Monogenetic Disease Therapy Market to Reach USD 96.6 Billion by 2035, Driven by Rapid Advancements in Gene-Editing Technologies

The global monogenetic disease therapy market (https://www.transparencymarketresearch.com/monogenetic-disease-therapy-market.html), valued at US$ 32.2 billion in 2024, is projected to expand at a compound annual growth rate (CAGR) of 11.0% from 2025 to 2035, reaching approximately US$ 96.6 billion by 2035. The surge in innovative gene-editing technologies such as CRISPR-Cas9, TALENs, and base editors, coupled with rising awareness of rare genetic disorders and robust regulatory support, is transforming how single-gene diseases are treated worldwide.

Discover Market Opportunities - Request Your Sample Copy Now: https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=86769

Analysts' Viewpoint

Analysts highlight that the monogenetic disease therapy industry is undergoing a fundamental transformation, fueled by the convergence of precision medicine, genomics, and AI-assisted diagnostics. Gene-based therapies are ushering in a new era of curative potential, replacing traditional symptom management approaches with permanent genetic correction.

A key trend is the expanding collaboration between biopharmaceutical companies and academic institutions, bridging the gap between laboratory discoveries and clinical applications. Increased investment incentives, including orphan-drug designations and accelerated approval pathways, are further driving innovation. Analysts predict that within the next decade, gene-editing therapies could become mainstream options for multiple monogenic disorders such as sickle cell anemia, cystic fibrosis, and polycystic kidney disease.

Market Introduction

The monogenetic disease therapy market is witnessing explosive growth as gene-editing and sequencing technologies evolve rapidly. With an estimated 10% of the global population affected by rare diseases, and approximately 80% of those being genetic in origin, there is a critical need for targeted therapies.

High-throughput sequencing, coupled with advanced viral vector production systems, has reduced costs and improved scalability. These advancements, combined with global initiatives for rare disease research funding, have created a robust innovation ecosystem focused on curative genetic solutions.

Furthermore, AI-powered bioinformatics tools now enhance mutation detection, optimize therapy design, and predict treatment efficacy - accelerating the pipeline of next-generation therapies.

Market Drivers

Advancements in Gene-Editing Technologies

The evolution of CRISPR-Cas9 and base-editing tools has revolutionized genetic therapy, offering precise correction of disease-causing mutations. These tools have become the foundation of numerous ongoing clinical trials targeting hematopoietic stem cells, retinal cells, and hepatocytes.

A landmark event in 2023 - the FDA approval of the world's first in vivo CRISPR therapy for sickle cell disease - has validated the safety and efficacy of gene-editing in humans. This milestone is widely regarded as a breakthrough for future therapies addressing hundreds of monogenic disorders.

As technologies continue to mature, gene-editing efficiency, delivery accuracy, and long-term durability are expected to improve significantly, solidifying their position as the backbone of future genetic medicine.

Rising Prevalence and Early Diagnosis of Rare Genetic Disorders
The growing global focus on early genetic screening has expanded the addressable patient pool for gene therapies. Countries across Europe, North America, and Asia are implementing newborn genomic screening programs that enable early diagnosis and intervention.

According to the Global Genes Alliance, one in ten people worldwide lives with a rare disease, and the majority stem from single-gene mutations. As next-generation sequencing (NGS) becomes more affordable and accessible, diagnosis timelines are shortening from months to mere days - drastically improving outcomes and boosting market demand.

Explore Strategies & Trends - Request Full Report Access - https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=86769

Dominance of Gene Therapy Segment

The gene therapy segment is expected to dominate the global monogenetic disease therapy market throughout the forecast period. Gene therapy offers long-lasting or potentially curative outcomes through single-dose administration, restoring defective gene functions and enabling sustained protein expression.

Innovations in adeno-associated viral (AAV) and lentiviral vectors, along with the rise of non-viral delivery mechanisms, have improved safety profiles and reduced immunogenicity risks.

Regulatory agencies are also endorsing these breakthroughs. For example, Zolgensma, approved for spinal muscular atrophy (SMA), has redefined treatment standards by offering life-saving efficacy in infants. Such success stories have prompted accelerated approvals for other genetic indications across neurology, hematology, and metabolic disorders.

Regional Outlook

North America leads the global monogenetic disease therapy market, accounting for the largest revenue share in 2024. The region benefits from:

A robust research ecosystem supported by the National Institutes of Health (NIH) and major academic centers.
A strong regulatory framework, including the FDA's orphan-drug and breakthrough therapy designations.
The world's largest number of gene therapy clinical trials, with over 60% conducted in the U.S. alone.
The U.S. in particular dominates due to established vector manufacturing capabilities, experienced researchers, and strong venture capital investments in biotechnology.

Meanwhile, Europe continues to expand its clinical pipeline, with Germany, the U.K., and France driving growth through national gene therapy programs and increased patient access initiatives. The Asia Pacific region, led by China and Japan, is also emerging as a promising market due to increased biotech investments and government-backed genome research programs.

Key Players and Competitive Landscape

The monogenetic disease therapy market features a dynamic mix of global pharmaceutical giants and specialized biotechnology firms. Leading players include:

Bayer AG
American Gene Technologies
CRISPR Therapeutics
Vertex Pharmaceuticals Incorporated
Sarepta Therapeutics, Inc.
MeiraGTx Limited
Verve Therapeutics, Inc.
Voyager Therapeutics, Inc.
Sanofi
Astellas Pharma Inc.
Orchard Therapeutics plc
Pfizer Inc.
Grifols, S.A.
Abeona Therapeutics Inc.

These companies are actively engaging in strategic collaborations, mergers, and licensing agreements to accelerate the commercialization of gene therapies.

In October 2025, Taysha Gene Therapies, Inc. received Breakthrough Therapy designation from the FDA for its AAV9-based therapy for Rett syndrome, signaling growing confidence in gene-based treatments for neurological diseases. Similarly, in September 2024, Vertex Pharmaceuticals secured Health Canada approval for PrCASGEVY, a genome-edited therapy targeting blood disorders.

The next decade will witness an exponential rise in gene therapy launches, supported by improved vector engineering, AI-guided treatment design, and personalized medicine approaches. As healthcare systems evolve to accommodate these high-value treatments, pricing models and reimbursement frameworks will also adapt, fostering long-term sustainability.

By 2035, monogenetic disease therapy is expected to shift from being experimental to mainstream, transforming the lives of millions worldwide. The integration of genomics, data analytics, and regulatory innovation will define the future of this high-impact sector.

Buy Full Report Now:

https://www.transparencymarketresearch.com/checkout.php?rep_id=86769<ype=S

Explore Latest Research Reports by Transparency Market Research:

Non-insulin Therapies for Diabetes Market: https://www.transparencymarketresearch.com/non-insulin-therapies-for-diabetes-market.html

Nocturia Market: https://www.transparencymarketresearch.com/nocturia-market.html

Neuroregeneration Therapy Market: https://www.transparencymarketresearch.com/neuroregeneration-therapy-market.html

Contact Us:
Transparency Market Research Inc.
CORPORATE HEADQUARTER DOWNTOWN,
1000 N. West Street,
Suite 1200, Wilmington, Delaware 19801 USA
Tel: +1-518-618-1030
USA - Canada Toll Free: 866-552-3453

About Transparency Market Research

Transparency Market Research, a global market research company registered at Wilmington, Delaware, United States, provides custom research and consulting services. Our exclusive blend of quantitative forecasting and trends analysis provides forward-looking insights for thousands of decision makers. Our experienced team of Analysts, Researchers, and Consultants use proprietary data sources and various tools & techniques to gather and analyses information.

Our data repository is continuously updated and revised by a team of research experts, so that it always reflects the latest trends and information. With a broad research and analysis capability, Transparency Market Research employs rigorous primary and secondary research techniques in developing distinctive data sets and research material for business reports.

This release was published on openPR.