine-biosynthesis enhancement, neurotrophic factor delivery, and genetic-risk modification (e.g., GBA).
o One-time dosing with potential multi-year effect supports premium pricing but demands robust safety and durability data.
• RNA and gene-expression modulators (RNAi, antisense)
o Focus on α-synuclein and other pathogenic targets; earlier stage but strategically important for truly disease-modifying strategies.

By disease stage / patient profile
• Genetically defined PD (e.g., PD-GBA)
o Attractive for gene-targeted therapies (CAP-003 and similar programs).
• Moderate-stage PD with motor complications
o Primary initial cell-therapy and GDNF-gene-therapy target group.
• Younger-onset and highly motivated patients
o Early adopters for invasive, high-value interventions such as neurosurgical gene and cell delivery.

By end user
• High-volume neurosurgical and movement-disorder centers
• Academic hospitals in North America, Europe and Japan
• Eventually, selected specialized centers in China, South Korea, Australia and other APAC markets as regulatory approvals expand.

Explore Full Report here: https://exactitudeconsultancy.com/reports/72098/cell-and-gene-therapy-in-parkinson-s-disease-market

Regional outlook
• North America and Europe
o Lead in clinical trial activity, infrastructure and investment for PD cell and gene therapies, with Bayer/BlueRock, AskBio, Capsida and multiple academic centers driving momentum.
• Japan and broader Asia-Pacific
o Strong academic leadership in dopaminergic cell replacement and iPSC science, with several early-phase trials already running.
• Rest of world
o Expected to follow once initial approvals and long-term safety data are established; time lag will depend on cost, regulatory capacity and neurosurgical expertise.

Key market drivers
• High unmet need: Current PD therapies are symptomatic and lose efficacy over time; there is no approved disease-modifying treatment.
• Strong scientific rationale for dopaminergic neuron replacement and targeted gene delivery, supported by encouraging Phase I safety and early efficacy signals.
• Large and aging patient population, with PD prevalence >10 million globally and rising, creating significant long-term demand.
• Macro growth of cell and gene therapy as a platform, attracting capital, manufacturing build-out and regulatory experience that will benefit PD indications.
• Strategic big-pharma involvement, including major investments by Bayer, Novartis and others, de-risking commercialization and market access pathways.
Key challenges include neurosurgical complexity, high upfront costs, long-term safety/oncogenicity monitoring, ethical issues around stem-cell sourcing, and payer hesitancy before robust real-world evidence is available.

Conclusion
The Cell and Gene Therapy in Parkinson's Disease market is still nascent in revenue terms but already strategically central within the broader PD landscape. Over the next decade, as:
• First dopaminergic cell therapies advance through Phase III,
• GDNF and GBA-targeted gene therapies mature, and
• α-synuclein and RNAi programs progress,
this segment is expected to evolve into a multi-billion-dollar, high-growth niche within the global cell and gene therapy market.
Companies that:
• Secure late-stage clinical success and regulatory approval for cell or gene therapies,
• Build specialized delivery networks and long-term follow-up infrastructure, and
• Integrate their products into comprehensive PD care pathways (alongside DBS, rehab and digital monitoring)
will be positioned to lead this next wave of Parkinson's disease treatment.

This report is also available in the following languages : Japanese (パーキンソン病市場における細胞・遺伝子治療), Korean (파킨슨병 시장의 세포 및 유전자 치료), Chinese (帕金森病细胞和基因疗法市场), French (Marché de la thérapie cellulaire et génique dans la maladie de Parkinson), German (Markt für Zell- und Gentherapie bei Parkinson), and Italian (Mercato della terapia cellulare e genica nel morbo di Parkinson), etc.

Request for a sample of this research report at (Use Corporate Mail ID for Quick Response) @ https://exactitudeconsultancy.com/request-sample/72098

Our More Reports:

Cell and Gene Therapy Manufacturing Market
https://exactitudeconsultancy.com/reports/72365/cell-and-gene-therapy-manufacturing-market

Cell and Gene Therapy Fundamental Analysis Market
https://exactitudeconsultancy.com/reports/72636/cell-and-gene-therapy-fundamental-analysis-market

Biologics Fill Finish Manufacturing Market
https://exactitudeconsultancy.com/reports/73133/biologics-fill-finish-manufacturing-market

Cell and Gene Therapy CRO Market
https://exactitudeconsultancy.com/reports/73143/cell-and-gene-therapy-cro-market

About Us
Exactitude Consultancy is a market research & consulting services firm which helps its client to address their most pressing strategic and business challenges. Our market research helps clients to address critical business challenges and also helps make optimized business decisions with our fact-based research insights, market intelligence, and accurate data.
https://bulletin.exactitudeconsultancy.com/

https://www.thehealthanalytics.com/

https://www.analytica.global/

https://www.marketintelligencedata.com/

https://www.marketinsightsreports.com/

https://exactitudeconsultancy.com/

Connect Us:
Irfan Tamboli
PHONE NUMBER +1 (704) 266-3234
EMAIL ADDRESS: sales@exactitudeconsultancy.com

This release was published on openPR.